Evidence-Based Health Care

Transkrypt

4th International Symposium
Evidence-Based
Health Care
HTA & Pricing
Skład / DTP
Maciej Dziadyk
Druk / Printed by
Centrum Druku GRAF – www.cdgraf.com.pl
Kraków, 7-8 XII 2009
Evidence-Based
Health Care
HTA & Pricing
Uniwersytet Jagielloński
Auditorium Maximum, ul. Krupnicza 35
Central and Eastern European Society
IV Międzynarodowe Sympozjum
Evidence-Based
of Technology Assessment in Health Care
Komitet Organizacyjny (LOC) / Local Organizing Committee (LOC)
Jacek Siwiec
Przewodniczący Komitetu Organizacyjnego / Chairman of LOC
Anna Bednarska
Maciej Dziadyk
Marcin Gąsiorowski
Urszula Gogołowicz
Katarzyna Katarzyńska
Małgorzata Karp
Anna Kordecka
Karolina Kucia
Magdalena Mrożek
Agnieszka Nadzieja-Kozioł
Radosław Rudź
Komitet Naukowy (SPC) / ScientiÞc Program Committee (SPC)
Jacek Ruszkowski
Przewodniczący Komitetu Naukowego / Chairman of SPC
Magdalena Władysiuk
Vice-przewodniczący Komitetu Naukowego / Vice-Chairman of SPC
Krzysztof Łanda
Vice-przewodniczący Komitetu Naukowego / Vice-Chairman of SPC
Robert Plisko
Przemysław Ryś
Kontakt / Contact
CEESTAHC
ul. Świętokrzyska 4/1, 30-015 Kraków, POLAND
tel. +48 (0) 12 357 76 34, fax +48 (0) 12 396 38 39
www.ceestahc.org , e-mail: [email protected]
4
Health Care
HTA & Pricing
Kraków 7-8 XII 2009
www.ceestahc.org
Prelegenci / Experts
Prof. Jack Dowie
Emeritus Professor of Health Impact Analysis
Public Health and Policy Dept London School of
Hygiene and Tropical Medicine, UK
Jim Furniss
Director, Global Market Access Strategy Bridgehead International Limited, Leicestershire,
UK
Prof. Michael Drummond
Professor of Health Economics Centre for Health Economics, University of York, UK
Prof. Pavel Vorobyev
President of Russia Society for Pharmacoeconomics Research, Russia
Prof. Bengt Jönsson
Professor of Health Economics, Stockholm
School of Economics, Sweden
Zoltan Kalo
CEO at Syreon Research Institute, Director of
Health Economics Research Centre at Eötvös
Loránd University, Hungary
Prof. Gert van der Wilt
Department of Epidemiology, Biostatistics and
HTA Radboud University Medical Centre Nijmegen, Nederlands
Dragana Atanasijevic
Local Consultant for HTA Project Coordination
Unit Ministry of Health of Serbia, Serbia
Chris Henshall
Pro Vice Chancellor for External Relations at
the University of York, UK
Mitchell Sugarman
Sr. Director of Health Economics, Policy and
Payment, Medtronic, USA
Prof. David Banta
Professor Emeritus, University of Maastricht,
Netherlands
Jorge Wernli
VP Global Pricing & Government Affairs at Vifor
Pharma, Switzerland
Alexandre Lemgruber
Head of the OfÞce of Economic Evaluation
of New Technologies, at the Brazilian Health
Agency - ANVISA, Brazil
Anita Burrell
Head, Health Economics & Reimbursement
PVD, SanoÞ Aventis, France
Prof. Rod Taylor
Associate Professor in Health Services Research & ScientiÞc Director of Peninsula Clinical
Trials Unit, Peninsula Medical School, UK
Rabia Kahveci
President of Turkish Evidence Based Medicine
Association, Turkey
J. Jaime Caro
President and Chief Executive OfÞcer, Senior
Vice President, UBC, USA
Joanna Mucha
Member of Parliament, academic lecturer,
Poland
Wojciech Matusewicz
Director, AHTAPol, Poland
Prof. Zbigniew Szawarski
Institute of Philosophy, University of Warsaw,
Poland
Prof. Jacek Ruszkowski
Director of the Public Health Center, Kozminski
University, Poland
Erin Huntington
Corporate Affairs Director Elli Lilly Europe, UK
Oleg Borisenko
Executive Director of Russia Society for Pharmacoeconomics Research, Russia
Joanna Lis
Manager of Health Economics Dept., SanoÞAventis Group, Poland
Krzysztof Łanda
CEO of HTA Audit, Poland
Precursor and promoter of EBM/HTA/EBHC in
Poland. Author and co-author of numerous
papers on methodology, guidelines and systemic studies. Initiator of education and training
activities in the Þeld of Health Technology
Assessment, including the EBHC Symposium.
Vice-president of HTA Consulting, Poland
An expert in EBM, HTA and PhE; author of
numerous training programs and research
analyses in HTA.
Katarzyna Bondaryk
Hogan & Hartson, Poland
5
Evidence-Based
Szanowni Państwo,
W imieniu Stowarzyszenia CEESTAHC oraz
Akademii Leona Koźmińskiego serdecznie zapraszamy do wzięcia udziału w:
On behalf of the organizers: CEESTAHC
and the Kozminski University we heartily invite you to take part in:
IV Międzynarodowym Sympozjum
the 4th International Symposium
Evidence-Based Health Care
Evidence-Based Health Care,
pt. „HTA & Pricing”
titled “HTA & Pricing”
Sympozjum odbędzie się w Krakowie w
dniach 7-8 grudnia 2009 roku w Auli Audytorium Maximum Uniwersytetu Jagiellońskiego.
The Symposium will take place on December 7th and 8th, 2009 in Krakow, at the Jagiellonian University Auditorium Maximum.
Organizowane po raz czwarty Sympozjum
EBHC jest wyjątkową inicjatywą edukacyjną
dla uczestników z Polski oraz innych krajów
Europy Centralnej i Wschodniej. Daje uczestnikom możliwość spotkania z ekspertami z
Polski, Europy i świata. Jest to również okazja
do swobodnej wymiany poglądów z przedstawicielami ministerstw i funduszów zdrowia
oraz reprezentantami europejskich środowisk
medycznych i biznesowych.
The EBHC Symposium, organized for the
fourth time, is a unique educational initiative for interested individuals from Poland and
other countries of Central and Eastern Europe.
Its participants have an opportunity to meet
experts from Poland, Europe and the whole
world. The Symposium provides also an opportunity to exchange opinions with representatives of ministries and public payers as well
as those of European medical and business
communities.
W roku ubiegłym mieliśmy przyjemność
zorganizować w Krakowie III Sympozjum
EBHC, które zaowocowało wymianą doświadczeń, nawiązaniem kontaktów i rozpoczęciem
współpracy pomiędzy polskimi oraz zagranicznymi środowiskami naukowymi. W roku 2008
odnotowaliśmy uczestnictwo ponad 300 specjalistów z zakresu EBM i HTA, decydentów,
menadżerów oraz przedstawicieli świata nauki
z kilkunastu krajów. Znaczącą grupę stanowili
światowej sławy eksperci reprezentujący europejskie oraz międzynarodowe organizacje
zajmujące się tematyką efektywności, opłacalności i jakości świadczeń medycznych.
Przewodnim motywem tegorocznego Sympozjum EBHC jest pricing, czyli kształtowanie
cen produktów leczniczych i wyrobów medycznych. Ustalanie cen stanowi ważny instrument
polityki zdrowotnej państwa.
6
Ladies and Gentlemen,
In the last year we had the pleasure of organizing the 3rd EBHC Symposium in Krakow,
which resulted in sharing of experience, establishing of new relations and further cooperation between scientists in Poland and worldwide. In 2008 our invitation was accepted by
more than 300 specialists in the Þeld of EBM
and HTA, decision-makers, managers and
scientists from Europe and the USA. We noted
among them a signiÞcant number of worldwide recognized experts, representing European and international organizations engaged in
problems of effectiveness, cost-effectiveness
and quality of health care services.
The keynote of this year’s EBHC Symposium is pricing of medicinal products and medical devices. Pricing is an important element
of the state health policy.
Health Care
HTA & Pricing
www.ceestahc.org
Ewolucja systemów zdrowotnych w krajach
wysokorozwiniętych zmierza w kierunku kontroli cen leków i wyrobów medycznych tak,
aby wydatki miały racjonalny charakter. Przejrzystość regulacji cen produktów leczniczych,
do której wprost odwołuje się dyrektywa Rady
UE (89/105/EEC z dnia 21.12.1988) zyskuje
coraz bardziej na znaczeniu w krajach Europy
Centralnej i Wschodniej.
Evolution of health care systems in developed countries tends towards price control of
medications and medicinal products in order
to rationalize expenses. Transparent regulation of medicinal product prices, mentioned
explicitly in the European Council directive
(89/105/EEC from 21.12.1988.) becomes a
more and more recognized issue in countries
of Central and Eastern Europe.
W ramach IV Sympozjum omówione zostaną systemy ustalania cen refundowanych produktów leczniczych i wyrobów medycznych,
zagadnienia wpływu aranżacji refundacyjnych
na poziom cen, mechanizmy i techniki negocjacyjne, a także zasady oceny innowacyjności oparte na HTA. Tematyka mechanizmów
pricing’owych i umów podziału ryzyka zostanie przedstawiona przez wybitnych praktyków
ze świata.
During the 4th Symposium systems of pricing for reimbursed medicinal products and
medical devices, problems related to reimbursement arrangements and its inßuence on prices, negotiation mechanisms and techniques
as well as HTA-based principles of assessment
of innovation will be discussed. Issues related
to pricing mechanisms and risk sharing schemes will be presented by worldwide recognized experts.
Program naukowy Sympozjum zostanie
zrealizowany w ciągu dwóch dni w ośmiu sesjach tematycznych.
The scientiÞc program of the Symposium
will be presented over two days in eight thematic sessions.
Przyjęta formuła Sympozjum zakłada czas
na dyskusję z wybitnymi ekspertami zagranicznymi, co pozwoli uczestnikom na interakcję w szerszym zakresie niż ma to zwykle
miejsce podczas tego typu konferencji. Komunikację ułatwi symultaniczne tłumaczenie
wszystkich wystąpień na język polski i angielski. W imieniu wszystkich osób i instytucji zaangażowanych w organizację Sympozjum serdecznie zachęcamy do udziału w tym ważnym
dla ekonomiki zdrowia wydarzeniu.
The form of Symposium assumes longer
time for discussion with eminent experts,
which will allow the participants to beneÞt
from their knowledge to a greater degree
than is usual for this kind of meetings. Communication will be facilitated by simultaneous
translation of all presentations into English
and Polish. On behalf of all the persons and
institutions engaged in organization of the
Symposium we heartily invite you to take part
in this event, so important for economic and
medical environment.
Łączymy wyrazy szacunku,
Yours faithfully,
prof. Jacek Ruszkowski
Jacek Siwiec
Przewodniczący Komitetu Naukowego
Chairman of the ScientiÞc Committee
Przewodniczący Komitetu Organizacyjnego
Chairman of the Organizing Committee
7
Evidence-Based
Poniedziałek, 7 grudnia 2009 / Monday, December 7th, 2009
Sesja / Session
Temat wykładu / Lecture topic
Prelegent
Expert
Otwarcie Sympozjum / Opening of the Symposium: David Banta, Jacek Ruszkowski, Jacek
Siwiec
Jack Dowie (opening presentation): Jak podejmować racjonalnie decyzje: analiza decyzyjna w oparciu o złożone kryteria, jako prawdopodobna przyszłość HTA oraz podejmowania
decyzji klinicznych / Deciding how to decide: Multi-Criteria Decision Analysis is probably the
future for both Health Technology Assessment and Shared Clinical Decision Making
1
Panel dyskusyjny
Discussion
panel
Potrzeba „dekalogu” dla decydentów
Need for decalogue for health care politician
2
HTA, kształtowanie cen i odpłatność za efekty
HTA, Pricing and Pay for Performance
25 min.
Bengt Jönsson
Zakupy centralne produktów leczniczych. Podział
ryzyk pomiędzy płatnika a Þrmę farmaceutyczną
Central purchase procedures for medicinal products. Risk sharing between the payer and the
pharmaceutical company
Katarzyna Bondaryk
Strategiczne kształtowanie cen leków w krajach
Unii Europejskiej
Strategic pricing of pharmaceuticals in the European Union
Zoltan Kalo
Przerwa na lunch / Lunch-break
Podział ryzyka: amerykański i europejski punkt
widzenia / Risk Sharing: US vs European Perspectives
3
Wartość
terapeutyczna
leków i rola
HTA w umowach podziału
ryzyka
Value of drug
therapy and
the role of
HTA in risk
sharing arrangements
Ocena stosowania szczepionki przeciwko HPV
(Cervarix) u kobiet w wieku 17-25 lat. Czy negatywne decyzje refundacyjne można uzasadnić
wobec opinii publicznej? / Appraisal of HPV vaccination (Cervarix) for girls/ young women aged
17-25 yrs. Can negative reimbursement decisions
be justiÞed to the public?
65 min.
9:30
David Banta
12:20
Jack Dowie
Joanna Mucha
Jorge Wernli
90 min.
Wojciech Matusewicz
Zbigniew Szawarski
Jacek Ruszkowski
Krzysztof Łanda
Przerwa na kawę / Coffee-break
Umowy podziału ryzyka:
cele, metody,
negocjacje
– perspektywa publiczna
/ Risk sharing
schemes:
objectives,
methods,
negotiations
– public perspective
Czas*
Timing*
25 min.
12:45
25 min. 14:10
25 min.
60 min.
Anita Burrell
30 min.
Gert van der Wilt
40 min.
Erin Huntington
30 min.
Wartość farmakoterapii i rola HTA w umowach
podziału ryzyka / The value of drug therapy and
the role of HTA in risk-sharing arrangements
Jim Furniss
25 min.
Spotkanie z ekspertami i uroczysta kolacja w „Pałacu pod Baranami” w Krakowie - Rynek Główny 27
(obok Ratusza) / ”Meet the Experts” dinner-party at the „Pałac pod Baranami” - Rynek Główny 27
(Main Square 27, by the Town Hall Tower)
8
14:10
15:10
15:10
17:25
Cena innowacyjnego produktu leczniczego z
perspektywy globalnego producenta farmaceutycznego
The price of the innovative drug from the perspective of global pharma company
* w czasy sesji wliczono czas dyskusji / discussion time included
12:20
12:45
20:00
Health Care
HTA & Pricing
www.ceestahc.org
Wtorek, 8 grudnia 2009 / Tuesday, December 8th, 2009
Sesja / Session
4
Wartość terapeutyczna
technologii
nielekowych z
perspektywy
przemysłu /
Value of nondrug technologies – industry
perspective
Prelegent
Expert
Ocena wyrobów medycznych jako wyzwanie dla HTA:
punkt widzenia oceniającego / The HTA challenge of
medical device assessment: The perspective of assessor
Rod Taylor
Aspekty polityczne refundacji technologii nielekowych
z perspektywy przemysłu / The Industry Perspective;
Policy and Reimbursement for Non-Drug Technologies
Mitchell
Sugarman
5
Rozwój HTA w
krajach Europy
Centralnej i
Wschodniej
- ostatnie osiągnięcia i zmiany
Developments
of HTA in CEE
countries
6
Wpływ farmakoekonomiki na kształtowanie cen w Rosji:
doświadczenia i perspektywy / Impact of pharmacoeconomics on pricing in Russia: experience and perspective
Pavel
Vorobyev
Znaczenie oceny nowego leku przy wprowadzaniu go na
rynek w Rosji / Value of new drug assessment during
market access in Russia
Oleg
Borisenko
7
8
30 min.
10:40
10:50
HTA w krajach rozwijających się – obciążenie czy
konieczność? / HTA – burden or need for developing
economies
Dragana
25 min. 10:50
Atanasijevic
12:40
Przepisy dotyczące kształtowania cen w Turcji i ich
wpływ na refundację. Jaka jest potencjalna rola HTA?
Pricing Regulations in Turkey, effects on reimbursement. What is the Potential Role for HTA?
Rabia Kahveci
25 min.
Rola HTA w regulacji cen nowych leków: doświadczenia
brazylijskie / The role of HTA in the price regulation of
new drugs: the Brazilian experience
Alexandre
Lemgruber
25 min.
Właściwe miejsce wydajności wśród priorytetów opieki
zdrowotnej / The proper role of efÞciency in “priority
setting” in health care
QALY: zło konieczne?
QALYs: a necessary evil?
10 min.
J. Jaime
Caro
Michael
Drummond
12:50
14:15
35 min.
Umowy CED i inne metody podziału ryzyka: pomoc czy
utrudnienie? / CED and other approaches to Managed
Entry: help or hindrance?
Chris Hens30 min.
hall
Studium wykonalności dla Agencji Cen ze szczególnym
uwzględnieniem działań na rzecz porozumień podziału
ryzyka / Feasibility of Pricing Agency in a CEE country
in handling Managed Entry Schemes
Magdalena
Władysiuk
Joanna Lis
Bolesne doświadczenia z HTA bez wcześniejszej analizy
problemu decyzyjnego – wskazówki dla wytwórców
raportów HTA / Painful lessons from stepping in the HTA
path without prior scoping – tips for HTA doers
Krzysztof
Łanda
Zakończenie Sympozjum / Closure of the Symposium: Jacek Siwiec
14:15
15:15
15:15
16:15
20 min.
5 min.
DeÞniowanie problemów decyzyjnych w procesie oceny
technologii medycznych: teoria i praktyka / Scoping in
health technology assessment: theory and practice
12:40
12:50
35 min.
60 min.
Przerwa / Technical break
DeÞniowanie
problemów decyzyjnych (APD)
Scoping
9:30
10:40
25 min.
Przerwa na lunch / Lunch-break
Refundacja
warunkowa w
ramach porozumień cenowych
Managed entry
schemes
30 min.
10 min.
Granice opłacalności w podejmowaniu decyzji
refundacyjnych
Cost-utility
thresholds vs efÞciency frontier
Czas*
Timing*
16:15
16:20
35 min.
16:20
25 min. 17:35
5 min.
* w czasy sesji wliczono czas dyskusji / discussion time included
9
Poniedziałek
7 grudnia 2009
Monday
December 7th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 1 / Session 1
Panel dyskusyjny: Potrzeba „dekalogu” dla decydentów
Discussion panel: Need for decalogue for health care politician
David Banta, Jack Dowie, Joanna Mucha, Jorge Wernli, Wojciech Matusewicz, Zbigniew Szawarski, Jacek Ruszkowski, Krzysztof Łanda
Opis sesji / About the Session
W tym roku panel dyskusyjny poświęcony będzie pożądanej postawie etycznej
polityków podejmujących decyzje dotyczące ochrony zdrowia. Ochrona zdrowia ma
szczególne i bardzo silne oddziaływanie na
społeczeństwo, ale też życie zwykłego „szarego człowieka”. Czy wobec tego wymagania
wobec osób podejmujących decyzje dotyczące tej dziedziny nie powinny być wyższe?
Może trzeba stworzyć coś na kształt dekalogu lub zbioru norm etycznych?
Przewidujemy gorącą dyskusję między
audytorium i zaproszonymi politykami oraz
decydentami.
This year discussion panel will raise questions concerning attitudes of health care politicians. Health care is a specially vulnerable
area with tremendous impact on society and
lives of individuals. If health care is so special in various aspects, maybe requirements
from health care politicians and decision
makers should be higher? Maybe could their
attitudes be appraised due to a “Decalogue”
or a special code of ethics? Should it be done
up front and/or periodically?
11
Evidence-Based
Sesja 1 / Session 1
A Global Perspective on Health Technology Assessment
HTA Development in the USA
• US Congressional Office of Technology
Assessment began HTA in 1975
• Growing attention to HTA in US in
various health programs
• Special interest in insurance coverage
and HTA - Blue Cross/Blue Shield,
Medicare program, Kaiser health plans
Early HTA in Europe
Supported by the European
Commission
• Sporadic HTA-related studies began in early
1980s, usually as part of health services
research
• Study groups and workshops on HTA-related
subjects began about 1985
Formation of National and
Regional Public HTA Programs
• 1987 - Swedish Council on Health
Technology Assessment (SBU)
• Early 1990s - France, Catalonia (Spain),
United Kingdom
• Active countries by early 1990s included
the Netherlands, Finland, Denmark,
Switzerland
12
Prelegent / Expert
David Banta
15 min.
HTA & Pricing
EUR-ASSESS Program 1994-1997
• First concerted attempt to coordinate
HTA in Europe (funded by European
Commission)
• Active partners included Sweden, the
Netherlands, France, the UK, Catalonia,
Switzerland
• Coordination itself was not funded
www.ceestahc.org
David Banta
Sesja 1 / Session 1
Health Care
Achievements of the EURASSESS Program
• Substantive group work
• More important - the process and
experience of working together
• Identification and recruitment of
members from other member states
•
International Journal of Technology Assessment in Health Care,
Volume 13, Spring 1997
Continued Efforts to Develop
a European Coordinating
Program
• HTA-Europe 1998-2000 - Commissioned
papers on the health systems and HTA in all
members of the EU - published in the
international journal of HTA in 2000
• ECHTA/ECAHI - 2000-2002
• EUnetHTA - 2004 - present
• Commitment of European Commission to
develop a permanent program
13
Evidence-Based
Sesja 1 / Session 1
Contributors to an
International View of HTA
• ISTAHC (and now HTAi) - 1985 on - Special
Interest Group on Developing Countries
(shaping of individuals)
• INAHTA - 1993 - 13 founding members; First
developing country member was Malaysia
(shaping of institutions - presently 46
members from 26 countries including Poland,
Brazil, Argentina, Chile, Taiwan)
Contributors to an
International View of HTA
(continued)
• The World Bank - substantial support to
developing countries beginning in China in
1986; Malaysia; Romania; Poland; other
Eastern European countries. Latin America
• The World Health Organization - support for
many individuals; PAHO more focus on
institutional development; WHO Collaborating
Centers
Asian Situation in HTA
• Only 2 members of INAHTA
• Asian Regional Network - around 10
country members; and a number of
Regional HTA Conferences 2000-on
• Korea, Philippines, Taiwan - Focus on
insurance coverage; programs set up as
offices in insurance organizations
14
David Banta
HTA & Pricing
Achievements of HTA
Worldwide
• Awareness in many countries and individuals
of the importance of evidence, especially
efficacy and safety
• A surprising common view of HTA and
common methods and approaches - probably
due to international organizations’ efforts
• Development of HTA programs in countries
with “emerging economies”
www.ceestahc.org
David Banta
Sesja 1 / Session 1
Health Care
Failures of HTA
• Almost no activity in countries at the lowest
level on the development scale - Africa, Latin
America and Caribbean, Asia and Pacific
countries, Eastern Europe
• Almost no activity in the world of Islam Malaysia, Iran, Lebanon are exceptions
• Only efficacy and cost-effectiveness seriously
considered in most HTA reports
The Challenge is Obvious
• Reaching out to countries and people
not involved in HTA at this moment
15
Evidence-Based
Sesja 1 / Session 1
16
Prelegent / Expert
Jak podejmować racjonalnie decyzje: analiza decyzyjna w oparciu o wiele
kryteriów jako prawdopodobna przyszłość HTA i wspólnego podejmowania
decyzji klinicznych / Deciding how to decide: Multi-Criteria Decision Analysis is
probably the future for both Health Technology Assessment and Shared Clinical
Decision Making
Jack Dowie
Przedstawiam JUDEMAKIA – mapę świata
Ocen i Podejmowania Decyzji, na której zaznaczono pełen zakres Technologii Zaufania (Belief
Technologies), Technologii Preferencji (Preference Technologies) i Technologii Decyzyjnych
(Decision Technologies) w ramach Kontinuum
Kognitywnego Kena Hammonda (od Analizy do
Intuicji). Mapa obejmuje też analizę decyzyjną
oraz szczególne przypadki implementacji MCDA
(Proces Hierarchii Analitycznej – Annalisa), jak
również niektóre zastosowania MCDA zarówno
w kontekście świadczeń zdrowotnych (ocena
technologii medycznych – HTA), jak i działań
klinicznych (wspólne podejmowanie decyzji klinicznych – SCDM). Podkreślone zostaną jej zalety w odniesieniu do rozwiązywania problemów
wynikających z chęci równoczesnego uwzględnienia w idealnym modelu opieki zdrowotnej
danych naukowych, preferencji pacjenta i efektywności kosztowej, zarówno na poziomie indywidualnym, jak i populacyjnym. Przedstawiam
również analizę efektywności zasobów decyzyjnych (DREA). Podczas gdy analiza efektywności kosztowej odpowiada na konkretne pytanie
– co powinniśmy zrobić?– DREA traktuje o sposobach podejmowania decyzji – jak powinniśmy zdecydować, co robić? Jaki jest właściwy
sposób podjęcia decyzji o tym, która technologia decyzyjna powinna być zastosowana w odniesieniu do konkretnej decyzji w konkretnym
kontekście, z uwzględnieniem wagi przypisanej
intuicji i analizie, ścisłości i związkowi z problemem, złożoności i praktyczności? MCDA stanowi najlepsze rozwiązanie tego „meta-decyzyjnego” problemu.
I introduce JUDEMAKIA, a map of the world
of Judgement and Decision Making, in which
we can locate the full range of Belief Technologies, Preference Technologies and Decision
Technologies in the framework provided by Ken
Hammond’s Cognitive Continuum of changing
Analysis to Intuition balances. Decision Analysis
and particular implementations of Multi-Criteria
Decision Analysis – MCDA (Analytic Hierarchy
Process, Annalisa) - are then located on this
map and some applications of MCDA in both
the health service context (Health Technology
Assessment - HTA) and clinical context (Shared Clinical Decision Making - SCDM) provided.
Its advantages in transparently addressing the
tensions in the simultaneous pursuit of Evidence-Informed, Preference-Based and Cost-Effective Care at both individual and population level are emphasised. Finally I introduce Decision
Resource-Effectiveness Analysis (DREA). While
Cost-Effectiveness Analysis focuses on the adoption decision - what should we do? - DREA
targets the decision decision - how should we
decide what to do? What is the appropriate way
to decide which Decision Technology should be
used for a speciÞc decision in a speciÞc context,
given the relative weights assigned to intuition
and analysis, rigour and relevance, complexity and practicality? MCDA emerges as the best
way to tackle this meta-decision.
40 min.
HTA & Pricing
Jak podejmować racjonalnie decyzje: analiza decyzyjna w oparciu o wiele kryteriów... / Deciding how to decide: Multi-Criteria Decision Analysis is probably
the future for both Health Technology Assessment and Shared Clinical Decision
Making
Deciding how to decide
Multi-Criteria Decision Analysis
is (probably) the future for
both
Health Technology Assessment
and
Shared Clinical Decision Making
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
Jack Dowie
Professor Emeritus of Health Impact Analysis
London School of Hygiene and Tropical Medicine
[email protected]
Abstract
• I introduce JUDEMAKIA, a map of the world of Judgement and
Decision Making, in which we can locate various Belief
Technologies, Preference Technologies and Decision Technologies in
the framework provided Ken Hammond’s Cognitive Continuum of
changing Analysis to Intuition balances.
• I locate (Single-Criterion) Decision Analysis – SCDA - and particular
implementations of Multi-Criteria Decision Analysis – MCDA
(Analytic Hierarchy Process, Annalisa) on this map
• I introduce some applications of MCDA in both the health service
context (Health Technology Assessment - HTA) and clinical context
(Shared Clinical Decision Making - SCDM)
• I suggest that Decision Resource-Effectiveness Analysis – DREA - is
the appropriate way to tackle the meta decision of how to decide to
decide – i.e. determine which Decision Technology should be used for
a specific decision in a specific context, given the relative weights
assigned to the high level criteria of rigour and practicality
• DREA is best implemented via MCDA
Some of the slides in the handout may not
appear in the presentation but they have
been included for later follow-up if desired.
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Evidence-Based
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Taxonomy of tasks and technologies
• To produce beliefs*, elicit beliefs and evaluate belief
claims we need a
Belief* Technology [*Knowledge/Evidence]
• To establish what preferences are held and elicit preference
judgements we need a
Preference* Technology [*Value]
• To make a decision we need a
Decision Technology
DTs need inputs from BTs and PTs
and BTs and PTs need inputs from DTs
and the transfer process requires ITs and CTs
• To transfer information we need an
Information Technology
• To communicate we need a
Communication Technology
Examples
• Belief or Knowledge Technologies
judgement, interview, panel, cohort study, RCT
• Preference or Values Technologies
judgement, interview, Visual Analog Scale,
Standard Gamble, Time Trade-Off, DCE
• Decision Technologies
judgement, coin toss, meeting, pro/con checklist,
F&F decision analysis, Comp Stoch Decision Model
• Information / Communication Technologies
body language, ppt presentation, Report with tables
and graphs, Youtube video, twitter
18
Jack Dowie
HTA & Pricing
Making
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Jack Dowie
Sesja 1 / Session 1
Health Care
A very inefficient BT
- Gary Larson
A very simple PT
– Randy Glasbergen
3 Risk factors
3 Risk factors
Black
White
Male
Male
Baseball cap
backwards
4 Mitigating
factors
Wrong
neighbourhood
4 Mitigating
factors
Loafers
White
Fed Ex
envelope
Groceries
Whistling
Sondheim
Humming
Motown
Over 40
Polo shirt
A moderately
analytic DT
– Garry Trudeau
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Evidence-Based
Sesja 1 / Session 1
Making
A fairly inappropriate ICT
– Nick D Kim
2005
2006
Underlying framework
• Cognitive Continuum Theory of Ken Hammond
Human Judgment and Social Policy: Irreducible Uncertainty,
Inevitable Error, Unavoidable Injustice (Oxford UP, 1996)
• 2 dimensions:
• COGNITIVE dimension (how we think about a JDM task)
which runs from highly Intuitively to highly Analytically
• TASK STRUCTURE dimension (how a JDM task presents itself to us)
which runs from Very well-structured to Very ill-structured
• Any instantiation of a BT, PT, DT or I/CT can be
• characterised by its Analysis-to-Intuition ratio / balance
• ‘quality’ assessed by the high-level criteria of Coherence and
Correspondence [not today]
• Location of a JDM on the Task Structure dimension does
induce (?) (should induce?) matching process on
Cognitive dimension
20
Jack Dowie
HTA & Pricing
Making
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
Highest demands and requirements
Control,
ability to
manipulate
variables
Time
and
resources
(??)
Analytical
skills
and
capacities
Lowest demands and requirements
21
Evidence-Based
Sesja 1 / Session 1
Making
InterventionalObservational
Border Conflicts
QualitativeQuantitative
Border Conflicts
INTERVENTION
INTERVENTION
OBSERVATION
MODELLING
ARGUMENTATION
INTUITION
‘INSTINCT’
22
Jack Dowie
HTA & Pricing
Making
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
‘Taking Into Account and Bearing In Mind’
•
•
•
•
•
•
•
•
•
•
•
‘Taking things into account’
‘Giving considerations due weight’
‘Establishing the right balance’
‘Keeping things in proportion’
‘Taking a measured view’
‘Bringing everything into the equation’
‘Figuring it out’
‘Seeking a degree of consensus’
‘Gauging the impact’
‘Making sure things add up’
‘Summing up…’
• note how TIABIM DT, a basically qualitative discourse,
is given a quantitative flavour
Key rule
• The products of BTs in B-land (beliefs =knowledge,
evidence, judgments, opinions) and of PTs in P-land
(preferences=values, importance weights) can only be
input into a DT in D-land at the same latitude as that DT
• E.g. an effect size from an RCT created at the A-I ratio
(level of explicitness and precision) of Randomistan can
only become an input into TIABIMIA by being moved to
the A-I ratio characteristic of Conferalot
(Don’t be deceived by appearances or claims)
• E.g. an intuitive probability judgement from Judgia can
only become an input into Analysia by being moved to the
A-I ratio of Modelia
• Analysing up/down = Dumbing up/down ??
23
Evidence-Based
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Making
HEALTH
IMPACT
ASSESS
MENT
Evidence
base
E-B
GUIDE
LINES
Patient’s
preferences
Note the position
of the apostrophe
24
Jack Dowie
HTA & Pricing
Making
HTA
I
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
HTA
I
TIABIM – the verdicts
• TIABIM is fine, no problems, flaws
• TIABIM is not perfect, it does produce lots of
bad/poor decisions, but this is because
• the wrong people with the wrong values dominate –
bring in the true/right ‘stakeholders’ and it will be fine
• we have the right people with the right values, but they
lack knowledge/information/evidence – supply them
with better k/i/e and it will be fine
• we have the right people but TIABIM processes need
improvement
• we have the right people but many currently lack the
relevant TIABIM skills - build their capacity in these
and it will be fine (classics education?)
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Evidence-Based
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Making
BELIEF
SYNTHESIS
HTA
PROB
II
ABILITIES
SCDA
?
VALUE
SYNTHESIS
HTA
PREFER
II
ENCES
SCDA
TreatEff
TestPos
0.90
X
0.20
Test and Treat if Positive
TestPos
NotX
0.80
disuTreat=1 5
priorprobX=.2
pTestNegg iv enNotX= .9
PTestNegg iv enX=1-pTestP osgiven X
pTestP osgiven No tX=1 -p TestNeg givenNotX
pTestP osgiven X= .9
pTreatEffective= .7
uF H= 100
uX=1 5
TreatEff
X
0.70
0.20
Treat without testing
0.80
X
Neither Test nor Treat
0.20
83.00
TreatNotEff
0.30
NotX
NotX
0.80
TreatWorthless
0.10
TestNeg
0.90
Test and Treat if Positive : 89.81
79.90
Xremains
0.10
89.81
Choice in
possible case
of X
0.70
TreatNotEff
0.30
TestNeg
NoTreatment
85.00; P = 0.13
0.00; P = 0.05
15.00; P = 0.02
85.00; P = 0.08
100.00; P = 0.72
85.00
0.00
85.00
15.00
100.00
Archetypal decis ion 2
An otherw ise fully healthy patient presents with symptoms and s igns which lead you to suspect either X or Z. Z is of no
clinical s ignificance and the patient will fully recover very quickly with an hour's res t. We can therefore define Z as NotX.
X has serious cons equences if not treated quickly and successfully.
A tes t exists for X but it is not perfect either in terms of s ensitivity or s pecificity. We w ill assume it has no underiable
as pects (disutility) as a proces s.
A treatment exists for X but it is not certainly effective. Moreover it has some undesirable as pects (disutility) as a proces s.
But if treated effectively the patient will return immediately to full health (FH )
Evaluate the 3 options.
26
Jack Dowie
HTA & Pricing
Making
George Bernard Shaw The Doctor’s Dilemma
Sir Colenso Ridgeon has developed Opsonin, ‘works’ (?) but only when a
test is done and result correctly interpreted
Louis Dubedat : a dissolute artist and ‘bounder’ (needs opsonin)
Blenkinsop : an outstanding GP who has dedicated his life to the poor …
and so is poor (needs opsonin)
BB : a very successful consultant who has dedicated his life to the rich and
so is rich and doesn’t believe in testing
Jennifer Dubedat : the beautiful wife who Ridgeon falls in love with
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
The problem of the Single Criterion
• Most common single criteria are Life
Expectancy/survival/mortality; ‘natural outcomes’;
individual biomarkers; generalised ‘utility’…
• The QALY is a multi-attribute measure, bringing
together Life Years and HRQOL (the Quality
Adjustment)
George Bernard Shaw The Doctor’s Dilemma
Colenso Ridgeon has developed Opsonin, ‘works’ (?) but on
• And the Sir
HRQOL
measure is often based on a
test is done and result correctly interpreted
multi-dimensional
Euroqol
hasopsonin
5)
Louis Dubedat :instrument
a dissolute artist(e.g.
and ‘bounder’
(needs
Blenkinsop : an outstanding GP who has dedicated his life to th
• But it is and
stillsoused
a single
is pooras
(needs
opsonin)criterion
BB : a very successful consultant who has dedicated his life to
so is rich and doesn’t believe in testing
• Many other
considerations
are wife
notwho
‘QALY-able’
– w
Jennifer
Dubedat : the beautiful
Ridgeon falls in love
not incorporable into a ‘super QALY’
27
Evidence-Based
Sesja 1 / Session 1
Making
Patient-centred care
Note the position
of the apostrophe!
• Requires patient’s preferences
• If we use a population derived QALY tariff (set of weights
over the 5 dimensions of health) we are applying those of
mean or median patient
• But it is not just that the QA is not for this patient… there
are other considerations in their mind…
• ‘Post-process’ Considerations (e.g. bother of follow-ups)
• ‘In-Process’ Considerations (e.g. experience of surgery)
• ‘Pre-Process’ Considerations (e.g. anxiety before surgery)
• Hazen’s GALY (Goal Achieved Life Years) is a recent
attempt to add ‘extrinsic’ considerations to QALY (e.g.
desire to live to see child’s wedding)
• Brazier, Dixon, Ratcliffe (2009) try to rescue SCDA
• But it can’t deal with multiple considerations and certainly
not in a specific decision support role.
Conclusion re SCDA
• In context of either the clinical consultation OR an HTA the
necessary condition is that the stakeholder/s are able to
indicate the relative importance of multiple considerations
in real time with minimal complexity
• Requiring them to make numerous pair-wise comparisons or
process alternative vignettes are simply not practical,
despite the impressive normative arguments for them as
rigorous elicitation methods
• Visual analog scales (or their equivalent) for separate
attributes are the only serious contender
• SCDA is simply not a viable basis for a Clinical Decision
Support System or a Health Technology Assessment
28
Jack Dowie
HTA & Pricing
Making
MCDA - variations
• MCDA covers a multitude of different techniques
• Mainly differ in the way they address issues like:
• the way criteria are assessed
• the application and computation of weights
• the algorithm used to derive the overall ranking
• the model to describe individual preferences (compensatory
versus non-compensatory criteria, linear versus non-linear
preferences)
• the uncertainty embedded in the data
• the ability of stakeholders to participate in the process.
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
• Two emerge from my ‘MCDA’ quest in both contexts
• Analytic Hierarchy Process
• Annalisa
Baltussen and Niessen
• The more analytical approaches that have been developed
over the past decades offer little guidance to policy makers.
• Concentrate on single criteria such as effectiveness, costeffectiveness, burden/impact, equity
• If address more than one criterion don’t advise on how to
integrate or judge the relative importance of each - policy
makers need to make choices taking into account such
multiple criteria simultaneously.
• Moreover, often do not cover all criteria that are relevant to
policy makers (particularly ‘soft’ ones)
• In other disciplines MCDA (transport, agriculture, natural
resource management) is routinely used in similar problems
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Evidence-Based
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ANALYSIA
TIABIMIA
Jack Dowie
HTA & Pricing
Making
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Jack Dowie
Sesja 1 / Session 1
Health Care
31
Evidence-Based
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Making
Jack Dowie
HTA & Pricing
Making
van Til et al. 2008 p461
• “As was shown by Hummel et al. patients
value different aspects of treatment compared
with health professionals.”
[and compared with other patients]
• “The AHP model used in this study could be a
way to include the personal aims, wishes and
demands of each patient… different trade-offs
regarding criteria importance could lead to a
different preference for treatment.”
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Jack Dowie
Sesja 1 / Session 1
Health Care
But…
• “The time requirements to complete the model [7
hours] were considered a disadvantage,
• and the AHP was thought to be bothersome for
use in day-to-day decision making. This
reservation might be a result of the large size of
the current model. Simpler models might be
possible, although care must be taken to maintain
important details.”
Annalisa
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Evidence-Based
Sesja 1 / Session 1
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Annalisa
• An “intermediate” Decision Technology
• Sits on boundary between Tiabimia and Analysia
- a ‘boundary object’ in itself
• Makes possible optimal practical balancing
• intuition and analysis
• rigour and relevance
• complexity and practicality
• One screen picture of whole decision with
interactivity
• Equally useful in HTA and CDM – important if
link is ever to be established
34
Jack Dowie
HTA & Pricing
Making
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
Reprise…
Step 1
TIABIMIA to ANALYSIA
to increase analysis to intuition ratio
Step 2
SCDA to MCDA
to increase coverage of attributes / outcomes /
considerations not able to be synthesised into Single
Criterion
Step 3
AHP to Annalisa
to increase practicality, communicability…
35
Evidence-Based
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Contexts
•
•
•
•
Clinical
Public
Public-clinical regulatory, e.g. NICE, PBAC, …
NICE currently struggling to deal with its responsibility
to ‘take into account’ multiple considerations in addition
to clinical and cost-effectiveness
• Increasing emphasis on rigour of the Cost-effectiveness
but with a QALY definition of effectiveness that leaves
‘other considerations’ to be processed with almost no
rigour… e.g. terminality!
• Why not MCDA?
NICE attributes/considerations to be TIA
• This exemplar set is derived from Guide to Technology Appraisal and other
NICE documents, including appeals (e.g. that on Bortezomib). Clearly it would
be NICE’s task to come up with an appropriate set (as well as
organize/outsource ratings and weightings) for an MCDA.
• pClEff= probability that the NT is clinically effective relative to the
Comparator
• pCostE20k= probability that the NT is cost effective relative to the
Comparator at a WTP below £20,000 per QALY
• Acceptability/Appropriateness/Preferences [of patients and professionals]
• Terminality= End of Life Use
• Orph/NoAlt/Rescue= NT is 'orphan drug' OR has no alternative besides Best
Supportive Care OR is used in a 'Rule of Rescue' situation
• OtherEq= Other Equity considerations
• DHpriorities= clinical priority area as designated by Secretary of State for
Health and Welsh Assembly Government
• HSFeasability/Impact= Health Service Feasibility/Impact
• Innovatoriness
• WiderSocietalConsiderations
36
Jack Dowie
HTA & Pricing
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Jack Dowie
Sesja 1 / Session 1
Health Care
37
Evidence-Based
Sesja 1 / Session 1
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DREA
• HTA in the form of Cost Effective Analysis (CEA)
addresses the adoption decision: what is the best
intervention to undertake, given this set of options, their
Effectiveness, and a Willingness to Pay (WTP) Cost
threshold per incremental unit of effectiveness?
• When undertaken stochastically and extended to VOIA
(Value of Information Analysis) it simultaneously
addresses the research decision: what is the best uncertain
parameter to find out more about and how?
• In contrast, DREA addresses the decision decision - what
is the best way to choose between intervention (and
research) possibilities, given the alternative decision
processes available and a decision resource threshold per
unit of decision effectiveness?
• We can think of it as Value of Analysis Analysis
38
Jack Dowie
HTA & Pricing
Making
100
Minimum
Usefulness
Threshold
'Scientific'
rigour
(multi-attribute index
based on
key high-level
criteria of
(a) logical coherence
(b) empirical
correspondence)
CBDM
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Jack Dowie
Sesja 1 / Session 1
Health Care
CDA
AHP
Annalisa
CJ
0
0
Practical usefulness
100
(multi-attribute index based on key high-level criteria
of (a) social/individual fit and (b) time/resource constraints )
Choosing a D(S)T
• The meta-decision faced by all practitioners and patients
and public policy makers is
how should we decide?
• There is no such thing as a ‘good decision process’ (or
DT) in the abstract, only one that performs well on
specified attributes with specified weights
• So MCDA can provide the framework within which to
evaluate Decision Technologies (for a specified task in a
specific context)
• DREA suggests that sometimes (often? usually?)
Annalisa will be the optimal DT given the Willingness
to Decision Resource
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Evidence-Based
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Jack Dowie
HTA & Pricing
Making
Why do economists ignore opportunity cost?
• CEA/HTA1 (like most analytical techniques) continues to be
developed in pursuit of ever-greater theoretical/conceptual
normativity and empirical/statistical rigour.
• It is taken for granted (e.g. Drummond, Schwartz, Jönsson et al
2008) that this continuous raising of the ‘best practice’ level by
scientific standards is a good thing and it is occurring with
remarkably little interest in, or attention to, either the ability and
willingness of decision owners to resource best/good practice
decision processes of this kind, or the complexity of decision
owner’s maximand.
• Rex Brown has been one of the few people exploring the reasons
for this growing gap and for the consequent failure of these
advanced techniques to spread as widely as the professionals
involved have urged and expected.
• His primary explanation lies in ‘analyst mismotivation /
misalignment’, in the form of analysts’ pursuit of professional
advancement/standing, mental comfort and financial well-being which interact of course…
How close should ‘prescriptive’ be
to ‘normative’ – and how far from
‘descriptive’?
www.ceestahc.org
Jack Dowie
Sesja 1 / Session 1
Health Care
Normative
Prescriptive?
Normative
Prescriptive?
Prescriptive?
DE
Prescriptive?
Prescriptive?
Prescriptive?
Prescriptive?
Prescriptive?
Prescriptive?
Prescriptive?
Descriptive
Descriptive
Predominantly Intuitive DT
Predominantly Analytical DT
WTDR
X
WTDR
Y
WTDR
Z
DR
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Evidence-Based
Sesja 1 / Session 1
Making
rigour oughtis
• an affliction in which the setting of unrealistically or
impractically rigorous requirements on some criterion
or criteria leads to rejection of realistic /
practical means of improvement … and hence
maintenance of status quo
• often self-inflicted and cultivated as a defence of
existing interests / competencies / processes
• but sometimes (as in many judgment and decision
making situations) simply a reflection of misguided
belief that 'scientific rigour' does not have to be
incrementally traded-off with 'practical usefulness'.
•http://cafeannalisa.org.uk
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Jack Dowie
HTA & Pricing
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References
• http://knol.google.com for my knols
(key ‘dowie’ into search box)
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Jack Dowie
Sesja 1 / Session 1
Health Care
Brown (2006) ‘Making decision research useful – not just rewarding’ Judgment
and Decision Research 1 (2) 162-173
Brazier, Dixon and Ratcliffe (2009) ‘The role of patient preferences in CostEffectiveness Analysis’ Pharmacoeconomics 27 (9) 705-712
Drummond, Schwartz, Jönsson et al (2008) ‘Key principles for the improved
conduct of Health Technology Assessments for resource allocation decisions’
International Journal of Technology Assessment in Health Care 24 (3) 244-258
Hazen (2007) ‘Adding Extrinsic Goals to the Quality-Adjusted Life Year Model’
Decision Analysis 4 (1) 3-16
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Evidence-Based
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Jack Dowie
Decision Resource-Effectiveness Analysis
towards Value of Analysis Analysis
Decision Resource-Effectiveness Analysis (DREA) is proposed as the appropriate technique for evaluating alternative
ways of making decisions (Decision Technologies - DTs) and supporting decision makers (Decision Support Technologies
- DSTs).
Contents
Summary
Background
DREA
MAUT Formulation
MCDA Formulation
Relationship between DE and DR
Why do analysts ignore Opportunity Cost?
Conclusion
Summary
Decision Resource-Effectiveness Analysis (DREA) is proposed as the appropriate technique for evaluating alternative ways of making
decisions (Decision Technologies - DTs) and supporting decision makers (Decision Support Technologies - DSTs).
Cost-Effectiveness Analysis (CEA) addresses the adoption decision: What is the best intervention to undertake, given a set of options,
given their Effectiveness, and given a Willingness to Pay (WTP) Cost threshold per incremental unit of intervention effectiveness?
When undertaken stochastically and extended to Value of Information Analysis (VOIA) it simultaneously addresses the research
decision: which uncertain parameter is it best to find out more about, and how?
In contrast, Decision Resource Effectiveness Analysis addresses the decision decision - what is the best way to choose between
intervention (and research) possibilities, given the alternative decision processes/technologies available and given a decision resource
threshold per unit of decision effectiveness?
In many ways we can think of DREA as Value of Analysis Analysis (VOAA).
Background
CEA (like most other analytical techniques) continues to be developed in pursuit of ever-greater theoretical/conceptual normativity
and empirical/statistical rigour, checklists of best practice flourishing .[1] [8] It is taken for granted that this continuous raising of the
‘best practice’ level by scientific standards is unquestionably a good thing. It is occurring with remarkably little interest, in or
attention to, either the ability and willingness of decision owners to resource decision processes of this kind on the one hand, or the
complexity of decision owner’s actual maximand in relation to their decision making processes on the other. Rex Brown has been one
of the few people exploring the reasons for this growing gap and for the consequent failure of these advanced techniques to spread as
widely as the professionals involved have urged and expected. [2] His primary explanation lies in ‘analyst
mismotivation/misalignment’, in the form of analysts’ pursuit of professional advancement/standing, mental comfort and financial
well-being – considerations which interact of course, particularly in the long term. But their pursuit, to be successful, clearly depends
on co-operation from professional groups (which is fairly simply explained) and collaboration by commissioning bodies (which
requires a more complex explanation).
DREA
DREA provides a means of evaluating alternative DTs and DSTs, including various types, levels and qualities of Decision Analysis as
well as more intuitive methods, in terms of Decision Effectiveness (DE) and Decision Resources (DR). Both DE and DR are to be
determined in specific contexts and cases by the specific decision owners. We find the most useful way to characterise different DTs
and DSTs is in terms of Hammond’s Cognitive Continuum of varying Analysis-to-Intuition ratios.[3] A key question is whether DREA
is best implemented within a MAUT (Multi-Attribute Utility Theory) or MCDA (Multi-Criteria Decision Analysis) framework. We
explore them in turn.
MAUT Formulation
In this formulation DREA is thought of as a direct parallel to CEA within a MAUT framework. The options become alternative ways
of making and/or supporting decisions, the baseline comparator being the status quo process (‘standard decision making practice’).
Effectiveness becomes Decision Effectiveness (DE) and Decision Resources (DR) replaces Cost. (Some might prefer the term Decision
Quality to Decision Effectiveness, but we prefer to keep the verbal link to CEA.)
Both DE and DR are multi-attribute indexes where the attributes, their definitions and their weights are those of the decision owner.
Note immediately that the ways of determining all these will be part of the analysis.
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The attributes for DE will almost certainly include conventional outcomes for the intervention decision itself (e.g. mean health gain,
health gain distribution/equity), but will probably also include attributes of the decision process, such as the structural quality of the
modelling (if any), the quality (coherence and correspondence) of the evidential inputs, the coherence and validity of the preference
inputs, the equity of the decision process, the way uncertainty is dealt with, and so on.
The attributes for DR may include time availability (e.g. arising from a deadline), financial costs, cognitive demands on the parties,
organisational fit, etc.
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To repeat, it is up to the decision owner/s (or their agents) to determine both the DE and DR attributes (including their definitions) and
to assign weights to them. If a ‘generic’ matrix/tariff were to be sought (e.g. for allocation decisions in the NHS) this would need to be
agreed independently of any particular decision.
In this MAUT formulation of DREA, where a strict parallel to CEA is envisaged, the required unit of DE might be called the DEU
(Decision Effectiveness Unit) and the required unit of DR the DRU (Decision Resource Unit). The Incremental Cost Effectiveness
Ratio or ICER (Willingness to Pay per QALY) would become the Incremental Decision Resource Effectiveness Ratio or IDRER
(Willingness to Decision Resource per DEU).
The existence of a WTDR per DEU threshold means that translation to the Net Benefit formulation is simple, but we present the
analysis here only in the conventional form of the DRDE plane (Figure 1) - not least because this has the benefit of emphasising the
existence of DR-Effective DTs and DSTs falling in the South-West quadrant. [4] Circles (filled green in colour versions) indicate DTs
which are DR-Effective, triangles (filled red in colour versions) indicates ones which are not.
Figure 1: The Decision Resource Decision Effectiveness plane
MCDA Formulation
In this formulation DREA is implemented as Multi-Criteria Decision Analysis (MCDA). The variety of scales on which the attributes
for both DE and DR will be measured make the practicality of constructing a DEU and a DRU suspect and means that Multi-Criteria
Decision Analysis will be the more appropriate method for DREA.
An example constructed in the Annalisa implementation of MCDA will suffice to convey the basic ideas of implementing DREA in
this way. Screenshots with varying weights and results appear in Figures 2-4 below.
Besides Annalisa herself, the options are ‘standard decision practice (via committees for instance), the Analytic Hierarchy Process
version of MCDA, and two formulations of conventional MAUT DA. We omit a random choice process on this occasion though this
often would be a useful baseline, equivalent to doing nothing in the intervention decision.
The criteria/attributes in this purely illustrative example are as follows, those to be maximised constituting DE and those to be
minimised constituting DR:
Health gain mean (maximise)
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Health gain equity (maximise)
Coherence (maximise)
Correspondence (maximise)
Transparency (maximise)
Time (minimise)
Economic resources (minimise)
Cognitive demands (minimise)
Organisational change (minimise)
Political loss (minimise)
Since most of these techniques can be carried out at various levels of DR it is important to specify the ‘dose’ in the option description,
especially time and financial cost.
Figure 2: equal weights lead to Annalisa emerging as optimal
Figure 3: varied weights lead to Standard Decision Process emerging as optimal
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Figure 4: varied weights lead to Comprehensive Decision Model emerging as optimal
How is risk/uncertainty dealt with? Conventional sensitivity analysis can be simply carried out, but if one wishes to build an
risk/uncertainty parameter into the model it will be introduced as a separate attribute alongside the mean attribute, to be weighted in
relation to the other attributes (including the mean) in the normal way.[5]
Since the question ‘where does the necessary data come from?’ inevitably makes its appearance at this stage, particularly from
analysts, it is worth stressing that analysis does not create data ‘problems’ that did not exist prior to analysis. In the case of decision
analysis these problems, whatever form they take, have the same implications for all alternative DTs/DSTs, including intuition-based
ones. Analysis reveals and clarifies the problems, it does not create them.
Those who find themselves arguing that some part or parts of an actual analysis of this sort has not been done ‘well enough’ will have
failed to grasp the essential point - unless they explicitly claim that their judgment is being made in the light of the practical constraints
implied by the WTDR threshold.
Relationship between DE and DR
While the essence of DREA lies in the decision-specific nature of the DE and DR elements and their weightings, it is tempting to
speculate about the likely shape of the relationship between DE and DR for different DTs (or DSTs).
Let’s postulate a simple situation where there is a predominantly Intuitive DT of the sort that characterises much current clinical and
public decision making and a predominantly Analytical DT alternative. We assume that both can be implemented at varying levels of
DR.
Largely intuitively I suggest the relationship for the IDT will be of the shape portrayed by the circles in the figure below, that for the
ADT as portrayed in the squares. The IDT provides finite DE with almost no DR and its DE rises over a limited range of increased DR,
achieved for example by combining/aggregating across a set of individual intuitive (e.g. expert) judgments. But at some point it plateaus
and eventually declines because the IDT cannot cope with the increased demands put on it, despite - or perhaps because of - the
increased resources devoted to it.
On the other hand the ADT has no DE at all until a finite amount of DR is devoted to it, but from this point on it follows an s-shaped
curve of increasing marginal returns, inflexion point and decreasing marginal returns. One implication of this picture is that there is
some point on the DR axis at which IDT and ADT exhibit equal DE. But the practically optimal DT will be determined by the WTDR
per unit on the horizontal axis. It will be the ADT to the right of the crossover and the IDT to the left. The left end of the diagram
explains why ‘clinical judgment’ is often the optimal DT in medicine.
What we observe in practice is (I suggest) the suboptimal use of ADT relative to IDT, given WTDR.
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Figure 5: Speculative relationships between DE and DR for alternative DTs
In our diagram (Figure 5) implementation of current best practice by scientific standards (the highest square at top right) yields the
maximum possible DE, but requires a WTDR per DEU of Z. However, actual WTDR is only Y, which should in principle lead to the
adoption of an ADT with somewhat, but not massively, lower DE. However the derogation and dismissal by analysts of the use of
ADTs inferior to best practice by scientific standards leads, not to the adoption of this practically optimal ADT at WTDR Y, but of
the IDT of even lower DE at that particular WTDR. Note that the practically optimal ADT even outperforms the DE-maximising IDT,
which - as we have drawn it - is available to decision makers at the even lower WTDR of X and accordingly dominates that at WTDR
Y.
Why do analysts ignore Opportunity Cost?
Why this attitude by analysts? And why the acceptance of their views by decision makers, which leads in many cases (we propose) to
an inefficient flip from an ADT to an IDT?
Brown’s primary explanation covers the former question – there is simply little or no mileage, professional or monetary – for analysts
to suggest that ADTs of less DE may be DR-Effective. This is interesting, and not a little paradoxical, in that the development and
success of Health Economics as a discipline has been largely built on making the case for Cost-Effectiveness as the allocation decision
criterion, rather than Effectiveness, on the basis that it reflects the opportunity cost of the resources. Here we find the opportunity cost
of Decision Resources is being ignored. The Law of Disciplinary Myopia, which I launched – clearly unsuccessfully - in 1985, states
that each social scientific discipline ignores its central message in relation to its own activities.[6] Economists will rarely if ever be
found suggesting there are diminishing returns to economists or economic analysis, or that they each have an opportunity cost.
But why would decision makers go along with this? The acceptance of the analysts’ line by decision makers can be explained
cognitively to a small extent – they do not know how to question the ‘use only best practice by scientific standards’ argument. But,
more likely, it can be best explained motivationally. Decision makers (even when agents for the individuals or population, who are the
decision owners) realise that their power resides ultimately in being able to take decisions intuitively and hence far from transparently.
They can have the best of both worlds by commissioning an ADT to best practice by scientific standards, but then using it only as a
DST that they will ‘take into account and bear in mind’. By the way, these are the same group of powerful people who give the biggest
points in Research Assessment Exercises to the scientifically rigorous and least points to the practically useful.
Conclusion
It is useful to think of the ‘normative’ as that which should happen in an ideal world, the ‘prescriptive’ as that which could happen in
the ‘real world’ with ‘practical’ changes, and the ‘descriptive’ as that which does happen. The essence of the above argument can be
captured by saying that most pressures lead to the analysts’ prescriptive being attracted ever-closer to their normative and ever more
distant from the descriptive. While it may be flawed in many ways the latter has (by definition) one certain virtue, that of being
regarded as ‘practical’.
‘Bounded rationality’ and ‘satisficing’ were important sources of Herbert Simon’s Nobel Prize in Economics. But ‘unbounded
rationality’ and ‘maximising without decision resource constraints’ have always been straw propositions as far as the real world is
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concerned. ‘Bounded rationality’ and ‘satisficing’ are no more than maximising within constraints on DR. While at one point (p251) in
their recent paper [8] Drummond et al acknowledge the need to use HTA resources in a cost-effective manner this is not followed up
in any way , leaving Banta and Hailey as commentators to emphasise the need to 'get real' in terms of timing and resource demands
[9][10].
DREA has three main functions:
1. to provide a moderately analytical answer to the question of just how unbounded ‘rationality’ should be in any particular
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case;
2. to provide the framework within which the unavoidable debates and disputes surrounding DE and DR can be pursued more
openly and fruitfully than they are at present;
3. to establish that there are a variety of DTs and DSTs available and that the choice amongst them can and should be
explicitly addressed, rather than the existence of choice being denied or the choice being portrayed as a ‘no-brainer’. (Both
tactics will typically be favoured by power-maximising decision makers - as contrasted with decision owners.)
The relevance of DREA and ‘Value of Analysis Analysis’ is nowhere better illustrated than in the introduction by the National
Institute for Health and Clinical Excellence (NICE) of its STA (‘Single Technology Appraisal’) process alongside the ‘standard’
Multiple Technology Appraisal.[7] While of course it would not be expressed this way the STA can clearly be interpreted as a move
to the SW quadrant in the DRDE plane – being lower on Decision Effectiveness but also lower on Decision Resources by the likely
structure of these two multi-attribute indexes.
References
1. Philips, Z., et al., Good practice guidelines for decision-analytic modelling in health technology assessment: a review and consolidation of quality
of assessment. Pharmacoeconomics, 2006. 24: p. 355-371
2. Brown, R.V., Making decision research useful - not just rewarding. Judgment and Decision Making, 2006. 1(2): p. 162-173.
3. Hammond, K.R., Human Judgment and Social Policy: Irreducible Uncertainty, Inevitable Error, Unavoidable Injustice 1996, New York: Oxford
University Press
4. Dowie, J., Why cost-effectiveness should trump (clinical) effectiveness: the ethical economics of the South West quadrant. Health Economics,
2004. 13(5): p. 453-459
5. Millet, I. and W. Wedley, Modeling Risk and Uncertainty with the Analytic Hierarchy Process. Journal of Multi-Criteria Decision Analysis, 2002.
11: p. 97-107.
6. Dowie, J., Education and decision theory: a personal view, in Behavioural Decision-Making: Theory and Analysis, G. Wright, Editor. 1985,
Plenum: New York. p. 363-377
7. National Institute for Clinical Excellence, Guide to the single technology appraisal (STA) process, 2006, NICE: London
8. Drummond MF, Schwartz JS, Jonsson B, Luce BR, Neumann P, Siebert U and Sullivan SD Key principles for the improved conduct of health
technology assessments for resource allocation decisions International Journal of Technology Assessment in Health Care 24:3 2008 pp244-258
9. H David Banta, Commentary on ref 8, pp 362-368
10. David Hailey, Commentary on ref 8, pp365-366
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JUDEMAKIA: a map of the world of judgement and decision making
in health
...................................................................................................
Judemakia is the world of JUdgement and DEcision MAKing, in which the flanking continents of Belief-land and
Prefer-land supply the resources for the central continent of Decision-land. All three have latitudinal regions that
embody differing balances of Intuition and Analysis in line with Hammond's Cognitive Continuum theory. The resulting
map, focused on health, can help us picture the nature of differing Decision Technologies and gain insight into the
disputes surrounding them.
Contents
Page 1
1 Introduction
2 The continents of Judemakia – tasks and technologies
3 The regions of Judemakia - Analysis and Intuition
4 Decision Technologies
5 Tribal loyalties and triple identities
6 Bayesian Decision Analysts and non-Bayesian Belief Technologists
7 Why is TIABIM so (overly?) dominant?
8 Deciding how to decide with Annalisa
9 Conclusion
more
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1
Introduction
While some of the confusion in contemporary health/medical discourses is intentional, cultivated and fomented by interested parties (politicians, professionals,
providers, patients, public), much of it reflects limited awareness and understanding of fundamental conceptual distinctions and philosophical divides.
These need to be explicitly acknowledged and addressed at every point if debate surrounding clinical and public health decision and policy making is ever to have
even the potential to be genuinely open and inclusive. Of course, the conflicts and tensions to which these distinctions and divides give rise must be, and always
somehow are, implicitly resolved in the making of any decision or policy. But this provides no guarantee that ‘best’ course of action/policy has been identified, let
alone identified transparently and accountably. If this is genuinely our aim we need to address these underlying sources of tension and conflict independently of any
particular case, as well as in every particular one.
I suggest there are 5 ‘meta-distinctions and divides’ relevant to judgement and decision making in health/medicine and while they are all more or less well known
individually I think their nature, relationship and significance can be better appreciated if they are pictured. The picture is a map of Judemakia, the world of
JUdgment and DEcision MAKIng. One of the five distinctions generates its continents (which vary in longitude), a second generates the regions within each
continent (which vary in latitude). The other three are best thought of as the basis of tribal loyalties and multidimensional identities among the inhabitants. We are
all Judemakians in all aspects of our life – work, rest and play.
2
The continents of Judemakia – tasks and technologies
We can summarise the relevant tasks and technologies in judgement and decision making as follows:
- To produce beliefs, to elicit beliefs and to evaluate belief claims we need Belief Technologies
(BTs), e.g. ‘clinical judgement (diagnostic)’, cohort study, RCT, lab test, autopsy. Non-Bayesian
Decision Theorists, and BDTs being diplomatic, will use the word ‘knowledge’ or ‘evidence’
rather than ‘belief/s’
- To establish what preferences are held and to elicit preference judgements we need Preference Technologies (PTs), e.g. ‘clinical judgement (of patient
preferences)’, interview, Standard Gamble, Time Trade-Off utility elicitation exercise, revealed preference study, discrete choice experiment. Non-Bayesian Decision
Theorists, and BDTs being diplomatic, will prefer to use the word ‘value/s’ rather than ‘preference/s’
- To make decisions (including forming policies) we need Decision Technologies (DTs) e.g.
‘clinical judgement’, coin toss, meeting, pro-con checklist with decision rule, Decision Analysis
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- Any application of any DT requires inputs from one or more BTs AND one or more PTs
- Any application of either a BT or a PT requires outputs from a DT (N.B.)
- The transfer of inputs within and between BTs, PTs and DTs requires Information and Communication Technologies (ICTs) e.g. nudge/wink and other
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body language, PowerPoint presentation, Report with tables and graphs
Mapping this in Figure 1 we can see that Judemakia is made up of 3 continents, with the supporting provinces of Belief-Land and Prefer-Land flanking the central
republic of Decision-Land. It will be evident that this is a decisio-centric mapping. To those who hold to either scio-centric or value-centric conceptions of the world
this will be heresy a la Copernicus. So be it.
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Figure 1
As we move ‘north’ the flanking continents first increasingly diverge from D-land and then, after the
equator, get closer and closer to it again. To explain this divergence and the difficulties for ICTs it
creates, as well explaining the internal ‘land use’ patterning that is obvious within each continent on this
map, we need to introduce the second meta-distinction.
Page 3
3 The regions of Judemakia - Analysis and Intuition
All the above tasks can be - and are in practice - implemented by technologies located throughout the Cognitive Continuum of changing Analysis-to-Intuition (A-I)
ratios. Very briefly, Cognitive Continuum theory [1] suggests that we have two basic types of cognition available to us - analysis and intuition. Contrary to those who
see these as binary and exclusive, Hammond suggests that we think of them as being combined, and combinable, in different ratios along a continuum running from
highly intuitive at one extreme to highly analytical at the other. While the continuum is indeed a continuum, broad ranges can be conceptualised as relatively
distinct modes of cognition and their boundaries have
received considerable reinforcement through social constructions (e.g. academic disciplines). Six modes seem sufficient to capture and locate the main types of
inquiry and practice but I have added ‘Instinct’ to the Continuum, without commitment to it being ‘cognitive’. (Figure 2).
This framework has obvious links with dual system (System1/System 2) theory, as well as other theories that distinguish between Implicit and Explicit processes,
but I will not pursue these here, beyond suggesting that the mapping is consistent with these alternatives if they abandon their binary character.
It is vital to emphasise at this point that there is no implication that the higher the A-I ratio the better - or the reverse – so neither the numbering scheme nor the
orientation of the map have any significance (as is confirmed by the illustrative inversion in the third part of this diagram).
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Figure 2
Broadly speaking, as we increase the A-I ratio, the definition of concepts, the specification of
relationships and the measurement of magnitudes becomes more explicit and precise—and ‘transparent’ in current parlance (Figure 3). In parallel, various
requirements change (Figure 4).
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Figure 3
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Figure 4
Not represented in these 2-D graphics is the quality dimension. Instantiations of both analysis and
intuition in any Technology may vary in quality and (if it helps) we can think of this as variation in
altitude.
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We are now in a position to identify and explain the provisional names for the regions that make up each of the continents (Figure 1). It is to be stressed that the
names have been chosen to represent the distinctive instantiations that occur in the region concerned rather than (necessarily) its typical ones. Examples of
inhabitants of the various regions are provided in Figure 5.
Figure 5
Of course, a mind is only in a region while specifically ‘in role’, and individual minds will move around the whole of Judemakia from moment to moment as they
utilize the BTs, PTs and DTs that are necessary to accomplish their task. For example, in order to design, perform or interpret a trial a ‘trialist’will have to make
preference judgements, and decisions, by definition outside Randomistan (sometimes called Cochrania).
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Numerous border and boundary disputes characterise life in Judemakia and two of the most familiar and vicious are those which focus on the line of latitude
separating Randomistan and Epidemia (the observational-interventional dispute) and that separating Modelia and Conferalot (the qualitative-quantitative dispute).
Note that these major border disputes cross all three continents, not just Belief-Land, though interestingly the same individual may typically be found on different
sides of the border on different continents (e.g. someone who is predominantly a modeller in Belief-land may be predominantly a moralist in Prefer-land and
predominantly a ‘TIABIMer’ in Decision-land). Both these border disputes and all of the many other ones within Judemakia are better understood when interpreted
as manifestations of the three other meta-divides, to which we turn – but only after establishing the character of the main regions of D-land, the place where
evidence and values (beliefs and preferences) must be integrated. In other words, where evidence-based medicine and values-based medicine and public health
must become simply the best medicine and public health.
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4 Decision Technologies
In our sort of society five broad types of DT are recognised in health decision making
• Instinct
“I was compelled to do it, the emotional drive was so powerful”
“I acted instinctively, without thinking”
• Intuition
“I simply felt I could trust him/her/them and followed their recommendation”
“After all that experience I recognised the pattern instantly and knew what to do”
• TIABIM - Verbal reasoning
“We Took Into Account and Bore In Mind all relevant considerations and made our
decision”
“I examined all the pros and cons in a balanced way and arrived at the best option”
• Analytical Decision Making
“ We carried out a cost-effectiveness analysis and implemented the action which
emerged as optimal”
• Algorithm
“I followed the flow chart/guidelines.”
The two more extremely analytical DTs to the ‘north’ of Algorithmia are conceptually possible (and real world examples do exist, especially in the mental health
field), but they represent increasingly de-humanised and ethically unacceptable modes for most of us.
The currently dominant DTs in both the clinical and public health contexts are various instantiations of Intuition and TIABIM.
We know little about what goes on in Intuition as a DT - recall that this is defined as a very low A-I ratio, not as ‘pure’ intuition. We do know that many health
providers and consumers believe and trust fervently in it, especially in ‘clinical’ contexts. However, since it cannot meet the transparency criteria for either shared
clinical decision making or accountable public health decision making, we leave it aside here as a DT. (Much of the most interesting and relevant work concerns the
importance of intuition in B-Land and P-land, where the Lens Model of Egon Brunswik has provided the basis for modelling the intuitive processing of ‘multiple
fallible indicators’ by human judges as Belief or Preference inputs into their selected DT. The intuitive processing of signs and symptoms into ‘diagnostic
possibilities’ is a classic example in the former case, the processing of verbal and non-verbal cues into ‘patient preferences’ the equivalent in the latter.)
Page 7
TIABIM, the DT mode with the second lowest A-I ratio, is verbal discursive reasoning and
argumentation that seeks to ‘Take Into Account and Bear In Mind’ all relevant considerations. Group versions of TIABIM discourse (such as most decision making
meetings) are peppered with assertions by the participants that they are
• ‘Taking things into account’
• ‘Giving considerations due weight’
• ‘Establishing the right balance’
• ‘Keeping things in proportion’
• ‘Taking a measured view’
• ‘Factoring everything into the equation’
• ‘Figuring it out’
• ‘Seeking a degree of consensus’
• ‘Gauging the impact’
• ‘Making sure things add up’
It is noteworthy that the italicised words suggest some sort of quantification is going on. This may well be occurring in intuitive form, but, if so, it is not very precise
or explicit, usually taking the form of ‘verbal quantifications’ such as ‘very likely’ and ‘good chance’ in Judgia or ‘paramount’ and ‘excessive’ in Ethicodia. No
equation actually ever provides the structure of a TIABIM discussion, even if some equations are ‘taken into account’ as inputs from more analytical BTs.
The magnitude of the task faced in TIABIMIA – and indeed of making decisions in general - is clearly huge, now we have it pictured in this way. As we can see in
Figure 6 TIABIM decision makers must take into account and bear in mind the outputs of BTs spanning the entire range of the A-I continuum. In the figure the
thickness of the lines linking the regions of B-land to TIABIMIA indicate what the present author sees as the observed preferences of TIABIMers for different
‘outsources’. Inputs from either end of the BT continuum are preferred on the basis that they offer certainty based on scientific authority in the north (hence
‘Evidence-based practice’) and certainty based on expert authority (‘Expertise-based practice’) in the south. There is manifest aversion to the outputs from
modelling in the middle, probably because this tends to maximise the exposure of our uncertainties. There is equivalent aversion to the elicitation and modelling of
preferences (values) in Elicitia, probably because this tends to maximise the exposure of our value conflicts (intra-personal as well as inter-personal). So the
equatorial provinces of Modelia and Elicitia are a little too hot and uncomfortable for many people! And we will find the same for mving decisions to Analysia
shortly.
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Figure 6
ICTs attempting to aid and facilitate the TIABIM process, especially transfers of highly analytical
research findings from the most northern regions of B-Land, face an almost impossible task, especially when the inputs come in classical statistical, non-Bayesian
form and therefore require translation for decision making (Dowie 2006). Their task is not helped by requests from TIABIMers that the specialist language used –
necessarily- at higher A-I ratios be ‘de-jargonised’. Usually, to be blunt, ‘dumbed-down’.
It will be noted (through the use of dashed lines) that the TIABIMing of inputs from P-land(‘values’) is carried out in much more fragmented and less rigorous ways
than those from B-land, being done largely intuitively and currently with little reference to (or funding of!) inputs from Elicitia. ‘Values-based practice’ is only
beginning to be spoken of in the same terms as its ‘Evidence-based’ equivalent and the key question here is which provincial source will be accorded greatest weight
in its promotion - Moralia, Ethicodia or Elicitia? If it is either of the former two the task in D-land will be integrating evidence from
the ‘far north’ with values from the ‘deep south’! The ‘hierarchy of evidence’ in D-land would be turned upside down in P-land.
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The diagram makes clear that the integration of Beliefs/Evidence and Preferences/Values in TIABIMIA
or indeed in decision making in general is an amazingly challenging task, given the range of A-I ratios
involved in supplying the inputs. Our key suggestion is that the information and communication
difficulties of accessing these inputs and integrating them in transparent and coherent fashion will often
– and much more often than is currently the case - be best achieved through a more analytic DT, located
in equatorial Analysia and implemented in some form of Decision Analysis.
Decision Analysis (Figure 7) is prescriptively concerned equally with Belief (Evidence) and (Value)
Preference inputs, insisting that they be synthesised separately to avoid cross-contamination in either
direction and that these syntheses to be performed at the same analytical level as itself operates as a DT.
Insisting that the separate syntheses and their subsequent integration all occur at the equator – the middle
A-I balance - in all 3 continents, minimizes the difficulty of intra- and inter-continental communication.
Minimises, not eliminates, since it is an essential implication of the cognitive continuum that
communication difficulties are inherent and impossible to eliminate entirely.
Figure 7
Analytical Decision Making (ADM) is interpreted here to embrace any procedure which either consists
of, or concludes with, the integration by a formally specified rule of separately quantified assessments of
beliefs and preferences, the integration producing a score for each option. Decision Analysis (including
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Cost-Effectiveness and Cost-Utility analysis) and Multi-Criteria Decision Analysis are the main forms.
5
Tribal loyalties and triple identities
As with any instantiation of any type of technology, DA has a multidimensional identity, reflecting the
position it takes on the three meta-divides that creates the tribal loyalties and divisions of Judemakia: (i)
on the nature of beliefs/knowledge, (ii) on the nature of goodness, and (iii) on the relative importance of
coherence and correspondence. It is important to stress that all implementations of all 3 types of
technologies – BT, PT or DT- necessarily have triple bases for their identities, reflecting the adoption of
positions on these divides, even though some seek to deny this and/or deny the need to admit and expose
their positions if an effective discourse is to occur.
On this occasion we shall restrict ourselves to establishing the triple bases of the identity of DA, leaving
the others to be inferred by comparison and contrast.
•
DA is fundamentally Bayesian in relation to the nature of empirical belief, where what is called
by most non-Bayesians ‘knowledge’ is that special case where a widely (intersubjectively)
agreed probability distribution is tight to the point of consisting only of the central point. To
Bayesians the task in B-land is to establish uncertain beliefs and hence probabilities, rather than
knowledge (which is seen as the special case where p=1). In a weaker form of this position the
DAist would allows non-Bayesian conceptions of knowledge to persist in B-land for BT
purposes (e.g. frequentist statistical methods that generate p values and/or effect sizes with
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confidence intervals) - so long as it is accepted that all DTs are inherently Bayesian, insofar as
they require probabilities that can only be ‘subjective’ since they relate to future and usually
unique events. The alternative DTs are hence Bayesian DA, Bayesian TIABIM and Bayesian
Intuition. (There is increasing evidence from neuroscience that our instincts are Bayes-based,
which would be no surprise to Bayesians.) This does not means that a Bayesian has no place for
frequencies, in fact the very opposite. Where they can be constructed, necessarily by subjective
specification of numerator and denominator, frequencies may well provide the best bases for
arriving at subjective probabilities. (To the Bayesian the adjective ‘subjective’ is, of course,
redundant). [2]
• DA is fundamentally consequentialist in relation to the nature of the good and the right. If we
crudely classify ethical positions into the absolutist-deontological (one does good by doing right)
and the consequentialist-utilitarian (one does right by doing good) DA adheres to the latter in
rejecting absolutist views as to options. Rejection of an option is achieved by assigning infinite
disutility to its consequences. But DA accepts that rights and duty considerations will determine
whose utilities are to be ‘taken into the count’ and with what weight. [3]
• DA privileges coherence over correspondence, but maximises correspondence within a
coherent framework. Illustrating the difference with a simple example, (internal) coherence
requires that one’s prior odds on a hypothesis be revised in the light of the likelihood ratio
[TPR/FPR] for new evidence by using Bayes theorem, while (external) correspondence requires
the three component probabilities to be empirically accurate (in relation to some
intersubjectively agreed frequency). Hammond argues convincingly that many of the major
conflicts and controversies in the social and other sciences can be viewed as conflicts between
coherence and correspondence theories of truth. In the Judgement and Decision Making (JDM)
field the heuristics and biases literature is based largely on internal coherence tests (e.g. was
Bayes theorem properly observed in revising a diagnosis in the light of new evidence?), while
the calibration and discrimination literature relies on external correspondence tests (e.g. did the
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patient survive on 70% of the set of occasions when the clinician gave the patient a 70% chance
of surviving?). While Hammond does not advance his argument separately in relation to B-land
and P-land it is important to do so. We can obviously test a set of values/preferences as well as
probabilities for their coherence (e.g. their transitivity). But while the application of the
correspondence test to probabilities is fairly uncontroversial (assuming we can agree on whether
the patient survived in the above example), its application to values/preferences is decidedly not.
Apart from those who are certain there is one correct set of values (which obviously they
possess) all we can do in the case of values is ask is how well somebody’s set of values
corresponds to somebody else’s set.
6 Bayesian Decision Analysts and non-Bayesian Belief Technologists
Why are non-Bayesian Belief Technologists (scientists, triallists, epidemiologists) not prepared to produce and offer their probabilities for the
unique events needed by decision makers using any DT?
Summarising what I see as the main reasons:
• some forget all that decisions need Preference inputs as well as Belief ones – and are
encouraged to do so by TIABIM politicians who wish to talk of ‘science or evidencebased policies’ rather than ‘science or evidence-informed’ ones [6]
• most assume that TIABIM decision makers have the necessary cognitive competences to
transform outputs from a scientific/frequentist paradigm into decision-relevant input
parameters (an assumption naturally encouraged by TIABIM decision makers!)
• most refuse to accept probabilities are always ultimately ‘subjective’ degrees of belief,
even if based on the construction of empirical (‘objective’) frequencies
• most refuse to talk of the probability of alternative hypotheses, given some evidence
(only of the probability of this evidence, given alternative hypotheses, as they have been
taught in mainstream stats courses)
These refusals means that decision makers have to transform what they are offered (e.g. effect sizes
with Confidence Intervals) into the probabilities needed for the decision. This transformation is done
covertly and/or unwittingly when they engage in Bayesian Decision Intuition (with the threats to
quality, transparency and accountability BDI involves), but has to be done explicitly and openly
when they (or their agents) engage in Bayesian Decision Analysis.
Many Decision Analysts would see little point in being Bayesian if they were not Decision Analysts,
while fully accepting that in order to be Decision Analysts they must be Bayesians. The key
difference is not between Bayesian and non-Bayesian approaches to statistical inference but between
the Decision Analytic and conventional approaches to decision making; conventional approaches
that, whatever their surface appearance, can all be characterised as some form of BDI (either simple
Intuition or TIABIM). The statistical conflict is important only because it is a major source of
quality problems in BDI as a DT, through the cognitive burden it imposes – albeit often unrealised on decision makers, as they struggle with the task of transforming the decision-irrelevant format of
mainstream scientific output (e.g. effect sizes from RCTs, or test sensitivity) into what they need
(probability of effect, predictive value positive of test). This cognitive burden is typically reduced in
BDI (Intuition and TIABIM) by the use of various inappropriate heuristics (e.g. stereotyping) and
unwitting misinterpretations (e.g. of p-values and Confidence Intervals)
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7
Why is TIABIM so (overly?) dominant?
Again we summarise briefly the various positions
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• TIABIM is fine, it has no general, major problems
• TIABIM is not perfect, it does produce lots of bad/poor decisions, but this is because
• the wrong people with the wrong values dominate – bring in the true/right ‘stakeholders’
and it will be fine
OR
• we have the right people with the right values, but they lack
knowledge/information/evidence – supply them with more and better and it will be fine
OR
• we have the right people but our TIABIM processes need improvement/refinement (better
rooms, room arrangements, chairing…)
OR
• we have the right people but many currently lack the relevant TIABIM skills - build their
capacity in these and all will be fine
www.ceestahc.org
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While it has many advantages the great potential disadvantages of TIABIM are that it is powerstructure friendly, hindsight-friendly and cognitive competence-friendly - because
• it does not require clear and explicit separation of beliefs and preferences
• it does not require explicit and precise statement of anyone’s beliefs (in form of
probabilities) and hence provides no serious basis for assessing their quality (using either
coherence or correspondence, or both, as the quality criteria)
• it does not require clear explicit and precise statement of anyone’s values as quantified
preferences and hence provides no serious basis for assessing their quality (using either
coherence or correspondence, or both, as the quality criteria)
One observes formidable reluctance and hostility to move to the middle regions of the continua – to
Analysia and its flanking supporting provinces of Modelia and Elicitia – which represent the most equal
balancing of analysis and intuition. Why this reluctance? Following an earlier hint, we suggest that,
apart from being power structure-friendly, it is because in the middle of all three continents one actually
maximises what psychologically and socio-psychologically most of us don’t want to know or accept. In
the middle of B-land we maximise uncertainty by exposing all its sources as completely as possible and
insisting that all the uncertainties be dealt with explicitly, transparently and quantitatively, rather than
denied or dealt with implicitly, covertly and qualitatively, as happens further south. Equally, in the
middle of P-land we maximise the extent to which we are confronted by the existence of incoherent
values within individuals and groups and value differences and conflicts between individuals and groups
(such as over uncertainty preferences and time discount rates). In the middle we are denied our denials
and exposed to the affective and emotional consequences of this loss.
But maybe the resistance, reluctance or hostility is not only – or even mainly – psychologically and
affectively motivated in this way. Maybe there are more practical reasons, including ignorance. Maybe ,
metaphorically, it’s not a question of not wanting to go to Analysia, but not knowing it exists, or not
knowing how to get there, or, having found out how to get there, of deciding the journey is too difficult
or expensive to make. I suggest we will only understand more about the relative importance of
‘motivated’ and ‘practical’ reasons through focusing on the meta-decision : deciding how to decide. In
other words focusing on comparative evaluation of alternative Decision Technologies. (This question
has been asked quite often recently, but only in the context of organizational arrangements and
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management styles. Here we are asking it in terms of Analysis-Intuition ratios, though of course these
have implications for organisation and management arrangements.)
There is only time and space here (practical considerations!) to adumbrate the argument and introduce
Annalisa as the test instrument.
8
Deciding how to decide with Annalisa
Let us take the overall goal to be identifying the best (i.e. most appropriate) Decision Technology for
this specified decision task. We suggest that this requires us to
• establish a set of attributes or criteria relevant to the appropriateness of a DT
• rate the DT options on each of these attributes/criteria
• weight the attributes/criteria in relation to each other
• integrate the ratings and weightings to produce a score for each DT option.
For present purposes we will suggest that the criteria relevant to the appropriateness of a DT may be
grouped under the high-level headings of ‘Scientific Rigour’ and ‘Practical Usefulness’, each of which
has two main sub-attributes. Scientific Rigour embraces Coherence and Correspondence, while Practical
Usefulness embraces Resource Demands (including time) and Institutional/Individual Fit (including
cognitive demands)
We can are now in a position to apply a multi-criteria decision analytic technique to the meta-decision
task, or rather to apply a specific implementation of MCDA, since the achievements in respect of
Scientific Rigour and Practical Usefulness will be determined by the specific way in which MCDA is
actually implemented in situ. These ways can range all the way from a 5 minute ‘back of the envelope’
implementation (becoming less practical as email reduces the availability of the key resource) to
employing highly sophisticated software such as Expert Choice to implement the Analytic Hierarchy
Process and use it to its fullest possible extent, therefore taking many days/weeks and considerable
amounts of resources [4] . The following 3 screen-grabs are from an ‘intermediate’
implementation of MCDA called Annalisa, a piece of software I developed specifically with the rigourpracticality trade-off of clinical medicine (and equivalent situations) in mind. The ratings and weightings
are personal conjectures (coming from the more southern regions of B-land and P-land) but will suffice
to show that the most appropriate way to decide is dependent on the weight attached to criteria and how
options rate on those criteria. This is intuitively obvious to most people in the abstract, but what is not
obvious is that failing to address the necessary trade-offs explicitly and openly will lead to inferior
decisions by most reasonable sets of weightings.
When the two Rigour criteria are given 50% weight each and Practicality is zero weighted it is the DTs
with higher A-I ratios which score best. The reverse applies when Fit and Resource demands are given
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50% weight each and Rigour is zero weighted. And Annalisa confirms – semi-analytically - the intuitive
conclusion that it will be the implementations which have the most equal balance of Analysis and
Intuition that emerge best when all four criteria are given equal weight.
Where is chez Annalisa? Immediately adjacent to the border with Tiabimia, so that that the difficulties
of border crossing – in both directions - are minimized.
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Figure 8 Rigour 100% weighted, Practicality zero weighted
Figure 9 Rigour zero weighted, Fit and Resource demands 100% weighted
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Figure 10 Rigour and practicality equally weighted
9
Conclusion
Both Judemakia (the map tool) and Annalisa (the software tool) can be thought of as ‘boundary objects’.
Boundary objects are those objects that inhabit several ‘Communities of Practice’ and are plastic enough
to adapt to local needs and constraints within each Community of Practice, but robust enough to
maintain a common identity across a ‘Community of Interest’ that embraces a number of Community of
Practices. They are weakly structured in common use when doing ‘boundary work’ within a Community
of Interest (e.g. all parties concerned with bowel cancer) and become strongly structured in use within
individual Communities of Practice (e.g. colorectal surgeons). [5] Boundary
objects may be things, ideas, processes, organisations, people, words, stories, diagrams, cartoons,
jokes... They may be maps. They may be metaphors. And they may be metaphorical maps. So I think of
Judemakia as a boundary object which is seeking to cross the boundaries of Communities of Practice
and Communities of Interest in health - and well beyond health, you will realise if you visit http://www.cafeannalisa.org.uk
- while self-consciously focusing on those boundaries. Which makes it a meta-boundary object, if you like.
Will it do its boundary work successfully? Boundaries/borders exist for many reasons, including
quality control within Communities of Practice (e.g. ‘scientific standards’, disciplinary methods), the
maintenance of material interests within a Community of Practice (e.g. income, prestige, power) and the
promotion of particular values within a Community of Interest (e.g. regarding the environment, a
culture, patients). Effective boundary objects must both (i) bring the diverse stakeholders within a
Community of Interest to the table and (ii) keep them there until an ‘acceptable’ resolution or common
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understanding is reached. Many boundary objects are very good at the first (e.g. words like ‘risk’ and
‘sustainable’) but do much less well, or fail disastrously, at the second. We will await the answer in
relation to Judemakia - and Annalisa - with obvious interest.
References
1. Hammond K (1996) Human Judgment and Social Policy: Irreducible Uncertainty, Inevitable Error, Unavoidable Injustice. New York, Oxford
University Press
2. Dowie J (2006) The Bayesian approach to decision making. in Killoran A, Swann C and Kelly M (eds.) Public Health Evidence: Tackling Health
Inequalities Oxford, University Press, 309-321
3. Dowie J (2007) Decision analysis: the ethical approach to most health decision making. in Ashcroft R, Dawson A, Draper H and McMillan J (eds.)
Principles of Health Care Ethics, 2nd edition Chichester, Wiley
4. Forman G and Selly M (2001) Decision by Objectives. World Scientific Press (download free from http://mdm.gwu.edu/forman)
5. Star, S and Griesemer, J (1989) Institutional ecology, 'translations' and boundary objects: amateurs and professionals in Berkeley's Museum of
Vertebrate Zoology, 1907-39 Social Studies of Science 19, 387-420
6. Dowie J (2004) Research implications of science-informed, value-based decision making. International Journal of Occupational Medicine and
Environmental Health 15: 83-90
Comments
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The future of HTA is MCDA
The future of Health Technology Assessment lies in the use of Multi-Criteria Decision Analysis
Contents
Introduction
The role of the 'other considerations'
The problem and the solutions
The Example
Attributes
Options
Weightings
Comment
The meta-decision
Attributes
less
Introduction
Health Technology Assessments are being used around the world to support regulatory bodies making decisions relevant to the
operation of health services. Among the most well-known of these bodies is the National Institute for Health and Clinical Excellence
(NICE) charged with appraising new technologies to determine whether the National Health Service (NHS) of England and Wales
should reimburse health authorities for expenditure on them.
The NICE Appraisal Committee is supplied with HTAs undertaken via the National Institute for Health Research. These HTAs are
made up of separate Clinical Effectiveness and Cost-Effectiveness Analyses, conducted within the fundamentally different paradigms
of Classical Meta-Analysis and Bayesian Multi-Attribute Utility Theory (MAUT) respectively.
Apart from dealing with these conceptually distinct products the Committee is also required to 'take into account' various
considerations. No formal procedure currently exists for the ‘taking into account’ of these ‘considerations’. They are dealt with by the
application of the committee's judgement and discretion to the limited amount of relevant 'evidence' brought to bear during the
meeting.
Since these considerations may increase the opportunity cost of the Committee’s decisions by 50% or more it is time to address them
more analytically. Multi Criteria Decision Analysis provides the obvious route.
The role of the 'other considerations'
While formally relevant in all Appraisals the 'other considerations' play an important role only when there is a question of raising the
Willingness to Pay (WTP) for an Incremental Quality Adjusted Life Year (QALY) above the normal 'threshold' of £20,000 - up to
£30,000 as a 'normal' maximum, or, 'exceptionally', beyond this figure (It is repeatedly stated that there is no official limit.) The current
suggestion that the Committee operates on a ‘range’ is actually conceptually inappropriate and misleading, given the previous wording
is an accurate representation of the position, and should be dropped.
The problem and the solutions
While these ‘other factors’ can increase the opportunity cost of approval by 50% or more (i.e. from £20,000 to £30,000 … or more),
the analytical level at which they are considered and justified contrasts starkly with that of the assessments. If their treatment to be
raised to a more credible and transparent level NICE has two broad options. One is to derive and apply a standard WTP tariff
adjustment for each such consideration and so (e.g.) bring a £33,000 ICER down to £28,000 if a consideration such as 'innovatory'
were to be tariff-rated at £5,000. The other is to move to Multi-Criteria Decision Analysis as the basis for Appraisal.
The exemplar ‘Annalisa’ developed and reported below illustrates the way MCDA could be used. The Annalisa implementation of
MCDA is obtainable at http://www.annalisa.org.uk and the exemplar file can be downloaded from http://www.cafeannalisa.org.uk.
[Go to the front page of the latter to get the basic ‘How to’ guide.] More complex MCDA programs, such as Hi-View and Expert
Choice (the latter implementing the Analytic Hierarchy Process version of MCDA), are also available.
The Example
Attributes
This exemplar set is derived from Guide to Methods of Technology Appraisal and other NICE documents, including appeals (e.g.
that on Bortezomib ). Obviously it would be NICE’s task to come up with an appropriate set (as well as organize/outsource ratings
and weightings).
pClEff= probability that the NT is clinically effective relative to the Comparator
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pCostE20k= probability that the NT is cost effective relative to the Comparator at a WTP below £20,000 per QALY
Acceptability/Appropriateness/Preferences [of patients and professionals]
Terminality= End of Life Use
Orph/NoAlt/Rescue= NT is 'orphan drug' OR has no alternative besides Best Supportive Care OR is used in a 'Rule of Rescue'
situation
OtherEq= Other Equity considerations
DHpriorities= clinical priority area as designated by Secretary of State for Health and Welsh Assembly Government
HSFeasability/Impact
Innovatoriness
WiderSocietalConsiderations
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Options
Approve New Technology
Confirm Comparator Technology
Ratings
Belief judgements,all assumed for this example. The pCostE20k rating would normally be available from the Assessment Report’s
Cost-Effectiveness Acceptability Curve.
Weightings
Straightforward value judgements in this example. Giving less weight to Cost-Effectiveness and greater weight to some 'other
considerations' flips the recommendation between Figures 1 and 2 below. Many patient and professional groups explicitly or implicitly
argue that Cost-Effectiveness should be given little or no weight in their case. Giving Clinical Effectiveness great weight and
Cost-Effectiveness very little will usually favour the New Technology, as in Figure 3, even without 'other considerations' being taken
into account.
We can note that the Chairman of the Bortezomib Appeal Panel recently denied that the Appraisal Committee had given
Cost-Effectiveness 'unreasonable weight', thereby implying that there is a 'reasonable weight'.
Comment
The Methods Guide is currently under review, but this appears to be focusing on the already sophisticated Assessment inputs into the
Appraisal, rather than the processing of the other considerations – which as already noted can increase the opportunity cost of
approval by 50% or more.
Figure 1
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Figure 2
Figure 3
The meta-decision
The choice among MCDA implementations and softwares is itself appropriately addressed through a MCDA. In such an analysis the
specification, rating and weighting of attributes relevant to effective communication among all stakeholders will almost certainly favour
less complex implementations, such as Annalisa, where scientific rigour can be explicitly traded off, as it must often be, with practical
usefulness...rather than this trading off being done implicitly and covertly, as it usually is. So while it is of relevance to note that (e.g.)
Annalisa involves a linear additive expectational model, it is irrelevant to go on to imply that this has any significance outside the
context of an MCDA in which model structure is one attribute of the different implementations being evaluated.
For further development of the meta-decision issue see my paper on ‘Decision Resource Effectiveness Analysis: towards Value of
Analysis Analysis’.
Figures 4 and 5 provide an exemplar MCDA for choice of Decision Technology.The attributes, their ratings and weightings are
obviously illustrative (though reflecting some personal inclinations).
Attributes
Time Demands
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Cost
Cognitive Demands
Transparency/Clarity
Separation of Belief and Value Judgments
Handling of Complexity
Transparency/Clarity
Produces an Optimal Option/Recommendation
Ease of Revision
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Note that while in Figure 4 a ‘15 minute Annalisa’ wins on these ratings and weightings there is no suggestion that an Annalisa must
be done quickly, merely that it can be. All aspects (option specification, attribute selection, rating, weighting) can be tackled as
rigorously as time and resources permit. In Figure 5, with time and expense less important, a 15 week £50k Comprehensive Bayesian
Decision Modelling comes out top.
Figure 4
Figure 5
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It will be noted that the multi-criteria WTP tariff approach mentioned at the beginning of the paper has not been included in the
contenders. It could, and probably should be, though intuitively it seems likely to be dominated on most attributes.
Comments
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Sesja 1 / Session 1
Notatki / Place for your notes
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Prelegent / Expert
Panel dyskusyjny: Potrzeba „dekalogu” dla decydentów
/ Discussion panel: Need for decalogue for health care
politician
David Banta, Jack Dowie, Joanna Mucha, Jorge
Wernli, Wojciech Matusewicz, Zbigniew Szawarski,
Jacek Ruszkowski, Krzysztof Łanda
10:20
11:30
Health Care
HTA & Pricing
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Sesja 1 / Session 1
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Poniedziałek
7 grudnia 2009
Monday
December 7th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 2 / Session 2
Umowy podziału ryzyka: cele, metody, negocjacje – perspektywa publiczna / Risk sharing schemes: objectives, methods, negotiations – public perspective
Bengt Jönsson – 25 min.
Katarzyna Bondaryk – 25 min.
Zoltan Kalo – 25 min.
Liczba umów podziału ryzyka systematycznie rośnie. Koresponduje to z rosnącymi
wydatkami w ochronie zdrowia, wzrastającymi oczekiwaniami społeczeństw względem postępu w medycynie oraz koniecznością zapewnienia dostępu do nowoczesnych
technologii medycznych w warunkach ograniczonych zasobów Þnansowych. Wzrost
świadomości klientów służby zdrowia oraz
presja producentów, prowadzi do zwiększenia presji Þnansowej i politycznej na
decydentów czy płatników. Wymusza to poszukiwanie metod społecznie bezpiecznego
zwiększania zakresu oferowanych świadczeń
zdrowotnych w ramach środków podstawowego ubezpieczenia zdrowotnego. Problem
równego dostępu do świadczeń zdrowotnych
i sprawiedliwego podziału ograniczonych zasobów Þnansowych na ochronę zdrowia staje się coraz bardziej drażliwy społecznie, a
politycy i decydenci w przypadku dokonania
nieracjonalnych wyborów coraz częściej stają pod pręgierzem opinii publicznej. Do porozumień podziału ryzyka (RSS; risk sharing
schemes) może dojść tylko wówczas, gdy jakieś ryzyko występuje po obydwu stronach:
regulatora/płatnika oraz producenta.
The number of risk sharing schemes
increases systematically. This corresponds to
growing expenses on health care, increasing
expectations related to progress in medicine
and necessity to ensure access to modern
health technologies within limited Þnancial
means. Increasing awareness of health care
consumers and pressure of manufacturers
result in increased Þnancial and political
pressure on decision makers and/or payers.
Thus socially secure methods to increase the
range of offered health care services within
the resources of basic health insurance must
be sought for. The problem of equal access
to health care services and just allocation
of limited resources in health care becomes
more and more socially sensitive, and politicians or decision makers must be prepared
for the wrath of public opinion in result of
their irrational choices. Risk sharing schemes (RSS) are practicable only if there is
some risk for both sides: the regulator/payer and the manufacturer.
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Dzięki mechanizmom przewidzianym w
stosownym porozumieniu każda ze stron
zdejmuje część ryzyka drugiej strony. Do
najważniejszych rodzajów ryzyka po stronie regulatora/płatnika należą m.in. podjęcie błędnej decyzji refundacyjnej czy cenowej, naruszenie dyscypliny Þnansowej, czy
wreszcie ograniczenie dostępności do świadczeń wysoce opłacalnych kosztem refundacji
nowego leku. Dodatkowo pojawić się może
ryzyko polityczne związane z nieprzewidzianymi wysokimi oczekiwaniami społecznymi, zarzuty o nierówne czy niesprawiedliwe
(preferowanie pewnych producentów) traktowanie produktów, czy dość częste oskarżenia o dyskryminację określonych grup
chorych. Warto podkreślić, że dzięki RSS
decydenci mogą zaoferować swoim obywatelom innowacyjne technologie medyczne
w koszyku podstawowym, jednocześnie zachowując kontrolę nad wydatkami z budżetu
na ochronę zdrowia. Umowy podziału ryzyka
mogą stać się szczególnie chętnie wykorzystywanym narzędziem systemowym w krajach średnio zamożnych, a zatem państwach
naszego regionu.
W ramach drugiej sesji poruszona zostanie tematyka podziału ryzyka postrzegana z
perspektywy płatnika/ubezpieczyciela.
The mechanisms speciÞed in an appropriate agreement make each side take some
risk of the other side. The most important
risks of the regulator/payer include wrong
reimbursement or pricing decisions, failure
to exercise Þnancial discipline or limitation
of access to highly cost-effective technologies in result of reimbursement of a new
medication. In addition, political risk related to unpredicted high social expectations,
charges of unequal or unjust treatment of
speciÞc products (i.e. preference of particular manufacturers), or quite common accusations of discrimination of speciÞc groups
of patients must be taken into account. It
should be stressed that RSS make it possible
for decision makers to offer innovative health technologies within the basic package,
at the same time maintaining control over
budget expenses on health care. Risk sharing schemes may become a useful systemic
tool, especially in mid-income countries, i.e.
in our region.
During the second session problems related to risk sharing as perceived by the payer/insurer will be discussed.
HTA & Pricing
25 min.
Zasada odpłatności za efekty (pay for performance – P4P) to narzędzie poprawy jakości opieki zdrowotnej, coraz szerzej stosowane w ciągu
ostatnich 5-10 lat. Początkowo było ono używane
głównie przez prywatnych i publicznych ubezpieczycieli w USA, ale ostatnio zyskuje popularność
w Anglii i innych krajach europejskich. Odpłatność za wyniki takich czy innych działań ma długą
tradycję w opiece zdrowotnej. Zasadą P4P jest
wynagradzanie zależne od zmierzonych efektów
zdrowotnych. Zasada ta jest całkowicie odmienna od systemu kapitacyjnego lub odpłatności za
świadczenie (fee for service), np. refundacji opartej na DRG lub rozmaitych mechanizmów alokacji
środków budżetowych przeznaczonych na opiekę
zdrowotną. Chociaż głównym celem P4P jest poprawa jakości opieki zdrowotnej, zasada ta znajduje również zastosowanie w działaniach ukierunkowanych na efektywność kosztową i osiąganie
oszczędności.
HTA jako narzędzie polityki zdrowotnej rozwija
się od ponad 30 lat. Początkowo oczekiwano, że
publikacja danych dotyczących efektów stosowania różnych technologii medycznych wystarczy,
aby zmienić codzienną praktykę. Doświadczenie
wskazuje, że nie zawsze tak jest, a zmiana praktyki wymaga podejmowania systematycznych
działań. Znaczenie efektywności kosztowej w HTA
wzrastało z czasem, a analizy HTA coraz ściślej
wiązały się z decyzjami dotyczącymi Þnansowania i refundacji. Zastosowanie zachęt ekonomicznych w celu zmiany praktyki i poprawy jakości
opieki zdrowotnej stanowi naturalny kolejny krok
w rozwoju HTA.
Podczas gdy początkowo HTA używano głównie
do oceny uznanych technologii, zastosowanie jej
jako narzędzia kształtowania cen i refundacji włączyło HTA do zagadnień związanych z wprowadzaniem na rynek nowych technologii medycznych,
zwłaszcza leków. W następstwie tego po pierwsze
wzrosła niepewność co do efektywności klinicznej
i kosztowej leków dotychczas nie stosowanych
poza badaniami klinicznymi, po drugie zaś nastąpiło powiązanie HTA z decyzjami dotyczącymi
kształtowania cen. Skoro decyzje dotyczące stosowania nowych technologii są obarczone znaczną
niepewnością zarówno po stronie sprzedawcy, jak
i kupującego, opracowano i zastosowano w praktyce rozmaite formy umów podziału ryzyka. Decyzja dotycząca refundacji, „czwarta przeszkoda”
w dostępie do rynku, rzadko ma postać prostego
www.ceestahc.org
Prelegent / Expert
Bengt Jönsson
Pay for performance is an instrument to
improve quality in health care that has gained
widespread use during the last 5-10 years.
Initially it was mainly used by US private and
public insurers, but lately it has spread to England and other European countries. While
payment for performance of different activities has a long tradition in health care, P4P is
directed towards measurement and reward of
health outcomes. It is thus distinctly different
from fee for service or capitation payment,
for example DRG based reimbursement and
different mechanisms for budget allocation in
health care. While focus is on improvement
of quality of care, recently P4P has also been
linked to initiatives for achieving cost-effectiveness and cost savings.
HTA as an instrument in health policy has
been developed over period of over 30 years.
Initially the expectation was that the publication of evidence on the performance of different health technologies should be enough
to change medical practice. Experiences have
revealed that this is not always the case, and
that systematic efforts for changing practice
are necessary. Over time cost-effectiveness
has become an increasingly important part
of an HTA, and HTA studies have been more
closely linked to reimbursement and funding
decisions. The use of economic incentives to
change practice and improve quality of care
is a natural development of the HTA movement.
While HTA in the beginning was mainly
used to assess established technologies, the
use of HTA as an instrument for pricing and
reimbursement has moved HTA into the Þeld
of market access for new health technologies,
in particular drugs. This has two consequences, Þrst an increasing uncertainty about the
effectiveness and cost-effectiveness of drugs
that have not yet been used outside clinical
studies, and secondly a linking of HTA into
decisions about pricing. Since decisions about use of new technologies are made under
great uncertainty for both seller and buyers, different schemes for risk sharing have
been developed and also used in practice. A
reimbursement decision, “the fourth hurdle”
for market access, is seldom a simple yes or
no decision. Increasingly different condition
Sesja 2 / Session 2
Health Care
71
Evidence-Based
Sesja 2 / Session 2
72
rozstrzygnięcia – tak lub nie. Refundacja może
być obwarowana coraz większą liczbą różnych
warunków, które można interpretować jako P4P;
aktualnie w użyciu jest szereg typów takich umów
(coverage by evidence development, conditional
treatment continuation, payment for responder,
payment for clinical outcome itp.).
P4P ma dwie składowe: pomiar efektu i reguły
wynagradzania dostawcy technologii w zależności
od wyników pomiaru. Mierzona wielkość powinna
bezpośrednio przekładać się na efekt zdrowotny.
Nie jest to zagadnienie trywialne, ponieważ stan
zdrowia zależy od wielu różnych czynników. Również poziom odpłatności (czy też cena) za jednostkę mierzonej wielkości musi być dobierana
ostrożnie w celu uzyskania zamierzonego efektu
zachęty. Oznacza to, że konieczne jest ustalenie
wartości różnych „mierników efektu”. Może to
być na przykład konieczność określenia wartości
zmian przeżywalności w odniesieniu do jakości życia. Chociaż P4P ma znaczny potencjał, skuteczne
działanie takiego systemu w praktyce natraÞa na
szereg przeszkód. Umowa taka może się wiązać
ze znacznymi kosztami, a jeśli jest wadliwie zaprojektowana, przynosi też wadliwe rezultaty. Lepiej jest stosować proste środki, których nie da
się poddać manipulacji; niestety, nie zawsze jest
to możliwe. Sporządzenie umowy zapewniającej
zarówno statyczną, jak i dynamiczną efektywność
alokacji środków na nowe technologie medyczne
jest jeszcze trudniejsze niż zastosowanie P4P
w celu poprawy jakości opieki zdrowotnej, przy
użyciu złożonego zestawu procesów i „mierników
efektu”.
Praktyczne doświadczenie w zakresie projektowania i wdrażania systemów P4P jest wciąż
ograniczone, a większość z nich wciąż funkcjonuje na zasadzie umów elastycznego kształtowania
cen. W ostatnim czasie podjęto jednak szereg
inicjatyw w celu ułatwienia rozwoju tej dziedziny, przede wszystkim poprzez systematyczne
gromadzenie danych dotyczących wyników leczenia po wprowadzeniu nowych leków na rynek.
Wzrastający stopień skomplikowania zagadnień
związanych z dostępem nowych leków do rynku,
na którym ryzyko ekonomiczne często staje się
ważniejsze od ryzyka działań niepożądanych, a
bardzo kosztowne interwencje bywają stosowane w niewielkich populacjach pacjentów, często
z ciężkimi schorzeniami, zachęca dostawców i
nabywców technologii do poszukiwania nowych
rozwiązań, korzystnych dla obu stron.
Bengt Jönsson
are linked to reimbursement and these conditions can be interpreted as P4P; coverage
by evidence development, conditional treatment continuation, payment for responder,
payment for clinical outcome etc.
P4P has two components, the measurement of the performance and the rules for
compensating the provider based on the
measurements. Outcome measures have to
be developed that are directly linked to the
performance. This is not trivial since health generally is determined by a number of
different factors. Similarly, the payment or
price for different quantities of the measured
outcome has to be carefully designed to give
the incentives. This means that the value of
the different performance indicators must be
established. This may for example make it
necessary to specify the value of changes in
survival relative to quality of life. While P4P
has a great potential, there are a number of
hurdles for such a system to work efÞciently in practice. The transaction costs involved
may be large, and if the design of the contract is ßawed, the result will also be ßawed.
Simple measures that cannot be manipulated
is to be preferred, but not always available.
To design contracts that achieve both static
and dynamic efÞciency in allocating resources for new health technologies is even more
difÞcult that using P4P to improve quality in
health care, where a combination of process
and outcome indicators is often used.
The practical experiences of designing and
executing such P4P systems are still limited, and most have the character of ßexible
pricing schemes. But there are a number of
initiatives under way that will facilitate the
development; the most important being the
systematic collection of outcome data after
market introduction of new drugs. There
are also strong incentives for providers and
purchasers to Þnd “win-win” solutions to the
increasingly difÞcult issues related to market
access for new drugs, where often the economic risk is overshadowing the risk of side effects, when high cost interventions are used
for very small patient populations, often with
severe illness.
HTA & Pricing
HTA, Pricing and Pay for
Performance
Bengt Jönsson
Stockholm School of Economics,
Sweden
www.ceestahc.org
Bengt Jönsson
Sesja 2 / Session 2
Health Care
Contents of presentation
! HTA
! The
link to pricing and reimbursement
! Pricing
! Value
in use versus value in exchange
! Pay for performance (P4P)
! The
road to flexible pricing according to
value?
HTA
The link to pricing and reimbursement
73
Evidence-Based
Sesja 2 / Session 2
Bengt Jönsson
HTA – The increasing role to inform decisions about
market access for new health technologies
Accumulated
knowledge
Production function
for knowledge about
a new health technology
Clinical practice
evidence
Time
MA
Time for traditional HTA
Complications added to the new role
!
Available evidence for undertaking an HTA are
mainly the same as those available for decisions
about market authorisation
!
!
!
Aimed at assessing safety and efficacy
Not sufficient for assessment of effectiveness
CostCost-effectiveness determined by price
!
!
Price a key parameter for reimbursement
Economic uncertainty is added to medical
uncertainty
Possible solutions to the dilemma
!
Ask innovators for more information
Will increase costs and time for development
Will make new innovations more expensive
! Evidence on relative effectiveness can often not be
derived from clinical trials
!
!
!
Systematic data collection after market
authorization
!
!
74
For assessment of risk and effectiveness
For assessment of costcost-effectiveness
HTA & Pricing
Some conclusions
!
HTA in the early development of new health
technologies is based on limited and uncertain
evidence
!
The costcost-effectiveness analysis links HTA to
pricing and reimbursement
!
!
Regulators and HTA agencies assess the same data
Value based pricing are based on incomplete and
uncertain data, mainly economic models
www.ceestahc.org
Bengt Jönsson
Sesja 2 / Session 2
Health Care
Pricing
Value in use versus value in exchange
Two aspects of pricing
!
Value in use
!
!
!
Value differs between indications
Value differs between jurisdictions
Value in exchange
Depends on competition in the market place
Patents and other property rights
! Market segmentation
!
!
! Opportunities
for parallel trade (arbitrage)
75
Evidence-Based
Sesja 2 / Session 2
Value based pricing
!
Has developed as a dominating principle over the last
30 years
!
!
!
Tagamet in 1976 started a new era
Replacing price control based on simple cost comparisons
and other cost based principles (R&D and production
investments in the country)
Based on a defined relation between price (cost) and
value
!
!
Often not explicitly assessed
Mechanisms for more explicit assessment developed over
time
!
Australia/Canada 1993, NICE 1999, LFN Sweden 2002
Some issues involved in VBP
!
Price is related to the product while value is
related to the use
CostCost-effectiveness varies between indications
CostCost-effectiveness varies between comparators
! CostCost-effectiveness varies between jurisdictions with
different price structures and income levels
!
!
!
The full application of value based pricing
requires differential pricing and market
segmentation (price discrimination)
Price dynamics in the market for
health technologies
!
!
An increasingly international and competitive
market
Many obstacles for a free flow of products
between countries have been lifted
!
!
!
76
The EU market as an example
Price differentials between countries for the
same product has been reduced over time
More transparent market makes it difficult to
apply different prices for different indications
Bengt Jönsson
HTA & Pricing
Some conclusions
!
While both innovators and payers accept the
principle of value based pricing, it has been
increasing difficult to apply it in practice
!
!
To the extent that we in some jurisdictions can see a
return to traditional price control
Further development requires a mechanism for
price discrimination
!
Which can be trusted by both parties
www.ceestahc.org
Bengt Jönsson
Sesja 2 / Session 2
Health Care
Pay for performance
(P4P)
The road to flexible pricing according
to value?
“If P4P is everything, maybe it is
nothing”
nothing”
!
!
CoCo-payments, prepre-use authorization, quantity and
dose limitations, coverage by evidence
development, restricted reimbursement, outcomes
guarantees, conditional treatment continuation ,
only in research, only with research, price volume
agreements, ..........
Reimbursement is not a hurdle (yes or no!)
!
!
Marketing under an open ended insurance is a thing of
the past(???)
Private and public insurers face resistance to premium
and tax increases
77
Evidence-Based
Sesja 2 / Session 2
“Trust is good but control is better”
better”
!
!
“However, lack of sufficient, timely and relevant
evidence makes HTAHTA-driven coverage and
reimbursement decisions difficult, if not
impossible.”
impossible.”
There is a lack of evidence but also a need to
follow up that decisions are implemented
Payers do not expect doctors and other health care
decision makers to use new technologies in a costcosteffective way
! Follow up data are demanded
!
Why are payers interested in P4P for
pharmaceuticals?
!
Waste of money is new major risk
!
!
!
Funding in health care shifting from input to output
!
!
!
High cost per patient
Uncertainty about outcome
From budgets and fee for service
To capitation and procurement of outcomes
Pay per pill or device not consistent with criteria and
incentives for costcost-effectiveness
!
Focus has shifted to indications and alternatives
P4P in health care
!
Was initially used among private and public health
insurers in US to provide incentives for improved
quality of care
!
!
!
Has in Europe been mainly developed in the UK
!
!
78
Stimulate the measurement of performance
Reward to providers according to prepre-defined targets
A tool for purchasers of care to get better value for money
Focus on measurement of performance rather than the
design of payment rules
Bengt Jönsson
HTA & Pricing
Examples from P4P in the US
!
Pay for participation dominates
!
!
!
!
Mainly for collection of performance data
Extra money to hospitals that report outcome data
Most projects relate to administrative innovations rather than
use of health technology; i.e. coordinated care
Experiences and concerns
!
!
!
Some evidence of improved quality of care, but no evidence
of cost savings
Some unun-intended consequences, for example avoidance of
high risk patients
Concerns about the validity of quality indicators, patient and
physician autonomy and privacy, and increased administrative
burdens
www.ceestahc.org
Bengt Jönsson
Sesja 2 / Session 2
Health Care
P4P for pharmaceuticals
As interpreted from reimbursement decisions
!
Restricted reimbursement
!
Limited to specifically defined patients within the licensed indication
indication
!
!
!
Reimbursement during evidence development
!
Requirement for new data
!
!
!
!
Performance measure: Patient characteristics
Payment decision: Continued of withdrawn reimbursement
From clinical practice or clinical studies
Performance measure: Effectiveness and/or costcost-effectiveness
Payment decision: Continued of withdrawn reimbursement
Performance guarantees
!
!
!
Payback or compensation if no effect (Proscar
(Proscar in BPH)
Payment for responders only (Velcade
(Velcade in Multiple Myeloma)
Myeloma)
Linking price to measure of effectiveness (Betaseron
(Betaseron in MS)
Examples from Sweden (1)
Restricted reimbursement to defined indications
!
Xenical for treatment of obesity
For patients with specific diagnostic criteria
Verification requested
! Implicit performance: Effectiveness and costcosteffectiveness
!
!
!
!
Champix in smoking cessation
Crestor and Ezetrol in hyperlipidemia (2nd line)
79
Evidence-Based
Sesja 2 / Session 2
The Swedish reimbursement system
Restricted reimbursement as a P4P tool
Evidence on effectiveness
and cost-effectivness.
Evaluating if the use in
clinical praxis follows the
specified indication.
Combination
Examples from Sweden (2)
Restricted reimbursement during evidence development
!
Exubera and Levemir for treatment of diabetes
!
!
!
Support of health economic assessment
Risk factor assessment in clinical practice
Implicit performance: Effectiveness and costcost-effectiveness
!
Accomplia and Zimulti (rimonaband)
rimonaband) in obesity and diabetes
Grazaxin in the management of grass pollen allergy
Azilect and Neupro in Parkinsson
!
Nexavar in renal cell carcinoma
!
Velcade for MM
!
!
!
!
!
!
Relevant clinical comparators and effectiveness in clinical practice
practice
Data for verification of survival estimates (register)
Two year restriction waiting for additional clinical data
After that reimbursement without restrictions
Some conclusions (1)
!
Restrictions on reimbursement in relation to licensed
indication based on costcost-effectiveness is common
!
!
!
!
!
80
Marketing must inform about this restriction
Follow up registration of patients treated according to
relevant characteristics (diagnosis, risk, factors, previous
treatments etc)
etc) is some times demanded
Many examples are “life style drugs”
drugs”
May or may not be combined with requests for data
collection to verify costcost-effectiveness in approved
indications
No detailed performance measures are specified and
open interpretation of results
Bengt Jönsson
HTA & Pricing
Some conclusions (2)
!
Requests for data collection to verify costcost-effectiveness
in approved indications address the following problems
!
!
!
Relevant comparator in clinical practice
Effectiveness and costcost-effectiveness in clinical practice
Uncertainty about long term effects
!
New data are decisive for continued reimbursement
!
For long term effects it may be enough that patients are
included in a registry
!
!
But no specified link between performance and payment
www.ceestahc.org
Bengt Jönsson
Sesja 2 / Session 2
Health Care
MS, RA, Cancer
P4P and optimal incentives for
innovation (1)
!
!
!
P4P is an instrument to provide incentives for
improved quality and costcost-effectiveness in health
care spending
While the trend is in the direction of P4P the
actual experiences so far are limited
Economic incentives are powerful and if they
are wrongly designed they can be dysfunctional
innovation (2)
!
!
!
The linking of HTA and reimbursement is an
example of how payers try to make sure that
they pay for performance
But HTA and reimbursement are only indirectly
linked to the resource allocation in health care
P4P requires more direct interaction between
payers (purchasers) and providers of health care
technologies
81
Evidence-Based
Sesja 2 / Session 2
innovation (3)
!
!
!
!
!
Performance payment make the innovative industry
partly responsible for efficiency in the health
services
Responsibility with limited influence?
New business model?
Are the potential benefits large enough to balance
the risks?
What is the alternative?
My conclusions
!
Focus in health care systems is shifting from
input and throughput to outcome
!
Innovation is increasingly a B2B activity, driven
by a search for winwin-win agreements
!
High transaction costs are on obstacle but they
can and will be reduced
!
!
82
The innovative industry will follow this trend
P4P is a mechanism for valued based pricing
Bengt Jönsson
HTA & Pricing
25 min.
Zakupy centralne produktów leczniczych. Podział ryzyk pomiędzy płatnika
a Þrmę farmaceutyczną / Central purchase procedures for medicinal products.
Risk sharing between the payer and the pharmaceutical company
www.ceestahc.org
Prelegent / Expert
Katarzyna Bondaryk
Sesja 2 / Session 2
Health Care
83
Evidence-Based
Prelegent / Expert
Sesja 2 / Session 2
Strategiczne kształtowanie cen leków w krajach Unii Europejskiej
84
W przypadku nowych leków dostępnych na
receptę dostęp do rynku zależy nie tylko od
uzyskania rejestracji, ale też od poziomu refundacji przez płatników. Ceny nowych leków
są przez płatników stale kwestionowane. Cena
optymalna z punktu widzenia Þrmy i jej udziałowców może być nie do zaakceptowania dla
społeczeństwa. W coraz większej liczbie krajów
refundacja nowego leku jest zatem uzależniona
od spełnienia kryteriów efektywności kosztowej
i możliwości Þnansowania.
Techniki tradycyjne, takie jak krzywe elastyczności cen wykreślane na podstawie wyników badań rynku, są niewystarczające dla
ustalenia strategii kształtowania cen przyszłych
leków. Określenie właściwej strategii kształtowania cen i refundacji jest kluczowym składnikiem procesu opracowywania nowego leku.
Strategiczne kształtowanie cen obejmuje istotne elementy ekonomiczno-zdrowotne. Wartość
nowego leku może być wyliczana w oparciu o
cenę komparatora i wartość różnicującą. Komparatorem jest zazwyczaj najbardziej prawdopodobny „złoty standard” leczenia w chwili oczekiwanego wejścia produktu na rynek.
Wartość różnicująca uwzględnia oszczędności,
ekonomiczny aspekt korzyści w odniesieniu do
stanu klinicznego, przeżywalności i jakości życia, jak również użyteczność (np. łatwość stosowania czy poprawę współpracy pacjenta).
W Unii Europejskiej obserwuje się konwergencję cen leków innowacyjnych. W strategicznym kształtowaniu cen nowych leków największą rolę odgrywają rynki o dużym potencjale
sprzedaży, a wprowadzanie leku na poszczególne rynki jest planowane w skali globalnej,
poczynając od krajów oferujących najwyższe
ceny. Konwergencja cen skutkuje zmniejszeniem środków przeznaczanych na badania i
rozwój w krajach zamożnych i ograniczeniem
dostępu do leku w krajach średnio zamożnych.
Pokonanie bariery dostępu do rynku w krajach
średnio zamożnych przy równoczesnym utrzymaniu ceny minimalnej w Europie wymaga
opracowania przez Þrmy farmaceutyczne specjalnych metod kształtowania cen.
Zoltan Kalo
Market access of new prescription medicines
is not only subject to approval by regulatory
agencies, but also depends on the reimbursement by payers. Pricing of new pharmaceuticals
has been constantly challenged by payers. The
optimal price for the company and their shareholders may not be acceptable for the society.
Consequently, the reimbursement of new pharmaceuticals is subject to fulÞlling the criteria of
cost-effectiveness and affordability in more and
more countries.
Traditional techniques, such as price elasticity curves elicited by market researchers, are
not sufÞcient to establish pricing strategies of
future drugs. Determining the right pricing and
reimbursement strategy in the drug development process is critical. Strategic pricing includes strong health economic elements. The
economic value of new medications can be calculated based upon the comparator price and
the differential value. The comparator is usually the most likely gold standard therapy at the
time of the expected launch of the product. Differential value includes cost savings; economic
value of clinical, survival and QoL beneÞts; and
the value in use, such as ease of administration, improved compliance.
In the European Union price convergence
of innovative medicines can be observed. The
strategic pricing of new drugs is based on markets with huge sales potential, and the launch
sequence is harmonised globally with early
launch in high price countries. The price convergence results in reduced R&D resources in
high income countries and reduced market access in middle income countries. Pharmaceutical companies have to develop special pricing
methods to tackle market access barriers in
middle income countries in parallel with maintaining the European ßoor price.
25 min.
HTA & Pricing
Strategic pricing of
pharmaceuticals in the
European Union
Zoltán Kaló
Director, Health Economics Research Centre (HERC)
Eötvös Loránd University
CEO, Syreon Research Institute
www.ceestahc.org
Zoltan Kalo
Sesja 2 / Session 2
Health Care
Krakow, 7 December 2009
Economic environment of
pharmaceutical innovation
• Global economic recession
• Reduced public spending
• Cost-containment of health expenditure →
focus on pharmaceutical expenditure
• Controlled reimbursement of innovative
medicines
• Reduced growth rate of the US pharma
market
Factors influencing the
market access and pricing
of pharmaceuticals
85
Evidence-Based
Sesja 2 / Session 2
Efficiency of pharmaceutical
innovation
Development cost of new chemical entities until
registration (million US$)
Increased cost of
pharmaceutical innovation
• Safety (longer trials, more
patients)
• Ethical standards (active
control vs placebo)
• Improved medicines (gold
standard comparator)
• Policy relevant comparator
• New endpoints (relevant for
payers – hard-endpoints,
naturalistic, QoL, resource
utilisation)
Ref: DiMasi JA, Grabowski HG. The Cost of Biopharmaceutical R&D: Is Biotech Different?’, Managerial and
Decision Economics 28 (2007): 469-479
Controlled pricing and
reimbursement of innovative drugs
• Cost-effectiveness (fourth hurdle)
• Budget impact → primary prevention (?)
• Reimbursement based on public health needs
• Financing protocols: first-line therapy → generic drugs
• Reference pricing: generic + therapeutic → me-too drugs
• International price referencing
• Cost-sharing:
– Outcome → conditional reimbursement
– Price-volume agreement (prevent rapid penetration)
– Claw-back
86
Zoltan Kalo
Health Care
HTA & Pricing
www.ceestahc.org
Zoltan Kalo
Sesja 2 / Session 2
Implications of R&D challenges
• Return on investment is the most important
question of pharmaceutical innovation
• Innovative companies have to improve the
– success rate of R&D
– sales of new medicines
• Time to peak sales in the new target for R&D
instead of time to registration
• This includes market access " pricing &
reimbursement
• Clinical development has to incorporate the
economic end-points
7
Optimal pricing is a
crucial success factor
• Optimal price has more impact on return
on investment (RoI) than
–
–
–
–
reduction of production costs
reduction of R&D costs
improved effectiveness of sales force
better targeting of marketing programmes
• Too high price: delayed or restricted
reimbursement
• Too low price: reduced profit (RoI)
Pricing of generic drugs
p(orig) = price of original drug
p(prod) = marginal cost of production
p(mand) = mandated price discount
p(opt) = optimal price – maximised revenue
p(comp) = price of generic competitors
0
p(prod)
p(opt) p(mand) p(orig)
ex-factory price of generic drugs (€)
87
Evidence-Based
Sesja 2 / Session 2
Pricing of innovative drugs
p(prod) = marginal cost of production
p(opt) = optimal price – maximised revenue
p(value) = economically justifiable price
0
p(prod)
p(opt)
p(value)
ex-factory price of original drugs (€)
Value based pricing
Economic value =
Economically justifiable price
Economic
Value
=
Comparator
Price
+
Differential
Value
Economic Value
Comparator
Price
Savings
in RU
QoL/
Utility
benefits
Productivity
Gains
Differential Value
88
Value
in use
Economic
Value
Zoltan Kalo
HTA & Pricing
Cost-effectiveness Analysis
Backwards
ICER =
∆Cost
=
∆ Effectiveness
( D2 - D 1) + (C 2 - C 1)
( E2 - E1 )
D = Drug Cost
C = Other treatment cost
E = Effectiveness
Solve for drug cost:
D2
Economic
Value
=
=
D1 + ( C1 - C2 ) + ICER · ( E2 - E1 )
Comparator
Price
+
+
Cost
Saving
www.ceestahc.org
Zoltan Kalo
Sesja 2 / Session 2
Health Care
Value of
clinical Benefits
Pricing research
S
Q
Q
u
a
n
t
i
t
y
P x Q = S [Sales]
Peak
Sales
S
a
l
e
s
Price
P
Price
Econ.
value
P
Value propositions
• unmet medical need – no alternative therapies
• rare disease (low incidence / prevalence →
orphan drugs)
• severe disease
• high cost of current therapies
• (new mechanism of action)
• significant efficacy improvement
• improved tolerability
• tailor made therapy (segmented patient groups)
• low cost-effectiveness ratio
89
Evidence-Based
Sesja 2 / Session 2
Economic value varies
across patient segments
900
80%
800
70%
700
60%
600
Market size
90%
50%
500
40%
400
30%
300
20%
200
10%
100
0%
0
Target Product Profile / Market Segment
Impact of EU and EMU on
pharmaceutical pricing
• transparent pricing strategy
• parallel trade + international price
referencing
• price convergence → no Ramsey pricing
• strategic pricing based on markets with
huge sales potential (Top 5 markets)
• global harmonisation of launch sequence
(early launch in high price countries)
90
Value of therapy
Order of potential indications
Zoltan Kalo
HTA & Pricing
Impact of transparent pricing
www.ceestahc.org
Zoltan Kalo
Sesja 2 / Session 2
Health Care
Ridley DB: Pharmacoeconomics 2005; 23 (7): 651-658
Middle-income countries
General framework:
• Payers want European floor price
• Optimal price for Top 5 European market is not
justifiable (ICER>threshold)
• Fourth hurdle is not implemented in all countries:
–
–
–
–
lack of professionals
small country
no budget for HTA
no political preference for transparent decisions
Middle-income countries
• Pharmaceutical HQ objective
– maintain European floor price
– prevent price erosion (currency rate fluctuations)
• Options for local pharmaceutical affiliates
– No fourth hurdle →
• launch as early as possible
• wait for the lowest price
– Fourth hurdle →
• non-transparent price discount (price-volume,
clawback, rebate etc.)
• narrow patient population
• risk-sharing / performance based agreement
91
Evidence-Based
Sesja 2 / Session 2
Conclusion
• Pharmaceutical R&D: cost-containment and
reduced efficiency
• Optimal pricing to ensure rapid market
access and profitability: critical success
factor
• Value based pricing for big markets
• Second-best strategy for middle-income
countries
• Need for trained pricing and health
economic professionals
92
Zoltan Kalo
Health Care
HTA & Pricing
www.ceestahc.org
Sesja 2 / Session 2
93
Poniedziałek
7 grudnia 2009
Monday
December 7th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 3 / Session 3
Wartość terapeutyczna leków i rola HTA w umowach podziału
ryzyka / Value of drug therapy and the role of HTA in risk sharing arrangements
Anita Burrell – 30 min.
Gert van der Wilt – 40 min.
Erin Huntington – 30 min.
Jim Furniss – 25 min.
Sensem umów podziału ryzyka z punktu
widzenia przemysłu jest podział ewentualnych kosztów (ryzyka Þnansowego) związanych z wprowadzeniem na rynek nowego
produktu. Ryzyko dla producentów jest tym
większe im wydatki na opracowanie danego
produktu były wyższe, a zdeÞniowana populacja (odbiorca) mniejsza. Stopień zmniejszenia ryzyka po każdej ze stron może być
Þnalnie różny, dlatego tak istotne jest określenie rodzajów ryzyka w ramach przygotowań do negocjacji i tworzenia strategii refundacyjnej czy cenowej. Gdyby można było
określić wartościowo sumę ryzyk po każdej
ze stron, to RSS (risk sharing schemes) mogły by niwelować to ryzyko w niewielkim (np.
15%) lub znaczącym (np. 50%) stopniu.
Do najczęściej występujących rodzajów
ryzyka (Þnansowe, polityczne, prawne) po
stronie producenta należą:
• brak uzyskania refundacji (radykalnie
niższe wpływy ze sprzedaży, niższa stopa zwrotu z inwestycji, zwiększone straty
itp.);
• uzyskanie refundacji na niekorzystnych
lub mniej korzystnych warunkach (niższy
limit, czyli wyższy stopień współpłacenia,
dołączenie produktu do jumbo group w
ramach substytucji terapeutycznej, zamiast substytucji generycznej czy oddzielnego listowania leku, restrykcyjne
kryteria preskrypcyjne lub np. kryteria
włączania pacjentów do programu terapeutycznego; ograniczenie terapią inicjującą itp.);
• poczucie Þaska strategii refundacyjnej i
cenowej – nieskuteczność w działaniach
na rzecz korporacji;
• wiele innych, specyÞcznych w danej sytuacji konkurencyjnej na rynku.
From the industry perspective risk sharing schemes are agreements made in order to share possible costs (Þnancial risk)
associated with marketing of a new product.
The higher are expenses associated with development of a new product and the smaller the target population, the higher is the
manufacturer’s risk. Decrease of risk may
be Þnally different between the sides; it is
therefore important to determine the risks
during preparation for negotiations and development of reimbursement or pricing strategy. If the summary risk on each side could
be quantitatively estimated, the RSS would
allow for decreasing this risk to a small (e.g.
15%) or signiÞcant (e.g. 50%) degree.
The most common manufacturer’s risks
(Þnancial, political or legal) include:
• failure to achieve reimbursement (radically lower sales income, lower return on
investment, increased losses etc.);
• achievement of reimbursement on unfavorable or less favorable terms (a lower
limit, i.e. higher co-payment; placing of
the product in a “jumbo group” on grounds of therapeutic substitution instead
of generic substitution resulting in separate listing of the product; restrictive
prescription criteria or introduction of a
therapeutic program with strict inclusion
criteria; limitations concerning initial treatment etc.);
• apparent Þasco of pricing and reimbursement strategy – ineffective activity for
the corporation;
• many other risks, speciÞc for particular
circumstances on a competitive market.
95
Evidence-Based
Sesja 3 / Session 3
96
W ramach tej części sesji przedstawiony
zostanie sposób postrzegania regulacji cen
leków i porozumień podziału ryzyka przez
przemysł farmaceutyczny. Wystąpienia będą
odnosić się do trybu i charakteru zawieranych umów, strategii cenowych i samych
negocjacji.
Podstawą porozumień cenowych jest ocena technologii medycznych, która pozwala
ocenić wartość terapeutyczną leku oraz adekwatność wyceny w stosunku do innowacyjności terapeutycznej. Strategie negocjacyjne, sposoby podziału ryzyka, mają wartość
wtórną wobec podstawowych analiz, jakimi
są przeglądy systematyczne i badania opłacalności. Należy podkreślić, że porozumienia
podziału ryzyka mogą być zawierane nie
tylko w przypadku bardzo drogich technologii medycznych, ale również wtedy, gdy
występuje istotna niepewność dotycząca
rzeczywistych korzyści zdrowotnych, proÞlu bezpieczeństwa interwencji oraz kosztów
generowanych w ramach terapii stosowanych w chorobach powszechnych. Ryzyko
związane z niepewnością oszacowań dotyczy
obydwu stron, zarówno regulatora/płatnika,
jak i producenta. Na podstawie analizy efektywności klinicznej, analizy ekonomicznej i
Þnansowej, zarówno regulator, jak też sama
Þrma zyskują wgląd w wartość terapeutyczną leku, stosunek tej wartości do ceny, ale
również w jakość dostępnych danych klinicznych i ekonomicznych. Im mniejsze ryzyko
oszacowań dla interwencji i jej komparatorów, tym mniejsze ryzyko podjęcia błędnej
decyzji refundacyjnej czy cenowej po stronie
urzędu, a także bardziej wiarygodne uzasadnienie dla ceny po stronie producenta. Jedno jest pewne - bez wysokiej jakości badań
klinicznych i prawidłowej oceny technologii
medycznych nie można mieć wglądu w rzeczywistą pozycję terapeutyczną i ekonomiczną leku, względem opcjonalnych sposobów
postępowania. Trudno jest wtedy producentowi ustalić cenę adekwatną do osiąganych
korzyści zdrowotnych, ale też trudno wymagać od decydenta, by bez wahania zgodził
się na bezwarunkową refundację leku po
proponowanej cenie.
During the session problems related to
price regulation and risk sharing schemes
will be presented from the pharmaceutical
industry’s point of view. The presentations
will discuss the scope and characteristics of
agreements made, pricing strategies and
negotiations.
Price agreements are based on health
technology assessment, which makes it possible to estimate therapeutic value of a medication and adequacy of pricing with respect to therapeutic innovativeness. Negotiation
strategies and risk sharing methods are secondary issues in relation to basic analyses
– systematic reviews and cost-effectiveness
studies. It must be stressed that risk sharing
schemes may be considered not only in case
of very expensive health technologies, but
also in situations of signiÞcant uncertainty as to actual health beneÞt or the safety
proÞle of an intervention, or costs related to
treatments applied in common diseases. The
risk associated with uncertain estimations
applies to both the regulator/payer and the
manufacturer. Analysis of efÞcacy and safety
as well as economic and Þnancial analyses
make it possible (both for the regulator and
the manufacturer) to assess therapeutic value of the product and its relation to the price as well as quality of available clinical and
economic data. The lower is the uncertainty of estimation for the intervention and its
comparators, the lower is the risk of making
a wrong reimbursement or pricing decision
for the authority, and the more rational is
justiÞcation of the price for the manufacturer. One thing is sure – without high-quality
clinical trials and proper health technology
assessment correct evaluation of the actual
therapeutic and economic value of a technology in relation to the optional methods of
treatment is not possible. In such circumstances it is difÞcult for the manufacturer to
propose a price adequate to health beneÞt,
but – on the other hand – the decision maker must not be expected to agree without
hesitation to reimburse the medication unconditionally at any proposed price.
Health Care
HTA & Pricing
www.ceestahc.org
Decyzja o przystąpieniu do umowy podziału ryzyka przez płatnika i podmiot odpowiedzialny dla produktu leczniczego, który ma być ewentualnie objęty refundacją,
może być związana m.in. z:
• wysokim kosztem technologii,
• nieznaną i trudną do przewidzenia docelową liczbą pacjentów, u których technologię można zastosować,
• niepewnością oszacowań dotyczących
skuteczności terapii,
• niepewnością oszacowań dotyczących
proÞlu bezpieczeństwa terapii (niedoszacowane lub niewykryte w badaniach klinicznych działania niepożądane),
• różną efektywnością kliniczną w różnych
podgrupach pacjentów, a co za tym idzie
różną opłacalnością terapii w zależności
od charakterystyki chorych,
• wysokimi kosztami związanymi z zastosowaniem dodatkowych technologii (dodatkowa diagnostyka, monitorowanie,
konieczność terapii skojarzonej),
• wielkością środków w budżecie państwa
przeznaczonych na Þnansowanie świadczeń zdrowotnych.
Druga część sesji poświęcona będzie roli
HTA w procesie ustalania cen i negocjacjach
cenowych. Przedstawione zostaną przykłady
efektywnego wykorzystania analiz HTA przy
zawieraniu umów o podziale ryzyka. Szczególna uwaga poświęcona będzie ocenie,
które rozwiązania sprawdzają się woptymalizacji gospodarki lekowej, a które mogą być
potencjalne ryzykowne dla prawidłowego
funkcjonowania systemu.
Decision on risk sharing between the payer and the product’s marketing authorization
holder may be based on:
• high cost of the technology under consideration,
• unknown and difÞcult to predict target
number of patients, in whom the technology may be used,
• uncertain estimation concerning efÞcacy
of treatment,
• uncertain estimation concerning the safety proÞle (adverse effects underestimated or not reported in clinical trials),
• differences between subgroups of patients with respect to efÞcacy and safety, entailing different cost-effectiveness
of treatment depending on the patients’
characteristics,
• high costs related to use of additional
technologies (diagnostics, monitoring,
necessity of combination treatment etc.),
• resources in the state budget allocated
for Þnancing of health care services.
During the third session the role of HTA
in pricing and price negotiations will be
discussed. Examples of effective use of HTA
analyses in risk sharing schemes will be presented. Special attention will be paid to evaluation of speciÞc solutions with respect to
optimization of drug policy or potential risk
to proper functioning of the system.
Sesja 3 / Session 3
97
Evidence-Based
Prelegent / Expert
Sesja 3 / Session 3
Podział ryzyka: amerykański i europejski punkt widzenia
Risk Sharing: US vs European Perspectives
98
Celem niniejszej prezentacji jest przegląd
różnych typów umów podziału ryzyka zawieranych w różnych systemach pod kątem ich podziału na dwie główne grupy: umowy oparte i
nie oparte na wynikach zdrowotnych. Jakkolwiek zarówno w USA, jak i w UE zawierane są
umowy różnych typów należących do obu tych
głównych grup, w amerykańskich systemach
typu „managed care” zaznacza się tendencja do
wybierania typów nie związanych z wynikami
zdrowotnymi, podczas gdy Center for Medicaid
and Medicare Services (CMS), podejmujące decyzje refundacyjne w ramach tych programów
rządowych, podjęło kilka inicjatyw opartych na
wynikach zdrowotnych. W Europie publikowane
są przykłady obu typów programów, przy czym
rozwiązania stricte Þnansowe są mniej popularne od umów odpłatności za efekt („pay for performance”), takich jak te zawierane przez NICE
w ramach systemu dostępu („access programme”) lub rejestr onkologiczny AIFA. Ostatnia
część sesji poświęcona będzie możliwym scenariuszom dalszego rozwoju tych inicjatyw oraz
przemyśleniom na temat ich implikacji.
Anita Burrell
This presentation seeks to lay out the different forms of risk sharing agreements which
are found in different jurisdictions under two
main headings; those that are health outcomes
based versus those that are non health outcomes based. Although there are various different forms of the two main types in both the
US and the EU there are tendencies for the US
managed care sector to focus on non-health
outcomes based examples whilst the Center for
Medicaid and Medicare Services (CMS) which
make coverage decisions for these government
programmes have several outcomes based initiatives ongoing. In Europe there are published
examples of both types of programmes with Þnancial based programmes being less popular
than a „pay for performance” type of guarantee such as those established under the NICE
Access programme or under the AIFA oncology
register. A Þnal section will deal with reviewing
potential scenarios for the future as a way to
promote thinking of the implications of these
initiatives.
30 min.
HTA & Pricing
Risk Sharing
US vs European Perspectives
Anita Burrell
Sanofi aventis
www.ceestahc.org
Anita Burrell
Sesja 3 / Session 3
Health Care
Agenda
!
Taxonomy of Agreements
– Non-Outcomes Based vs Outcomes Based
Contracting, Market and Price Agreements
Performance Guarantees
! Conditional reimbursement
! Pay for performance/short term effectiveness
!
!
– Differences between EU and US in types of
agreements
– Potential future scenarios
Taxonomy of Agreements
Performance-based agreements between health care payers and manufacturers
Non-outcomes based modelsb
Population level
Market
share
Patient level
Price
volume
Utilization
caps
Health outcomes-based agreements
Coverage with
evidence
generationa
Manufacturer
funded
treatment
initiation
Source: Pharmaceutical Outcomes Research &
Policy, University of Washington
Performance guaranteesb
Conditional reimbursement
Experimental
Studies (e.g.
RCTs)
Short-term
effectiveness
Outcomes
guarantee
Observational
Studies
Clinical
Endpoint
Pattern or
process of
care
Surrogate
Endpoint
a Includes CMS coverage with evidence development initiative
b Also termed “risk-sharing” in certain contexts
c
Includes UK’s Office of Fair Trading reform proposal for the PPRS toward value-based
pricing
99
Evidence-Based
Non Outcomes Based Risk
Sharing
Sesja 3 / Session 3
!
Financial Risk Sharing is popular in the
USA
– Discounting schemes (including rebates) Risk
is also shared with the patient for high cost
therapies
!
!
!
Co-payments
Co-insurance
In Europe these are most often formed as
utilization caps or price/volume
agreements (e.g. France)
Performance Based
Schemes
Performance-based health outcomes schemes:
Arrangements between a payer and a pharmaceutical, device, or diagnostic manufacturer where the price, level and/or nature of reimbursement are tied
to future measures ultimately related to patient quality or quantity of life.
Conditional reimbursement: Binary coverage
determination is conditioned upon patient participation in research
or evaluation of short-term effectiveness
Coverage with evidence developmenta:
Coverage conditioned upon the collection of
additional evidence to support continued, expanded,
or withdrawal of coverage.
Experimental Studies
(e.g. RCTs):
Reimbursement tied to
collection of additional
evidence related to safety and
efficacy
Performance guaranteesb: Level of
reimbursement is tied to measure of clinical
outcome in “real world” and includes pre
determined consequences. Can be evaluated at the
population or patient level
Short-term
effectiveness: Coverage
continuation tied to
achieving short-term
effectiveness goals
Outcomes guarantee: Refunds,
rebates, or price adjustments if the
product fails to meet agreed outcome
targets
Pattern of care:
the impact on clinical
decision making or
practice patterns
Observational Studies:
Reimbursement tied to collection of
additional evidence related to
effectiveness, cost-effectiveness,
safety, utilization, and/or clinical
impact
Clinical Endpoint:
Surrogate endpoint:
A characteristic or variable that reflects
how a patient feels or functions, or how
long a patient survives
A biomarker intended to substitute for
a clinical endpoint, i.e., a biomarker
that is expected to predict clinical
benefit, harm, or lack of benefit or
harm.
a
b
Includes CMS coverage with evidence development initiative
Also termed “risk-sharing” in certain contexts
Key Elements of Performance
Based Schemes
!
!
!
!
Agreements between a payer and a pharmaceutical,
device, or diagnostic manufacturer where the price level
and/or nature of reimbursement is related to the actual
future performance of the product in either the research
or “real world” environment rather than the expected
future performance.
Agreements are linking coverage and/or net price to
health outcomes and/or value
Issue is the level of uncertainty at launch regarding the
benefits of allowing access versus the price/cost of new
interventions
Two main types of agreement
– Performance Guarantees
– Conditional Reimbursement
100
Anita Burrell
Health Care
HTA & Pricing
www.ceestahc.org
Anita Burrell
Source,
Year
Disease
area
Manufactur
er
Moldrup,
1995
High
cholesterol
Merck
Pollack, 2007
Breast
Cancer
Genomic
Health
Payer
Agreement
Patients
and
Insurers
Merck promised to refund patients and insurers
up to six months of their prescription costs if
simvastatin plus diet did not help them lower
LDL cholesterol to target concentrations
identified by their doctors.
United
Healthca
re
United Healthcare agreed to reimburse the
OncotypeDx test for 18 months while it and
Genomic Health monitor the results. If the
number of women receiving chemotherapy
exceeds an agreed upon threshold, even if the
test suggests they do not need it, the insurer will
negotiate a lower price.
Sesja 3 / Session 3
Published Performance
Guarantees in the USA
Published Performance
Guarantees in Europe
Source, Year
Country
Disease area
Manufacturer
Payer
Agreement
Chapman20,
2003
UK
High cholesterol
Park Davis
(Pfizer)
North
Staffordshire
health authority
Park Davis (Pfizer) agreed to rebate the North Staffordshire health authority if
a defined patient population did not achieve a low density lipoprotein
cholesterol concentration target of < 3 mmol/l after using statins.
Sparrowhawk21
, 2007
UK
Asthma
Novartis
National health
service
Novartis offers UK hospitals replacement product for appropriately diagnosed,
high-need Xolair (omalizumab) patients who fail to achieve target clinical
response.
Thomson, 2008
UK
Colorectal cancer
Merck
Primary care trust
Rebate direct to primary care trust on the cost of any vials of Cetuximab used
for patients who do not achieve a pre-agreed clinical outcome
(‘nonresponders’) at up to 6 weeks (up to an agreed maximum of 3200
milligrams).
Novartis and DAK (a German insurance company) have agreement to refund
money for Sandimmun Optoral (Cyclosporin), Myfortic (mycophenol acid)
or Certican (Everolimus) if a patient loses his/her donor kidney.
Anonymous22,
2008
Germany
Kidney transplantation
Novartis
Deutsche
AngestelltenKrankenkasse
(DAK)
Anonymous22,
2008
Germany
Osteoporosis
Novartis
DAK and Barmer
DAK and Barmer (a German insurance company) have a money back
guarantee for Aclasta (Zoledronat) if an osteoporosis related fracture occurs
Green24, 2006
UK
Multiple myeloma
Johnson and
Johnson
National health
service
J & J agreed to reimburse the NHS in either cash or product for patients who
do not respond (Response measure: 50% decrease in serum M protein) after
4 cycles of treatment with Velcade. Responding patients receive additional 4
cycles.
Chadwick7,
2003
UK
Multiple Sclerosis
Biogen, Schering,
Teva/Aventis,
Serono
National health
service
Patients using Interferon beta’s or glatiramer acetate are followed for 10
years with treatment effects determined every two years. Drug price reduced
to maintain cost effectiveness at £36,000/QALY
MS Risk-sharing : Key Elements
!
!
!
!
Detailed monitoring over 10 years of a cohort of
patients to confirm the cost-effectiveness of the MS
treatments, various beta interferons and glatiramer.
Treatments initiated by specialist MS centres based
on ABN guidelines; no bar to clinicians prescribing
for patients falling outside these guidelines.
Central features are target outcome measures;
agreed NHS price; threshold cost per QALY of
£36,000.
Outcome measure is Expanded Disability Status
Score. Actual outcomes reviewed every 2 years
against standard MS disease (non-treated)
progression model through the EDSS scale.
See Health Services Circular 2002/004
http://www.doh.gov.uk/publications/coinh.html
101
Evidence-Based
Issues in the MS scheme
!
!
Sesja 3 / Session 3
!
!
!
!
!
Long negotiation process
Danger of gold plating data requirements
Difficulty in recruitment
Difficulty in monitoring
Awaiting interim results
Untried reconciliation process and adjudication
group /process
Is this unique or endemic to risk sharing?
Conditional
Reimbursement
!
!
!
Main example of conditional reimbursement
in US and EU is Coverage with Evidence
Development (CED)
Allows a technology to be made available
under specific conditions, usually a defined
period, after which the benefits of the
technology are reviewed.
Objective of the additional data generation
is to reduce uncertainty around a specific
aspect of the evidence base
CMS and Coverage with
Evidence Development
« The purpose of CED is to generate data on the
utilization and impact of the item or service
evaluated in the National Coverage Determination,
so that Medicare can
a) document the appropriateness of use of that item
or service in Medicare beneficiaries under current
coverage
b) consider future changes in coverage for the item or
service
c) generate clinical information that will improve the
evidence base on which providers base their
recommendations to Medicare beneficiaries
regarding the item or service ».
102
Anita Burrell
Health Care
HTA & Pricing
www.ceestahc.org
Anita Burrell
CMS Coverage with
Evidence Development
!
Actually covers two concepts:
!
Coverage conditioned on specific additional data
collection
– Coverage with Study Participation (CSP)
!
Coverage conditioned on care being delivered in a
setting with a pre-specified data collection process and
additional protections in place such as are present in
some research studies
– Laid out in on-line document from CMS (see
http://www.cms.hhs.gov/mcd/ncpc_view_docum
ent.asp?id=8)
Sesja 3 / Session 3
– Coverage with Appropriate Determination (CAD)
CMS Coverage with Evidence
Development Programmes
Source,
Year
Disease area
Manufacturer
Payer
Agreement
CMS, 2004
Cognitive impairment
Multiple
CMS
An FDG-PET scan is covered in patients with mild cognitive impairment or early
dementia in the context of an approved clinical trial.
CMS, 2005
Hearing loss
Multiple
CMS
CMS may cover cochlear implantation for treatment of hearing loss when the
provider is participating in, and patients are enrolled in, an approved clinical trial
CMS, 2005
Oncology
Multiple
CMS
An FDG-PET scan is covered in patients with brain, ovarian, pancreatic, small cell
lung, testicular cancers, and certain indications for cervical cancer in the context of
an approved clinical trial.
CMS, 2005
Tachyarrhythmia’s
Multiple
CMS
Implantable Cardioverter Defibrillators are covered in the context of an approved
clinical trial or registry.
CMS, 2006
Chronic hypoxemia
Multiple
CMS
The home use of oxygen is covered for those beneficiaries with arterial oxygen
partial pressure measurements from 56 to 65 mmHg or oxygen saturation at or
above 89% who are enrolled subjects in clinical trials approved by CMS and
sponsored by the National Heart, Lung & Blood Institute (NHLBI).
CMS, 2006
Atherosclerotic
disease
Multiple
CMS
CMS covers Percutaneous Transluminal Angioplasty and Stenting of intracranial
arteries for the treatment of cerebral artery stenosis !50% in patients with
intracranial atherosclerotic disease when furnished in an approved clinical trial.
CMS, 2008
Colorectal cancer
SanofiAventis, BMS,
Pfizer,
Genentech
CMS
Oxaliplatin, irinotecan, cetuximab, or bevacizumab for the treatment of colorectal
cancer are covered in the context of an approved clinical trial.
Coverage with Evidence
Development in Europe
Source,
Year
Country
Disease area
Manufacturer
Payer
Whalen10,
2007
Fr.
Schizophrenia
Johnson and
Johnson
French health
authority
Agreement
France's health care authority agreed to cover Risperdal Consta at
J&J's asking price if J&J performed studies to evaluate whether
Risperdal Consta helps patients stay on their medications. If the
studies show otherwise, J&J will reimburse France a portion of the
money it spent on the drug.
Chadwick7,
2003
UK
Multiple
Sclerosis
Biogen, Schering,
Teva/
Aventis, Serono
NHS
Patients using Interferon beta’s or glatiramer acetate are followed
for 10 years with treatment effects determined every two years. Drug
price reduced to maintain cost effectiveness at £36,000/QALY
Should also note that there is increasing use of CED in hospital settings for
pharmaceuticals by the DHCIB in the Netherlands (see for example Retèl et
al, IJTAHC 2009;25(1): 73-83)
Catalan Agency for HTA in Spain has also made CED recommendations
103
Evidence-Based
Performance Based
Schemes
Performance-based health outcomes schemes:
Arrangements between a payer and a pharmaceutical, device, or diagnostic manufacturer where the price, level and/or nature of reimbursement are tied
to future measures ultimately related to patient quality or quantity of life.
Conditional reimbursement: Binary coverage
determination is conditioned upon patient participation in research
or evaluation of short-term effectiveness
Sesja 3 / Session 3
Coverage with evidence developmenta:
Coverage conditioned upon the collection of
additional evidence to support continued, expanded,
or withdrawal of coverage.
Experimental Studies
(e.g. RCTs):
collection of additional
evidence related to safety and
efficacy
Performance guaranteesb: Level of
reimbursement is tied to measure of clinical
outcome in “real world” and includes pre
determined consequences. Can be evaluated at the
population or patient level
Short-term
effectiveness: Coverage
continuation tied to
achieving short-term
effectiveness goals
Outcomes guarantee: Refunds,
rebates, or price adjustments if the
product fails to meet agreed outcome
targets
Pattern of care:
the impact on clinical
decision making or
practice patterns
Observational Studies:
Reimbursement tied to collection of
additional evidence related to
effectiveness, cost-effectiveness,
safety, utilization, and/or clinical
impact
Clinical Endpoint:
Surrogate endpoint:
A characteristic or variable that reflects
how a patient feels or functions, or how
long a patient survives
A biomarker intended to substitute for
a clinical endpoint, i.e., a biomarker
that is expected to predict clinical
benefit, harm, or lack of benefit or
harm.
a Includes
b Also
CMS coverage with evidence development initiative
termed “risk-sharing” in certain contexts
Pay for Performance
!
!
!
!
NHCQ pushed quality measures
including HEDIS
Center for Payment Reform
CMS Value Based Purchasing
No payment for « never events »
– Avoidable rehospitalisations
– Nosocomial infections
– VTE
Short-term effectiveness
Country
Disease
area
Product
Manufacturer
Payer
Agreement
Italy
Renal cell
carcinoma
Sunitinib,
Sorafenib
Pfizer, Bayer
Italian health
authority
A hospital discount of 50% applies to the first two/three months of
treatment with Nexavar (sorafenib) and Sutent (sunitinib). For
responding patients, the treatment is then reimbursed and the
discount dropped.
Sparrowhawk2
1, 2007
Italy
Alzheimer's
disease
Alzheimer
’s disease
drugs
Multiple
Italian health
authority
During first 3 months, patients starting Alzheimer's disease
drugs are assessed for short-term effectiveness. Drug provided
free by manufacturer. If treatment goals are met after 3 months,
treatment is continued for a max of 2 years – drug costs
reimbursed by national health service. Evaluation by UVA every 6
months
Green24, 2006
UK
Johnson and
Johnson
National
health service
Source, Year
IMS
Health25,
2007
Multiple
myeloma
Velcade
104
J & J agreed to reimburse the NHS in either cash or product for
patients who do not respond (Response measure: 50% decrease
in serum M protein) after 4 cycles of treatment with Velcade.
Responding patients receive additional 4 cycles.
Anita Burrell
Health Care
HTA & Pricing
www.ceestahc.org
Anita Burrell
NICE recommended Velcade as a possible treatment for
progressive multiple myeloma for people:
– Who have relapsed for the first time after one treatment, and
– who have had a bone marrow transplant, if suitable for them.
After not more than four cycles of treatment, a blood or urine test
should be done to check how well the cancer has responded to
bortezomib.
Treatment should be continued only if there has been at least a
partial response to the drug.
A response-rebate scheme will allow patients at first relapse who
show a full or partial response to Velcade to carry on with the
treatment, fully funded by the NHS, and patients who show no or
minimal response to be taken off the drug and the drug costs
refunded by the drug’s manufacturer.
“This is a win-win situation for patients and the NHS.”
!
!
!
!
!
Sesja 3 / Session 3
Velcade (bortezomib)
Lucentis (ranibizumab)
!
!
!
!
!
NICE recommends that the NHS should pay for a
maximum of 14 injections of Lucentis per eye, which
should result in stable vision for most patients and
improved vision for around a quarter of patients.
It recommends that the manufacturer should pay if
any further doses are needed.
A dose-capping scheme will need to be agreed by both
the manufacturer and the Department of Health.
Responses to earlier consultation made clear that
many people felt it was unacceptable for NICE to
recommend treating only the second affected eye.
NICE has taken these concerns on board, and now
recommends treating the first eye to come to clinical
attention.
OFT on risk sharing
!
!
!
!
!
Where data at the time of launch is insufficient to take an
informed view on cost effectiveness, then, in a limited number
of cases, a risk sharing approach could be adopted.
This would require the company and payer to agree a contract
in which the drug is reimbursed, contingent on claims of
clinical effectiveness being realised in practice. This would be
assessed through information on the use of the drug in clinical
practice.
If expected outcomes are not realised, prices would be
changed and / or repayments made.
Risk sharing arrangements could in principle be particularly
relevant for the treatment of chronic (as opposed to acute)
conditions, where final clinical outcomes may only become
clear after several years of use.
However, challenges for implementation remain and risk
sharing would be the exception rather than the norm under an
ex ante approach to pricing
105
Evidence-Based
OFT on non-linear pricing
!
Sesja 3 / Session 3
!
!
!
!
We feel much could be achieved by allowing for more flexible
price structures such as price volume agreements and rebate
systems.
This would be particularly useful for drugs for which cost
effectiveness differs markedly by indication and patient subgroup.
A higher price could apply for a particular prescription volume,
reflecting the subgroup for which the drug will be particularly
effective, and a lower price for excess volumes.
The same outcome could be achieved through rebates between
companies and payers and in practice, this may be a more
practical solution.
A more flexible pricing structure would help address the concerns
that companies have incentives to incur marketing expenditure in
an attempt to increase volumes beyond those for which the drug
is cost effective.
Changes to the price structure would therefore help ensure the
incentives of firms are much more closely aligned with those of
the NHS.
“Fast Track” Back to NICE
New Process Stage 1
Company questions
decision
! -ve (10%)
! restr. (0-60%)
Existing Process
Referred to
NICE (free
pricing)
Scoping
Process to be
confidential
Consultation only if
guidance change
proposed
* Proposed price to be
implemented post +ve
NICE review. Company
reserves right to retain
original price with –
ve/restricted guidance if rereview negative
NICE
appraisal
No further
action
NICE
guidance
stands
Restricted/
No NHS
funding
Company accepts
decision
! +ve (30%)
! restr. (0-60%)
Company
proposes
flexible
option
! price/vol
! risk share
! proven
value/price
! expected
value/price
No/restricted
NHS funding
Appeal
Funding
direction
Company
proposes
revised
price*
Feedback
meeting with
NICE + DH
Company
Company
considers
considers
options
options
" re-review
! re-review
evidence
evidence
Company
meets with
DH to discuss
options
New Process Stage 2
NICE Re-review
(shortened process)
Previous
guidance
stands
+ve/restricted
guidance
Funding
direction
Financially-Based Patient Access
Schemes
These are where:
! The company does not alter the list price of the drug,
but offers effective discounts or rebates which may be
linked to (for example) the:
– Numbers or type of patients treated
– Response of patients treated
– Numbers of doses required
! Within these schemes the simplest type is one involving
an adjustment to the price the NHS pays without a need
for additional reporting of patient data as this places the
least burden on the NHS
106
Anita Burrell
Health Care
HTA & Pricing
www.ceestahc.org
Anita Burrell
Outcome-Based Patient Access
Schemes
Outcome-based Schemes can be split into three
sub-groups:
!
– Expected value: rebate. The company seeks agreement to a
price subject to the collection of additional evidence as agreed
with NICE. Such an arrangement would be subject to a rebate
and subsequent reduction in list price in the event of the
additional evidence not supporting the current price
– Risk Sharing: Outcomes are measured and price adjustments
and/or cash transfers are made in one or both directions
(between the company and the NHS) in the light of the
outcomes identified relative to those anticipated in line with the
terms of the scheme
Sesja 3 / Session 3
– Proven value: price increase: The company seeks agreement to
a later increase in price subject to a re-review of the drug in the
light of additional evidence collection as agreed with NICE.
Summary on Patient Access Schemes
!
!
!
!
!
!
Introduces a “fast track” confidential post-Guidance route into
the NHS
Financially-based Schemes offer a tool for companies to offer
flexibility to get value whilst maintaining international pricing
policies
Outcomes-based Schemes also offer a pre-agreed tool to
adjust price to reflect better evidence
Ends current ad hoc arrangements within the NICE process
Maintains company control of price setting. NICE will not set
or indicate price.
Two year review point to assess operations in practice
Potential Future
Scenarios
!
Option One:
– Increased use of the all types of the risk sharing
framework but in the same ad hoc manner as
currently observed
!
Option Two
– Decreased use of the performance
guarantee/outcomes types of risk sharing frameworks
but increased financial risk sharing
!
Option Three
– Move towards a “NICE-like” use of the risk sharing
framework so that it is an integrated part of the
pricing and/or reimbursement process
107
Evidence-Based
Sesja 3 / Session 3
108
Thank you for your
attention
[email protected]
Anita Burrell
HTA & Pricing
40 min.
www.ceestahc.org
Prelegent / Expert
Ocena stosowania szczepionki przeciwko HPV (Cervarix) u kobiet w wieku 17-25
lat. Czy negatywne decyzje refundacyjne można uzasadnić wobec opinii publicznej? / Appraisal of HPV vaccination (Cervarix) for girls/ young women aged 17-25
yrs. Can negative reimbursement decisions be justiÞed to the public?
W Holandii Narodowa Komisja Oceniająca doradza Ministerstwu Zdrowia w sprawach
związanych z refundacją. Należy tu rozróżnić
ocenę wstępną (ang. assessment) od ostatecznej (ang. appraisal). Przedmiotem oceny
wstępnej jest inkrementalny wskaźnik kosztużyteczność dla interwencji, a jej efektem są
wstępne zalecenia. Ocena ostateczna natomiast obejmuje również uwarunkowania jakościowe (np. sprawiedliwość, solidarność), co
może skutkować zmianą zaleceń wstępnych.
Aktualnie ten system oceny nie uwzględnia zagadnień kształtowania cen i podziału ryzyka.
Paradoksalnie wydaje się, że wynika to głównie
z podejmowanych przez rząd prób wzmocnienia mechanizmów rynkowych w systemie opieki
zdrowotnej. Ponieważ jednak oczekuje się, że
kryzys Þnansowy spowoduje istotne ograniczenie wydatków na ochronę zdrowia, zainteresowanie kształtowaniem cen i umowami podziału
ryzyka (popieranymi w UK, a stosowanymi np.
w Nowej Zelandii) wzrasta. W niniejszym dokumencie omówione zostaną niedawne zmiany w
holenderskim systemie oceny, w tym kryteria
stosowane aktualnie przy podejmowaniu decyzji refundacyjnych. Przedstawione zostanie
studium przypadku odrzucenia wniosku (dotyczącego stosowania szczepionki przeciwko HPV
u kobiet w wieku 17-25 lat). Przedyskutowane
będą główne argumenty za odrzuceniem wniosku oraz pytanie, czy mechanizmy kształtowania cen lub podziału ryzyka mogłyby zmienić
ten werdykt. Można podnieść argument, że
podstawowym problemem jest uzasadnienie
negatywnej decyzji refundacyjnej wobec opinii publicznej. W dokumencie podjęte zostaną
rozważania, czy ujęcie sprawiedliwości zgodnie
z teorią możliwości (ang. capabilities) Amartyi
Sena jest dostatecznie atrakcyjne i przekonujące, by spełnić tę funkcję. Omówiony zostanie
zakres informacji niezbędny dla zastosowania
teorii Sena. Zaproponowany zostanie sposób
oceny funkcjonowania systemu opieki zdrowotnej będący próbą praktycznego zastosowania
zasady sprawiedliwości w ujęciu Sena – oceny
poprzez łączną osiąganą korzyść zdrowotną
oraz (nie)równość dystrybucji tejże korzyści.
Gert van der Wilt
In the Netherlands, a National Appraisal
Committee advises the Ministry of Health on
coverage issues. A distinction is made between
assessment and appraisal: assessment results
in an estimate of the incremental cost utility
of an intervention. This leads to a provisional
advice. During appraisal, qualitative considerations (e.g., justice, solidarity) may lead to a
deviation of this provisional advice. Pricing and
risk-sharing are currently not part of this assessment – appraisal system. Paradoxically, this
seems to result largely from the government’s
attempts to reinforce market mechanisms in
the health care system. However, the Þnancial
crisis is expected to necessitate very substantial cuts in the health care budget, and the interest in pricing and risk-sharing schemes as
have been advocated in the UK and practiced
in for instance New Zealand, has considerably
increased. In this paper, recent developments
in the Dutch assessment – appraisal system
will be explained, including the criteria that are
currently used in coverage decisions. A case
study will be explored, where an application
was turned down (HPV vaccination for girls /
young women aged 17 – 25 years). The main
arguments for turning down the application will
be presented, and the question whether pricing
or risk-sharing might have made a difference
will be discussed. It will be argued that the key
problem is justiÞcation of negative reimbursement decisions to the public. The paper will explore whether Amartya Sen’s capability account
of justice could be sufÞciently appealing and
convincing to fulÞll this role. The informational
requirements of Sen’s capability account will be
discussed. A framework in which a health acre
system’s achievement is expressed in terms of
aggregate health gains and (in)equality of the
distribution of these gains will be proposed as
a possible operationalisation of Sen’s account
of justice.
Sesja 3 / Session 3
Health Care
109
Evidence-Based
CEESTAHC, Krakow 2009
Sesja 3 / Session 3
Appraisal of HPV vaccination
(Cervarix) for girls/ young women
aged 17 – 25 yrs
Prof dr G J van der Wilt
Department of Epidemiology, Biostatistics & HTA,
Radboud University Nijmegen Medical Centre/
Athena Institute, VU University, Amsterdam, NL
Outline
• Appraisal system in the Netherlands
• No pricing / risk-sharing (yet)
• Case study: appraisal of HPV
vaccination for 17 – 25 yrs (turned
down)
• Would pricing / risk-sharing have
made a difference?
• Discussion: how can negative
reimbursement decisions be
justified to the public?
Appraisal in the Netherlands
Council for Public Health &
Health
Care
National Health Insurance Board
Ministry of Health
National Appraisal Committee
BENEFIT
PACKAGE
110
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
aim of the National Health
Insurance Board: “to enable
universal access to an affordable
basic benefit package that provides
for necessary care”
Criteria for coverage: necessity,
effectiveness, cost effectiveness,
and sustainability
Council for Public Health
Sesja 3 / Session 3
National Appraisal Committee
Recommendations for appraisal committee
Assessment: provisional advice, based on costeffectiveness
Appraisal: may deviate from provisional advice
on the basis of qualitative considerations
(e.g., solidarity, justice)
Severity of disease / costeffectiveness
111
Evidence-Based
Stakeholder consultation
CVZ staff
Sesja 3 / Session 3
draft recommendation
stakeholder consultation
appraisal committee
final recommendation
Ministry of Health
Case study: HPV vaccination, 17 – 25 yrs
Woodman et al. Nature Reviews Cancer 7, 11–22 (January 2007) | doi:10.1038/nrc2050
112
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
Schiffman et al,
The Lancet 2007;
370: 890 - 907
High spontaneous clearance rate
Sesja 3 / Session 3
Progression of disease
Schiffman et al,
The Lancet 2007;
370: 890 - 907
Modeling future impact of
vaccination
Overview of epidemiologic
structure of multi-HPV type
model. HPV infection may
progress to either genital
warts or cervical disease, with
regression possible for HPV
infection, CIN grades 1–3 and
genital warts. Only cervical
cancer confers an added risk
of mortality, as depicted in the
figure. However, in the full
model (not shown for
simplicity) all individuals face
an underlying age and sexspecific mortality rate due to
non-cervical cancer-related
causes. CIN = Cervical
Intraepithelial Neoplasia; HPV
= Human Papillomavirus.
BMC Infect Dis. 2009; 9: 119
113
Evidence-Based
Sesja 3 / Session 3
Most Common and Other Selected qHPV Adverse Events Following
Immunizationin the United States, Reported to VAERS, 2006 - 2008
Slade, B. A. et al. JAMA 2009;302:750-757.
Copyright restrictions may apply.
Application turned down; main
arguments
• Cost effectiveness not sufficiently supported by
data (too optimistic assumptions regarding
efficacy, duration of protection, cross
protection, compliance)
• Budget impact
• No reliable method for risk stratification
• False sense of security
• National screening programme
• Ultimate impact on risk of cervical cancer
unknown
• Model not transparent
Valuation of health gain
Expected health gain per woman:
0.0127 – 0.0145 QALYs
(4 - 5 Quality Adjusted Life Days)
114
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
Incremental costs
• Incremental costs per woman (lifetime):
Incremental Cost Utility Ratio
Sesja 3 / Session 3
€274,51 - €277,06
Costs / QALY:
€ 18.930 / QALY - € 21.815 / QALY
Could negotiations with the
manufacturer have made a
difference?
115
Evidence-Based
Sesja 3 / Session 3
Model assumptions, baseline
Duration of protection: life long
Efficacy of the vaccine in preventing
HPV infection:95%
Overall cross protection: 31%
Attendance: 100% (all women, 3
vacinations)
Overly optimistic?
Sensitivity analysis
Sensitivity analysis of 209 different
parameter sets that reproduced
Canadian data specific to human
papillomavirus (HPV) type for infection,
cervical intraepithelial neoplasia, cervical
cancer and genital warts. These graphs
plot the numbers needed to vaccinate to
prevent an episode of genital warts and
a case of cervical cancer. For the base
case, it is assumed that the vaccine
efficacy is 95%, the duration of
protection is lifelong and the age at
vaccination is 12 years. *DNP = does
not prevent outcome.
CMAJ. 2007 August 28; 177(5): 464–
468.
Justifying negative decisions
116
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
How can a decision to exclude an
intervention from coverage be justified?
(if at all)
Sesja 3 / Session 3
Key question:
David Wiggins (Winchester, Oxford):
Whatever decision is reached, or
whatever recommendation is made, it
should be such that it can be seen, or
recognized as just by citizens who are
committed to justice.
Inconsistency in appraisal system
• First, estimate incremental cost - utility
• Decide, provisionally, on the basis of ICUR
• Deviate from provisional advice, if this seems
appropriate on the basis of considerations of
justice or solidarity
Problem:
• Allocation of resources on the basis of ICUR is a
specific interpretation of distributive justice:
utilitarianism
117
Evidence-Based
Sesja 3 / Session 3
Utilitarian principle:
Allocate resources such, that
the ICUR in all directions of
expenditure is equal (Pareto
optimality);
In practice: cover costs only
when ICUR < threshold level (a
society’s monetary valuation of
a QALY)
Utilitarianism
Egalitarian? Yes: “A QALY is a QALY is a
QALY” (Alan Williams)
Basic problems with utilitarianism:
• Assumption of commensurability
• Insenstive to distribution (concerned
with utility maximization at the
aggregate level)
Alternatives:
John Rawls
A theory of justice (1971) (Justice as fairness)
Thought experiment:
“veil of ignorance”
Principles:
• maximal individual freedom, compatible with
equal degree of freedom for all;
• Fair equality of opportunity;
• “maxi-min”
118
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
Alternatives: Sen
Capability approach
Sesja 3 / Session 3
• Amartya Sen: the capability approach
• Capability: the capacity and
opportunity that people have to do
the things that they have reason to
value;
• Circumstances that can have a
major impact on people’s
capabilities (poverty, disease)
• Unequal distribution of capabilities
• Health care (policy): aimed at
reducing these inequalities
HPV, cervical cancer, and capabilities
• Are women’s capabilities
importantly affected by cervical
cancer?
• Does cervical cancer contribute to
significant inequalities in
capabilities?
• Should interventions be aimed at
reducing such inequalities?
• Are there any opportunities to do
this?
• If so, how (cost)effective are these?
119
Evidence-Based
Sesja 3 / Session 3
• Vaccination or improvement of
current screening programme?
• The Netherlands (population: ca. 16
Million)
• Annual incidence of cervical cancer:
ca. 700
• Annual mortality from cervical
cancer: ca. 200
Who benefits from screening?
Active participation rates according to age
and the combinations of country of birth
(Nl, The Netherlands; N.West, nonWestern countries; West, Western
countries) and socioeconomic status. Van
Leeuwen et al, Cancer 2005; 105: 270-6.
Achievement: health gains + its
distribution
Achievement plane. (Clarke & Hayes, 2009)
120
Gert van der Wilt
Health Care
HTA & Pricing
www.ceestahc.org
Gert van der Wilt
Bootstrap replications for the incremental change in absolute inequalities and in the
difference in mean health. (Clarke & Hayes, 2009)
Changes in population health and its
distribution over time
Sesja 3 / Session 3
Uncertainty
Achievement plane for changes in
risk factor prevalence and
inequality compared to 1989 in 3
successive National Health
Surveys. a) Risk factors smoking,
high cholesterol, high blood
pressure and heart disease; and
b) obesity, overweight, diabetes
and no exercise. The dashed line
represents the line of no change
in achievement, with points above
this line representing increasing
achievement compared to 1989.
(Clarke & Hayes, 2009)
Conclusions
• Negative reimbursement decisions:
socially & politically extremely sensitive
• Pricing / risk-sharing schemes would, in
this particular case, not have made the
difference
• This is partly so because cost-utility
provides an insufficient basis for public
justification
• Alternative conceptions of justice
worthwhile to explore
• Different informational requirements!
121
Evidence-Based
Sesja 3 / Session 3
• From a capability perspective:
• Use resources to improve
attendance to screening among
disadvantaged groups, rather than
to extend the vaccination
programme to 17 – 25 year old
girls / women, provided that (cost)
effective interventions are available
to achieve this
• Normative issue: which guiding
principle for our health care
system?
• Measurement problem
• Issue of causality (and effective
intervention)
Bertrand de Jouvenel (1903 – 1987)
“Every immediate fields of choice open to us, in
either private or public capacity, offers us
opportunity for the exercise of justice. Whenever
we miss this opportunity we feed the sum of
social injustice –a sum which it is comfortable but
untrue to regard as the product of some single
institution or mode of arrangement.”
122
Gert van der Wilt
HTA & Pricing
Thank you for your attention!!
www.ceestahc.org
Gert van der Wilt
Sesja 3 / Session 3
Health Care
123
Evidence-Based
Prelegent / Expert
Cena innowacyjnego produktu leczniczego z perspektywy globalnego producenta farmaceutycznego / The price of the innovative drug from the perspective of global pharma company
Erin Huntington
Sesja 3 / Session 3
Cena innowacyjnego produktu leczniczego z perspektywy
globalnego producenta farmaceutycznego
The price of the innovative drug from the perspective
of global pharma company
ERIN HUNTINGTON
What the innovative drug is?
- for a company
- for a medicine
- for a patient
- for a payer…
Declining number of innovative molecules during the last
years because of:
- increasing R&D costs
- individuality of treatment – „personalized medicines”
proposal: Slides from EFPIA
124
30 min.
Health Care
HTA & Pricing
www.ceestahc.org
Erin Huntington
Current status of practices across EU countries
(some coutries regulatory approval + pricing negotiation,
role of HTA in the reimbursement process especially in
the light of pricing)- I believe that David’s input may be
tremendously valid
Sesja 3 / Session 3
EU different policy towards pricing of medicines
The price of innovative drugs is not only the production cost…
Broder policy implication…
New stakeholders:
- Ministry of Economy
- Ministry of Finance
- Prime Minister
As the outcome of the variety in our environment…
The most valid practices on the markets outside Europe
Different definition:
- Floor Price…
- Public Price…
- Fixed Prices vs. Maximum Prices
125
Evidence-Based
Sesja 3 / Session 3
Different definition of prices:
- Floor Price…
- Public Price…
- Fixed Prices vs. Maximum Prices
The price versus overall spendings
Risk Sharing systems from pharma company perspective…
Lilly position statement
Polish challanges for proper drug policy in the coming
years – Duda’s input
The role of national payer negotiation body…
Industry attempt:
… oficial negotiation: the price, reimbursement level & risk sharing
agreement with MoH…
… establishment of the national negotiation body should be ensured by
the reimbursement act,
- it was placed in the draft of reimbursement act prepared and applied
by INFARMA in 2008
- in parallel, MoH is working on a new Reimbursement Act – still no
further information on the contents
126
Erin Huntington
HTA & Pricing
25 min.
www.ceestahc.org
Prelegent / Expert
Wartość farmakoterapii i rola HTA w umowach podziału ryzyka
The value of drug therapy and the role of HTA in risk-sharing arrangements
W najnowszej umowie zbiorowej dotyczącej
kształtowania cen leków (Pharmaceutical Price
Regulation Scheme) wprowadzonej w Zjednoczonym Królestwie uwzględniono zasadę wyceniania leków zgodnie z ich wartością terapeutyczną. Zasada ta umożliwia zarówno elastyczne
kształtowanie cen leków, jak i zawieranie umów
typu „patient access schemes” (termin zalecany
zamiast „umowa podziału ryzyka”). W obu przypadkach procedury odnoszą się do ocen dokonywanych przez NICE, tj. agencję oceny technologii
medycznych. Jak dotąd nie mamy doświadczenia w zakresie elastycznego kształtowania cen w
oparciu o ocenę technologii medycznych. Omówiono szereg przykładów umów podziału ryzyka
zawartych zgodnie z nowymi zasadami.
Chociaż zawarte umowy są uzasadnione efektywnością kosztową (zgodnie z ocenami NICE),
to jednak pozostają bardziej rozwiązaniami Þnansowymi niż umowami podziału ryzyka w
oparciu o rzeczywistą wartość leków.
Jim Furniss
The new Pharmaceutical Price Regulation
Scheme in the UK recognises the arguments for
a value-based approach to pricing, and adopts
the principle in its provisions on both ßexible
pricing and patient access schemes (the preferred term of risk sharing agreements). In both
cases the procedures are speciÞcally linked to
assessments by NICE, the Health Technology
Assessment agency. As yet there has been no
experience of ßexible pricing based on health
technology assessment, but a number of examples of risk sharing under the new arrangements
are reviewed. While the risk sharing schemes
that have been adopted have been justiÞed on
the basis of the impact on cost effectiveness, as
assessed by NICE, they are all Þnancially-based
schemes rather than true performance-based
risk sharing.
Sesja 3 / Session 3
Health Care
127
Evidence-Based
Sesja 3 / Session 3
The value of drug therapy and the role of HTA
in risk-sharing arrangements
4th International EBHC Symposium
Krakow
7th December 2009
1
Confidential © Bridgehead International Ltd
Bridgehead International
Contents
Slide number
1
The OFT and value based pricing
3
2
The new 2009 PPRS scheme
8
3
Flexible pricing
10
4
Patient access schemes(risk sharing)
14
5
Conclusions: The potential and limitations for companies with
innovative products
28
2
The Office of Fair Trading (OFT) was critical of the
pricing scheme for drugs in the UK
The Pharmaceutical Price
Regulation Scheme
An OFT market study
Published in
February 2007
Conclusions:
• Profit and price controls do not reflect the value of drugs
• The PPRS does not benefit patients
• The PPRS does not encourage investment
Recommendations:
• Value based pricing for branded medicines
• Reference pricing for generic medicines
http://www.oft.gov.uk/shared_oft/reports/comp_policy/oft885.pdf
128
3
Jim Furniss
Health Care
HTA & Pricing
www.ceestahc.org
Jim Furniss
The OFT proposed two main options for reform, to
deliver value based pricing
• Option 1 - Ex Post Value Based Pricing
#
#
Retaining up-front freedom of pricing for companies
Replace company wide profit controls and price cuts with a series of expost reviews of the cost effectiveness of individual drugs or drug classes
• Option 2 - Ex Ante Value Based Pricing
#
#
In addition to aspects of option 1, it would involve a fast track ex-ante
assessment of a new drug’s cost effectiveness.
Rapid decision could be made on appropriate pricing
− NICE Single Technology Appraisal model
4
Sesja 3 / Session 3
− NICE standard technology appraisal model
How would it operate?
• The maximum price of a product would be set according to the clinical
benefit it delivers relative to an appropriate comparator
#
The comparator might be generic (innovation would not be rewarded per
se)
− Therapeutic reference pricing?
− A possible brand premium of up to 50% may apply
• Manufacturers would submit a suggested price, along with cost
effectiveness evidence, which might differ across indications
• An analysis of value-reflective prices would be undertaken using
existing HTA agencies
#
#
#
National Institute for Health and Clinical Excellence (NICE)
Scottish Medicines Consortium
All Wales Medicines Strategy Group
• The evidence would then be used by the Department of Health to
negotiate price
• Products and therapy areas would be subject to periodic review
5
Commission on the Value of Medicines
• For the longer term, the OFT recommends a Commission on the Value
of Medicines (CVM)
#
#
To integrate HTA bodies
To take account of industry and patient group views on HTA
• This could initially be introduced without legislation
#
But any formal merging of NICE, SMC and AWMSG would require
legislation
• This body could evolve (again with legislation) to become a Medicines
Pricing Commission which also carried out the DoH price function
#
#
#
This would add to regulatory stability in principle (akin to Bank of England
independence)
This would require legislation to implement
This is therefore considered a very long term option
6
129
Evidence-Based
Advantages and challenges
Advantages
• Value based pricing
#
Price reflects the value to patients and the NHS
• Fairness to companies and recognition for effectiveness
• More flexible price structures
Sesja 3 / Session 3
#
A risk sharing approach could be adopted when data is not sufficient on cost
effectiveness
Challenges
• Principles for Assessing Cost Effectiveness
#
Value Based Pricing will need to take into account the incremental benefits the
drugs will bring, which may be different for different patients or indications
• Informational Requirements
#
It is difficult to demonstrate the clinical and cost effectiveness of a drug,
especially at launch when clinical experience is limited
• Institutional Design
#
The credibility of the institution carrying cost effectiveness should be high e.g.
NICE
7
The 2009 PPRS agreement
8
The 2009 PPRS is a radical change from previous
agreements
• The new PPRS agreement1, effective from 1st January 2009, includes
a number of new elements:
#
#
#
#
#
A price cut from 1st February 2009, and scheduled price changes in future
years to 2013
Consultation on the introduction of generic substitution
Flexible pricing
Patient access schemes (risk sharing)
A study on the uptake of innovative products
• In addition, NICE has:
#
#
Implemented a review of procedures, with a view to speeding up
assessments
Made new provision for the treatment of “end-of-life medicines”
• This presentation focuses on flexible pricing and patient access
schemes
1
http://www.dh.gov.uk/en/Healthcare/Medicinespharmacyandindustry/Pharmaceuticalpriceregulationscheme/2009PPRS/index.htm
130
9
Jim Furniss
HTA & Pricing
Flexible pricing
10
www.ceestahc.org
Jim Furniss
Sesja 3 / Session 3
Health Care
The new flexible pricing provisions reflect one of
the criticisms made by the OFT
• Recognises that the initial launch indication of a medicine, and the
supporting evidence available at that time, may not fully reflect its
longer-term value to patients
• Allows a company to propose an initial price that reflects value at
launch, but with the freedom to increase (or decrease) price as further
evidence or new indications emerge
• Flexible pricing will only apply to medicines subject to NICE appraisal
#
#
A review by NICE will be required to determine whether the revised price
provides value to the NHS
Prices for medicines not selected for NICE review may only be adjusted
using the normal modulation provisions
• Price increases under flexible pricing are limited to 30%, and only one
increase is permitted during a product’s life
11
The process for gaining approval for a price increase is
complex and hedged with restrictions
No
Is medicine subject to
NICE appraisal?
Price adjustment only via
modulations
Yes
Major new indication likely to have
significantly different value
Flexible pricing applies
Significant new evidence
Launched after
1/1/2009
Company initiates
procedure (STA or MTA
Launched before
1/1/2009
NICE initiates procedure
(STA or MTA)
NICE appraisal using
current procedure
Launched before
1/9/2007
No NICE
appraisal
No increase
possible
Positive NICE appraisal
for new indication
Price increase up to 30% possible,
once in product lifecycle
Positive NICE
assessment
Negative NICE
assessment
Price increase
No price increase
Launched after
1/9/2007
Price increase for new
indication
Implement price
increase for new
indication after 12
months
Discount or rebate for
existing indications
12
131
Evidence-Based
The process for gaining approval for a price increase is
complex and hedged with restrictions
No
Is medicine subject to
NICE appraisal?
Price adjustment only via
modulations
Yes
Major new indication likely to have
significantly different value
e of
enc ns
i
r
e
p
Launched after
before
visio Launched
1/9/2007
1/9/2007
o ex
Launched before
is n ese pro
e
1/1/2009
r
e
h
t using
h
No NICE
No increase
ofappraisal
et, t tionNICE
current procedure
appraisal
possible
NICE initiates
s yprocedure
a
A
(STA or MTA) er
p
o
Positive NICE appraisal
the
Flexible pricing applies
Sesja 3 / Session 3
Significant new evidence
Launched after
1/1/2009
Company initiates
procedure (STA or MTA
Price increase up to 30% possible,
once in product lifecycle
for new indication
Price increase for new
indication
Positive NICE
assessment
Negative NICE
assessment
Price increase
No price increase
Implement price
increase for new
indication after 12
months
Discount or rebate for
existing indications
13
Patient access schemes
(risk sharing)
Definitions
The process
Some case studies
• Revlimid
• Sutent
• Erbitux
• Stelara
14
The new PPRS agreement did not initiate risk
sharing schemes in the UK
A number of risk sharing schemes had already been introduced
• Treatments for multiple sclerosis (2002)
#
#
An example (the only one?) of a true risk sharing scheme
Patient outcomes to be monitored over a ten year period, and prices
adjusted according to the outcomes achieved in practice
• Lucentis (2008)
#
Novartis agreed with NICE to pay for the drug cost of treatment beyond 14
injections per patient
• Tarceva (2008)
#
#
Following initial rejection by NICE, Roche announced 27.5% interim price
cut to make it equivalently priced with competitor pending results of appeal
Tarceva accepted by NICE on appeal at discounted price
• Velcade (2008)
#
#
Janssen renegotiated with NHS to refund cost of the first 4 cycles if there is
no clear patient benefit
If there is benefit, a patient can continue with next 4 cycles
132
15
Jim Furniss
Health Care
HTA & Pricing
www.ceestahc.org
Jim Furniss
Definition
• To facilitate earlier patient access to medicines that are not in the first
instance found to be cost and clinically effective by NICE
• “Schemes proposed by a pharmaceutical company and agreed with The
Department [of Health] (with input from NICE) to improve the cost
effectiveness of a drug and enable patients to receive access to
innovative medicines”
• Applies only to England and Wales (not Scotland)
Key principles: Schemes should be:
• Clinically robust, clinically plausible, appropriate and monitorable
• Operationally manageable for the NHS without unduly complex
monitoring, disproportionate additional costs and bureaucracy
• Consistent with existing financial flows in the NHS (costs and savings
must accrue to local services making commissioning and treatment
decisions)
There will be a review of patient access schemes after not more than 2 years
16
Sesja 3 / Session 3
“Patient access schemes” is the preferred
terminology for “risk sharing”
Four types of “patient access schemes” are
identified
Financially-based schemes:
• The company offers effective discounts or rebates which may be
linked to:
#
#
#
Numbers or types of patients treated (price-volume agreements)
Response of patients treated (includes an “outcome” dimension)
Numbers of doses required
Outcome-based schemes:
• Proven value: price increase
# Price is agreed subject to re-review and increase in the light of
additional evidence collection as agreed with NICE
• Expected value: rebate
# Agreement to a price subject to additional data collection and rereview; rebate and/or subsequent price reduction if additional
evidence does not support the current price
• Risk sharing
# Clinical or patient reported outcomes (PROs) are measured and
price adjustments and/or cash transfers made (in either direction)
in the light of actual versus anticipated outcomes
Based on
assessed costeffectiveness
Based on direct
measurement of
outcomes (true
risk sharing)
17
The process for patient access schemes includes both the
DH and NICE, but DH has the power of veto
NICE single
technology appraisal
At outset
(with
initial NICE
submission)
NICE multiple
After final
NICE guidance
Short fixed period
for application
Agree in
principle
with DH
Includes
consultation
with NHS
Agree in
principle
with DH
Includes
consultation
with NHS
Agree in
principle
with DH
Submit to NICE at start of
MTA process
NICE determines detail of scheme
(including duration, review and termination)
18
133
Evidence-Based
Patient access schemes: the Revlimid example
Revlimid (lenalidomide)
followed the single technology
appraisal process
NICE single
At outset
Sesja 3 / Session 3
(with
initial NICE
submission)
Celgene proposed the risk
sharing after the draft FAD
After final
NICE guidance
Short fixed period
for application
Agree in
principle with
DH
Includes
consultation
with NHS
The scheme was agreed by DH
(presumably after consultation
with the NHS)
NICE updated their assessment
in the light of:
Agree in
principle
with DH
• The risk sharing proposal
• The new criteria for end-of-life
medicines
Outcome
Costs to the NHS are capped
Patients have access to Revlimid
19
Revlimid: The outcome
The recommendation
• “Lenalidomide in combination with dexamethasone is recommended, within its licensed
indication, as an option for the treatment of multiple myeloma in people who have
received two or more prior therapies, under the following circumstances:
#
#
The NHS will cover the cost of the drug for the first 2 years (26 cycles of 28 days) of treatment
The drug cost of lenalidomide (excluding any related costs) for people who remain on treatment
for longer than 2 years will be met by the manufacturer”
End of life treatment
• “The Committee was satisfied that the population and the technology of interest meet
the criteria for accepting that this is an appraisal of a life-extending, end-of-life treatment
and that the evidence presented for this consideration was supported by robust data”
The numbers
• Taking into account the limitation on patient numbers, and the price capping scheme,
the assessed cost per QALY was in the range £41,300 to £43,800 (the company’s
corresponding figures were £28,941 to £30,350)
• The predicted average savings from the cost capping were in the range £3,500 to
£8,000, applying to between 11% and 17% of patients.
• Average lifetime treatment costs (with the cost cap) were estimated at between £46,300
and £51,800.
http://www.nice.org.uk/guidance/index.jsp?action=article&o=43041
20
Treatments for renal cell carcinoma were being assessed
using the multiple technology appraisal process
NICE single
At outset
(with
initial NICE
submission)
NICE multiple
After final
NICE guidance
Short fixed period
for application
Agree in
principle
with DH
Includes
consultation
with NHS
Agree in
principle
with DH
Sutent
Includes
consultation
with NHS
Agree in
principle
with DH
Submit to NICE at start of
MTA process
But Sutent has been extracted and treated as if it were a single technology appraisal
134
21
Jim Furniss
Health Care
HTA & Pricing
www.ceestahc.org
Jim Furniss
Sutent benefited from both the patient
access scheme and the end-of-life medicine
provisions
• Sutent for first-line treatment of advanced and/or metastatic renal cell
carcinoma was taken out of the MTA process and treated as an STA
#
“The manufacturer of sunitinib (Pfizer) has agreed a patient access scheme
with the Department of Health, in which the first treatment cycle of sunitinib is
free to the NHS. The Department of Health considered that this patient access
scheme does not constitute an excessive administrative burden on the NHS”
• Cost per QALY for the target patient population was calculated
#
“The agreed pricing strategy of the first cycle of sunitinib being free to the NHS
resulted in an ICER of £54,366 per QALY gained for sunitinib compared with
IFN#$”
• The technology was then assessed against the end-of-life treatment
criteria
#
“The Committee was satisfied that sunitinib currently meets the criteria for
being a life-extending end-of-life treatment, and that the evidence presented for
this consideration was sufficiently robust”
22
Sesja 3 / Session 3
• A risk sharing scheme was agreed with DH
Erbitux (cetuximab) followed the single
technology appraisal process
• The appraisal considers the use of cetuximab in combination with
FOLFOX and in combination with FOLFIRI as possible first treatments for
patients with metastatic colorectal cancer
• Following ACD* consultation, Merck-Serono (the manufacturer) submitted
revised analyses which incorporated a patient access scheme
#
#
#
#
This analysed cetuximab in combination with FOLFOX compared with FOLFOX
alone
The scheme is based on a 16% rebate of the amount of cetuximab used when
given in combination with FOLFOX for people with KRAS wild-type metastatic
colorectal cancer who have metastases confined to the liver
Cetuximab would normally be rebated in the form of free stock at a rate of 16%
for all patients in the scheme on a per patient basis, with an option for rebate
via credit note or cash
The scheme requires that patients are treated according the final NICE
guidance and that data should be provided to the manufacturer to show that
NICE guidance has been followed
*appraisal consultation document
23
Erbitux (cetuximab) followed the single
technology appraisal process
• The patient access scheme does not apply to patients treated with
cetuximab in combination with FOLFIRI
• Length of treatment with cetuximab is limited
#
Patients should not receive cetuximab for more than 16 weeks (whether
treated with FOLFIRI or FOLFOX); at this time the patient should be
assessed for resection of liver metastases
• Cost per QALY was calculated for both combination treatments
#
#
For cetuximab in combination with FOLFOX compared with FOLFOX alone
gave ICER of between £26,700 and £33,300 per QALY gained (with the
rebate)
For cetuximab in combination with FOLFIRI compared with FOLFIRI alone
an ICER of £23, 500 per QALY gained
24
135
Evidence-Based
Ustekinumab for the treatment of adults
with moderate to severe psoriasis
• Following a single technology appraisal, Stelara
(ustekinumab) is recommended as a treatment option for adults with
moderate to severe plaque psoriasis
Sesja 3 / Session 3
• The patient access scheme means that the cost to the NHS will be the
same for any size patient - whether they require a 45mg dose or a
90mg dose
#
#
#
The SPC recommends that people whose body weight exceeds 100kg
should receive a dose of 90 mg of ustekinumab; for those under 100kg, the
dose is 45 mg
Under the scheme, for people who weigh more than 100 kg and who are
prescribed the 90 mg dose (two 45 mg vials), the manufacturer will provide
both vials at a total cost of £2147 (the cost of a single vial)
The Department of Health considered that this patient access scheme
does not constitute an excessive administrative burden on the NHS
25
Without the patient access scheme
ustekinumab could not be considered a costeffective use of NHS resources
• Without the patient access scheme the ICERs for ustekinumab would be
£41,000 per QALY gained compared with supportive care, £102,000 per
QALY gained compared with intermittent etanercept 25 mg, and £300,000
per QALY gained compared with adalimumab
#
#
The Committee concluded that ustekinumab could not be considered a costeffective use of NHS resources without the patient access scheme
The manufacturer proposed that the patient access scheme is to remain in
place until either a review of the guidance by NICE or the introduction of any
new formulations that would render the scheme obsolete, and that it would not
be withdrawn without the agreement of NICE and the Department of Health
• The estimates of cost effectiveness that included the patient access
scheme were considered as reasonable
#
In the manufacturer’s base-case analysis (including the patient access
scheme) ustekinumab had an ICER of £29,600 per QALY gained compared
with supportive care, an ICER of £27,100 per QALY gained compared with
etanercept 25 mg given intermittently
26
Potential patient numbers in each Patient Access
Scheme
Drug
Indication
Eligible patient group
Estimated no.
of patients
Revlimid
(lenalidomide)
Multiple myeloma in people
who have received two or
more prior therapies
All patients
2100 (1)
Sutent
(sunitinib)
Advanced and/or metastatic
renal cell carcinoma (RCC)
Those suitable for
immunotherapy and with
an ECOG performance
score of 0 or 1
Total no. with
advanced and/or
metastatic RCC
is ~4000 (1)
Sutent
(sunitinib)
Unresectable and/or
metastatic GIST after failure
of imatinib treatment
All patients
90 to 150 (1)
Erbitux
(cetuximab)
Metastatic colorectal cancer
(MCC)
Patients receiving
cetuximab in
combination with
FOLFOX
1,632 (2)
Moderate to severe plaque
psoriasis
Patients weighing
>100kg
20 (in 2009) to
785 (in 2013) (2)
Stelara
(ustekinumab)
Estimates taken from (1) NICE guidance documents and (2) manufacturer submissions
136
27
Jim Furniss
HTA & Pricing
Conclusions: The potential and limitations for
companies with innovative products
28
www.ceestahc.org
Jim Furniss
Sesja 3 / Session 3
Health Care
Conclusions: The PPRS provisions can work
• Revlimid, Sutent ,Erbitux and Stelara provide the first examples of the
operation of the new “patient access schemes” provisions in the 2009
PPRS agreement
#
All are essentially financial schemes
− Revlimid and Stelara are effectively cost cap schemes
− With Sutent the company provides the first month of treatment without charge –
effectively a discount
− Erbitux is a simple discount
#
These are not different in essence from some previous agreements in the UK
#
They illustrate the flexibility of the new schemes
#
They illustrate the limitations of the new schemes
− Lucentis was a cost cap
− Tarceva was a simple discount
− Sutent was separated out from the initial multiple technology appraisal
− Avastin was not included the patient access scheme for mRCC because of its other
indications, making monitoring difficult
• Revlimid and Sutent also provide the first examples of the use of the new
“end-of-life” criteria by NICE
29
Conclusions: Implications for companies with
innovative products
• Risk-sharing will remain the exception, not the rule, for most products
#
But may become the preferred pricing mechanism for high priced products
with small patient populations and limited evidence of efficacy or costeffectiveness IF current schemes prove successful to the DoH or the NHS
− Should current schemes become too difficult or costly to manage, or show no
appreciable impact on cost, risk-sharing will be supplanted by another costcontrol mechanism
#
No systematic evaluation of the impact of risk-sharing has yet been
conducted
− A review is promised after two years
• The onus is on companies to decide whether to propose risk sharing
schemes, and what kinds of scheme to propose
#
The scheme must
− Reflect the characteristics of the product and therapy area
− Offer benefits to the payer
− Be workable within the context of the NHS
#
Where there are (or will be) competing products, a well-designed risk
sharing scheme may provide a competitive advantage
30
137
Evidence-Based
Sesja 3 / Session 3
138
Contact
Jim Furniss
Bridgehead International Ltd
Pera Innovation Park
Nottingham Road
Melton Mowbray
Leicestershire, LE13 0PB
United Kingdom
T: +44 1664 503 700
F: +44 1664 503 705
www.bridgehead.com
[email protected]
31
Jim Furniss
Health Care
HTA & Pricing
www.ceestahc.org
Sesja 3 / Session 3
139
Wtorek
8 grudnia 2009
Tuesday
December 8th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 4 / Session 4
Wartość terapeutyczna technologii nielekowych z perspektywy
przemysłu / Value of non-drug technologies – industry perspective
Rod Taylor – 30 min.
Mitchell Sugarman – 30 min.
Choć polityka cenowa w państwach Europy Centralnej i Wschodniej zmienia się w
ostatnich latach, wciąż daleko do spełnienia
wymogów Dyrektywy Transparentności UE w
tym zakresie. Decydenci stosują różnorodne
metody mające na celu ustalenie cen produktów wyrobów medycznych na rodzimych
rynkach. Decyzje dotyczące ustalania cen
leków i wyrobów medycznych często podejmowane są w oparciu o niepełne informacje
dotyczące skuteczności danej technologii lub
algorytmy trudne do zastosowania w praktyce klinicznej. Istotną rolę w takich sytuacjach odgrywają nowoczesne rozwiązania
„inżynierii Þnansowej” w obszarze świadczeń
Þnansowanych ze środków publicznych takie
jak: porozumienia podziału ryzyka (risk sharing agreements), refundacja warunkowa
(CED, coverage with evidence development)
lub ich zmodyÞkowane formy tzw. payback
czy price-volume agreement. W przypadku wyrobów medycznych instrumenty te są
szczególnie przydatne ze względu na to, że
w momencie wprowadzania na rynek nowego wyrobu, informacje o jego skuteczności
bywają bardzo ograniczane. Jest to związane ze stosunkowo łagodnymi w porównaniu
z produktami leczniczymi wymogami w zakresie niezbędnej dokumentacji, a z drugiej
strony z trudnościami w przeprowadzeniu
badań RCT dla takich technologii (szczególnie tych stosowanych w chirurgii).
W czasie sesji przedstawione zostaną
możliwe rozwiązania w zakresie oceny wartości terapeutycznej i wyceny technologii
nielekowych oraz możliwości i zagrożenia
związane z ich specyÞką.
Although pricing policy in countries of
Central and Eastern Europe has changed
over the last years, there is still much to
be done in order to fulÞll demands of the
EU Transparency Directive with this respect. Decision makers apply various methods
for pricing of medicinal products on their
home markets. Decisions concerning pricing
of medications and medicinal products are
often based on incomplete information concerning efÞcacy of a speciÞc technology or
algorithms difÞcult to implement in clinical
practice. In such circumstances modern solutions of “Þnancial engineering” concerning
services Þnanced from public means, such
as: risk sharing agreements, conditional reimbursement (coverage with evidence development – CED) or – in a modiÞed form
– payback or price-volume agreements have
an increasingly important role to play. These
instruments are especially useful in case of
medical devices, taking into account the fact
that at the time of marketing of a speciÞc
product information concerning its efÞcacy
may be very limited. This is due to requirements regarding necessary documentation,
relatively less rigorous as compared to those concerning medicinal products, as well as
difÞculties associated with RCTs in case of
such technologies (especially those used in
surgery).
During the session possible solutions concerning assessment of therapeutic value and
pricing of non-drug technologies as well as
opportunities and threats related to speciÞcity of non-drug technologies will be presented.
141
Evidence-Based
Prelegent / Expert
Sesja 4 / Session 4
Ocena wyrobów medycznych jako wyzwanie dla HTA: punkt widzenia oceniającego / The HTA challenge of medical device assessment: The perspective of
assessor
142
Głównym celem oceny technologii medycznych (HTA) jest ocena efektywności klinicznej
i kosztowej technologii medycznych, tj. leków,
wyrobów medycznych i badań diagnostycznych. Wiele agencji, jak np. brytyjski National Institute for Health and Clinical Excellence
(NICE), stawia te same wymagania w dziedzinie HTA wszystkim technologiom medycznym.
Wyrobów medycznych dotyczą jednak istotne
uwarunkowania wpływające na ocenę ich efektywności klinicznej i kosztowej. Obejmują one
zakres danych dotyczących licencjonowania,
interakcje operator-urządzenie oraz innowacje
inkrementalne.
Na przykładzie stymulatora rdzenia kręgowego (urządzenia stosowanego w leczeniu
bólu) zilustrowane zostaną wyzwania w zakresie HTA, jakie wyroby medyczne stawiają oceniającemu.
Rod Taylor
Health technology assessment (HTA) primarily seeks to assess the clinical and cost effectiveness of medical technologies that can include
drugs, devices and diagnostic tests. For many
agencies, such as the National Institute for Health and Clinical Excellence (NICE) in UK, the
HTA requirements are the same for all medical technologies. However, there are important
speciÞc medical devices considerations that
impact on the assessment of their clinical and
cost effectiveness. These include the evidence
requirements of licensing, device-operator interaction and incremental innovation.
Using the example of the spinal cord stimulation (a medical device technology designed to manage various pain indications), this
presentation will illustrate the HTA challenges
of medical devices from the perspective of the
assessor.
30 min.
HTA & Pricing
30 min.
www.ceestahc.org
Prelegent / Expert
Aspekty polityczne refundacji technologii nielekowych z perspektywy przemysłu
The Industry Perspective; Policy and Reimbursement for Non-Drug Technologies
Kontynuując temat omówiony przez profesora Roda Taylora, tj. różnice pomiędzy technologiami lekowymi i nielekowymi, w niniejszej sesji
skupimy się na związanych z nim zagadnieniach
politycznych. Przyjrzymy się szczególnie, jak
producenci urządzeń radzą sobie w warunkach
różnych systemów refundacji obowiązujących w
różnych krajach i jak opracowywana jest ogólna
strategia refundacji. Jako przykład posłuży stymulacja rdzenia kręgowego.
Mitchell Sugarman
Continuing along the theme outlined by
Professor Rod Taylor about the differences between drug and non-drug technologies, this
session will examine the associated policy issues. SpeciÞcally, the session will address how
device companies must navigate the differing
reimbursement systems from country to country and how a broad reimbursement strategy is
developed. Spinal cord stimulation will be used
as an example.
Sesja 4 / Session 4
Health Care
143
Wtorek
8 grudnia 2009
Tuesday
December 8th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 5 / Session 5
Rozwój HTA w krajach Europy Centralnej i Wschodniej
– ostatnie osiągnięcia i zmiany
Developments of HTA in CEE countries
Paweł Vorobyev, Oleg Borisenko – 25 min.
Dragana Atanasijevic – 25 min.
Rabia Kahveci – 25 min.
Alexandre Lemgruber – 25 min.
W trakcie sesji przedstawione zostaną
ostatnie dokonania na polu implementacji
EBHC i HTA w poszczególnych krajach naszego regionu. Sytuacja w krajach Europy
Centralnej i Wschodniej w odniesieniu do
rozwoju instytucji zajmujących się HTA jest
bardzo zróżnicowana. Wymiana doświadczeń w zakresie kreowania praktyki HTA, ze
względu na podobne doświadczenia, nie tylko o charakterze społeczno-gospodarczym,
ale także historycznym, wydaje się szczególnie cenna. Z jednej strony pozwala w dłuższej perspektywie uniknąć kosztownych niepowodzeń korzystając z doświadczeń innych
państw, z drugiej jest pomocna w promowaniu nowych, często nowatorskich rozwiązań,
które mogą stanowić wkład własny państw
naszego regionu w rozwój HTA.
W czasie sesji poruszony zostanie szeroki
wachlarz problemów, od rozwiązań w zakresie organizacji agencji HTA, tworzenia listy
leków refundowanych, polityki cenowej po
edukację w zakresie HTA/EBM. Na sesje zaproszeni zostali reprezentanci agencji HTA,
ministerstw zdrowia oraz innych instytucji
zajmujących się tą dziedziną.
During the session recent achievements
in the Þeld of EBHC and HTA implementation
in countries of our region will be presented.
The situation with respect to development of
HTA institutions varies considerably among
speciÞc countries of Central and Eastern Europe. Regarding common experience, not
only in socioeconomic but also historical sense, exchange of experience concerning HTA
practice seems especially valuable. On one
hand, learning from each other’s experience
can make it possible to avoid costly failures
in a longer perspective; on the other, such
exchange is helpful with respect to promotion of new, innovative solutions which may
become our region’s own contribution to HTA
development.
During the session a wide range of problems will be discussed, including solutions
in organization of HTA agencies, creation
of lists of reimbursed drugs, pricing policy
and education in HTA/EBM. Representatives
of HTA agencies, ministries of health and
other institutions involved in HTA development have been invited to take part in this
session.
145
Evidence-Based
Prelegent / Expert
Sesja 5 / Session 5
Wpływ farmakoekonomiki na kształtowanie cen w Rosji: doświadczenia i perspektywy / Impact of pharmacoeconomics on pricing in Russia: experience and
perspective
Przedmiotem niniejszego raportu jest kształtowanie cen leków w Rosji. W Rosji regulacja cen
odbywa się poprzez ustalanie ograniczeń ceny
sprzedaży oraz marży hurtowej i detalicznej,
co dotyczy tylko leków o znaczeniu podstawowym. Ceny leków nie wpisanych na Listę Leków
o Podstawowym Znaczeniu nie są w ogóle regulowane. Istniejące mechanizmy kontroli wzrastających kosztów leków nie działają – w ciągu
8 miesięcy roku 2009 średnia cena opakowania leku wzrosła o 35%, a sprzedaż zmalała o
7,7%. System kształtowania cen jest niestabilny
z uwagi na znaczny wpływ wahań kursów głównych walut. Farmakoekonomika nie ma zastosowania przy kształtowaniu cen; nie stworzono też
systemu cen referencyjnych. Refundacja leków
dotycząca tylko 10% populacji oraz słaba regulacja cen znacznie ograniczają dostępność leków
dla pacjentów. W sierpniu 2009 r. Rząd FR ogłosił nowe zarządzenie (nr 654) mające zmienić
istniejące mechanizmy kształtowania cen leków.
Zarządzenie to nie wprowadza jednak fundamentalnych zmian w zakresie regulacji cen.
Paweł Vorobyev
This report focuses on drug pricing issues in Russia. In Russia price regulation is the
registration of limiting selling prices and the
establishment of wholesale and retail markup
which affects only vital and essential drugs. Pricing of drugs which are not included into the
Vital and Essential Drug List is not regulated
at all. Existing mechanisms of controlling the
increasing cost of drugs do not work – in the
pharmacy segment the average cost of package has grown by 35% and sales has fallen by
7,7% for 8 months of 2009. Pricing system is
unstable; it is signiÞcantly affected by changes
in the major currencies. Pharmacoeconomics is
not used in the price registration, there is no
reference price system. Drug reimbursement
provided only for 10% of the population and
poorly regulated pricing signiÞcantly reduce the
availability of medicines for the population. In
August 2009 the Government of RF approved
new enactment (№654), which should change
existing mechanisms to drug pricing, however
it does not include fundamentally new approaches to the price regulation.
* łączny czas z wykładem Olega Borisenki / joined time for two experts: Pavel Vorobyev and Oleg Borisenko
146
25 min.*
Health Care
HTA & Pricing
perspective
www.ceestahc.org
Paweł Vorobyev
Impact of pharmacoeconomics
on pricing in Russia:
experience and perspective
How are expenses of medications
paid in Russia (ambulatory)?
• Provision with essential drugs (ONLS,
former DLO) – 2 mln. of people (1.4% of
population of Russia)
• Regional benefit – 8 mln. of people (5.6%
of population of Russia)
• “7 nosologies” – 76.000 of people (0.05%
of population of Russia)
• Purchasing of drugs by population
Sesja 5 / Session 5
Professor Pavel Vorobyev
President of ISPOR Russian Chapter
How are prices on medications
regulated?
• Registration of marginal price for vital and
essential medications
• Fixation of marginal wholesale and retail
bidding to sale prices on medications from
Vital and Essential Drug List
• Pricing of medication, not included in Vital
and Essential Drug List (previously – in
DLO) is not regulated absolutely
* Decree of Government of Russian Federation from 09, November, 2001, "782
147
Evidence-Based
perspective
Pricing in pharmacy (free sale)
• Custom price
• Custom duty (11%) + value added tax
(VAT, 10%)
• Price markup of distributor (not less than 2
distributors) (practically 30% from each
one)* + VAT (10%)
• Price markup of pharmacy (up to 30%) +
VAT (10%)
* Despite of data of Stature on State Regulation of Prices, approved by Decree of Government of Russian
Federation, 9, November, 2001, # 782 (III.16): the sum of wholesale markups of all wholesale
organizations, taking part in selling of medication on the territory of region of Russian Federation must
not exceed relevant marginal wholesale markup, proved by executive legislative body of this region of
Russian Federation
Sesja 5 / Session 5
Forming of wholesale and retail
markups
*
• It can be fixed by executive legislative body of
regions of Russian Federation only for the
medications from the Vital and Essential Drug
List
• It is regulated by Decree of Government of RF
from 7, March, 1995 #239 and Decree of
Government of RF from 9, November, 2001
#782
• Prices and assortment of production in hospitals
and pharmacies are controlled by
Roszdravnadzor selectively for 130 drugs (Order
of Ministry of Public Health and Social
Development of RF from 27, May,2009 #277n )
In practice, markups are overpriced
• Rostov Region*: retail markups are 25-45%
despite of limited 23%; wholesale markups are
27.5-68.6% despite of limited 10%.
• Kaluga Region**: retail markups are 34-60%, at
the same time fixed one is 30%
• Izhevsk***: retail markups are 40%, fixed ones –
35%
• Nizhniy Novgorod****: wholesale markups are
17-25%, fixed ones –15%
* http://www.donland.ru/rst/Inform_organizatsijam/2065.pdf
** http://www.regnum.ru/news/fd-central/kaluga/medicine/1195636.html
*** http://www.kprf-udm.ru/news-archive/2586-lekarstva
**** http://www.niann.ru/?id=360408
148
Paweł Vorobyev
Health Care
HTA & Pricing
perspective
www.ceestahc.org
Paweł Vorobyev
Total markups for the sale
•
•
•
•
•
328% - Detralex (Servier)
196% - Actovegin (Nycomed)
108% - Essentiale forte (Sanofi Aventis)
122% - Linex (Lek)
45% - Viagra (Pfizer)
Forming of price
•
•
•
•
ONLS – tender
“7 nosologies” - tender
Regional benefit - tender
Custom are performed according Federal
Law #94
• Conditions: the lowest price of drugs, in
one region – the presence of data on
bioequivalence for generics
Sesja 5 / Session 5
* DSM Group, 09.2009
Chaotic energies on the
regulation of pricing
• Decree of President of RF, 28, February, 1995 #221
• Regulation of Government of RF, 07, May, 1995
#239
• Regulation of Government of RF, 9, November,
2001, #782
• Order of Ministry of Public Health and Social
Development of RF, 19, October, 2004 #165
Development of RF, 31, December, 2006 #907
Development of RF, 27, May, 2009 #277n
• Regulation of Government of RF, 8, August, 2009 #
654
149
Evidence-Based
perspective
Registration of prices
• Mandatory registration of sale price of
producer on medication from Vital and
Essential Drug List (for the present time –
only 25% of drugs have registered price)
• Registered prices are reflected in State
Register of Drug Prices (24 issue) *
• Registered prices sometimes are higher
than real sale prices, prices are not newly
registered for several years
*http://www.roszdravnadzor.ru/i/upload/files/1256753238.44581-27164.pdf
Sesja 5 / Session 5
Comparative evaluation of prices of drugs, rub.
Registered price according to LVSD,
rubles
Price in pharmacy (Moscow), rubles
Captopril
Tab.25 mg #20
6,00 - 26,37
4,22 39,00
(Capoten 57,00 - 160,00)
Enalapril
Tab.2.5 mg #20
3,89 - 47,99
4,09 - 125,10
Atenolol
Tab.50 mg #30
16,50 - 48,00
5,10 - 50,00
Formoterol
Caps.pulv. For inject.
12 µg #30
825,35
611,50 - 1098,48
Amoxicillin
Caps.250 mg #20
46,25
12,14 - 45,00
Azithromycin
Caps.250 mg #6
78,53 - 220,00
79,50 - 590
Co0trimoxazole
Tabl.480 mg #20
10, 20 39,91
7,00 - 25,90
IUN
At the present times
• Present mechanisms of price growth containment
are ineffective – during 8 months of 2009 year the
medium price of drug raised on 35%, and sales
volume decreased on 7.7% in pharmacy*
• System of prices is not stable, it is liable to
significant influence of rates of exchange
• Pharmacoeconomics is not used in registration of
price, the system of referent pricing is absent
• Reimbursement of drugs for only 10% of
population of Russia and lack of regulation of
pricing significantly decreases of access to drugs
for people
* DSM Group, 09.2009
150
Paweł Vorobyev
Health Care
HTA & Pricing
perspective
New initiatives
of Government of RF*
www.ceestahc.org
Paweł Vorobyev
Who can develop it and
which will they be?
• 2 methods are in development: determination of
fixed sales prices of producer for medicines from
Vital and Essential Drug List, method of
definition of wholesale and retail markups to
Vital and Essential Drug List
• Monthly monitoring of prices in pharmacies by
Roszdravnadzor
• Re-registration of prices of all medicines from
Vital and Essential Drug List up to 1, March,
2010 according new method
• New mechanisms of control over innovations are
developed
New initiatives of Government of
RF (2)
• Substantiation during registration of price of
Russian producer must include a data on price
of drug with information of IUN and form of
production in Russia
• Substantiation during registration of price of
foreign producer must include reference data on
price of drug in country-producer
• Executive legislative body of regions of Russia
can fix wholesale and retail markups to all drugs,
but not only to ones included in Vital and
Essential Drug List
Sesja 5 / Session 5
* Decree 8, August, 2009 #654 “On perfection of state regulation of prices of vital and sufficient drugs”
151
Evidence-Based
Prelegent / Expert
Sesja 5 / Session 5
Znaczenie oceny nowego leku przy wprowadzaniu go na rynek w Rosji
Value of new drug assessment during market access in Russia
Jak dotąd nie udowodniono efektywności
znacznej liczby leków Þnansowanych w Rosji
(dla 41 z 655 leków wpisanych na Listę Leków o
Podstawowym Znaczeniu nie ma danych o skuteczności). Co więcej, żaden zespół zajmujący
się (formalnie lub nieformalnie) oceną technologii medycznych nie działa w ramach Ministerstwa Zdrowia i Rozwoju Społecznego ani innej
instytucji publicznej. W roku 2007 Ministerstwo
Zdrowia i Rozwoju Społecznego zawiesiło również prace nad standaryzacją w opiece zdrowotnej, w tym standaryzacją wyboru leków. Decyzje dotyczące Þnansowania leków podejmowane
są arbitralnie. W ciągu ostatnich 5 lat program
Þnansowania leków nie objął żadnego nowego,
efektywnego leku. Jednocześnie w ramach Rosyjskiej Akademii Nauk Medycznych powstała organizacja zajmująca się oceną technologii
medycznych, która opracowała metody podejmowania decyzji dotyczących wyboru technologii medycznych. Wyniki tych prac nie zostały
jednak wzięte pod uwagę przez władze.
Oleg Borisenko
A great number of drugs without proven effectiveness are Þnanced in Russia (41 of 655
drugs in the Vital and Essential Drug List have
no evidence of efÞcacy). Moreover there is no
any formal or informal service for health technology assessment in the Ministry of Health and
Social Development or in other public authorities. Also since 2007 the Ministry of Health
and Social Development has suspended work
on standardization in health care, including the
standardization in the selection of drugs. Decisions on Þnancing drugs are taken arbitrarily.
For the past 5 years the program of drug Þnancing has not included any new effective drug. At
the same time there is an organization for assessment of medical technology in the Russian
Academy of Medical Sciences, which had worked out approaches to the decision making for
choice of medical technology, however public
authorities do not take into account the results
of its work.
* łączny czas z wykładem Pavla Vorobyeva / joined time for two experts: Pavel Vorobyev and Oleg Borisenko
152
25 min.*
Health Care
HTA & Pricing
www.ceestahc.org
Oleg Borisenko
Value of new drug
assessment during market
access in Russia
Which drugs are paid in Russia?
• Provision with essential drugs (ONLS) – ONLS
list
• Regional benefit – region’s own list of drugs
• «7 nosologies» - there’s 17 medications
• Also there is separate Vital and Essential Drug
List (approved by Government of RF in the latter
case in 2007), hospital drugs are paid according
to this list - 655 medications. It correlates with
ONLS
Sesja 5 / Session 5
Oleg Borisenko
Executive Director
ISPOR Russian Chapter
Large quantity of drugs without proven
effectiveness is used in Russia
• 40 from 655 drugs from
Vital and Essential Drug
List have no proven
effectiveness
Table – top 10 drugs
• In 2007, about 20% of
outlay in addition
provision of drugs (DLO)
were spent on drugs
without proven
effectiveness, according
the opinion of experts of
Formulary Committee of
Russian Academy of
Medical Sciences
• Population spends
money for nonsense at
all
153
Evidence-Based
Ministry of Health Care and Social
Development breaks of all scientific activity
Sesja 5 / Session 5
• In 2007, MoH stopped standardization in public health in
fact, standards of medical care are not approved
• Specialists of standardization are separated from their work
• Normative documents, regulating the development of Vital
and Essential Drug List on the basis of proves of
effectiveness and pharmacoeconomics, are not used. They
are replaced with “copying”, but inexact documents
• In 2009, the development of protocols of care was stopped
• MoH does not interact with progressive scientists, councils,
and structures, namely with Russian Academy of Medical
Sciences
How decisions are made?
• In Russia the treatment of Gaucher's disease is
financed (200 000 EURO in year per patient,
160 patients)
• In Russia the treatment of inhibitor haemophilia
is financed (1.3 billion EURO in year per patient,
40 patients)
• But mucopolysaccharidosis (I and II types) is not
financed (200 000 EURO in year per patient,
100 patients) – several children die every year
How decisions are made?
• Arbitrarily, according to politic moment
• DURING LAST 5 YEARS THE PROGRAM OF
FINANCING DID NOT INCLUDE ANY NEW
EFFECTIVE DRUG (ONCOLOGY,
RHEUMATOLOGY, HEMATOLOGY)
• MoH propose searching charitable organizations
as an answer for all requests for help
• A FORMAL OR UNFORMAL SERVICE FOR
HEALTH TECHNOLOGY ASSESSMENT IN
STRUCTURE OF MINISTRY OF PUBLIC HEALTH
OND SOCIAL DEVELOPMENT IS ABSENT
154
Oleg Borisenko
Health Care
HTA & Pricing
www.ceestahc.org
Oleg Borisenko
Health technology assessment in
Russia
The Formulary Committee – HTA
activities
• Evaluation of applications for inclusion of drugs
in the List of essential drugs, Orphan drugs and
in Negative List from producers and experts
• Placing applications at the web-site
• Standard procedure of evaluation
• Three levels of examination (secretariat,
professional group, presidium)
• Decision-making by consensus
Sesja 5 / Session 5
• There is no any formal service in Ministry
of health structure
• There is no understanding that such
service is necessary
• The Formulary Committee has been
working at the evaluation of medical
technologies for 12 years
155
Evidence-Based
Prelegent / Expert
Sesja 5 / Session 5
HTA w krajach rozwijających się – obciążenie czy konieczność?
HTA – burden or need for developing economies
156
Systemy opieki zdrowotnej w krajach bałkańskich zorganizowane są w oparciu o tradycyjny, trójstopniowy podział na jednostki opieki
podstawowej oraz drugo- i trzeciorzędowej. W
niektórych krajach dokonano ostatnio decentralizacji podstawowej opieki zdrowotnej pozostawiając ją w gestii samorządów lokalnych; w
innych krajach nastąpi to wkrótce. Niezależni
lekarze podstawowej opieki zdrowotnej są (lub
wkrótce będą) wynagradzani w systemie kapitacyjnym. Systemy opieki zdrowotnej są Þnansowane z wielu źródeł, w tym donacji, ale
przede wszystkim przez Fundusze Ubezpieczenia Zdrowotnego stanowiące podstawowe
źródło Þnansowania świadczeń na wszystkich
poziomach opieki. Jak dotąd w dziedzinie opieki
zdrowotnej nie działają inne instytucje ubezpieczeniowe (rządowe ani prywatne). Osoby
ubezpieczone w Funduszu otrzymują „książeczkę zdrowia” umożliwiającą bezpłatny dostęp do
publicznego systemu opieki zdrowotnej oraz
nabywanie leków z częściową lub całkowitą
refundacją. Aktualnie nie ma pewnych danych
statystycznych dotyczących odsetka populacji
objętego ubezpieczeniem zdrowotnym w krajach bałkańskich. Ocenia się, że ok. 30-90%
osób pracujących jest objęte ubezpieczeniem
(w zależności od kraju).
Podział systemu na poziomy opieki stwarza
wyjątkowo dogodne warunki do oceny technologii medycznych. W takim systemie oferowane
świadczenia i wyposażenie ośrodków powinny
być dostosowane do problemów zdrowotnych
występujących w populacji. W rzeczywistości
jednak systemy opieki zdrowotnej borykają się
z problemami „okresu przejściowego”. Oprócz
braku standardów wyposażenia ośrodków na
różnych poziomach brak jest również standardów świadczeń. Standardy takie powinny
stanowić „serce” systemu i być oparte na tak
ścisłych danych, jak to możliwe. Skutkuje to
zatarciem podziału ośrodków na poszczególne
stopnie referencyjności. Pacjent może na przykład przejść całą drogę od najniższego poziomu opieki (pomoc w przypadkach nagłych) do
szpitala uniwersyteckiego, ale też może od razu
znaleźć się w ośrodku o najwyższym poziomie
referencyjności, pomijając etapy pośrednie.
Zaburza to gromadzenie danych statystycznych dotyczących świadczeń. Innym trudnym
do oceny czynnikiem jest liczba pacjentów nie-
The health systems of the Balkan countries
are organized on the traditional three levels of
care-primary, secondary and tertiary. Primary
health care in some countries have recently
been decentralized to the local governments or,
in some other countries it is going to be very
soon. Independent general practitioners are
paid by a capitation system or they are going
to face it very soon as well. The health care
systems are Þnanced from the number of sources including donations, but mostly by Health
Insurance Funds as a major player in the recurrent Þnancing of services at all levels, since
there are no other governmental or private insurance funds providing health coverage to the
population jet. Members of the HIF are issued a
‘health booklet’ which allows them free access
to the public healthcare system, as well as to
purchase drugs with or without co-payment.
There are currently no hard statistics as to
what percentage of the population has health
insurance coverage; it is estimated though that
approximately from 30% to 90% of the ‘active’
(working) population is covered in different countries of Balkan.
That kind of systems, split at the levels of
care, potentially provides an extremely useful
framework for health technology assessment.
In such system, the provision of services and
equipment follows the health problems of the
population. But what we can see in reality characterizes health systems in transition countries. Apart of lacking standards for equipment
that is appropriate at each level, there is lack
of standards of level of services as well. Such
standards are part of the “heart” of a system
and should be based on good evidence whenever is possible. As a result of that, there is
no clear picture of the referral system among
the levels of care. Namely, the patient may go
regularly through the system from the bottom
level (emergency service) to the University hospital, or may go directly to the third level of
care skipping the all previous steps, and that
number of services stays unidentiÞed from
the routine statistics. Another unknown fact
is number of unnecessary referred cases sent
from secondary to tertiary level of care.
The consequence of Þnancing modiÞcation
in some countries is that top hospital management started to think about services that their
25 min.
HTA & Pricing
potrzebnie kierowanych z ośrodków drugo- do
trzeciorzędowych.
Zmiany systemu Þnansowania w niektórych
krajach skutkują zmianami sposobu postrzegania przez dyrekcje szpitali udzielanych przez nie
świadczeń oraz problemu zakupu wyposażenia.
Pojawiają się zespoły złożone z techników, ekonomistów i klinicystów, których zadaniem jest
ocena wartości sprzętu i wyposażenia oraz potrzeb w tym zakresie w kontekście efektywności
i opłacalności. Mogłoby się wydawać, że jesteśmy na najlepszej drodze do zastąpienia dominującego dziś w większości systemów podejmowania decyzji w oparciu o opinię ekspertów
podejściem systematycznym, uwzględniającym
zasady EBM, EBHC i HTA. W rzeczywistości jednak wciąż możliwe jest wprowadzenie do systemu całkowicie nowej procedury bez uprzedniej oceny jej skuteczności i bezpieczeństwa
względnie oszacowania, czy jej Þnansowanie
jest możliwe w ramach dostępnych środków.
Istotnym aspektem bezpiecznej i skutecznej
opieki zdrowotnej opartej na danych naukowych jest rynek leków. Wszystkie wspomniane
kraje dokładają starań, by dostosować obowiązujące w tym zakresie regulacje prawne do dyrektyw Unii Europejskiej. Mimo że w niektórych
krajach działania regulacyjne pozostawiono w
gestii Ministerstwa Zdrowia, w innych powołano
niezależne agencje lekowe w celu uczynienia
systemu bardziej transparentnym.
www.ceestahc.org
institutions provide or equipment that should
be purchased. In that sense, there is a very
new trend of having “cost ofÞces” composed
of engineers, economists and clinicians, with a
common task to assess the need for new equipment and its value in a term of effectiveness
and cost effectiveness. At a Þrst glance it sounds as a basic systematic approach and should
be considered as a potential starting point for
EBM, EBHC and HTA development in these countries since that nowadays their systems work
mostly on opinion based expertise. However, in
realty if someone wants to introduce completely new procedure to the system it may be done
without asking for any permission or examination of its efÞcacy and safety, or is it justiÞed to
Þnance this service within available resources.
Going deeper to the issue of providing evidence
based, safe and effective care we are approaching pharmaceutical service. All mentioned
countries show strong orientation towards the
European Union wishing to bring into line pharmaceutical legislation with EU directives. And
even that some countries retained the technical
regulation functions within the Ministries of Health others set up independent drug agencies
as a way to the better transparency.
Sesja 5 / Session 5
Health Care
157
Evidence-Based
HTA
burden or need for developing
economies
Sesja 5 / Session 5
Dr Dragana Atanasijevic
[email protected]
Presentation outline
• HTA countries, who and where are they?
• Why is HTA needed?
How to better use available resources?
• Everything In Its Own TimeTime to do things properly
HTA countries, who and where are they?
European countries in respect to the
development and use of HTA in
decision-making
158
Health Care
HTA & Pricing
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Indexes that measure the
development of a country
GDP per capita - Life expectancy - Literacy rate - …..
HDI - the level of human development
•Life expectancy at birth, as an index of population health
and longevity
•Knowledge and education, as measured by the adult
literacy rate and the combined primary, secondary, and
tertiary gross enrollment ratio
•Standard of living as measured by the natural logarithm of
GDP per capita at PPP
0.950 and over
0.900–0.949
0.850–0.899
0.800–0. 849
0.750–0.799
0.700–0.749
0.650–0.699
0.600–0.649
0.550–0.599
0.500–0.549
Sesja 5 / Session 5
World map indicating the Human
Development Index
0.450–0.499
0.400–0.449
0.350–0.399
under 0.350
Data unavailable
Source: UN Development Program , Human Development Report 2009, compiled on the
basis of data from 2007
Some European countries by Human
Development Index
10 highest HDIs
10 lowest HDIs
HDI
HDI
Rank
Country
Change
2007 data compared to
2006 data
1
Norway
2
Iceland
0.969
3
Ireland
0.965
0.971
4
Netherlands
0.964
5
Sweden
0.963
6
France
0.961
7
8
Switzerland
Luxembourg
0.960
9
Finland
0.959
10
Austria
0.955
0.960
% +0.001
% +0.002
% +0.001
% +0.003
% +0.002
% +0.003
% +0.001
% +0.001
% +0.004
% +0.003
Rank
Country
Change
2007 data compared to
2006 data
1
Moldova
0.720
2
Ukraine
0.796
% +0.002
% +0.007
3
Bosnia and Herzegovina
0.812
% +0.005
4
Macedonia
5
Russia
6
Albania
7
Belarus
8
Serbia
0.826
9
Montenegro
0.834
10
Romania
0.837
0.817
% +0.004
0.817 [nb 2] % +0.011
0.818
% +0.004
0.826
% +0.007
% +0.005
% +0.006
% +0.005
Source: UN Development Program , Human Development Report 2009, compiled on the
basis of data from 2007
159
Evidence-Based
Income divisions
Sesja 5 / Session 5
Source: Valerie Moran, Human Development Network, World Bank, 2009
Predominance of formal HTA agencies
in high-income European countries
Source: EUNETHTA WP8. Systems to support Health Technology Assessment (HTA) in member states
with limited institutionalization of HTA
Funding of HTA organization
Funding of HTA organization
Income/ available budget
Source of funding
N
%
Agency
Government
33
80.5
UK NCCHTA
Research funding bodies
19
46.3
GERMANY
GERMANY IQWIG
Private industries (e.g. pharmaceutical industry)
10
24.4
Academia/University
10
24.4
Donor agencies (foundations, patient associations, charity, 7
others)
17.1
Public health care providers
7
17.1
Compulsory health care insurance (public)
6
14.6
Intergovernmental organizations
3
7.3
Private medical insurance
3
7.3
Private health care providers
3
7.3
Amounts
21,6 MIL USD
DAHTA 1,5 MIL USD
11 MIL Euro
AUSTRIA
LBI of HTA
BELGIUM
0,93 MIL USD
KCE
3,06 MIL USD
LATVIA VSMTVA
0,05 MIL USD
NETHERLANDS
CVZ
10,3 MIL USD
FRANCE
HAS
60 MIL Euro
Source: EUNETHTA WP8. Systems to support Health Technology Assessment (HTA) in member states
with limited institutionalization of HTA
160
Health Care
HTA & Pricing
www.ceestahc.org
Total Health Expenditures Per Capita
in US $
4000
3500
3000
2500
2003
2004
2000
2005
1500
1000
500
M
RO
E
BU
G
GR
NG
AC
M
M
IT
A
AU
S
HU
NG
CH
R
CY
P
CR
O
B
BH
SL
AL
SL
SR
K
B
0
Sesja 5 / Session 5
Why is HTA needed?
How to better use available resources?
Total Health Expenditures Per Capita as
Purchasing Power Parity (US $)
i.e. in the
currency that has the same purchasing power in every country (PPP)
5000
4500
4000
3500
3000
2500
2000
1500
1000
500
q
M
ac
ed
Gr
.
ee
ce
d,
k
Bu
lga
ri a
Ro
m
an
ia
f
gr
o
y
tri
a
Au
s
te
ne
on
M
Ita
l
ru
s
ga
ry
Cy
p
Hu
n
ep
ck
R
BI
H
at
ia
Cr
o
Ch
e
ia
an
g
Al
b
a
va
ve
ni
Slo
Slo
Se
r
bi
aq
,r
kia
0
161
Evidence-Based
How to reconcile the principles of solidarity and
efficiency?
•
•
•
•
•
•
•
Ongoing decentralization
Changing of payment mechanisms
Social health insurance is the dominant model
No other governmental or private insurance funds
Coverage is uneven (30% to 90% )
Health spending is low
To visit specialists, patients need to be referred by a general
practitioner , three levels referral system
• Patients who visit specialists without a referral are required to
pay out-of-pocket money
• Services purchased at the private centers - 100% out of pocket
• Transitional economies , no transparency, corruption …
Sesja 5 / Session 5
Health Survey, Serbia 2006
Utilization of health services
Ways of obtaining drugs in the adult population
1.1
2000
No purchasing –
drugs are expensive
Buying in pharmacy
57.1
39.4
0.2
By prescription
44.4
2006
54.1
0
10
20
30
40
50
60
% population
Utilization of health services
Obtaining drugs in the adult population by the wealth index
% population
0
10
20
30
40
50
60
70
Serbia
Poorest
Poorer
Middle class
Rich
Richest
162
By prescription
Buying
Health Care
HTA & Pricing
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HEALTH CARE PAYMENTS
“out of pocket”
% total expenditures
Outpatent sevices (public)
Outpatent sevices (private)
Dental sevices (public)
Dental sevices (public)
Diagnostic sevices (public)
Diagnostic sevices (public)
Payments for drugs
Complementary medicines
Alternative medicine
Hospital care (public)
Hospital care private)
Rehabilitation
Medical divaces- othopaedic and other
Direct (informal) payment
Others
5
10
15
20
25
30
35
40
45
50
5.5
9.4
4,0
17.7
3.9
7.9
3.3
0.4
1.3
0.5
0.5
0.5
1.4
.
42.4
.
1.2
Lack of knowledge, experience,
transparency… (1)
•
•
•
•
•
•
No inventory of medical equipment
No standards for medical equipment
No needs assessment
No priorities approach in health policy
No cost effectiveness analysis
No estimates of the future recurrent costs for the nation and the
health care facility
• No commitment on how to cover these recurrent costs
• No clear picture of the referral system among the levels of care
• No number of unnecessary referred cases sent from secondary to
tertiary level of care
Sesja 5 / Session 5
0
Lack of knowledge, experience,
transparency… (2)
• No awareness of counterfeit medicines problem and
weaknesses in inspection and detection ability
• No provision for Pharmacovigilance
• No regulatory provision for Clinical Trials
• No regulatory provisions exist for import and control of
Active Pharmaceutical Ingredients
• Hospital sector drug procurement organised centrally
failures in decentralization because of lack of staff training
in drug procurement practices
• Drug Needs Assessment is difficult in view of the absence
of hospital protocols / clinical guidelines
• Drug Usage Evaluation is not carried out at any level of the
system
163
Evidence-Based
What are the expert’s remarks?
Sesja 5 / Session 5
• Complexity of the local context and decisionmaking process
• Political instability
• Poor communication between stakeholders
• No legislative framework
• Low decision-making transparency
• Attitudes of decision-makers (bureaucracy)
• Financial discouragement
• Inconsistency in following expert
recommendations leads to the limited
sustainability of results
Everything In Its Own Time
Time to do things properly
Two ways demands
Increasing pressure on governments
In-country
• Decentralisation
• Health reform initiated – new
financing mechanisms
• Lack of resources - “cost
offices” composed of
engineers, economists and
clinicians
• Increased demand for
purchasing newer and newer
technologies
• Requirements from the new
structures: licensing,
accreditation or quality
assurance bodies
164
Out-country
• Raising awareness through
different projects
• International contacts
• Links wit the HTA
community
• Skilled and committed
groups of “working bees”
• Demand for more
transparent decisionmaking
Health Care
HTA & Pricing
www.ceestahc.org
“value for money” principle and transparency requirements
– introducing health technology assessment
HTA provides evidence for
• Effective benefits package
and public health
methodologies
• Appropriate distribution of
technology over the
country, region or level of
care (buildings, equipment,
drugs, supplies)
• Referral criteria and it
fosters cooperation
between levels of care
Key question
If HTA Agency - then what kind of HTA Agency?
– Developing an ‘arms length’ multi skilled public agency for HTA, funded
primarily from public resources, as adopted in e.g. the UK: The National
Institute for Clinical Excellence – a model which is being adopted widely
throughout Europe;
– The development of an EBM/HTA network (real or ‘virtual’) linking
various competent agencies/university departments, which could be
moulded into a single service;
– A commissioning model with the Ministry of Health/Health Insurance
Fund/ Agency contracting out various aspects of HTA to competent
agencies and authorities in different fields, as undertaken in USA;
– The development of unit to specifically collaborating in a broader
international network such as Cochrane Collaborating Centre, EUnetHTA
etc;
– Developing a ‘twinning’ approach involving a well-established
international agency such as NICE to provide specific HTA, EBM and CEA
expertise, using that Agency’s materials for initial activities as well as
undertaking substantial knowledge transfer activities leading to
independent operation
– Or, the adoption of a combination of these approaches.
Sesja 5 / Session 5
• Structural aspects of the
quality of health care
• Clinical practice guidelines
and clinical pathways
(which technology, on what
indication, by when and to
which patient)
• Prevention from patients
exposure to harmful
interventions
The decision -Heavy, light or mixeddepends on the health system needs and
available funds
165
Evidence-Based
Estimated costs of a HTA Report
Costs per individual
analysis depending
on the type of the HTA
institution
Heavy
€
26 654
Mixed
€
10 316
Light
€
5 926
Costs of
of overall
analysis and
developing a
report
Costs of quality
control of an
individual
report
Sesja 5 / Session 5
Source: Feasibility study on HTA Institutionalization in Serbia, 2008
166
Health does not know for borders
• EBM/HTA awareness raising
• Create EBM/HTA Journals/Bulletins
• Access to international full text medical literature
databases
• PhD HTA-related research
• EBM / HTA Reference library (electronic and print)
• Cochrane on-line library access
• Formal networks: e.g. INAHTA, HTAi , EUNetHTA, or
regional CEESTAHC (concept of leapfrogging- reduction
in duplication, new and improved methodological
developments…)
HTA & Pricing
25 min.
www.ceestahc.org
Prelegent / Expert
Przepisy dotyczące kształtowania cen w Turcji i ich wpływ na refundację. Jaka jest
potencjalna rola HTA? / Pricing Regulations in Turkey, effects on reimbursement.
What is the Potential Role for HTA?
Zapoczątkowany w roku 2003 Projekt Transformacji Opieki Zdrowotnej przyniósł radykalne
zmiany w tureckim systemie opieki zdrowotnej.
Jego celem było zwiększenie efektywności systemu poprzez poprawę zarządzania, skuteczności,
satysfakcji pacjentów i świadczeniodawców oraz
osiągnięcie trwałej równowagi budżetowej. Jego
kluczowe elementy obejmowały: wprowadzenie
Powszechnego Ubezpieczenia Zdrowotnego, powołanie pojedynczego Instytutu Bezpieczeństwa
Społecznego, rozszerzenie i ułatwienie dostępu
do opieki zdrowotnej oraz uczynienie jej bardziej
przyjazną, jak również wprowadzenie racjonalnego zarządzania zużyciem leków oraz materiałów i wyrobów medycznych.
Powszechne ubezpieczenie zdrowotne zapewnia pełną opiekę 72-milionowej populacji, której
znaczna część przed jego wprowadzeniem nie
była objęta ubezpieczeniem. Wydatki na opiekę
zdrowotną, już szybko rosnące, wzrosły jeszcze
bardziej wraz ze wzrostem liczby uprawnionych
do niej, jak również starzeniem się populacji i
rosnącym zapotrzebowaniem na nowe technologie. Alarmująco wzrosły wydatki na leki, osiągając około 40% całkowitych wydatków na opiekę
zdrowotną. W roku 2004 dostrzeżono potrzebę
wprowadzenia Polityki Cenowej w odniesieniu do
leków. Ceny leków były bardzo wysokie w porównaniu z innymi krajami, a refundacja obejmowała niemal wszystkie leki. Można wręcz powiedzieć, że nie istniała polityka refundacyjna ani
jakiekolwiek kryteria włączenia lub wykluczenia
leków z list. Główny Zarząd Leków i Farmacji,
komórka Ministerstwa Zdrowia odpowiedzialna
za kształtowanie cen leków, wprowadził w 2004
roku system cen referencyjnych. Zgodnie z nowymi regulacjami ministerstwo wyznacza 5 referencyjnych państw członkowskich UE, a cena
detaliczna leku w aptece w Turcji nie może być
wyższa niż najniższa cena wśród tych 5 państw.
Zmiana ta dała Turcji szansę wprowadzenia
standardowego podejścia do kształtowania cen
wszystkich leków oraz spowodowała znaczny
spadek cen, nawet do 75% w przypadku niektórych leków. Może to sprawiać wrażenie, że ceny
w Turcji są bardzo niskie w porównaniu z innymi
krajami. Taki obraz jest jednak całkowicie mylny. Podstawą systemu cen referencyjnych jest
cena detaliczna leku w aptece, a zatem, jakkolwiek ta cena w Turcji może być niższa, cena
refundacyjna określonego leku może nadal być
Rabia Kahveci
Health Care Transformation Project which
started in 2003 made very radical changes in
Turkish health care system. It started with the
objective to make the health system more effective by improving governance, efÞciency,
user and provider satisfaction and long term
Þscal sustainability. The key elements included
implementing General Health Insurance, a single Social Security Institute, expanding the delivery of health care and making it more easily
accessible and friendly, implementing rational
drug use and management of medical materials
and devices.
General health insurance provided a whole coverage of a 72 million population, where
health care expenses of many millions had not
been covered before the change. Health care
expenditures which already had a high rate of
increase raised even further with a broader coverage of the population as well as an aging
population and the increaased demand for new
technologies. The increase in pharmaceutical
expenditure was giving an alarm, which was
around 40 % of total health care expenditures.
In 2004 noticed was a need in regulating the
Pricing Policy of pharmaceuticals. The pharmaceutical prices was very high compared to other
countries and there was a reimbursement policy where almost all drugs were reimbursed. It
could even be said that there was no reimbursement policy with any criteria for inclusion or
exclusion of drugs in the lists. The Ministry of
Health, General Directorate of Pharmaceuticals
and Pharmacy, the responsible body for pharmaceutical pricing, started reference pricing in
2004. According to the new policy the ministry
would take 5 reference EU member countries
and the pharmacy retail price of a drug in Turkey would not be higher than the lowest price among these 5 countries. This change gave
Turkey the chance to have a standard approach to all drugs for pricing and also provided a
big decrease in prices, even upto 75% in some
drugs. Although this change gives the impression that Turkey might have very low prices
compared to other countries, this is totally misleading. The reference pricing is based on the
pharmacy retail price so, although this price
might be lower in Turkey, reimbursement price
for a speciÞc drug could still be much higher
than several other countries. In 2004 the re-
Sesja 5 / Session 5
Health Care
167
Evidence-Based
Sesja 5 / Session 5
168
znacznie wyższa niż w innych krajach. W roku
2004 powołano również komisję refundacyjną
przy Ministerstwie Finansów w celu uregulowania problemu refundacji tych leków.
W roku 2005 Ministerstwo Zdrowia ustanowiło procedurę licencjonowania leków. Równolegle z tymi zmianami powołano Instytut Bezpieczeństwa Społecznego (SSI), odpowiedzialny
za regulacje dotyczące pracy, powszechnego
ubezpieczenia zdrowotnego i politykę refundacyjną. Personel SSI zaczął stopniowo przejmować odpowiedzialność za refundację od Ministerstwa Finansów, porządkując skomplikowaną
listę obejmującą tysiące refundowanych leków o
bardzo różnych cenach, nie ograniczoną niemal
żadnymi restrykcjami. W roku 2006 SSI wprowadził system MEDULA umożliwiający monitorowanie wydatków na leki w całym kraju. W roku
2007 komisję refundacyjną przeniesiono pod
nadzór SSI. Obejmuje ona przedstawicieli Ministerstwa Zdrowia, Ministerstwa Finansów oraz
świata nauki i przemysłu, a jej sekretariat jest
obsługiwany przez SSI. Wkrótce po rozpoczęciu
prac komisji odczuwalna stała się potrzeba uregulowania polityki refundacyjnej. Nadal nie było
obiektywnych, opublikowanych kryteriów wpisywania leków na listy, a podejmowane decyzje
nie były oparte na danych farmakoekonomicznych. Wydatki na leki nadal wykazywały co roku
dwucyfrowy wzrost. Pierwszą połowę 2008 roku
zdominowały dyskusje nad wprowadzeniem
ewentualnych wytycznych dotyczących wnioskowania o refundację leków. Czas ten i dyskusja nad przyszłością polityki refundacji leków w
Turcji przyniosły również bogate doświadczenie
zarówno urzędom publicznym, jak i przemysłowi. W połowie roku 2008 opublikowano wytyczne, na mocy których po raz pierwszy w historii
wprowadzono obowiązek przedkładania analizy
farmakoekonomicznej wraz z wnioskiem o refundację. Wytyczne weszły w życie z końcem
2008 roku, a teraz zbliża się koniec pierwszego
roku ich obowiązywania.
W roku 2009, po dokonaniu obserwacji działania nowych przepisów i przeglądu aktualnych
list refundacyjnych, rząd stwierdził, że ceny refundacyjne w Turcji wciąż są wyższe niż w wielu innych krajach, istnieją znaczne wewnętrzne
różnice cen pomiędzy lekami o podobnej efektywności, a wydatki na leki nadal szybko rosną.
Światowy kryzys ekonomiczny, który nie ominął
Rabia Kahveci
imbursement commission was also established
under the Ministry of Finance in order to regulate reimbursement of these drugs.
In 2005 the Ministry of Health regulated
the licencing procedure of the drugs. Parallel
to these changes the Social Security Institution
(SSI) was established with the responsibility
of regulations related to labor, general health
insurance and reimbursement policies. A complicated list of reimbursed thousands of drugs
with many different prices and almost no restriction rules was given to the hands of SSI
staff, taking the reimbursement responsibility
over slowly from Ministry of Finance. In 2006
SSI established a system called MEDULA which
gave SSI the opportunity to follow pharmaceutical expenditures throughout the country.
In 2007 the reimbursement commission was
under SSI with representatives from Ministry
of Health, Ministry of Finance, academics and
industry, secratariat run by SSI. Soon after
this commission started working, felt was the
need to make a regulation in reimbursement
policy. Still there was no objective published
criteria for inclusion of the pharmaceuticals in
the lists and the decisions were not based on
pharmacoeconomical data. Still there was a double-digit increase each year in pharmaceutical
expenditures. First half of 2008 there were big
discussions around the potential guidelines for
submission of pharmaceuticals for reimbursement. This period has also been a great experience for both public and industry sides, sitting around a table and discussing the future of
Turkish pharmaceutical reimbursement policy.
And mid-2008 guidelines were published making pharmacoeconomical analysis mandatory
for submissions, Þrst time in reimbursement
history. The guidelines were valid starting by
the end of 2008 and has now completed its Þrst
year.
In 2009, after careful watching of new regulations and reviewing the current reimbursement lists the government noticed that the
reimbursement prices in Turkey are still higher
than many other countries, there is internally big differences in prices of drugs that are
comparably effective and the pharmaceutical
expenditure continues to rise rapidly. The global economical crisis, which also affected Turkey, also pressed the government to take some
HTA & Pricing
Turcji, również zmusił rząd do podjęcia kroków
zaradczych. Znów doszło do dyskusji pomiędzy
rządem (na szczeblu nawet wyższym niż poprzednio) a przemysłem nad możliwymi rozwiązaniami zaistniałej sytuacji. Koniec roku 2009
przynosi kolejne zmiany przepisów dotyczących
kształtowania cen i refundacji, z wprowadzeniem
systemu wewnętrznych cen referencyjnych na
czele.
W niniejszej prezentacji przedstawione zostaną przepisy dotyczące kształtowania cen i
refundacji w Turcji po wprowadzeniu nowych
rozwiązań w roku 2009, ich wpływ na politykę
refundacyjną, potencjalne korzyści i zagrożenia
wynikające z ich wprowadzenia, możliwości dalszych zmian na listach refundacyjnych oraz potencjalna rola HTA w powstającym systemie.
www.ceestahc.org
Rabia Kahveci
precautions. Again this time the government,
and even at much higher policymaker level this
time, and the industry sat around the table to
discuss possible solutions for this situation. The
end of 2009 gives birth to changes in pricing
and reimbursement regulations again, internal
reference pricing being one of the hot topics.
With this introduction to the Turkish health
care system, pricing and reimbursement regulations, during the presentation, focus will
be on the changes in the system in 2009, how
price regulations would affect reimbursement
policy, what could be beneÞts and risks of such
regulations, what else could be done to revise
current lists and whether HTA could play any
potential role during these regulations.
Sesja 5 / Session 5
Health Care
169
Evidence-Based
Rola HTA w regulacji cen nowych leków: doświadczenia brazylijskie / The role
of HTA in the price regulation of new drugs: the Brazilian experience
The Role of HTA in the price regulation
of new drugs:
the Brazilian experience
Alexandre Lemgruber
Office Of Economic Evaluation of New Technologies
Brazilian Health Agency (ANVISA)
Sesja 5 / Session 5
4th International Symposium – Evidence-Based Health Care
HTA and Pricing
Krakow, December 2009
HTA and Pricing
$
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The great majority of new drugs does
not show clinical benefits over the
best treatment available
High budget impact of new drugs but
no value for money
Growing use of HTA to assess the
value of new drugs and to help
pricing decisions
In some countries pharmaceuticals
are classified according to the benefit
to the treatment (France, Canada,
Brazil)
International experience
$
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170
France: 67% of the drugs evaluated
did not add anything to the
treatment (1981 and 2003)
Sweden: 58% with no additional
benefit (1987-2000)
USA: 77% considered as mee-too
drugs (1998-2002)
Canada: only 5,9% of the new drugs
were considered to bring significant
clinical benefit over the current
treatment (1990-2003)
Prelegent / Expert
Alexandre Lemgruber
25 min.
Health Care
HTA & Pricing
www.ceestahc.org
Alexandre Lemgruber
What is an innovative drug?
- New process?
- New mechanism of action?
- New molecule?
Price regulation of new drugs in
Brazil - criteria evolution
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In the early stage of the regulation,
patented medicines had their prices out of
the control
Afterwards these prices were limited by the
average of prices in 5 reference countries
In 2004, important changes were made in
the regulatory framework, with the
application of health technology assessment
to the pricing decisions: evidence based
price regulation policy
Sesja 5 / Session 5
patented medicine=really
innovative medicine in terms of
the benefit for the treatment?
Economic regulation and economic
evaluation of new technologies
$
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Branch of the Brazilian Health Surveillance
Agency (ANVISA)
Responsible for HTA to support pricing
decisions
Participation in the National Commission
that gives the recommendation to the
Minister about which technologies should be
paid by the Public Health System
Dissemination strategies
Budget impact analysis
International cooperation
171
Evidence-Based
Evidence Based Price
Regulation
New pharmaceuticals (new chemical
entities) are classified according to their
benefits over the comparators, and their
ceiling prices are defined based on a rapid
HTA (3 months)
If the new drug has no benefit over the
chosen comparator (best treatment), then it
is classified as a Category 2 drug, and their
ceiling price is defined based on costminimization analysis
If the new drug is considered to be better
than the comparator (Category 1), then a
premium price is allowed, but this price
cannot be higher than the lowest price
among 9 reference countries
This analysis is prior to the drug launch
$
$
$
Sesja 5 / Session 5
$
Main characteristics of the
model
$
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Fourth hurdle (besides efficacy,
safety and quality) before marketing
approval
Application of HTA to define the
ceiling prices for new drugs
Does not allow that mee-too drugs be
more expensive than the best
treatment option (85% of the cases)
Not only limited to the Public Health
System (SUS), since around 75% of
the market is private
Challenges
$
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172
Lack of head-to-head studies
- Should we accept indirect
comparisons?
Studies against the wrong comparator
- Should we demand new clinical
trials?
Surrogate end-points
- Should we accept them for pricing
decisions?
Alexandre Lemgruber
Health Care
HTA & Pricing
www.ceestahc.org
Alexandre Lemgruber
Reimbursement Decisions
The National Commission for Technology
Incorporation (CITEC) is responsible for
making the recommendation to the Minister
of Health, who makes the final call about
which technologies should be paid by the
Public Health System
The Commission has 5 members, 3 from the
Ministry of Health and 2 from the agencies
(ANVISA and the Agency that regulates the
health private sector-ANS)
The recommendation is based on a HTA
report
4 votes are needed to approve a
recommendation
Budget impact analysis is required
$
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Two important publications:
- Brazilian Bulletin of Health Technology
Assessment (BRATS)
- Health and Economics
The main goal is to help decision-makers
making informed decisions
BRATS is published every 3 months
8 issues have been published so far
The Bulletin has around 22.000 subscribers,
in more than 30 countries
Health and Economics had its first edition
last month, with great acceptance
Both are avaiable at www.anvisa.gov.br
(publicações - boletins eletrônicos)
Sesja 5 / Session 5
Dissemination
Working in Collaboration
$
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Joint projects on economic evaluation
(Argentina, Cuba)
Anvisa has been asked to offer training
programs on HTA, Regulation and Pricing
(Colombia, Uruguai, Cuba, Cabo Verde,
Moçambique)
HTAi group of developing countries
Project to strengthen HTA in the
MERCOSUL region
Working group on Economic Regulation in
the PAHO region
Department of Science and Technology
(Ministry of Health) is member of the
INAHTA
173
Evidence-Based
An Invitation
$
$
$
Rio de Janeiro will host the 2011 HTAi
meeting
It will be the first time that a LatinAmerican country hosts this meeting
The theme will be “HTA and Health Systems
Sustainability”
Sesja 5 / Session 5
We are looking forward to seeing you!
174
Alexandre Lemgruber
Health Care
HTA & Pricing
www.ceestahc.org
Sesja 5 / Session 5
175
Wtorek
8 grudnia 2009
Tuesday
December 8th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 6 / Session 6
Granice opłacalności w podejmowaniu decyzji refundacyjnych
Cost-utility thresholds vs efÞciency frontier
J. Jaime Caro – 35 min.
Michael Drummond – 35 min.
Niemiecki Instytut Jakości i Efektywności w Opiece Zdrowotnej (IQWiG) to niezależna instytucja naukowa, której zadaniem
jest ocena wartości świadczeń zdrowotnych
z punktu widzenia pacjentów. Został on powołany 1 czerwca 2004 r. jako pozarządowa
instytucja typu non-proÞt będąca fundacją
prawa prywatnego i działająca z upoważnienia Federalnej Komisji Wspólnej i Federalnego Ministerstwa Zdrowia.
Jak dotąd w Niemczech ocena leków
sprowadza się do określenia ich wartości klinicznej. IQWiG zaproponował metodę oceny
w oparciu o zależność koszt-korzyść, nazwaną „analizą granicy efektywności”. Metoda ta
ma zastosowanie do określania cen maksymalnych dla określonych leków, które nie
mogą być włączone do grup referencyjnych.
Dla najlepszej technologii w danym obszarze
terapeutycznym powinna zostać określona
cena maksymalna. Jest to warunek wstępny umożliwiający podejmowanie decyzji,
jakie koszty w odniesieniu do uzyskanych
korzyści są uzasadnione i możliwe do przyjęcia w przypadku pacjentów powszechnego
ubezpieczenia zdrowotnego w Niemczech.
Oznacza on także, że w różnych obszarach
terapeutycznych można ustalić różne wartości progowe dla efektywności kosztowej. W
większości krajów europejskich przyjmuje
się jedną wartość progową współczynnika
koszt-użyteczność dla wszystkich technologii w systemie opieki zdrowotnej. Podczas
sesji omówione zostaną różnice pomiędzy
metodami oraz ich zalety i wady.
The Institute for Quality and EfÞciency in
Health Care (IQWiG) in Germany is an independent scientiÞc institute that assesses the
beneÞts of health care services for patients.
It was established on 1 June 2004 as a nongovernment and non-proÞt private law foundation, and is commissioned by the Federal
Joint Committee or the Federal Ministry of
Health.
So far in Germany, the assessment of
drugs has been limited to their clinical beneÞt. IQWiG has presented a concept for
cost-beneÞt assessment methods, called
“EfÞciency Frontier Analysis”. The methods
are used to set the ceiling price for speciÞc
drugs that cannot be included in a reference price group. A ceiling price should be set
for a superior health technology in a given
therapeutic area. This is the precondition
for enabling a decision as to which costs for
which beneÞt are appropriate and reasonable for the community of citizens insured by
SHI in Germany. It means also that in different therapeutic areas the different cost
effectiveness thresholds could be set. Generally in most of European countries one costutility threshold are established for all technologies in health care system. During the
session differences between the methods
will be discussed including their advantages
and disadvantages.
[IQWiK, http://www.iqwig.de/iqwig-presents-aconcept-for-cost-beneÞt.738.en.html]
[IQWiK, http://www.iqwig.de/iqwig-presents-aconcept-for-cost-beneÞt.738.en.html]
177
Evidence-Based
Sesja 6 / Session 6
178
Pojęcie „granicy efektywności” zostało
wprowadzone przez międzynarodową grupę
ekspertów w dziedzinie ekonomiki zdrowia.
Za jego pomocą można porównywać zależność koszt-korzyść dla dowolnej liczby opcji
terapeutycznych. Określona opcja w porównaniu z inną jest określana jako „efektywna”, jeżeli przy tym samym koszcie pozwala
osiągnąć wyższą korzyść lub przy tej samej
korzyści jest bardziej efektywna kosztowo.
GraÞczne odwzorowanie „granicy efektywności” ma postać krzywej łączącej punkty
odpowiadające najbardziej efektywnym interwencjom. Za pomocą takiego wykresu
można również określić właściwą (maksymalną) cenę dla nowego leku. Zastosowanie
pojęcia „granicy efektywności”, odnoszącego
się tylko do technologii uznanych za najlepsze, ma następujące implikacje:
• brak miejsca dla nowych, „gorszych” opcji
(nawet jeśli są tańsze od istniejących),
• nowe, „równoważne” opcje nie są oceniane (równoważność ceny),
• efektywność musi odpowiadać oszacowaniom IQWiG.
Dla leków skuteczniejszych lecz droższych niż dotychczas stosowane można
oszacować cenę, dla której zależność kosztkorzyść mieściłaby się w przyjętym zakresie
efektywności.
W styczniu 2008 r. IQWiG opublikował
dokument dotyczący „metod oceny relacji
pomiędzy kosztami i korzyściami w niemieckim systemie powszechnego ubezpieczenia
zdrowotnego”. Poniższy cytat stanowi podsumowanie komentarza Michaela Drummonda i wsp. na temat metod stosowanych
przez IQWiG.
The concept of “efÞciency frontier” has
been developed with the support of a group
of international health economists. It can be
used to compare the cost-beneÞt relation of
any number of therapy alternatives. Health economists describe one intervention in
comparison to another as being “efÞcient” if,
at the same cost, it displays a higher beneÞt
or is more cost-efÞcient at the same beneÞt.
This „efÞciency frontier” is presented graphically as a curve that joins the most efÞcient
interventions. With the help of this graph, an
appropriate price for a new drug (ceiling price) can also be described. Implication of the
efÞciency frontier, which is addressed only
health technologies judged superior:
• new “inferior”therapies have no place (even if less expensive than existing
ones)
• new “equivalent”therapies not assessed
– price equivalence
• effectiveness component must reßect
IQWiG estimates.
For drugs that are more beneÞcial but
more costly than those so far used, it can
be determined where their price would have
to lie so that the cost-beneÞt relation lies
within the accepted efÞciency range.
In January 2008 a consultation document
on ‘the methods for assessment of the relation of beneÞts to costs in the German statutory health care system’ was published by
IQWiG. Micheal Drummond with colleague
have commented the methods to be used by
IQWiG (please Þnd the below the citation of
conclusions).
Health Care
HTA & Pricing
www.ceestahc.org
[ Michael Drummond M, Rutten F The IQWiG
methodology paper version 1.0 ]
“By imposing the restriction to only consider the efÞciency of resource allocation within a therapeutic area and not across therapeutic areas IQWiG has maneuvered itself
into a difÞcult position. This restriction makes it impossible to conduct economic evaluations to international standards and only
allows the presentation of information which
is of limited value to the decision maker and
gives little guidance on how to decide on the
introduction and pricing of medical technologies. Furthermore, by not considering the
relative efÞciency of interventions across
different therapeutic areas it runs the risk
of allowing clearly inefÞcient technologies or
rejecting clearly efÞcient technologies. Finally, constructing the efÞciency frontiers for
each therapeutic area will consume many
resources, only a small part of which would
be needed to conduct a standard economic
analysis, especially as available information
on cost-effectiveness from studies abroad
can be used. In summary, we are in full support of IQWiG’s efforts to conduct economic
analyses but, unfortunately the methods
currently proposed are not up to the task.”
[Michael Drummond M, Rutten F The IQWiG
methodology paper version 1.0]
Sesja 6 / Session 6
„Poprzez wprowadzenie zasady uwzględniania efektywności przy alokacji zasobów
wyłącznie w określonym obszarze terapeutycznym, a nie pomiędzy obszarami, IQWiG
postawił się w trudnej sytuacji. Zastrzeżenie to uniemożliwia prowadzenie analiz ekonomicznych zgodnie z międzynarodowymi
standardami, zaś prezentowane zgodnie z
nim informacje mają ograniczoną wartość dla
decydentów i niewiele wnoszą przy podejmowaniu decyzji dotyczących wprowadzania
nowych technologii medycznych i kształtowania ich cen. Co więcej, wobec braku możliwości porównania względnej efektywności
interwencji pomiędzy różnymi obszarami terapeutycznymi wzrasta ryzyko dopuszczenia
technologii zdecydowanie nieefektywnej lub
odrzucenia technologii efektywnej. Po ostatnie, określenie granicy efektywności dla
każdego obszaru terapeutycznego spowoduje znaczne zużycie zasobów, których niewielka część wystarczyłaby dla przeprowadzenia standardowej analizy ekonomicznej,
szczególnie przy uwzględnieniu dostępnych
informacji na temat efektywności kosztowej
pochodzących z badań przeprowadzonych w
innych krajach. W pełni popieramy wysiłki
IQWiG na polu analiz ekonomicznych; niestety, zaproponowane metody nie odpowiadają podjętemu zadaniu.”
179
Evidence-Based
Prelegent / Expert
Sesja 6 / Session 6
Właściwe miejsce wydajności wśród priorytetów opieki zdrowotnej
The proper role of efÞciency in “priority setting” in health care
180
W większości krajów przyjęto do wiadomości,
że model zakładający zapewnianie opieki zdrowotnej niezależnie od kosztów jest niemożliwy do
utrzymania, co pociąga za sobą konieczność dokonywania wyborów – jakie świadczenia można
zapewnić i za jaką cenę. Z uwagi na emocjonalny aspekt wszystkiego, co wiąże się z chorobą,
oraz ryzyko polityczne związane z wprowadzaniem ograniczeń w tym obszarze, istnieje silny
nacisk na ustanowienie jasnych zasad dokonywania tych wyborów w oparciu o dane naukowe
i w sposób tak obiektywny, jak to możliwe. Bardzo atrakcyjne wydaje się przyjęcie wydajności
jako głównego kryterium, zgodnie z zasadą, że
wybierać należy bardziej wydajne interwencje,
dzięki czemu środki będą wydatkowane zgodnie
z priorytetami społecznymi, zapewniona zostanie
spójność i utrzymane ograniczenia budżetowe.
Takie czysto utylitarystyczne podejście wymaga
jednolitego sposobu wartościowania pozwalającego oszacować wszystkie możliwe skutki interwencji – dobre i złe – ponieważ (przynajmniej co
do zasady) wymagane jest porównanie wydajności interwencji w całym systemie opieki zdrowotnej. We wszystkich innych rodzajach analiz
ekonomicznych wartość taką można wyrazić po
prostu w jednostkach monetarnych. W odniesieniu do zdrowia wprowadzono wielkość będącą
iloczynem oczekiwanej długości życia przez jego
jakość, czyli QALY. Pomimo swej atrakcyjności
ta stosunkowo prosta metoda ułatwiająca podejmowanie obiektywnych, racjonalnych decyzji
dotyczących Þnansowania opieki zdrowotnej w
ramach ograniczonego budżetu nie sprawdza
się. Decyzje podejmowane w oparciu o koszt za
QALY ani nie ułatwiają utrzymania systemu w ramach założonego budżetu, ani też nie są zgodne
z priorytetami społecznymi. Co więcej, QALY to
sposób oceny, który ze swej zasady wprowadza
najgorszy rodzaj dyskryminacji: osób starszych,
niepełnosprawnych, pokrzywdzonych. Takie postępowanie jest sprzeczne z najbardziej podstawowymi zasadami etycznymi przyjętymi w
naszych społeczeństwach. Gdyby rzeczywiście
rozumiano jego implikacje, byłoby ono nielegalne w wielu krajach. W niniejszej prezentacji
zostanie omówiona właściwa rola wydajności w
podejmowaniu decyzji dotyczących Þnansowania świadczeń oraz przedstawiona alternatywna
wobec kosztu za QALY metoda oceny wydajności
interwencji.
J. Jaime Caro
Most countries have recognized that supplying health care without regard to its cost is not
sustainable and, thus, that some choices are
necessary in terms of what is covered and at
what price. Given the highly emotional nature
of dealing with illness and the political risks of
making rationing decisions in this area, there
has been a strong push for an explicit means of
guiding these judgments that grounds them on
scientiÞc evidence and renders them as objective as possible. One very attractive approach
is to use efÞciency as the main criterion, with
the underlying idea that the decisions should
favor more efÞcient interventions and by doing so the investments will accord with social
priorities, respect limited budgets and ensure
consistency. This pure utilitarian view requires
a universal measure of value that can cover
all possible intervention effects — good and
bad — because it requires comparisons of efÞciency (at least in principle) across the health
care system. Although in all other economic
analyses it is understood that monetary units
fulÞll the requirement, in health these were rejected in favour of weighted average survival:
the sum product of life expectancy and quality
of that life, or QALY. Despite the great attractiveness of a relatively simple method that facilitates objective, rational health care coverage
decisions consistent with a limited budget, the
cost per QALY approach does not work. It does
not help the system stay within a budget and
fails to accord with social priorities. Moreover,
the QALY measure that is at its core embodies
the worst kind of discrimination: against the
elderly, the disabled, the disadvantaged — it
goes against the most basic ethical principles
our societies have embraced. Indeed, in many
countries its use would be illegal if the implications were understood. In this talk, the proper
role of efÞciency in coverage decisions will be
discussed and an alternative to the cost per
QALY approach will be presented.
35 min.
HTA & Pricing
www.ceestahc.org
J. Jaime Caro
Sesja 6 / Session 6
Health Care
181
Evidence-Based
Sesja 6 / Session 6
182
J. Jaime Caro
HTA & Pricing
www.ceestahc.org
J. Jaime Caro
Sesja 6 / Session 6
Health Care
183
Evidence-Based
Sesja 6 / Session 6
184
J. Jaime Caro
HTA & Pricing
www.ceestahc.org
J. Jaime Caro
Sesja 6 / Session 6
Health Care
185
Evidence-Based
Sesja 6 / Session 6
186
Lata życia skorygowane jakością (QALY) to
szeroko stosowany sposób pomiaru korzyści
zdrowotnych w analizach ekonomicznych, zalecany w wielu krajowych wytycznych. Metoda ta jest jednak także przedmiotem istotnej
krytyki. Co więcej, w Zjednoczonym Królestwie
NICE (National Institute for Health and Clinical
Excellence), jeden z głównych propagatorów
stosowania QALY, wydał niedawno dodatkowe
zalecenia, w których sugeruje wyższe szacowanie QALY dla dwóch ostatnich lat życia.
Stosowanie QALY przy podejmowaniu decyzji dotyczących opieki zdrowotnej ma kilka
zalet. Po pierwsze, metoda ta uwzględnia występowanie wielorakich skutków interwencji
wpływających na długość i jakość życia. Po
drugie, w sposób bezpośredni odwołuje się do
własnego osądu pacjentów na temat efektów
zdrowotnych. Po trzecie, umożliwia modelowanie korzyści i kosztów w czasie oraz, po ostatnie, może stanowić bezpośrednie uzasadnienie
podjętej decyzji.
Z drugiej strony metoda ta może być niedogodna zarówno ze względów metodologicznych, jak i politycznych. Różne sposoby pomiaru (ocena preferencji związanych z danym
stanem zdrowia) skutkują różnymi wynikami.
Różne „generyczne” narzędzia dają różne oszacowania zyskanych QALY.
Niektóre kluczowe założenia QALY (np. stała
i proporcjonalna wymiana, addytywność i niezależność) wyraźnie nie wytrzymały próby czasu. Co więcej, ze społecznego punktu widzenia
wcale nie jest oczywiste, że jednakowe szacowanie QALY u różnych pacjentów to właściwe
podejście. Rozstrzygnięcia dotyczące „wartości
za pieniądze” albo wynikają z wcześniejszych
decyzji Þnansowych, albo opierają się na arbitralnie przyjętym progu.
Prelegent / Expert
Michael Drummond
The quality-adjusted life-year (QALY) is a widely-used measure of health gain in economic
evaluation and is recommended in several sets
of national guidelines for conducting studies.
However, it also attracts a considerable amount
of criticism. In addition, in the United Kingdom,
The National Institute for Health and Clinical
Excellence (NICE), one of the main advocates
of the use of QALYs, has recently issued supplementary guidance indicating that QALYs in the
last two years of life can be valued at a higher
level.
There are several advantages of using the
QALY approach in health care decision-making.
First, it acknowledges that there are multiple
outcomes from interventions, impacting on
length and quality of life. Secondly it explicitly
incorporates value judgments from individuals
about health outcomes. Thirdly, it models beneÞts and costs of interventions over time and,
Þnally, it as an explicit decision rule.
On the other hand, there are several methodological and policy drawbacks. Different measurement approaches (for estimating health
state preference values) give different results.
Different ‘generic’ instruments give different
estimates of QALYs gained.
Several key assumptions of the QALY (ie
constant proportional trade-off, additive independence) clearly do not hold. In addition,
Also, if the concern is social value, it is not at all
clear that equal weighting of QALYs across individuals is the preferred approach. Judgments
of value for money are either linked to past funding decisions, or made based on an arbitrary
threshold.
35 min.
HTA & Pricing
Istnieje kilka alternatywnych metod oceny
ekonomicznej wartości leczenia, takich jak „gotowość do płacenia” (ang. willingness-to-pay,
contingent valuation) lub doświadczenia dyskretnego wyboru. W praktyce jednak podstawową alternatywą dla QALY pozostaje analiza
koszt-konsekwencje, która decydentowi pozostawia określenie materialnej wartości uzyskanej korzyści. Przy bliższym zbadaniu okazuje
się jednak, że metoda ta ma te same ograniczenia, co QALY.
Ogólnie rzecz ujmując, QALY pozostaje najbardziej użyteczną metodą w krajach o ściśle
ograniczonym budżecie przeznaczonym na
opiekę zdrowotną, w których decydenci uważają, że korzyść uzyskana z leczenia to dobry
punkt wyjścia dla dyskusji o alokacji zasobów,
a społeczeństwo ceni sobie bezpośrednie uzasadnienie decyzji dotyczących opieki zdrowotnej.
www.ceestahc.org
Michael Drummond
There are several alternative economic approaches to assessing the economic value of
treatment, such as contingent valuation (ie
willingness-to-pay) and discrete choice experiments. However, in practice the main alternative to the use of QALYs is to conduct a ‘cost-consequences analysis’, which leaves the valuation
of the beneÞt to the decision-maker. However,
on close examination, this approach is found to
have of the same problems as QALYs.
In general, QALYS are likely to be most useful if the country has a ‘hard’ healthcare budget
constraint, if decision-makers feel that the health gain from treatments is a useful starting
point for discussing resource allocation, and if
the community values explicitness in healthcare decision-making.
Sesja 6 / Session 6
Health Care
187
Evidence-Based
Is the QALY a Necessary
Evil?
Michael Drummond
Centre for Health Economics,
University of York
Outline of Presentation
• Some background.
Sesja 6 / Session 6
• What’s good about the QALY?
• What adjustments are required to QALYs?
• Are there suitable alternatives to QALYs?
• What are the issues we have to resolve, QALYs
or no QALYs?
Some Background
• The QALY has been the favoured outcome
measure for most health economists for 30 years.
• It is recommended in several sets of economic
evaluation guidelines (eg Washington Panel,
CADTH, NICE).
• Recently, the IQWiG guidelines, and possibly
others, reject QALYs.
• NICE has departed from standard QALY
methodology in its supplementary guidance for
‘end of life’ therapies.
188
Michael Drummond
Health Care
HTA & Pricing
www.ceestahc.org
Michael Drummond
NICE’s Most Recent Controversy
• In August 2008, NICE published its Appraisal
Consultative Document on four new drugs for treating
advanced renal carcinoma: bevacizumab, sorafenib,
sunitinib, temsirolimus.
• It recommended that none of the four drugs should be
used in the NHS on the grounds that they were not costeffective.
• Oncologists and patient organizations were outraged,
since these drugs are widely used in many other
countries and offer benefit to patients for whom no other
effective treatments are available.
Independent Evaluation of Drugs for
Advanced Renal Carcinoma
(First-line Treatments for Patients Suitable for
Immunotherapy)
Cost
QALYs
Sunitinib versus IFNalpha
£31,185
0.44
£71,462
Bevacizumab added
to IFN-alpha
£45,435
0.27
£171,301
Temsirolimus versus
IFN-alpha*
£22,272
0.24
£94,385
(* patients with poor prognosis)
Cost/QALY
Source: NICE, 2008
Supplementary Guidance for ‘End
of Life’ Therapies
Sesja 6 / Session 6
Drug Comparison
• If the therapy:
-is for a small patient population with life expectancy of
less than 24 months;
-where no equivalent therapy exists;
-where the therapy adds three months or more to life
expectancy.
• Then:
-the QALYs gained should assume full quality of life in
the added months;
-in addition the Committee can consider that the QALYs
gained should be weighted sufficiently high for the
therapy to be approved given NICE’s current threshold.
189
Evidence-Based
What are the Desirable Features
of the QALY Approach?
• Acknowledges that there are multiple
outcomes from interventions, impacting on
length and quality of life.
• Explicitly incorporates value judgments
from individuals about health outcomes.
• Models benefits and costs of interventions
over time.
• Has an explicit decision rule.
Issues Arising from the Use of the
QALY Approach
Sesja 6 / Session 6
• Methodological issues
• Policy issues
Methodological Issues
• Different measurement approaches (for
estimating health state preference values)
give different answers.
• Different ‘generic’ instruments give
different estimates of QALYs gained.
• Several key assumptions of the QALY (ie
constant proportional trade-off, additive
independence) clearly do not hold.
190
Michael Drummond
Health Care
HTA & Pricing
www.ceestahc.org
Michael Drummond
Policy Issues
• If the concern is social value, it is not at all clear
that equal weighting of QALYs across individuals
is the preferred approach.
• Judgments of value for money are either linked
to past funding decisions, or made based on an
arbitrary threshold.
• The ICER does not tell us about the opportunity
cost of adopting the new technology
(Birch and Gafni; 2006).
• Lack of, or inadequacy of, alternative
treatments.
• Seriousness of the condition.
• Affordability from the patient perspective.
• Overall financial implications for
government.
• Equity objectives.
Sesja 6 / Session 6
Factors Considered Alongside
Cost-Effectiveness
The Relationship Between Social
Value and Incremental Cost Per
Quality-Adjusted Life-Year (QALY)
191
Evidence-Based
So What Do We Do?
• Develop a series of distributive weights for
QALYs?
• Establish a ‘deliberative’ decision-making
process to incorporate other relevant factors
(beyond the incremental cost per QALY)?
• Establish a stronger basis for cost-effectiveness
threshold(s)?
• Encourage more transparency and public
debate about healthcare resource allocation
decisions?
Alternatives to QALYs
Sesja 6 / Session 6
• Perform a ‘cost-consequences analysis’
and leave the rest up to the decisionmaker.
• Use contingent valuation or discrete
choice experiments.
IQWiG’s Efficiency Frontier
Source: IQWiG 2008
192
Michael Drummond
Health Care
HTA & Pricing
www.ceestahc.org
Michael Drummond
Issues Apparently ‘Avoided’ by
IQWiG’s Approach
• Assumptions about the link between
clinical outcomes (as observed in trials)
and long term health benefit (as modelling
is not necessary required).
• Relative valuations of states of health.
• Specification of a ‘threshold’ of
willingness-to-pay.
• Explicit discrimination between patient
groups.
• Consideration of all relevant alternatives.
• Dealing with multiple health outcomes.
• Reliability of clinical measures for
predicting long-term health benefit and
value.
• Implicit valuation of health outcomes.
• Relationship between efficiency and
equity.
Consideration of all Relevant
Alternatives
Sesja 6 / Session 6
Key Issues Raised by IQWiG’s
Approach
• Efficiency frontier approach is good for
eliminating ‘dominated’ alternatives.
• Selection of alternatives can change the
shape of the frontier.
• Data limitations may inhibit the calculation
of the frontier for ‘older’ interventions.
• The most critical choice appears to be that
of the last intervention on the frontier, prior
to the new intervention.
193
Evidence-Based
Reliability of Clinical Measures for
Predicting Long-Term Health
Benefit and Value
• A problem for all approaches to economic
evaluation.
• Typically a model is used to project long-term
outcome, using a mixture of trial-based and
observational data.
• Often it is important to recognize non-linearities
in the relation between short-term and long-term
outcomes.
• In the IQWiG approach will future benefits be
sometimes ignored, or ‘modelled’ implicitly?
Sesja 6 / Session 6
Implicit Valuation of Health
Outcomes
• Explicit ‘thresholds’, like that used by
NICE, have been criticized.
• Also, it is clear that a threshold range is
required.
• In making a decision about a ceiling price
for a new drug, IQWiG will implicitly be
setting a threshold willingness-to-pay for
additional value.
IQWiG’s Efficiency Frontier:
Decision zones above the superiority boundary
Source: IQWiG 2008
194
Michael Drummond
Health Care
HTA & Pricing
www.ceestahc.org
Michael Drummond
The Cost-effectiveness Plane
More Costly
$1
00
,00
0/Q
E
(Intervention is less
effective and more costly)
AL
Y
Ca
Da
$2
00
0,0
Y
AL
/Q
Ba
Decrease in QALYs
Increase in QALYs
Db
A
Cb
Y
AL
/Q
(Intervention is more
effective and less costly)
AL
Y
00
0,0
$1
00
,00
0/Q
$2
Bb
Less Costly
Ontario Cost/QALY criteria
New treatment
Which
curve ?
Standard of Care
Who by, and how,
will the decision
be made ?
i
t
s
Costs
And what decision, when more than one outcome
is relevant ?
Sesja 6 / Session 6
B
e
n
e
f
195
Evidence-Based
Issues We Need to Resolve:
QALYs or No QALYs
• Trade-offs among multiple outcomes.
• Projections of long-term benefit.
• Discrimination among different patient
groups.
Sesja 6 / Session 6
How is IQWiG’s Approach
Discriminatory?
• As recommendations are made independently in
different disease areas, it is likely that the
implied amount paid for a unit of health gain (eg
a year of life gained) will differ between diseases
• The willingness-to-pay for more health benefit is
likely to be determined largely by the slope of
the line between the last two drugs on the
frontier
• As in the case of NICE, the recommendations
from the assessment are accompanied by a
deliberative decision-making process
When Are QALYs Useful?
• If you have a ‘hard’ healthcare budget
constraint
• If you feel that the health gain from
treatments is a useful starting point for
discussing resource allocation
• If you value explicitness in healthcare
decision-making
196
Michael Drummond
Health Care
HTA & Pricing
www.ceestahc.org
Michael Drummond
Is There Convergence at Last?
• NICE
- adjustments to QALYs for end-of-life therapies.
• IQWiG
- modeling of costs and outcomes over the same
time horizon;
- combination of outcomes (aka QALYs) within
therapeutic areas.
Sesja 6 / Session 6
The Future for Europe?
Conclusions
• The challenges to QALYs posed by the IQWiG
guidelines should be taken seriously.
• Advocates of the ‘standard’ QALY approach
suggest that ,while adjustments are required, it
is not immediately obvious what these should
be.
• Other approaches to resolving resource
allocation decisions raise their own challenges
and more experience needs to be accumulated.
197
Wtorek
8 grudnia 2009
Tuesday
December 8th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 7 / Session 7
Refundacja warunkowa w ramach porozumień cenowych
Managed entry schemes
Chris Henshall – 30 min.
Magdalena Władysiuk – 20 min.
Evidence-Based Health Care (EBHC) zakłada podejmowanie decyzji na podstawie
rzetelnych analiz. Zbierane dane i analizy
mają służyć zmniejszeniu niepewności urzędu podejmującego decyzje, w tym decyzje
refundacyjne i cenowe. Decyzje te w zasadniczym stopniu zależą od wyników analiz
ekonomicznych i ich odniesienia do granicy
opłacalności w danym kraju. Z kolei wyniki
analiz ekonomicznych w szczególny sposób
zależą od wyników analiz efektywności, a
więc jakości i ilości dostępnych danych dotyczących skuteczności i bezpieczeństwa porównywanych technologii medycznych. Niepewność oszacowań w tym zakresie jest więc
szczególnie istotna, gdyż powoduje wzrost
ryzyka związanego z decyzją refundacyjną i
cenową, zarówno po stronie regulatora, jak i
producenta. Do metod podziału ryzyka, które ukierunkowane są na zmniejszenie ryzyka związanego z niepewnością oszacowań,
dzięki zgromadzeniu dodatkowych danych,
w wyniku prowadzenia dalszych badań, należą tzw. Managed Access Schemes (MAS),
a wśród nich: Coverage with Evidence Development (CED), Only in Research (OIR)
oraz Conditionally Funded Field Evaluations
(CFFE).
In Evidence-Based Health Care (EBHC)
decisions should be based on valid analyses.
Data collection and analysis should be aimed
at reduction of the decision-maker’s uncertainty, especially with respect to reimbursement and pricing. Such decisions depend
to a high degree on the results of economic
analyses and their relation to the cost-effectiveness threshold assumed in a speciÞc
country. The results of economic analyses
depend in turn on the results of efÞcacy and
safety analyses, i.e. the quality and quantity of available data concerning efÞcacy and
safety of the compared health technologies.
Uncertainty of evaluations in this area is therefore of special importance as it increases
the risk associated with reimbursement and
pricing decisions, affecting both the manufacturer and the regulatory authority. Risk
sharing methods, aimed at reduction of the
risk associated with uncertainty of the estimations through continuous collection of additional data from ongoing trials, include socalled Managed Access Schemes (MAS), and
among them: „Coverage with Evidence Development” (CED), „Only in Research” (OIR)
and „Conditionally Funded Field Evaluations”
(CFFE) methods.
199
Evidence-Based
Sesja 7 / Session 7
Wymienione wyżej metody zakładają
przyznanie warunkowej refundacji, a więc
przyjmuje się, że z czasem dojdzie do weryÞkacji zasadności decyzji refundacyjnej oraz
ew. ceny produktu, na podstawie gromadzonych danych, w przewidzianym umową okresie. Zakłada się, że zebrane z czasem dane
pozwolą na zmniejszenie niepewności oszacowań w stopniu pozwalającym na podjęcie
jednoznacznej decyzji refundacyjnej i ustalenie ceny produktu, adekwatnej do uzyskiwanych korzyści zdrowotnych. Gromadzenie
danych może odbywać się w ramach badań
obserwacyjnych (w tym rejestrów), ale również np. w ramach dodatkowych badań randomizowanych, przy czym koszt tych badań
ponosi albo regulator, albo producent, albo
są one dzielone między strony w ramach porozumienia.
Jednym z głównych celów warunkowego
Þnansowania jest wspieranie dalszego rozwoju innowacji oraz postępu technologicznego, poprzez częściowy zwrot nakładów
poniesionych w trakcie realizacji procesu badawczo-rozwojowego.
200
The methods listed above are based on
conditional reimbursement, i.e. it is assumed
that accumulation of additional data within
a period of time speciÞed in the agreement
will allow for veriÞcation of the reimbursement decision and/or the product price. It
is assumed that data accumulated with time
will allow for reduction of the uncertainty of
estimation and therefore for an unequivocal
reimbursement decision and pricing of the
product, adequate to the achieved beneÞts.
Accumulation of data may be based on observational studies (including registries) as
well as additional randomized trials; costs
of such trials may be incurred either by the
regulatory authority or the manufacturer,
or they may be shared between the parties
according to a speciÞc agreement.
One of the main objectives of conditional
Þnancing is to support innovativeness and
technological progress through partial reimbursement of expenditure associated with
the research and development process.
HTA & Pricing
30 min.
www.ceestahc.org
Prelegent / Expert
Umowy CED i inne metody podziału ryzyka: pomoc czy utrudnienie?
CED and other approaches to Managed Entry: help or hindrance?
Umowy typu Coverage with Evidence Development (CED) in inne metody podziału ryzyka
w przypadku nowych technologii medycznych
cieszą się w ostatnich latach wzrastającym zainteresowaniem. Pojawia się szereg nowych zagadnień, istotnych dla twórców, płatników oraz
odbiorców nowych technologii. Ważne jest, aby
zagadnienia te były otwarcie i konstruktywnie
dyskutowane przez wszystkie zainteresowane
strony w celu określenia właściwych zastosowań
różnych propagowanych metod.
Health Technology Assessment International
(HTAi) to międzynarodowe towarzystwo naukowe powołane w celu wspierania i promocji rozwoju, propagowania, rozumienia i stosowania
oceny technologii medycznych (HTA) na całym
świecie. W ramach HTAi Policy Forum najważniejsze osoby reprezentujące sektor publiczny i
prywatny mają wyjątkową okazję do wymiany
informacji oraz nieformalnych, strategicznych
dyskusji na temat aktualnego stanu HTA, jego
rozwoju i implikacji dla systemów opieki zdrowotnej, przemysłu, pacjentów i innych podmiotów, zarówno między sobą, jak i z zaproszonymi
ekspertami z całego świata. W roku 2007 Policy
Forum poświęcono umowom CED, a prowadzone dyskusje i sformułowane zalecenia stały się
podstawą opublikowanego w tym samym roku
artykułu . W roku 2010 dyskusje w ramach Policy Forum dotyczyć będą umów podziału ryzyka
(Managed Entry).
W niniejszej prezentacji przedstawione zostaną zalecenia Policy Forum dotyczące umów
CED oraz omówione niektóre zagadnienia poruszone podczas dyskusji na ten temat na Forum.
Przedstawione zostanie także wstępne stanowisko Forum w kwestii umów podziału ryzyka oraz
(przed podjęciem dyskusji na Forum) pewne
reßeksje osobiste dotyczące związanych z nimi
szans i wyzwań.
Chris Henshall
Coverage with Evidence Development (CED)
and other forms of Managed Entry for new health care technologies have received increasing
attention in recent years. They raise many issues for those developing, covering, using and
receiving new technologies. It is important that
these are discussed openly and constructively
by all stakeholders to identify the appropriate
applications of the various approaches being
advocated.
Health Technology Assessment International (HTAi) is an international scientiÞc society
that aims to support and promote the development, communication, understanding and use
of health technology assessment (HTA) around
the world. The HTAi Policy Forum provides a
unique opportunity for senior Þgures from the
public and private sectors to meet one another
and invited international experts for informal,
strategic discussions about the present state of
HTA, its development and implications for health care systems, industry, patients and other
stakeholders. The Policy Forum discussed CED
in 2007 and a paper based on its deliberations
and recommendations was published later that
year . The Policy Forum will discuss Managed
Entry in 2010.
This presentation will review the recommendations of the Policy Forum on CED and discuss
some of the issues that arose in the Forum’s
discussion of this topic. It will also set out the
approach that the Forum intends to take to Managed Entry and offer some personal reßections
on the opportunities and challenges that these
approaches present, ahead of the Forum’s deliberations.
Sesja 7 / Session 7
Health Care
201
Evidence-Based
Coverage with Evidence Development and other
approaches to managed entry: help or hindrance?
Chris Henshall
University of York
Sesja 7 / Session 7
Plan of presentation
!
HTAi and the HTAi Policy Forum
!
Policy Forum paper on CED
!
Pros and cons of CED as an option
!
Identifying technologies for CED
!
Practical issues in a CED decision
!
Subsequent use of evidence developed
!
Comments on CED
!
Other forms of agreement between producers
and funders of technologies to manage entry
Health Technology Assessment
International (HTAi)
!
!
The mission of HTAi is to support and promote
the development, communication, understanding
and use of health technology assessment (HTA)
around the world, as a scientifically based and
multidisciplinary means of informing decision
making regarding the introduction of effective
innovations and the efficient use of resources in
health care
HTAi activities
!
!
202
Annual Scientific Meeting; Journal; Interest
Groups; Strategic Alliances; Members Services
HTAi Policy Forum
Chris Henshall
Health Care
HTA & Pricing
www.ceestahc.org
Chris Henshall
HTAi Policy Forum
!
The HTAi Policy Forum provides a unique opportunity for
senior people from public and private sector organizations
with strategic interests in HTA to meet one another,
members of the HTAi Board, and invited international
experts, for strategic discussions about the present state
of HTA, its development and implications for health care
systems, industry, patients and other stakeholders
!
The aim of the Forum is to provide an environment where
senior people can engage in strategic discussions
informed by the perspectives of their different
organizations without the constraints associated with
discussions of specific products or organizational policies
!
!
!
!
!
!
!
Comments on CED
!
Policy Forum Paper on CED
Hutton J, Trueman P, Henshall, C. Coverage with
Evidence Development: An examination of
conceptual and policy issues. International
Journal of Technology Assessment in Health
Care, 23:4 (2007), 425–435
Sesja 7 / Session 7
203
Evidence-Based
Pros and cons of CED
Group
Pros
Funders
Monitoring and review;
Meet patient
possible investment in
demands; influence
evidence development ineffective technology;
challenge of stopping
ineffective technology
Manufacturers
Adoption of
technology that might
otherwise be rejected
Delay to full market
access; additional
burden of data
collection/analysis
Patients
Early access to
promising technology
Technology may prove
ineffective or have
more disbenefit than
benefit
Cons
!
Restrict CED to
potentially significant advances
with promising indications, but uncertainties
that are material to a coverage decision
! which can be addressed satisfactorily
through a study whose methods, timescale
and costs are feasible within the CED
paradigm
!
!
Sesja 7 / Session 7
!
Avoid CCED if it means that we will have to
forgo evidence that is needed to determine
appropriate use
Practical issues in CED decision
!
Must be clear agreement on
!
!
!
!
204
Evidence development study design
! To resolve identified uncertainties
! In acceptable timescale
Responsibility for funding treatment(s)
Responsibility for evidence development
! Funding
! Managing
! Access
! Analysis and reporting
Rules and responsibilities for decisions on
termination/modification of study in the light of
emerging evidence from study and elsewhere
Chris Henshall
Health Care
HTA & Pricing
www.ceestahc.org
Chris Henshall
Use of the evidence developed
!
Should be agreement from the outset on
responsibilities for coverage decision in
light of new evidence, including processes
for input and consultation
!
Will need to reflect and utilize systems and
criteria for related coverage decisions in
healthcare system – which may have
evolved since CED decision
!
!
!
!
!
!
!
Comments on CED
!
Comments on CED
!
Its not easy
!
All parties have concerns
!
!
!
!
To decide when and how to do it; to implement; or to manage
and make final decisions
Industry fears payers will use CED when the evidence is sufficient
to approve use; and/or demand unreasonable levels of certainty at
first or subsequent review points
Payers fear that industry will do less to ensure that costeffectiveness information is available at launch, hoping for a CED
decision to get market access and public funding for evidence
collection; and that system will get bogged down in large number
of studies and further reviews of evidence
Sesja 7 / Session 7
Industry analyses suggest
!
!
CCED reduces net present value of a technology
Hence incentives to
! improve outcome measures in Phase III trials
! Shift portfolio to technologies less likely to be subject to CED
205
Evidence-Based
!
!
!
!
!
!
!
Comments on CED
!
Other forms of agreement
!
!
2010 Forum meeting is on Managed Entry
Funders can
!
!
!
!
Conditions can include
!
!
!
!
!
Sesja 7 / Session 7
!
206
approve use of a treatment (at a given price)
not approve use (at a given price)
approve use subject to certain conditions
Collection of further evidence (CED)
Limitation to defined clinical indications
Limitation to those showing a defined response to initial
treatment/tests
Limitation on total volume per patient
Limitation on total volume for a population
Limitations on funding and/or repayment by company if agreed
health outcomes not achieved at patient and/or population level
Issues for discussion
!
Many agreements are being developed and
discussed – can we agree a clear typology?
!
Can health care delivery systems meet the
logistical demands of managed entry schemes?
!
How to handle multiple, competing or interacting
proposals for particular diseases?
!
What incentives do CED and ME create for
payers and manufacturers
!
Are there simpler ways to promote access?
Chris Henshall
Health Care
HTA & Pricing
www.ceestahc.org
Chris Henshall
Thank you
www.htai.org
Sesja 7 / Session 7
[email protected]
207
Evidence-Based
Sesja 7 / Session 7
208
Prelegent / Expert
Studium wykonalności dla Agencji Cen ze szczególnym uwzględnieniem działań
na rzecz porozumień podziału ryzyka / Feasibility of Pricing Agency in a CEE
country in handling Managed Entry Schemes
Wzmocnienie roli AOTM i Rady Konsultacyjnej
w polskim systemie ochrony zdrowia poprzez
wprowadzenie nowych zmian legislacyjnych w
2009 roku, czy projekt ustawy refundacyjnej
z prawdopodobnie nowoczesnymi kryteriami
refundacji napawają optymizmem. Jednocześnie rodzą się pytania w jakim zakresie zostaną
rozwiązane problemy negocjacji cenowych oraz
czy ewentualnie umowy podziału ryzyka będą
możliwe w polskim systemie ochrony zdrowia.
Zarzuty Komisji Europejskiej z dnia 29.06.2007
r. wobec Polski dotyczące m.in. braku kryteriów
cenowych i uzasadnień do rozstrzygnięć negocjacji cenowych budzą dodatkowy niepokój.
Kto zajmie się tymi aspektami w Polsce – Ministerstwo Zdrowia, Narodowy Fundusz Zdrowia,
czy może jakaś nowa instytucja?
Analizując obecną sytuację, potencjalne zadania (w tym negocjacje umów o podziale ryzyka), koszty powołania i koszty funkcjonowania
stosownej instytucji, autorzy książki „Pricing
– ceny leków refundowanych, negocjacje i podział ryzyka” napisanej pod redakcją Krzysztofa Łandy (Kraków 2009) przedstawili studium
wykonalności dla Agencji Cen w Polsce. Autorzy
dokonali przeglądu sytuacji w obszarze ustalania cen w Polsce oraz ocenę rozwiązań, które
były już testowane i/lub sprawdziły się w innych krajach. Na podstawie ankiet rozesłanych
do członków PPRI Network (Pharmaceutical Pricing and Reimbursement Information) opracowano pytania dotyczące idei, formy i technicznej strony prowadzenia aktywnej i racjonalnej
polityki cenowej. Studium wykonalności dotyczy kilku możliwych scenariuszy w zależności
od organizacji Agencji Cen i jej umiejscowienia
w systemie.
Jednym z głównym zadań Agencji, oprócz
wyceny świadczeń i negocjowania ich cen, powinno być zawieranie, monitorowanie i analiza
umów podziału ryzyka (risk sharing schemes,
RSS). Indywidualne porozumienia o podziale
ryzyka powstały w odpowiedzi na wzrastającą
niepewność co do wyników dotyczących skuteczności, bezpieczeństwa, czy kosztów wprowadzania nowych terapii i równocześnie umożliwiły realizację oczekiwań społecznych czy
politycznych. . W tradycyjnym systemie refundacji ryzykiem nie uzyskania efektu zdrowotnego i podjęcia złej decyzji refundacyjnej czy nadwyrężenia budżetu przeznaczonego na ochronę
Legislation changes introduced in 2009, enhancing the role of the AHTAPol and its Consultative Council in the Polish health care system,
or development of a new reimbursement act,
probably introducing modern reimbursement
criteria, are certainly optimistic signs. On the
other hand, important questions – to what degree problems related to price negotiations will
be solved and whether risk sharing schemes
will be permitted in the Polish health care system – remain open. Charges of the European Commission against Poland, formulated on
29.06.2007., including the lack of pricing criteria and justiÞcation of reimbursement decisions, may cause additional anxiety. Who will
be in charge of these issues in Poland – the
Ministry of Health, the National Health Fund, or
perhaps a new authority?
Having analyzed the current situation, potential tasks (including negotiation of risk sharing
schemes), and costs of creation and functioning
of an appropriate institution, the authors of “Pricing – prices of reimbursed drugs, negotiations
and risk sharing”, a book by Krzysztof Łanda
(ed.) et al. (Krakow 2009) presented a feasibility study for the Pricing Agency in Poland. The
authors reviewed the situation in the area of
pricing in Poland and evaluated solutions tested
and/or proven successful in other countries.
Based on a survey among the Pharmaceutical
Pricing and Reimbursement Information (PPRI)
Network members questions concerning the
idea, forms and technical aspects of active and
rational pricing policy were formulated. The feasibility study includes several scenarios, depending on organization of the Pricing Agency
and its place in the system.
One of the main tasks of the Agency, apart
from pricing of the services and negotiation of
prices, should be entering into, monitoring and
analysis of risk sharing schemes (RSS). Individual risk sharing schemes have been introduced in response to increasing uncertainty as
to results concerning safety, efÞcacy or costs
of introduction of new treatments, in order to
make it possible to meet social or political expectations. In a traditional reimbursement system the risk of failing to achieve the expected
health beneÞt, making a wrong reimbursement
decision or exceeding the budget allocated for
health care (among many other risks) is in-
20 min.
HTA & Pricing
zdrowia (jak i wielu innych ryzyk) obarczony
jest ubezpieczyciel (publiczny czy prywatny).
Umowy typy RSS dzielą ryzyko między ubezpieczyciela i producenta, ale zasadne są jedynie w
przypadku, gdy ryzyko występuje po obydwu
stronach. Podjęcie analizowanych aktywności
wymaga jednak umiejętności gromadzenia i
analizy odpowiednich danych, oraz kompetencji w zakresie negocjacji, monitorowania i implementowania umów RS w systemie.
Funkcjonowanie Agencji Cen może prowadzić
do zwiększenia przejrzystości polityki cenowej
państwa oraz stworzenia ram dla rozmów i wymiany informacji pomiędzy uczestnikami procesu negocjacji cen. Jednocześnie stworzenie
silnego centrum analityczno-negocjacyjnego
(Agencji Cen) pozwoliłoby na wprowadzenie
mechanizmów - z jednej strony – utrzymujących dyscyplinę budżetową, z drugiej strony
- pozwalających na wprowadzenie nowych lub
innowacyjnych technologii do systemu ochrony
zdrowia.
www.ceestahc.org
curred by the payer (public or private). In a
RSS the risk is divided between the insurer and
the manufacturer; however, such a scheme is
feasible only if the risk affects both parties.
Activities associated with RSS require speciÞc
skills related to collection and analysis of relevant data, as well as competent negotiation,
monitoring and implementation of RSS in the
system.
Functioning of the Pricing Agency may
increase transparency of the state’s pricing policy as well as create framework for information
exchange between the parties to price negotiations. At the same time the Pricing Agency,
being a strong center for analysis and negotiation, would make it possible to introduce
mechanisms keeping the budget discipline on
one hand, while on the other allowing for introduction of new or innovative technologies into
the health care system.
Sesja 7 / Session 7
Health Care
209
Evidence-Based
Kraków, 8 grudnia 2009 r.
Studium wykonalno!ci dla
Agencji Cen ze szczególnym
uwzgl"dnieniem dzia#a$ na
rzecz porozumie$ podzia#u
ryzyka
Zarzuty Komisji Europejskiej z dnia 29.06.2007 r.
w stosunku do Polski
%
%
%
%
%
%
Sesja 7 / Session 7
%
%
210
%
niedotrzymywanie terminów rozstrzygni!" refundacyjnych oraz cenowych
brak uzasadniania rozstrzygni!" refundacyjnych oraz cenowych
brak weryfikowalnych kryteriów dotycz#cych rozstrzygni!" refundacyjnych
oraz cenowych
brak uzasadniania dla kwalifikacji do poszczególnych poziomów
refundacyjnych
brak definicji leków na choroby zaka$ne lub psychiczne dla osób z
upo%ledzeniem umys&owym
brak okre%lenia warunków, wg których nast!puje kwalifikacja do wykazu
niektórych chorób przewlek&ych
brak zapewnienia procedury odwo&awczej
dzia&ania protekcyjne w stosunku do polskich producentów leków
generycznych
dzia&ania ograniczaj#ce umieszczanie na li%cie leków innowacyjnych
Aktualna sytuacja w Polsce
% Ustawa o #wiadczeniach opieki zdrowotnej
finansowanych ze #rodków publicznych (Dz. U. 2008, Nr
164, Poz. 1027
z pó!n. zm.)
% Ustawa o cenach (Dz. U. 2001, Nr 97, Poz. 1050 z pó!n.
zm.)
% Ustawa refundacyjna ?
Health Care
HTA & Pricing
www.ceestahc.org
Czy zmiany legislacyjne pozwoli#y
na wprowadzenie:
% racjonalnej polityki cenowej?
% przejrzysto%ci ustalania cen i wyceny %wiadcze'
zdrowotnych?
% poprawy wydajno%ci procesów oceny wniosków
i ustalania cen?
% mo(liwo%ci wprowadzenia umów indywidualnych
(w tym umów podzia& ryzyka) do systemu?
Studium wykonalno!ci dla
Polskiej Agencji Cen
Aktualna sytuacja w Polsce
%ramy prawne
%instytucje kszta&tuj#ce i maj#ce
wp&yw na polityk! lekow#
(cenow# i refundacyjn#)
Funkcjonowanie Agencji Cen w krajach UE
Sesja 7 / Session 7
Funkcjonowanie Agencji Cen
w Europie
&Struktura
&Zadania
&Pracownicy
211
Evidence-Based
Agencja cen
jako funkcjonalno!% systemu
a nie okre%lona organizacja czy instytucja
Funkcjonalno!% systemu: „Agencji Cen”
Agencja Cen
w MZ
Agencja Cen
w NFZ
Agencja Cen
w AOTM
Sesja 7 / Session 7
Agencja Cen
212
Aspekty rozpatrywane w ramach tworzenia
modeli Agencji Cen
Health Care
HTA & Pricing
www.ceestahc.org
Potencjalne zadania Agencji
1. negocjowanie oraz ustalanie cen urz!dowych leków
i wyrobów medycznych
2. wycena %wiadcze'
3. monitorowanie cen
4. analiz! propozycji i zawieranie umów podzia&u
ryzyka (RSS)
5. monitorowanie wykonywania umów RSS
6. mi!dzynarodowa wymiana informacji cenowych
7. zakupy centralne
Model funkcjonowania Agencji Cen
Zadania
Wycena
1 substancja – 2 osoby
(kontrola jako%ci danych)
1 osoba – 100 substancji
Sesja 7 / Session 7
Co ocenia
Agencja
Cen
Negocjacje
Zespó& negocjacyjny – 3 osoby
- osoba decyzyjna
- osoba wykonuj#ca wycen!
- negocjator
213
Evidence-Based
Model funkcjonowania Agencji Cen
Zadania
Wycena
Min. - leki Top 100 + ocenione przez
AOTM
Max. - leki finansowane ze %rodków
publicznych+ ocenione przez
AOTM
Negocjacje
• 100 – 150 wniosków o ustalenie
nowej ceny
• 800 wniosków o zmian! ceny
(generyki – obni(enie referencji)
Sesja 7 / Session 7
Koszty funkcjonowania Agencji Cen
z bud&etu pa$stwa
214
Analiza ryzyka
Health Care
HTA & Pricing
www.ceestahc.org
Dzi"kuj"
Cezaremu G&ogowskiemu
Krzysztofowi )andzie
Jakubowi Adamskiemu
Robertowi Plisko
Joannie Lis
Kamili Wendykowskiej
Sesja 7 / Session 7
autorom rozdzia#u „Studium
wykonalno!ci dla Agencji Cen w Polsce”
215
Wtorek
8 grudnia 2009
Tuesday
December 8th, 2009
EBHC HTA & Pricing
www.ceestahc.org
Sesja 8 / Session 8
DeÞniowanie problemów decyzyjnych (APD)
Scoping
Joanna Lis – 35 min.
Krzysztof Łanda – 25 min.
Ostatnia sesja IV Sympozjum poświęcona będzie problematyce deÞniowania problemów decyzyjnych (scoping). Wszelkie decyzje podejmowane w ochronie zdrowia nie
powinny być wyborami osobistymi, losowymi czy nieświadomymi. Każda z nich niesie
za sobą bardzo poważne konsekwencje.
Często błędem jest podejmowanie decyzji
bez przeprowadzenia rzetelnych analiz, czy
bez wykonania studiów wykonalności dla
opcjonalnych rozwiązań. Z drugiej strony
czasem zdarza się, że dostępne analizy nie
odpowiadają na potrzeby decyzyjne. Ich
zakres jest niepełny, kierunki analityczne
błędne, zastosowane narzędzia analityczne
pozostawiają wiele do życzenia, a wyciągnięte wnioski nie odpowiadają na pytania
decydenta.
The last session of the 4th Symposium
will focus on deÞning of decision problems
(scoping). In decisions concerning health
care there should be no room for personal, random or subconscious choices. Each
decision entails extremely serious consequences. Making a decision without proper
analyses or feasibility studies regarding
optional solutions is a common mistake.
On the other hand, available analyses are
sometimes inadequate for decision making. Their scope may be incomplete, analysis wrongly directed, applied analytical
tools may leave much to be desired and
the conclusions drawn may not answer the
decision maker’s questions.
217
Evidence-Based
Sesja 8 / Session 8
Jeśli chodzi o refundację czy wycenę,
każda ocena technologii medycznej powinna być a priori precyzyjnie ukierunkowana,
by nie stać się oderwaną od rzeczywistości i potrzeb decyzyjnych. Te potrzeby decyzyjne muszą więc być określone zanim
analitycy rozpoczną zbieranie danych, czy
syntezę. Wytyczne oceny technologii medycznych poszczególnych krajów przedstawiają różne drogi osiągnięcia wysokiej
jakości analiz HTA. Zgodnie ze zaktualizowanym wytycznymi AOTM w Polsce, na
wzór brytyjskiego NICE, istotną wagę przywiązuje się do scoping’u. Należy podkreślić, że deÞniowanie problemu decyzyjnego
nie stanowi wyłącznie opisu opcjonalnych
technologii medycznych i wskazania, ale
jest fundamentem wiarygodnej, a nade
wszystko użytecznej ich oceny. Do określenia zakresu porównań wykorzystuje się
schemat PICO (population, intervention,
comparison, outcome). Często dodatkowo
ująć należy subpopulacje, w których dana
technologia może być szczególnie opłacalna, specyÞczne warunki stosowania interwencji, faktycznie stosowaną praktykę,
dłuższy niż w badaniach klinicznych horyzont czasowy, w którym korzyści i koszty
są rozważane, czy część koszyka świadczeń gwarantowanych, o wpisanie do którego producent aplikuje.
Sesja w całości poświęcona będzie teorii i praktyce deÞniowania problemów decyzyjnych na rzecz oceny technologii medycznych.
218
As to reimbursement or pricing, evaluation of each health technology should be
precisely directed a priori, so as not to become unrealistic or irrelevant to decision
needs. These decision needs must therefore be determined before the analysts begin
to collect or synthesize data. Guidelines
concerning health technology assessment
implemented in speciÞc countries represent
different ways to high-quality HTA analyses. According to updated Polish (AHTAPol) guidelines, based on those published
by the NICE, scoping plays an important
role. It must be stressed that deÞnition of
a decision problem is not just a description
of optional health technologies and the indication, but a foundation for their credible
and – in the Þrst place – useful assessment. The scope of comparison is usually
deÞned using the PICO (population, intervention, comparator, outcome) formula. In
addition, speciÞc subpopulations, in which
a particular technology may be especially cost-effective, speciÞc circumstances,
in which an intervention is used, current
practice, a time horizon, longer than that
of clinical trials, in which costs and beneÞts
are evaluated, or a part of the guaranteed
beneÞt package, in which the technology
under consideration will be placed, should
often also be taken into account.
During the whole session theory and
practice of deÞning of decision problems
in health technology assessment will be
discussed.
HTA & Pricing
35 min.
www.ceestahc.org
Prelegent / Expert
DeÞniowanie problemów decyzyjnych w procesie oceny technologii medycznych:
teoria i praktyka / Scoping in health technology assessment: theory and practice
Według teorii decyzji, sformułowanie problemu decyzyjnego jest zazwyczaj pierwszym
krokiem do zbudowania modelu decyzyjnego.
Dobrze sformułowany problem powinien szczegółowo deÞniować: decydenta lub decydentów,
warunek ograniczający decyzje, zbiór decyzji
dopuszczalnych oraz kryteria oceny decyzji.
Procesy decyzyjne w ochronie zdrowia, która
stanowi jeden z najbardziej złożonych strukturalnie i funkcjonalnie systemów, podejmowane
są na różnych poziomach, przez liczne podmioty. Użytecznym narzędziem w podejmowaniu decyzji w ochronie zdrowia jest ocena
technologii medycznych (HTA). Celem HTA jest
dostarczanie opartych na racjonalnych podstawach informacji, które są niezbędne do podejmowania decyzji z zakresu polityki zdrowotnej
oraz wspomaganie racjonalnego wykorzystania
zasobów przeznaczonych na ochronę zdrowia i
uzyskaniu optymalnych efektów o jak największej wartości dla pacjentów.
Analiza procesu decyzyjnego jest jedną z
części procesu oceny technologii medycznych
w Polsce i na świecie
W Polsce, pierwszym etapem przeprowadzanej analizy procesu decyzyjnego jest jasne
sprecyzowanie badanej technologii, interwencji
diagnostycznej, proÞlaktycznej lub terapeutycznej, stosowanych w określonej sytuacji klinicznej.
Wymagany jest pełny opis zagadnień kontekstu klinicznego według schematu PICO:
• populacji, w której dana interwencja ma być
stosowana (P);
• proponowanej interwencji (I);
• komparatorów (C);
• efektów zdrowotnych, czyli punktów końcowych badań klinicznych (O).
Joanna Lis
According to the theory of decision, formulation of the decision problem is usually the Þrst
step in construction of a decision model. A wellformulated problem should deÞne in detail: the
decision maker(s), conditions limiting the decision, a set of possible decisions and the criteria
for assessment of a decision.
Decision processes in health care, constituting one of the most complex systems in terms
of structure and function, are made by numerous entities at various levels. Health technology assessment (HTA) is a useful tool for decision-making in health care. The aim of HTA is to
provide rationally based information, necessary
for decision-making in health care policy, and to
support rational utilization of resources allocated for health care in order to obtain optimum
effects and the highest value for patients.
Decision process analysis is a part of health
technology assessment in Poland and worldwide.
In Poland the Þrst stage of the decision problem analysis is clear deÞnition of the investigated technology, i.e. a diagnostic, preventive
or therapeutic intervention used in a speciÞc
clinical situation.
The clinical context should be presented in
detail, according to the PICO formula:
• the population, in which the intervention
should be used (P);
• the proposed intervention (I);
• comparators (C);
• outcomes, i.e. endpoints of clinical trials
(O).
Sesja 8 / Session 8
Health Care
219
Evidence-Based
Sesja 8 / Session 8
W Wielkiej Brytanii, NICE opracowuje najpierw zakres oceny problemu decyzyjnego tzw.
draft scope a następnie zleca ocenę tematu tzw.
Þnal scope. DeÞniowane są następujące parametry: problem kliniczny i populacja docelowa,
oceniana technologia, komparatory, istotne klinicznie punkty końcowe dla danej technologii,
koszty, horyzont czasowy, w którym powinny
być oceniane koszty i efekty zdrowotne. Ponadto identyÞkowane są subpopulacje pacjentów,
dla których technologia mogłaby być klinicznie
i kosztowo efektywna. Brane są także pod uwagę ilościowe oszacowanie istniejących dowodów
naukowych dla ocenianej technologii oraz zagadnienia związane z równością w dostępie do
świadczeń medycznych i/lub sposób zapobiegania nierówności, jak również inne, potencjalnie
istotne dla tworzenia wytycznych elementy
Wytyczne przeprowadzenia oceny technologii medycznych w innych krajach, zawierają
także najważniejsze informacje, które pozwalają w mniejszym lub większym stopniu zidentyÞkować i ocenić problem decyzyjny.
Jasno zdeÞniowany problem decyzyjny pozwala na rzetelne przygotowanie wiarygodnych
analiz HTA, które umożliwiają racjonalne podejmowanie decyzji w ochronie zdrowia.
220
Joanna Lis
In the United Kingdom, the NICE deÞnes Þrst
the scope of a decision problem analysis, so
called “draft scope”, and then proceeds to the
analysis itself, so-called “Þnal scope”. The following issues are deÞned: the clinical problem
and target population, the evaluated technology, the comparators, clinically important endpoints for a speciÞc technology, costs, and the
time horizon, in which costs and health effects
will be evaluated. In addition, subpopulations
of patients are identiÞed, in whom the technology could be effective and cost-effective.
Quantitative estimations of existing evidence
for the evaluated technology are also taken into
account, as well as problems related to equal
access to health care services and/or measures
to prevent inequity, and other issues potentially
important for development of guidelines.
Guidelines on health technology assessment
published in other countries also contain essential information making it possible (more
or less) to identify and evaluate the decision
problem.
Clearly deÞned decision problems allow for
reliable development of credible HTA analyses,
thus making it possible to make rational decisions in health care.
Health Care
HTA & Pricing
www.ceestahc.org
Joanna Lis
Definiowanie PROBLEMÓW DECYZYJNYCH
w procesie oceny technologii medycznych:
teoria i praktyka
SCOPING in
Health Technology Assessment:
theory & practice
Joanna Lis
Health Economics & Market Access Manager
Sanofi-Aventis
4th International Symposium EBHC: HTA & Pricing
Kraków, December 8th, 2009
Problem decyzyjny
Sytuacja problemowa, w której podmiot
(DECYDENT) staje przed konieczno!ci"
wyboru jednego z przynajmniej dwóch
mo#liwych wariantów dzia$ania
Problem decyzyjny
'
'
Sformu$owanie problemu decyzyjnego jest
zazwyczaj pierwszym krokiem do
zbudowania MODELU DECYZYJNEGO
Dobrze sformu$owany problem powinien
szczegó$owo definiowa%:
•
•
•
•
decydenta lub decydentów
warunek ograniczaj"cy decyzje
zbiór decyzji dopuszczalnych
kryteria oceny decyzji
Sesja 8 / Session 8
'
221
Evidence-Based
Problem decyzyjny
'
decydent
'
'
'
podmiot dokonuj"cy wyboru ostatecznego wariantu decyzji
w przypadku kiedy decydent jest zbiorowy, nale#y ustali% odpowiednie
procedury umo#liwiaj"ce podj&cie decyzji, kiedy poszczególne osoby
uwa#aj" ró#ne warianty za optymalne (np. g$osowanie)
warunek ograniczaj"cy decyzje
'
'
Warunek taki ogranicza przestrze' decyzyjn" do pewnego podzbioru
decyzji
Warunki ograniczaj"ce mo#na podzieli% ze wzgl&du na ich wp$yw na
decyzje na:
• warunki sztywne - usuni&cie warunku powoduje zmian& zbioru decyzji
optymalnych
• warunki lu!ne - usuni&cie warunku nie powoduje zmiany zbioru decyzji
optymalnych
'
zbiór decyzji dopuszczalnych
'
kryteria oceny decyzji:
'
'
'
zbiór wszystkich decyzji, która spe$niaj" wszystkie warunki
ograniczaj"ce decyzje
przyporz"dkowanie ka#dej dopuszczalnej decyzji, ilo!ciowej lub
jako!ciowej oceny korzy!ci, wynikaj"cych z podj&cia takiej decyzji
cz&sto kryterium oceny nazywane jest celem decyzji
Problemy decyzyjne w ochronie
zdrowia
Sesja 8 / Session 8
Procesy decyzyjne w ochronie zdrowia,
która stanowi jeden z najbardziej z$o#onych
strukturalnie i funkcjonalnie systemów,
podejmowane s" na ró#nych poziomach,
przez liczne podmioty
222
HTA a Problemy decyzyjne w
ochronie zdrowia
'
(INAHTA 2008):
• HTA - mi&dzydyscyplinarna analiza medycznych,
ekonomicznych, socjalnych i etycznych implikacji
rozwoju, rozpowszechniania i u#ycia technologii
medycznych
'
Celem HTA jest:
• dostarczanie opartych na racjonalych
podstawach informacji, które s" niezb&dne do
podejmowania decyzji z zakresu polityki
zdrowotnej
• wspomaganie racjonalnego wykorzystania
zasobów przeznaczonych na ochron& zdrowia i
uzyskaniu optymalnych efektów o jak
najwi&kszej warto!ci dla pacjentów
Joanna Lis
Health Care
HTA & Pricing
www.ceestahc.org
Joanna Lis
I
A
IZ "C
AL NO J
AN YW ZNE
T
C
EK NI
EF KLI
A
N NA
O A S LIZ
CH Y A
RO ST W
N EM P#
Y
YW
ZD
U
RO
W
IA
HTA: Ocena Technologii Medycznych
ANALIZA
PROBLEMU
DECYZYJNEGO
ANALIZA
EKONOMICZNA
Analiza problemu
decyzyjnego w Polsce
AOTM
Sesja 8 / Session 8
ANALIZA PROBLEMU DECYZYJNEGO - Scoping the project - mind map
223
Evidence-Based
Analiza problemu decyzyjnego w
Polsce – wytyczne AOTM
'
•
Definiowanie problemu:
Pierwszym etapem przeprowadzanej analizy jest jasne
sprecyzowanie badanej technologii, interwencji
diagnostycznej, profilaktycznej lub terapeutycznej,
stosowanych w okre!lonej sytuacji klinicznej.
•
Wymagany jest pe$ny opis zagadnie' kontekstu
klinicznego wed$ug schematu PICO
•
W przypadku analiz do$"czanych do wniosków
o finansowanie technologii ze !rodków publicznych,
kontekst kliniczny analiz musi odpowiada% opisanemu
we wniosku
•
Nale#y równie# wskaza%, które technologie, i w jakim
stopniu mog" zosta% zast"pione przez technologi&
ocenian".
'
Analiza problemu decyzyjnego zgodnie ze
schematem PICO (S):
(P) Populacja
(I) Interwencja
(C) Komparatory (interwencje do porównania)
(O) Efekty zdrowotne, czyli punkty ko'cowe
bada' klinicznych
• (S) typy bada' klinicznych (modyfikacja PICO
do PICOS)
Sesja 8 / Session 8
•
•
•
•
224
Populacja:
'
•
Nale#y przedstawi% charakterystyk& docelowej populacji
lub populacji, która b&dzie poddawana ocenianej
interwencji.
•
Opis ów powinien obejmowa% podstawowe informacje
o chorobie lub problemie zdrowotnym, z uwzgl&dnieniem
historii naturalnej choroby, rokowania i stosowanych
obecnie metod diagnostycznych lub terapeutycznych.
•
Trzeba okre!li% potencjaln" liczebno!% populacji i opisa%
metod& jej oszacowania oraz uzasadni% j".
Joanna Lis
Health Care
HTA & Pricing
www.ceestahc.org
Joanna Lis
Interwencja
'
•
Powinno si& scharakteryzowa% ocenian" interwencj&
zdrowotn"
•
W przypadku interwencji zarejestrowanej w Polsce
nale#y poda% dat& rejestracji lub dat& pierwszej
deklaracji zgodno!ci wyrobu medycznego
i zarejestrowane wskazania, porównuj"c je
ze wskazaniami rozpatrywanymi w analizie
•
Dla technologii niezarejestrowanych w Polsce,
a zarejestrowanych w innych krajach, w$a!ciwe jest
podanie wybranych dat i miejsc ich rejestracji w tych#e
krajach oraz warunków okre!lonych przez instytucje
rejestruj"ce – w szczególno!ci przez EMEA i FDA
•
Komparatory
Analiza kliniczna polega na porównaniu skuteczno!ci
i bezpiecze'stwa stosowania ocenianej interwencji (sposobu
post&powania) z wynikami innych interwencji (opcjonalnych
sposobów post&powania), stosowanych w docelowej populacji.
•
Komparatorem dla ocenianej interwencji w pierwszej kolejno!ci
musi by% tzw. istniej$ca praktyka. Jest to sposób
post&powania, który w rzeczywistej praktyce medycznej
prawdopodobnie zostanie zast"piony przez ocenian" technologi&.
•
Zaleca si& przeprowadzenie porównania równie# z innymi
komparatorami, czyli z technologiami:
najcz&!ciej stosowan",
najta'sz",
najskuteczniejsz"
zgodn" ze standardami i wytycznymi post&powania klinicznego
'
'
'
'
•
Istotne jest, aby wybrane komparatory odpowiada$y warunkom
polskim. Ich wybór powinien by% rzetelnie uzasadniony oraz
opatrzony (ród$ami danych.
'
•
Efekty zdrowotne
W analizie klinicznej powinny by% ocenianie efekty zdrowotne, które
stanowi" istotne klinicznie punkty ko'cowe, odgrywaj"ce znaczn" rol&
w danej jednostce chorobowej, tj.:
•
•
•
•
•
zgony,
zachorowania b"d( wyleczenia,
jako!% #ycia
dzia$ania niepo#"dane (z podzia$em na ci&#kie i pozosta$e) i/lub incydenty medyczne
Punkty ko'cowe w analizie klinicznej powinny:
'
'
wi"za% si& z ocenian" jednostk" chorobow" i jej przebiegiem, odzwierciedla%
wszystkie medycznie istotne aspekty problemu zdrowotnego i jednocze!nie
umo#liwia% wykrycie potencjalnych ró#nic mi&dzy porównywanymi interwencjami;
mie% zasadnicze znaczenie dla podejmowania racjonalnej decyzji (punkty
krytyczne danego problemu zdrowotnego)
•
W przypadku, kiedy nie stwierdzono bada' klinicznych z klinicznie istotnymi
dla pacjenta punktami ko'cowymi, jako wyniki mog" by% oceniane
surogaty Zalecane jest wtedy przedstawienie w analizie zwi"zku pomi&dzy
u#ytymi surogatami, a klinicznie istotnymi punktami ko'cowymi.
•
W przypadku, kiedy wyniki oceny klinicznej uzyskane s" przy u#yciu skal
lub kwestionariuszy, nale#y przedstawi% informacje o ich walidacji, oraz
istotno!ci klinicznej wyników
Sesja 8 / Session 8
'
225
Evidence-Based
Istotny klinicznie punkt ko%cowy maj$cy znaczenia
dla pacjenta * - parametr/wynik, którego zmiana pod
wp$ywem leczenia sprawi$aby, #e to leczenie b&dzie
po#"dane przez chorych
'
'
•
•
•
Odzwierciedlaj" wp$yw leczenia:
przed$u#aj"cy #ycie,
poprawiaj"cy samopoczucie chorego b"d(
pozwalaj"cy #y% bez powik$a' choroby lub jej leczenia
* (ang. clinically important endpoint, clinically relevant endpoint, patient
important outcome, patient-oriented endpoint)
Ocena Technologii Medycznych w Polsce:
AOTM i HT ’A’
'
'
Sesja 8 / Session 8
'
226
A – APD - analiza problemu
decyzyjnego
A - Assessment – ocena analityczna
wykonywana przez Wydzia$ AOTM
A - Appraisal - ocena warto!ciuj"ca
przygotowywana przez Rad&
Konsultacyjn" przy AOTM
Scoping
w Wielkiej Brytanii
NICE
Joanna Lis
Health Care
HTA & Pricing
www.ceestahc.org
Joanna Lis
NICE: Etapy procesu oceny technologii
medycznych i wydawania rekomendacji w
Wielkiej Brytanii
2.
3.
4.
5.
6.
7.
8.
9.
10.
Wst&pny wybór tematów do oceny
Zidentyfikowanie „konsultantów” oraz „komentatorów”
Opracowanie zakresu oceny – draft scope
Zlecenie NICE oceny tematu – final scope
Opracowanie raportu oceny przez niezale#ny o!rodek akademicki oraz
poddanie raportu konsultacji
Pierwsze spotkanie niezale#nego Komitetu Oceniaj"cego i opracowanie
wst&pnej rekomendacji (ACD)
Poddanie dokumentu ACD procesowi konsultacji - 4 tygodnie
Uwzgl&dnienie uwag po poddaniu dokumentu konsultacji i opracowanie
ostatecznej rekomendacji (FAD).
Przekazanie FAD do akceptacji NICE; mo#liwo!% odwo$ania si& od
decyzji Komitetu (tylko „konsultanci”)
Opublikowanie ostatecznej rekomendacji jako „NICE Guidance”
Wielkiej Brytanii - NICE
Wybór tematu
Identyfikacja zainteresowanych
organizacji i podmiotów
Przeszukiwanie literatury
i kompilacja informacji
Stworzenie Draft Remit&Scope i
Identyfikacja wa#nych w"tków
Konsultanci/
Komentatorzy
Konsultacje
Assessment Appraisal
Final Remit&Scope
Scoping w UK/NICE:
Definiowane parametry
'
'
'
'
'
'
'
'
'
'
Problem kliniczny i populacja docelowa
Oceniana technologia
Komparatory
Istotne klinicznie punkty ko'cowe dla danej technologii
Koszty
Horyzont czasowy, w którym powinny by% oceniane koszty i
efekty zdrowotne
Identyfikacja subpopulacji pacjentów, dla których
technologia mog$aby by% klinicznie i kosztowo efektywna
Ilo!ciowe oszacowanie istniej"cych dowodów naukowych
dla ocenianej technologii
Zagadnienia zwi"zane z równo!ci" w dost&pie do !wiadcze'
medycznych i/lub sposób zapobiegania nierówno!ci
Inne, potencjalnie istotne dla tworzenia wytycznych
elementy
Sesja 8 / Session 8
1.
227
Evidence-Based
O scopingu w innych
agencjach HTA
Spain
Netherlands
Switzerland
Austria
Slovak Republic
Yes
Approved indication; offlabel use is considered for
comparator
Yes
Yes
Approved indication
Yes
Be clearly specify
Yes, usually is
determined by a
precise indication
Be clearly specify
Relevant groups or subgroups need to be
defined.
Most efficient
options. Most
used options.
Real situation.
Do nothing
option
Treatment in clinical
guidelines of GPs; if not
available most prevalent
treatment
Closest alternative
technology, first
choice treatment,
non-intervention
Standard
therapy, most
frequent therapy
or most effective
therapy
The most relevant
alternative treatment
which is either the the
treatment that is most
likely to be replaced by
the new treatment
Years of life
gained. QALYs
Effectiveness by intentionto-treat principle, and
expressed in natural units
(pref life-years gained) or
QALY
Life years gained or
lost, health related
quality of life, quality
corrected life years
gained or lost
Depends on
research question
Final outcome
parameters: life years
gained (CEA) or QALYs
gained (CUA) for
chronic conditions
INDICATION
POPULATION
COMPARATORS
OUTCOMES
Sesja 8 / Session 8
Belgium
228
France
Germany
Hungary
Italy
Portugal
Approved
indication
Approved indication
Approved within
the health-care
system
Licensed one(s)
Yes
Target of the
treatment
Consistent with
the clinical file
Relevant subgroups need to be
defined.
The target population
of the evaluation
must be clearly
described.
Be clearly
specified
Yes
Yes
Yes, described in great
detail
The most frequently
used (inc nontreatment) or newer
strategies which may
legitimately be
deemed likely to
become reference
strategies in the very
near future
Standard
treatment
(others may
apply but have to
be justified)
Current
accepted
standard
therapy that
could be
replaced. The
selection should
be justified.
Most wide-spread
treatment. Solid
evidence for a
scientifically-based
comparison must be
available.
The most common
treatment, less
expensive and most
efficacious
COMPARATORS
The most relevant
alternative
treatment which is
either the the
treatment that is
msot likely to be
replaced by the
new treatment
Final outcomes
preferred
Valid and reliable
profiling and
indexing
instruments
should be used
for measuring
quality of life
Final outcome
and changes in
QoL, QALY
Effectiveness by
intention-to-treat
principle, and
expressed in natural
units or QALY
Can not say which one
is better than the
other. Be validated for
Portugal and justify
the choice.
OUTCOMES
Final outcome
parameters: life
years gained
(CEA) or QALYs
gained (CUA) for
chronic conditions
INDICATION
POPULATION
Joanna Lis
Health Care
HTA & Pricing
Scotland
Sweden
England & Wales
Yes
Clearly define the spectrum of
diseases
Not specified
Not stated
Yes
Yes, includes age and sex
distribution and co-morbidities
It should be clearly defined
which patients are expected to
be treated with the analysed
pharmaceutical
Most likely to be
displaced in
Scotland
Most used
Relevant comparators for the
technology being appraised
are those routinely used in
the NHS, and therapies
regarded as best practice
when this differs from routine
practice.
Not specified
Need clearly
explained
QALY. If CBAWTP
Given its widespread use, the
QALY is considered to be the
most appropriate generic
measure of health benefit that
reflects both mortality and
HRQL effects.
Outcome measures relevant to
specific treatement
recommended (successfull
treatments, time without
symptoms or pain, life years,
quality adjusted life years.
Willingness to pay estimates
should not be only outcomess
but can be used as a supplement
POPULATION
COMPARATORS
OUTCOMES
Baltic (Latvia,
Lithuania, Estonia)
Finland
Ireland
Norway
Approved one(s)
Indication approved for the
medicinal product for which
reimbursement status is applied
or, if there are several indications,
the most important one or ones
Study question be
clearly stated
Approved
indication
Yes
Clearly specified
Not stated
Yes
Most commonly used
alternative or practice. Be
justified.
To be replaced product, most
commonly used treatment, the
best or minimum therapy. Need
justification
Rationale be given,
explain the
alternative in detail
Most prevalent
treatment, most
inexpensive
treatment, no
treatment
Change in the health
state. Absolute risk
difference calculated
Not stated
Be clearly stated.
Not stated
INDICATION
www.ceestahc.org
Joanna Lis
Denmark
Not stated
INDICATION
POPULATION
OUTCOMES
Analiza problemu decyzyjnego current practice
'
'
Streszczenie
Analiza problemu
• Cel i metodyka
• Problem decyzyjny
• Populacja
'
'
Opis choroby, epidemiologia, etiopatogeneza, symptomatologia,
rozpoznanie, czynniki ryzyka, rokowanie, leczenie, waga problemu
zdrowotnego
Wytyczne praktyki klinicznej
• Interwencja
'
'
'
•
•
•
•
Status finansowania danej technologii
Propozycje finansowania ze !rodków publicznych
Aktualne rekomendacji z innych agencji HTA
Wst&pna analiza kliniczna
Analiza kosztów- g$ówne dane kosztowe
Proponowany zakres analiz HTA
Identyfikacja nowych informacji mog"cych mie% wp$yw na
problem decyzyjny w najbli#szym czasie
Sesja 8 / Session 8
COMPARATORS
229
Evidence-Based
Definiowanie PROBLEMÓW DECYZYJNYCH
w procesie oceny technologii medycznych
pozwala na !wiadome i racjonalne
podejmowanie decyzji w ochronie
zdrowia
' pozwala na przygotowanie analiz
HTA, które odpowiadaj" na pytania
decydenta
' Pozwalaj" na prowadzenie
racjonalnej polityki zdrowotnej
Sesja 8 / Session 8
'
230
Joanna Lis
Health Care
HTA & Pricing
www.ceestahc.org
Prelegent / Expert
Krzysztof Łanda
Painful lessons from stepping in the HTA path
without prior scoping – tips for HTA doers
Bolesne do!wiadczenia z HTA bez
wcze!niejszej analizy problemu decyzyjnego
– wskazówki dla wytwórców raportów HTA
Krzysztof "anda
HTA Audit
Bardzo precyzyjna i
przejrzysta mapa Polski, ale
… jeste"my przecie# w
Marrakeszu …
Wysokiej jako"ci analizy,
zgodne z wytycznymi, ale …
nie odpowiadaj$ na nasz
problem decyzyjny!!!
Dokumenty #ród$owe
Procedury wewn$trzne CMJ (2000-2002), HTA Consulting
(2002-2006) i HTA Audit (2007-2009)
Guide to the methods of technology appraisal, NICE, June 2008
Wytyczne Oceny Technologii Medycznych (HTA), AOTM, luty 2009
________________________________________________
Przyk%ady na podstawie audytów wykonanych przez HTA Audit
Sesja 8 / Session 8
25 min.
Bolesne doświadczenia z HTA bez wcześniejszej analizy problemu decyzyjnego
– wskazówki dla wytwórców raportów HTA / Painful lessons from stepping in the
HTA path without prior scoping – tips for HTA doers
231
Evidence-Based
Technology appraisal by NICE - etapy
Jasne i precyzyjne
zdefiniowanie
problemu decyzyjnego
SCOPING
ASSESMENT
APPRAISAL
Ocena analityczna
zebranych dowodów /
informacji
Ocena warto#ciuj&ca,
interpretacja wyników i
wnioskowanie
Etapy procesu oceny technologii
medycznych i wydawania
rekomendacji
1.
2.
3.
4.
Wst%pny wybór tematów do oceny (DH)
Zidentyfikowanie „konsultantów” oraz „komentatorów”
Opracowanie zakresu oceny (DH, NICE) – draft scope
Zlecenie NICE oceny tematu (DH) – final scope
Opracowanie raportu oceny przez niezale'ny o#rodek akademicki oraz
poddanie raportu konsultacji
6. Pierwsze spotkanie niezale'nego Komitetu Oceniaj&cego i opracowanie
wst%pnej rekomendacji (ACD)
7. Poddanie dokumentu ACD procesowi konsultacji - 4 tygodnie
8. Uwzgl$dnienie uwag po poddaniu dokumentu konsultacji i opracowanie
ostatecznej rekomendacji (FAD).
9. Przekazanie FAD do akceptacji NICE; mo&liwo!' odwo$ania si% od
decyzji Komitetu (tylko „konsultanci”)
10. Opublikowanie ostatecznej rekomendacji jako „NICE Guidance”
Sesja 8 / Session 8
5.
232
Scoping
vs „ekspertyzy wst%pne”
Ekspertyzy wst%pne tworzone by$y
• od samego pocz&tku HTA w Polsce od roku
2000 w CMJ (Centrum Monitorowania Jako#ci
w Ochronie Zdrowia w Biurze Standaryzacji)
• a nast$pnie w HTA Consulting od 2002 roku
do 2006 i pó(niej
• od 2009 wytyczne AOTM wprowadzi%y
konieczno#) opracowywania APD (analiz
problemu decyzyjnego)
Krzysztof Łanda
Health Care
HTA & Pricing
www.ceestahc.org
Krzysztof Łanda
Ekspertyza wst%pna (EW)
stanowi …
… co# w rodzaju swoistego protoko$u
analizy – podobnie jak opracowuje si$
protoko%y przed przeprowadzeniem
bada* klinicznych, czy protoko%y przed
rozpocz$ciem przegl&du
systematycznego w przypadku
Cochrane Collaboration, ale EW jest
czym# wi$cej ni' protoko%em ….
Zapisy SOP HTA C z 2002 roku:
Standardowa ekspertyza wst!pna
obejmuje:
•
•
•
•
•
zapoznanie si% Zespo&u Projektowego z problemem zdrowotnym oraz
porównywanymi opcjami b%d$cymi przedmiotem analizy
przedstawienie wyników wyszukiwania bada' klinicznych dla
porównywanych opcji po zastosowaniu wst!pnie opracowanej strategii
wyszukiwania
przedstawienie mo"liwych kierunków dalszych prac
przedstawienie podstawowych kategorii kosztowych, które
analizowane b%d$ w pe&nym opracowaniu oraz proponowanych #róde$
danych kosztowych, co do warto"ci kosztów oraz cz%sto"ci zdarze'
generuj$cych koszty
przedstawienie wst%pnej oceny prawdopodobnych wyników raportu HTA
(ostatecznego opracowania), o ile jest to mo#liwe na etapie ekspertyzy
wst%pnej
przedstawienie kalkulacji ca$kowitej kwoty kontraktu za realizacj%
projektu (ekspertyza wst%pna i opracowanie ostateczne)
SCOPING NICE
– zakres i zagadnienia
•
•
•
•
•
•
•
•
•
•
Problem kliniczny i populacja docelowa
Oceniana technologia
Komparatory
Istotne klinicznie punkty ko*cowe dla danej technologii
Koszty
Horyzont czasowy, w którym powinny by) oceniane koszty i efekty
zdrowotne
Identyfikacja subpopulacji pacjentów, dla których technologia mog%aby by)
klinicznie i kosztowo efektywna
Ilo#ciowe oszacowanie istniej&cych dowodów naukowych dla ocenianej
technologii
Zagadnienia zwi&zane z równo#ci& w dost$pie do #wiadcze* medycznych
i/lub sposób zapobiegania nierówno#ci
Inne, potencjalnie istotne dla tworzenia wytycznych elementy
Sesja 8 / Session 8
•
233
Evidence-Based
The evidence base
Wytyczne NICE podkre#laj&, 'e scoping jest
dobrym momentem do ilo!ciowego
oszacowania dost%pnych dowodów
naukowych dla ocenianej technologii, w tym
kluczowych i/lub najnowszych bada* dla
ocenianego leku oraz komparatorów
Aspekt praktyczny ekspertyz
wst%pnych czy APD w relacji
wykonawca - zamawiaj(cy
Wysoko#) wynagrodzenia
Sesja 8 / Session 8
Nak%ad pracy
234
Problem badawczy
Krzysztof Łanda
Health Care
HTA & Pricing
www.ceestahc.org
Krzysztof Łanda
Populacja / wskazanie
Szczepienia przeciw grypie
Metaanaliza „ca&a evidence do jednego kot&a”
vs
Uj$cie wielu aspektów w APD – efektywno#) szczepionki zale'y
m.in. od:
1. Trafno#ci przewidywa* WHO co do atakuj&cych szczepów wirusa grypy w
nadchodz&cym sezonie
2. Rzeczywistego rozpowszechnienia wirusa grypy: high season / low season
Fragment raportu z audytu
Problem zdrowotny, którego dotyczy opiniowana analiza
stanowi wyj$tkowo obszerne i wielow$tkowe zagadnienie,
st$d wykonanie kompleksowej oceny technologii
medycznych jest w tym przypadku szczególnie trudne.
Kwestia szczepie' przeciwko grypie obejmuje bardzo
szeroki zakres bada' i wymaga uwzgl%dnienia wielu
dodatkowych, wa#nych aspektów.
Audyt opracowania musi zatem bra( pod uwag%
rozleg&o"( i stopie' skomplikowania problemu
decyzyjnego oraz wykonanej pracy analitycznej.
Sesja 8 / Session 8
„Konieczna eksploracja zmienno"ci skuteczno"ci szczepie' w zale#no"ci od
wyst%powania epidemii, jej zakresu i intensywno"ci oraz dostosowania szczepionek
w poszczególnych latach do kr$#$cych szczepów wirusa .Obydwa zagadnienia maj$
równie# kluczowe znaczenie dla oceny ekonomicznej szczepie'.”
235
Evidence-Based
Interwencja
„Zapomniane” komparatory
=
>
=
>
>
=
>
MAGIC?
Sesja 8 / Session 8
Jedna z najcz$stszych manipulacji, dlatego tak WA)NE jest
wymaganie przedstawienia wszystkich istotnych opcji post$powania
i uzasadnienia wyboru komparatorów w ramach APD
236
Komparatory
Krzysztof Łanda
Health Care
HTA & Pricing
www.ceestahc.org
Krzysztof Łanda
Efekty zdrowotne
Przejrzysto!' – wydaje si%, &e
wszelkie APD w Polsce kierowane do
urz%dów powinny by' publikowane
Na stronie internetowej NICE mo&na !ledzi' kolejne etapy
oceny danej technologii
Publikowane s& m.in. krótka charakterystyka oceny, sk%ad komitetu
oceniaj&cego, post$p prac, draft scope, final scope oraz pozosta%e
wa'ne dokumenty dotycz&ce oceny
Bardzo precyzyjna i
przejrzysta mapa Polski, ale
… jeste"my przecie# w
Marrakeszu …
Wysokiej jako"ci analizy,
zgodne z wytycznymi, ale …
nie odpowiadaj$ na nasz
problem decyzyjny!!!
Sesja 8 / Session 8
Wszystkie informacje o opublikowanych raportach dost$pne s& na
stronie: http://www.nice.org.uk/Guidance/TA/Published
Informacje o raportach jeszcze nieopublikowanych dost$pne na
stronie: http://www.nice.org.uk/Guidance/TA/InDevelopment
237
[email protected]
Bardzo dzi%kuj% za uwag%!
Sesja 8 / Session 8
W prezentacji wykorzystano kilka slajdów przygotowanych
przez Ann% Bednarsk( i Ma$gorzat% Karp na rzecz
intelektualnych obiadów czwartkowych CEESTAHC
238
Evidence-Based
Health Care
HTA & Pricing
www.ceestahc.org
Sesja 8 / Session 8
239

Evidence-Based Health Care

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