Relative Effectiveness Assessment: wie hilfreich sind die EUnetHTA

Transcription

Relative Effectiveness Assessment: wie hilfreich sind
die EUnetHTA Konzepte
Alric Rüther1, Annette Zentner²
1 IQWiG:
Ressort Versorgungsqualität
Internationale Beziehungen
² FG Management im Gesundheitswesen,
TU Berlin
2
HTA Europa: Hintergrund
Report der High Level Group on Health Services and Medical Care (HLG) der
Europäischen Kommission (30. November 2004):
 “HTA has become a political priority and there is an urgent
need for establishing a sustainable European network for
HTA”.
DG SANCO Public Health Work Plan 2005:

HTA wird Prioritätsgebiet
Kommissar Kyprianou : (HTA 1 der 7 Haupt-Gebiete):
“….the usefulness of establishing a sustainable European
health technology assessment network has been recognised
…..
I hope to see this network being set up this year, with support
from the public health programme.”
European Network for HTA
EUnetHTA
 Projekt der Europäischen Kommission
 Ziel:
 Aufbau eines effektiven und zukunftsfähigen
Netzwerks für HTA in Europa
 Strategische Ziele
 Basis für politische Entscheidungen
 Verbindung öffentlicher HTA-Agenturen,
Forschungseinrichtungen und
Gesundheitsministerien
 Effektiver Informationsaustausch
 Unterstützung politischer Entscheidungen durch Mitgliedsstaaten
EUnetHTA Project
2006-2008
 64 Partners: 55 Partners from 29 countries (21 EU
Member States) including 9 International Partner
Organisations
 Final Report published by European Commission
http://ec.europa.eu/health/ph_systems/docs/eunethta_report_en.pdf
 Fourteen scientific articles published in Int J Technol
Assess Health Care, 2009: 25 (Suppl. 2)
http://journals.cambridge.org/action/displayIssue?jid=THC&volumeId=2
5&issueId=S2
5
The time-line of reaching a sustainable and
permanent HTA network in Europe
Health
Programme
2005
2009
2011
Call for
project
proposals
Call for
Joint
Action
Call for
Joint
Action
2006-08
EUnetHTA
Project
Legislation
DG R&I
6
2009
EUnetHTA
Collaboration
2010 -12
EUnetHTA
JA
HTA Core Model® and its
application
Pool of
Structured HTA Information
HTA Core Model
AE
AE
= Assessment element
AE
= Core
AE
= Non-core
INFO
CORE HTA
AE
AE
AE
AE
element
element
= Locally
produced
information that does not
follow HTA Core model
LOCAL REPORT
AE
AE
AE
INFO
INFO
AE
AE
Domain 1
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
AE
Domain 2
AE
INFO
Rapid HTA
AE
Domain 3
AE
AE
AE
AE
Domain 4
AE
AE
AE
AE
AE
REA
AE
AE
AE
Kristensen FB_2 Croatia 20101207
AE
AE
Takes into
account local
epidemiology,
resources,
values, etc.
HTA Core Model®: structure
Health problem and current use of
technology
Technical characteristics
Domain –Topics- Issue
Safety
Clinical effectiveness
Topic 1: Mortality
Issue 1: What is the effect of the
intervention on overall
Topic
2: Morbidity
mortality?
Clinical effectiveness
Costs and economic evaluation
Ethical analysis
Issue 2: What is the effect of the
intervention
on mortality caused
Topic 3 etc…
by the target disease?
Organisational aspects
Issue 3: etc…
Social aspects
Legal aspects
8
Core HTA Structure
Kristensen FB_2 Croatia 20101207
9
European network for HTA
Joint Action between European
Commission and EU Member States
A total of 34 government appointed organisations from 23 EU Member
States, Norway and Croatia and a large number of relevant regional
agencies and non-for-profit organisations that produce or contribute to
HTA
EUnetHTA Joint Action 2010–2012 | www.eunethta.eu
EUnetHTA JA organisation
WP1 – Coordination (Lead Partner (LP) – NBoH, Denmark)
WP2 – Dissemination (LP – IPH-RS, Slovenia; Co-LP – SBU, Sweden)
WP3 – Evaluation (LP – NETSCC, United Kingdom)
WP4 – Core HTA (LP – THL, Finland; Co-LP – AGENAS, Italy)
WP5 – Relative Effectiveness Assessment (REA) of Pharmaceuticals
(LP – CVZ, Netherlands; Co-LP – HAS, France)
WP6 – Information Managment System (LP – KCE, Belgium, Co-LP –
DIMDI, Germany)
WP7 – New Technologies (LP – HAS, France, Co-LP – LBI-HTA,
Austria)
WP8 – Strategy and Business Model Development (LP – NBoH,
Denmark)
• a) HTA training and capacity building (focus on EUnetHTA tools)
and
• b) facilitation of national HTA strategies.
• Responsible organisation for a) – ISCIII, Spain
• Responsible for b) – AHTAPol, Poland
EUnetHTA JA Strategic position
All activities of the EUnetHTA Joint Action arise
from the premise that its outputs will be used to
inform but not mandate the content of national /
regional / institutional HTA reports
Kriterien für Bewertung
und Entscheidung
Kriterium
A A C C D E F F G H I I N N N P P S S U
T U A Z K S I R R U T R L O Z L T E K K
Patientenrelevanter
(Zusatz)Nutzen
X X X X X X X X X X X X
X X X X X X X X
Kosten-Nutzen
X X X
Auswirkung auf Budget
X X X X
Preise in anderen Ländern
X
X X X
Zu erwartender Umsatz
X
X
Behandlungsalternativen
X X
Soziale, ethische Erwägungen
X X X X X X
X X X
X X X
X X X X X
X X
X X
X
X
X
X
X
X
Public Health Auswirkungen
F&E-Kosten der Hersteller
X X X
X X
X
X
X
X
X
X
X
X
X X
X
X
X
X
X
X X
X
X
X X X
Bedarf der Gesellschaft
Pharmakologische Merkmale
X
X
X
X
X
X
X X X
Prioritäten der Regierung
X
Quelle: Zentner et al. 2005; Kanavos et al. 2009
Dr. med. Annette Zentner MPH
Level of welfare effects
Levels of European HTA collaboration
Scenario 3:
Joint
Assessments
Common European assessments
Scenario 2:
Guideline
development
Common European guidelines
Scenario 1:
ICT platform
Sharing national assessment
reports in Europe
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu
Joint Action 2010–2012
EUnetHTA JA1 WP5 REA Pharmaceuticals
 EUnetHTA was asked to take this work forward by the
Steering Committee of the HL PF in autumn of 2008.
 EUnetHTA WP on REA started in 2010
 EUnetHTA decided to work with the definitions that had
been agreed in PF2008
 According to the Pharmaceutical Forum:
• Relative effectiveness can be defined as the extent to which
an intervention does more good than harm compared to one
or more intervention alternatives for achieving the desired
results when provided under the usual circumstances of
health care practice.
The WP5 Partners JA1
1 Lead
1 Co-lead
17 Associated Partners
13 Collaborative Partners
EUnetHTA WP5 REA Pharmaceuticals
Objective:
Development of HTA tools
and methods for REA
Background
review on
national REA (CVZ)
Development
of model
for Rapid REA (CVZ)
Pilot of
Rapid REA (CVZ)
Methodology
guidelines
for REA (HAS)
Collaboration
with EMA (HAS)
Background review on national REA#
Objective: provides an overview of the processes and
methodologies used in national practice
29 countries included*
Data gathered:
• Health care system
• Process
• Methodology
Australia Austria Belgium Canada
Czech Republic Denmark England& Wales (UK)
Estonia Finland France Germany Hungary
Ireland Italy Latvia Luxembourg Malta Netherlands
New Zealand Norway Poland Portugal Scotland (UK)
Slovakia Slovenia Spain Sweden Switzerland
Turkey United States of America
*Separate data abstraction for England/Wales & Scotland
#Kleijnen et al. http://www.eunethta.eu/upload/WP5/Link1.pdf
Background review on national REA
Conclusions and challenges
 Most of countries carry
out some form of REA to
support national reimbursement
decisions of pharmaceuticals
 The scope and the
methodology used vary across
countries to some extent >>
however not that much (TERMINOLOGY!)
 Rapid assessments are most common and relevant for reimbursement
Purposes
 The differences between counties, as well as the reasons behind them,
need to be considered in the development of a common European
methodology for REA
Development of REA model
Should be based on HTA Core Model (HTA Core Model = A structured
manner of creating and presenting HTA information)
WP5
HTA Core Model
Model for Full REA
Model for Rapid REA
Health problem and current
use of technology
use of technology
use of technology
Description and technical
characteristics of the
technology
Safety
Effectiveness
technology
Safety
Effectiveness
technology
Safety
Effectiveness
Cost and economic
considerations
Cost and economic
considerations
Cost and economic
considerations
Ethical analysis
Ethical analysis
Ethical analysis
Organisational analysis
Social aspects
Social aspects
Social aspects
Legal aspects
Legal aspects
Legal aspects
 Mutliple comparators
 Years after market
authorisation
 Indication based
 Limited number of
comparators
 Soon after market
authorisation
WP5 pilot of a rapid assessment
Objective: Test the usability of the draft model for rapid REA and
the draft guidelines
Topic selection:
 List was produced of all pharmaceuticals that received market
authorization between June 2010 and February 2011
 Selection made based on exclusion criteria
 Manufacturers approached for willingness to provide submission file (2 out
of 4 were willing)
 Pazopanib for the first-line treatment of metastatic renal cell cancer.
Basic documentation:
 Manufacturer submission file
 Rapid REA model
 Methodological guidelines developed in WP5
WP5 pilot of a rapid assessment
 Experience
• Participation of 29 organisations requires intense coordination on
several levels
• 3,5 months timelines for scope/assessment phase if possible but
very intense
• Relevance of all research questions (assessment elements) for a
rapid assessment?!
• Harm/benefit analysis is still in development (synthesis).
• This model of collaboration is not sustainable if rapid assessments
needs to be done within three months
22
EUnetHTA methodology
guidelines
Primary objective: help assessors to make
rapid assessment of pharmaceuticals
- e.g. give clear recommendations, useful for HTA assessors
• Scope
• Terminology
• Structure of documents
• Consistency between guidelines
– e.g. clinical endpoints: should be consistent with other « endpoints » guidelines
• Define the most important concepts
-
e.g. (relative) efficacy and effectiveness, types of endpoints, comparator,
applicability, surrogate endpoints
23
23
EUnetHTA methodology
guidelines
9 guidelines:
• Criteria for choice of most appropriate comparator(s)
• Methods of comparison: direct and indirect comparisons
• Endpoints used for REA of pharmaceuticals:
–
–
–
–
–
Clinical endpoints
Surrogate endpoints
Composite endpoints
Health-related quality of life
Safety
• Levels of evidence:
– Internal validity
– External Validity (Applicability)
Elaboration and review process
Draft by individuals in partner HTA organisations
Review and comments by HAS and WP5 members
Comments incorporated by original authors
Workshops on specific topics
Revisions by original authors and HAS
Consultation with Stakeholders and Public
25
EUnetHTA methodology guidelines
Consultation process
Internal consultation
Guidelines
•
•
•
•
Composite endpoints
Surrogate endpoints
Choice of comparator
Applicability
•
•
•
•
HRQoL
Safety
Internal validity
Direct and indirect
comparisons
Clinical endpoints
•
(EUnetHTA partners and
stakeholders
representatives)
Public
consultation
(including EMA)
March 7 – April 13
June 29 – Sept 10
April 16 – May 25
Sept 3 – Oct 31
EUnetHTA methodology guidelines
Clinical endpoints
–
–
–
–
–
–
–
–
–
Well defined and justified, clinically relevant to the disease.
Both statistical and clinical relevance
Objective EP, subjective EP and Patient-relevant EP
Long-term or final EP where possible (OS, all-cause
mortality); short-term endpoints are acceptable for acute
conditions with no long-term consequences
Sensitive to changes due to treatment
Free from measurement or assessment error, unbiased
Relative and absolute measures to be presented
Adjustment for multiple hypothesis testing
EP data analysis to explicitly state the handling of missing
data
27
HTA Methoden und Qualität?
Methoden auf Basis internationaler
Standards der evidenzbasierten
Medizin (§139a SGB-V)
•
•
•
•
•
•
•
Kausalitätsprinzip
Transparenz
Reliabilität
Surrogate
Dramatischer Effekt
Bias
……….
Schwarzer R, et al. Health Policy 2013 [in preparation]
Europäische Union
Artikel 15
Zusammenarbeit bei
der Bewertung von
Gesundheitstechnologien
Die Union unterstützt und erleichtert die
Zusammenarbeit und den Austausch
wissenschaftlicher Informationen
zwischen den Mitgliedstaaten im
Rahmen eines freiwilligen Netzwerks,
das die von den Mitgliedstaaten
benannten […] nationalen Behörden
[…] verbindet.
Ziele des EU-Netzwerks
www.eunethta.net
a) die Unterstützung der Zusammenarbeit zwischen
nationalen Behörden
beziehungsweise anderen Stellen;
b) die Unterstützung der Mitgliedstaaten bei der Bereitstellung objektiver,
zuverlässiger, rechtzeitiger, transparenter, vergleichbarer und übertragbarer
Informationen über die relative Wirksamkeit sowie gegebenenfalls über die
kurz- und langfristige Wirksamkeit von Gesundheitstechnologien und die
Schaffung der Voraussetzungen für einen effizienten Austausch dieser
Informationen zwischen den nationalen Behörden beziehungsweise anderen
Stellen;
c) Unterstützung der Analyse des Inhalts und der Art der Informationen, die
ausgetauscht werden können;
d) Vermeidung von Doppelarbeit bei den Bewertungen.
The time-line of reaching a sustainable and
permanent HTA network in Europe
Health
Programme
2005
2009
2011
Call for
project
proposals
Call for
Joint
Action
Call for
Joint
Action
2006-08
EUnetHTA
Project
2009
EUnetHTA
Collaboration
2008 - 11
Legislation
DG R&I
31
2015+
European
network for
HTA
2010 -12 2012 -14
EUnetHTAEUnetHTA
JA
JA 2
2011
Legal basis:
Cross Border
Healthcare
Directive
2011 +
CBHC CBHC Directive
Draft Cross Border Directive Expert Group
for
Healthcare Directive
now
HTA network
Article on HTA network decided implementation
Financial
support:
1) EU Budget
2) Member
States
2011 FP7-Health
2012-Innovation-1
New methodologies
for HTA
WP5 Joint Action 2 (2012-2015)
 Ten pilots on rapid REA of pharmaceuticals
between 2012-2015
Collaboration model:
 One organisation is author and one organisation is co-author
 A number of WP5 partners will be dedicated reviewers
 Other WP5 partners will be consulted after this dedicated review
WP5 Joint Action 2 (2012-2015)
Important issues:
 Input of submission files is central part of assessment
 Active involvement of industry is essential
 European submission file format needs to be developed (WP7)
 Close collaboration with EMA is needed
 Implementation of (parts of) rapid assessment in national
assessments must be stimulated
 Analysis of implementation hurdles (started in JA1)
 Timing of assessment is crucial; ideally REA report should
become available just after the publication EPAR
 Broad selection of pilot pharmaceuticals
 Inclusion of assessments of orphan drugs!
Ausblick




Länderübergreifende Zusammenarbeit wird in der EU mit Priorität gefördert
Die Bewertungsprozesse der einzelnen Länder unterscheiden sich im Detail
Methodische Anforderungen sind oft unterschiedlich
EUnetHTA versucht über das Core-Modell:
 Einfache Werkzeuge zur Zusammenarbeit zu etablieren und
bereitzustellen
 Methodisch einen kleinsten gemeinsamen Nenner zu definieren
 Akzeptanz herzustellen

Hilfreich für uns?
Dank an Wim Goettsch (CVZ) und
Mira Pavlovic (HAS) für
Unterstützung und Slides
Institut für Qualität und Wirtschaftlichkeit
im Gesundheitswesen (IQWiG)
•
•
Im Mediapark 8
D-50672 Köln
•
•
Telefon +49-221/3 56 85-0
Telefax +49-221/3 56 85-1
•
www.iqwig.de
Photo: http://img.fotocommunity.com/images/Koeln-Rhein-Erftkreis/Koeln/KoelnTurm-im-Mediapark-a24731976.jpg
REA
CER
REA versus CER
REA
CER
[email protected]
europa.eu
• Arzneimittel
• alle Verfahren
• Erstattung/Preis
• Patientenversorgung/
Prävention
• politische Steuerung
• Forschungsförderung
• Sekundäranalysen
• Sekundäranalysen
und Primärstudien
HTA

Relative Effectiveness Assessment: wie hilfreich sind die EUnetHTA

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