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EUnetHTA Joint Action 2010–2012 | www.eunethta.eu
REA of Pharmaceuticals
Collaboration between EMA and EUnetHTA
Development of consensus on HTA methodologies
Mira Pavlovic, MD
Deputy Head, HTA Division, HAS, France
EUnetHTA WP-5 co-lead, WP7 lead
Moscou, May 21, 2012
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20122
Development of consensus on HTA
methodologies
One of the aims of European Collaboration in the field of HTA
EUnetHTA JA1 (2009-12)
WP5: REA of pharmaceuticals
Pilot of joint REA assessment
Core HTA model for REA of pharmaceuticals
Methodology guidelines
WP7:
Planned and ongoing projects (POP) database
Additional evidence generation (EVIDENT database)
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20123
Development of consensus on HTA
methodologies
European collaboration (EUnetHTA)
EunetHTA JA2 (2012-15)
• WP7 : Increase the quality of evidence generation (all HT)
– Initial evidence:
• « Early Dialogue » between developers and HTA bodies
• « Disease-specific guidelines »
– Additional evidence : « Common core protocol »
• Harmonise methods of assessment (all HT)
– Methodology guidelines
– « Core HTA information »
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20124
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)
Primary objective: help HTA assessors in rapid REA of pharmaceuticals
16
WP5 Methodology guidelines
(EunetHTA JA1, REA of pharmaceuticals)
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20125
Process of developing methodological guidelines
5
Draft by individuals in partner HTA organisations
Review and comments by work package 5 members
Comments incorporated by original authors
Workshops on specific topics
Revisions by original authors and HAS
Consultation with Stakeholders and Public
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20126
WP5 Methodology guidelinesProcess of guidelines development
Guidelines WP5 and SAG
consultation
(3rd drafts)
EMA and Public
consultation
(4th drafts)
• Clinical EP
• Composite EP
• Surrogate EP
• Choice of comparator
• Direct and indirect
comparisons
March 7- April 13
• Comments received
• Revised versions
expected by May 7
May 14 – July 14
• HRQoL
• Safety
• Internal validity
• Applicablity
Just started:
April 18 – May 25
June 29 – Sept. 14
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20127
WP5 Methodology guidelinesProcess of guidelines development
• 1st internal review: 1274 comments received
• 2nd internal review (ongoing)– 15 – 170 comments per guideline
• Nature of comments received
– Structure,
– Scope,
– terminology,
– define most important concepts,
– Consistency between guidelines
– Give clear and useful recommendations for HTA assessors
– Content and recommendations challenged
Real input of guidelines on REA of pharmaceuticals reviewed by HTA doers themselves
Final documents: before the end of 2012
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20128 88
New EUnetHTA initiative on
Early dialogue
• Part of EUnetHTA JA2 WP7 (starts: October 2012)
• One of the EU priorities
• Preparatory Pilots conducted (HAS Coordinator)
• Supposed added benefit for patients
• All HEALTH TECHNOLOGIES
• Questions on clinical relative effectiveness, economical
and other aspects, relevant for the development plan
• Prospective in nature: development strategies, e.g.
planned phase III studies, cost-effectiveness studies
• Non binding and confidential
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–20129 99
New EUnetHTA initiative on
Early dialogue
Excellent possibility to test:
– HTA prospective thinking on evidence
requirements based on concrete example(s)
– Consolidated view/agreement (?) on the choice
of comparators and endpoints for REA
basis for the development of disease-specific
guidelines (WP7 JA2)
– Test EUnetHTA guidelines recommendations in
real examples of product development
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201210
New EUnetHTA initiative on
Early dialogue: PILOTS
• Gain experience
• Prepare JA2 pilots
• Define how we want to work in future
• Timetable
– 1st pilot early dialogue meeting: end of May 2012
– 2nd pilot early dialogue meeting: end of June 2012
– Feedback from HTA and company: Sept. 2012, before start of JA2
– Procedure to be revised accordingly: Oct. 2012
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201211
EUnetHTA – EMA collaboration
• Methodology guidelines
– EMA comments on EUnetHTA guidelines
– EUnetHTA members can comment on EMA
guidelines
• EMA – HTA scientific advice
– EMA SA (drugs) + several HTA bodies
• EPARs improvement project
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201212
EMEA Scientific advice (SAWP)
The SAWP (CHMP working party): Multidisciplinary Expert Group, 28 members
Advice to companies on• all aspects of drug development: quality, non-
clinical, clinical
• non-product related issues = “broad advice”, (statistics, validation of a new scale)
• qualification of biomarkers
Prospective, optional, non-binding
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201213
EMA Scientific Advice Procedure
• 2 coordinators for each request
• 1 month to prepare the report
• Internal discussion at the SAWP at D30
• Discussion with the company if necessary at D60
• Draft joint report
• 2 peer-reviewers (1 from SAWP, 1 from CHMP)
• Presented, discussed and officially validated at the CHMP
• Sent to the company
• 400 advices per year
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201214
EUnetHTA – EMA collaboration
EMA – HTA parallel scientific advice
for drugs
• Pilot phase
• 6 advices organised by Tapestry network
• More and more advices requested directly by companies
• HTA agencies chosen by the company requesting advice
• HTA input within the EMA SA procedure at the time of the discussion meeting with the company (D60) only (minutes)
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201215
EUnetHTA – EMA collaboration
EMA – HTA parallel scientific advice
for drugs - content
EMA - targeted questions – product development plan
HTA – targeted questions – active comparisons, outcomes, added value,
design of pragmatic trials, COST-EFFECTIVENESS +++
Common questions
Final advice:• by each HTA body participating in the exercise ?
• compiled document ?
• Exchange of final advices? (EMA, HTA)
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201216
EUnetHTA – EMA collaboration
EMA – HTA parallel scientific advice
for drugs
• Possibility to test:
– Agreement on the choice of comparators and endpoints both for MA and REA
– EUnetHTA and EMA guidelines recommendations in real examples of product development
– Consistency with final decisions
• EMA: MA opinion
• HTA: reimbursement
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201217
EUnetHTA – EMA collaboration
EPARs improvement project
• The High Level Pharmaceutical Forum:
– the EMA to consider how the EPAR can further contribute to relative effectiveness assessments
• EMA - EUnetHTA (2010-2011) meetings:
– adapt assessment report template (in line with comments from MEDEV/EUnetHTA)
– assessment report templates revised by EMA and used for Opinions from November 2010.
– Develop questionnaire for monitoring of the implementation
– Perform parallel review and compare results
• Results presented and compared (Feb 22, 2012, Paris)
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201218
EUnetHTA – EMA collaboration
EPARs improvement project methodology
• Checklist (questionnaire) based on the action items agreed by EUnetHTA and EMA
• 36 questions targeting areas for improvement
• Two types of answers depending on the question:– Yes (1) / No (0) / Not Applicable (13 questions)
– Excellent (3) / Good (2) / Could be improved (1) / No (0) / Not Applicable (23 questions)
• Scope: Format / Scientific content / Support for SPC
• Compliance of the EPAR for each question
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201219
EUnetHTA – EMA collaboration
EPARs improvement project – parallel
review
Pumarix, Esbriet, Teysuno, Xeplion, Xiapex, Gilenya, Halaven, Jevtana, Pravafenix, Trobalt
EUnetHTA
• Each EPAR reviewed by two HTA organisations,
EMA
• First review (all EPARs): single reviewer
• Peer review: senior member of staff
Same questionnaire
Same assessment scales
Same evaluation tool
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201220
EPAR improvement project
EUnetHTA Observations
• Space for improvement in discussion of the key elements of the clinical study design.
– Patient population, Comparators, Duration of the study, Endpoints and/or composite endpoint use
• Shortcomings of efficacy data would benefit from more discussion in the assessment report.
• Additional analysis requested by the CHMP should be better identified and explained in the assessment report
• EUnetHTA (coordinated by HAS) to make a consolidated proposal for further improvements of the EPAR, highlight expectations
EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu Joint Action 2010–201221
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