ISPOR 18th Annual European Congress Tuesday 10 November,13:45-14:45

Transcription

1 ISPOR 18th Annual European Congress Tuesday 10 November,13:45-14:45 Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session 1

2 Throughout the session, we are taking your questions via Twitter. Ask #realworldevidence #ISPORMilan. Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session 2

3 Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session Jacco Keja: Recent HTA Decisions Jacco Keja, PhD Global CoE Leader in HEOR, Real World Evidence Solutions IMS Health, Rotterdam, The Netherlands 3

4 Impact on Reimbursed Price No Yes Health Technology Assessments: Complexity HTA agencies have been playing a meaningful role in pharmaceutical pricing & reimbursement in Europe, but there is wide variation in assessment & implementation Complexity in HTA is driven by many factors: Scope and impact of HTA by market 1. Different country-specific HTA systems 2. Different assessment criteria (e.g. measures of clinical effectiveness and safety; quality of life; cost) 3. Variation in medical practice DE CZ FR GR AU BEL BUL EST IT FI LI NE NO PL PO RO SP CR CY IR HU LV SW DK 4. Differences in implementation in terms of access or prices; different scopes of influence SL TU MT SK UK No Yes Impact on Access CH Addressing differences in assessment approach has become the focus of efforts for convergence, with the goal of achieving greater consistency in evaluation of the underlying evidence 6 Debate over coordinated EU Relative Efficacy Assessments Arguments for the need for coordinate EU relative efficacy assessments Transparency Manufacturers desire transparency regarding the evaluation of evidence packages for planning development and launch strategies There is consistency in the requirement for evidence between countries Political acceptability Payers want an evidence-based and politically acceptable process that balances efforts to increase access to effective new technologies against fiscal constraints Challenges with implementation Healthcare systems Inherent variation in healthcare systems and patient pathways Value assessment criteria differ (e.g. clinical effectiveness & safety; quality of life; cost (including cost-effectiveness or budget impact) Health policy European health policies & healthcare budget remain a national responsibility Common standards Common standards: More difficult for countries with formalized procedures to accept a change in standards Lack of trust between pharma /HTA 7 Convergence will, of course, require resources... leadership... among government officials... individuals in professional organizations, including the ISPOR membership. The goal should be to improve the science and to reduce uncertainty. (Neumann, 2009) 1. Neumann, 2009, Value in Health 4

5 Examples of ongoing activities supporting European convergence Example of organisations promoting elements convergence: Established to create an effective and sustainable network for HTA across Europe Examples of activities: EUnetHTA s methodological guidelines Endpoints used in Relative Effectiveness Assessment - Safety Collaborations: Major EU organizations: EFPIA, EMA, EUCERD, EUPATI, HTAi The HTA network: Created in 2013 by the European Commission as a permanent, voluntary network connecting national authorities responsible for HTA Aims: To facilitate efficient use of HTA resources in Europe To create a sustainable system of HTA knowledge sharing To promote good practice in HTA methods and processes 8 HTA Position Paper of the European organisations of patients, consumers, healthcare providers and payers Summary view: Sharing information and using transparent approaches in the assessment and decision-making are likely to shorten the time-to-market make the reimbursement decision more understandable Final reimbursement decisions will reflect the national context and logically differ across Member States IMS Analysis: Recent HTA assessments to determine extent of convergence in practice Methodology IMS Health analyzed the degree of convergence in the outcomes results for: Launches: 12 recent launches across Type 2 Diabetes; Multiple Sclerosis; Prostate Cancer; Hepatitis C Considering: o Similarity of evidence submission o Evaluation of products o Evaluation using a consistent set of criteria Markets: France, Germany, Sweden, UK Key Findings Divergence Convergence NATURE OF THE TA CRITICAL ENDPOINTS High cost Low unmet need, limited differentiation Patient-reported outcomes Epidemiology Surrogate endpoints High unmet need, strong product differentiation Hard endpoints 9 TRIAL DESIGN Several plausible comparators Superiority vs. inferiority Subgroup definition Clear comparator 5

6 Case example: MS HTA evaluations Patient group: RRMS Positive Positive with limitations Negative Country Aubagio (teriflunomide) Gilenya (fingolimod) Lemtrada (alemtuzumab) Tecfidera (dimethyl fumarate) FRANCE ASMR V: No conclusive H2H study; oral benefit (Note: SMR- Important) ASMR IV: Relapse rate, reassessment for LT tolerance Not reviewed (evaluation will proceed) ASMR V: no superiority study vs. active Tx; oral benefit (Note: SMR- Important) GERMANY No added benefit: lack of clarity on side effects, conclusions on mortality Minor : fewer flu-like symptoms (RRMS patients) Not reviewed No added benefit: No suitable data; inappropriate comparator SWEDEN Fewer side effects Not cost effective vs. Extavia (initial) Cost effective vs. Copaxone (appeal) Relapse rate Cost effective relapse rate Small gain in QoL vs. Tysabri, at a lower cost Relapse rate Cost effective; economic assessment Dec 2016 with RWE UK (NICE) Contingent on PAS relapse rate Cost effective Contingent on PAS relapse rate Cost effective More effective (disability / relapse rates) Cost effective Contingent on PAS proportion patients with a relapse at 2 yrs Cost effective UK (SMC) Contingent upon PAS rate of relapse For RRMS* Contingent on PAS; Relapse rate Cost savings over 5 years vs. Tysabri rate of relapse Cost effective Contingent on PAS proportion patients with a relapse at 2 yrs 10 *SMR, Actual Benefit vs. ASMR, Improvement in Actual Benefit ** Decision was accepted for RRMS patients with high disease activity despite treatment with at least one disease modifying therapy in 2015 Abbreviations: RRMS, Relapsing-remitting multiple sclerosis; PAS, patient access scheme; IFN-β, beta-interferon Source: HTA published guidance, assessments, and reimbursement decisions; synthesis based on interpretation of clinical and economic information provided in manufacturer HTA submissions and published guidance Summary Factors contributing to divergence: Different country-specific HTA systems Different assessment criteria Variation in medical practice Differences in implementation The extent of divergence is related to: Nature of therapeutic area (cost, epidemiology, unmet need) Critical endpoints (PROs, surrogate / hard clinical endpoints) Trial design (comparators, superiority vs. inferiority) What level of convergence is achievable and realistic for evidence evaluation? (e.g. understanding of endpoints, benchmarks and trial design elements)... for cost-effectiveness and budget impact?... for value placed on softer endpoints?... For weight of importance placed on a TA, (disease epidemiology)? 11 6

7 Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session European assessment of relative efficacy at time of launch: a value for Europe? Industry views 7

8 European access problem driven by fragmented clinical assessments European assessment can improve access if performed in parallel to market authorisation and in lieu of national assessment Industry views Large access differentials across Europe New medicines reach some countries with long delays In some countries, certain products are not available at all Different assessment approaches lead to inconsistent access decisions Inconsistent evidence requirements create duplication and market access delays Harmonisation of data requirements and assessment approach can accelerate assessments and end duplication EU-wide view on clinical performance can reduce access differentials European clinical assessment must be separate from marketing authorization process, but done concurrently to save time It must be integrated in national market access processes to avoid duplication covering clinical aspects only Economic assessments must be local to account for diversity in health systems Industry remains opposed to economic value assessment at EU level 14 EU REA what does it mean? Rapid REA report Description of the health problem The technology & its place in treatment pathway Local considerations Local issues related to the health problem Local treatment pathway Decision-maker General Clinical Efficacy / Effectiveness Safety Appraisal recommendation Local coverage decision Key Centralized Local Economic considerations Social values / ethical & legal issues 15 8

9 Process (ideal) timeline: EUnetHTA process (with EFPIA comments) The process should initiate 180 days before the CHMP positive opinion The scoping should take place close to the EoI and should determine PICO A final draft submission review meeting before the CHMP positive opinion The assessment should take no longer than 100 days Expression of interest Participation Choice of product Scoping meeting PICO Final draft submission review meeting Assessment Draft assessment Revisions Publication -180 CHMP opinion EPAR Timeline (days) 16 Re-use Assessment Appraisal Decision-making National appraisal National decision-making + National context-specific elements

10 Pilots so far Pilot Author Co-author Zostavax (SP-MSD) Canagliflozin (J&J) Sorafenib (Bayer) ZIN (national HTA agency) FIMEA (Medicines Agency producing evaluations of economic and therapeutic value) AIFA (national HTA agency) A. Gemelli (hospital conducting HTA and advising regional coverage decisions) AAZ (national HTA agency) Regione Veneto (regional HTA agency) INFARMED (national HTA agency) Ramucirumab (Lilly) NOKC (HTA centre proving reports to the national Medicine Agency, NoMA) AAZ (national HTA agency) Vorapaxar (MSD) HAS (national HTA agency) Ministry of Health (national HTA) 18 Lessons from pilots Overall satisfied, but Need some process and methodology improvements Review the process to integrate an early scoping meeting (shortly after EoI) and a later final draft submission review meeting Discuss how to handle confidentiality issues Discuss involvement of other stakeholders Tailor the EUnetHTA template to fit European assessments (focus on transferrable information, not a collection of all national requirements) Discuss safety section Re-use is a major issue Timelines did not warrant re-use in early pilots But Member State commitment is a major concern

11 15 recommendations for improving the rapid REA #1 1. The industry should propose the current timetable should be followed. Only pilots where there 1 is an expectation of this being met should be initiated in JA3 21. A project alignment meeting 60 days prior to the scoping meeting should be introduced 3 The lead author should be chosen on based experience and should be planning to assess the product in their own market. This would imply that the lead author is directly involved in a national HTA process. The role of lead and co-author should be made explicit 1. To understand the benefits in terms of re-use, the pilots in JA3 should reflect different types of 4 product. This should be more explicit than JA2 1. Voluntary participation should improve if pilots based on re-use offer benefits to participating 5 companies, however, the process for including pilots should be made more transparent 61. The inclusion of patients and physicians in the process should be piloted in JA 3 1. The primary objective of JA3 pilots should be re-use but other process and methodological 7 issues still need to be resolved recommendations for improving the rapid REA #2 8. The objective of different pilots, at least at a high level, should be transparent and discussed 8 with the MAH 1. Feedback should be a formal part of the process and lessons from the pilots shared with MAH 9 and WP5 members 8. The EUnetHTA methodology should continue to a best practice model and not a collation of all 10 the methodological approaches used by the national HTA frameworks 8. The guidelines should be incrementally improved and where authors take a different position, 11 there should be a requirement to explain the rationale 128. The role of safety analysis needs to be reconsidered and tested in JA3 8. The tracking of re-use requires consistent definitions, a focus on whether this reduces 13 duplication and more consistent reporting 8. If re-use is to occur, all stakeholders need to commit to encourage its use. This includes 14 EUnetHTA, authors and reviewers and the industry The pilots under JA3 should investigate the value of explicitly defining where the Rapid 15 Assessment should replace elements of the national assessment. It seems most realistic this could be through a coalition of the willing 21 11

12 EFPIA views for JA3 JA3 is a crucial step in setting up a permanent system of European assessments JA3 should focus on delivering a limited number of high quality European assessments based on a manufacturer submission These European assessments need to be fully integrated in national HTA processes. This requires that: National agencies in charge of value assessment of pharmaceuticals at time of launch are directly involved in JA3 A set of MS with processes aligned with EUnetHTA methodologies commit to replacing some elements of their national assessments with the European assessments Other Member States engage in the reflection on how to modify processes to integrate European assessments 22 End of presentation 12

13 Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session 13

14 The cost of NO Europe Perspective of a Member State Wim Goettsch* Advisor International Affairs and Academia Milan, November Reasons for European Collaboration Technologies become more international and patients become more European Decrease duplication on HTA assessments Increase consistency between different national HTA assessments 27 BUT DECISIONS ON REIMBURSEMENT SHOULD BE MADE ON A NATIONAL LEVEL!! 14

15 Experiences of a middle-sized European country -Willingness to use European assessments - Have used three of the REA pilots; zostavax, canagliflozin and ramucirumab in more or less extent - Growing internal trust in the quality and consistency of the EUnetHTA REA reports - Have developed a format to most efficiently use the EUnetHTA report for our reimbursement commitee (joint report with extended summary in NL) - The language is not an issue -Timeliness is most important for pharma, 90-days after MA. 28 Leading to a national advice? 15

16 Issues for discussion Decision maker (Ministry of Health) supports use European reports as part of national assessment Can be used in alignment with clinical guideline development (national and international) More discussion on interpretation of EUnetHTA assessment for national reports Early advice collaboration with EMA is no brainer, but important equal playing field between HTA and EMA and sufficient involvement of smaller countries 30 Introduction Alastair Kent, OBE, Genetic Alliance UK, London, UK Recent HTA decisions Jacco Keja, PhD, IMS Health, Rotterdam, The Netherlands Industry view Member State view Overall conclusion Andrea Rappagliosi, LLM, Sanofi Pasteur MSD, Lyon, France Wim Goettsch, EUnetHTA JA2, Zorginstituut Nederland Alastair Kent - Moderator Q & A Session Q & A Session 16

17 Questions? 17

18 What actions need to be taken? Who are the key stakeholders required to drive this? What level of convergence is achievable vs. what level should be implemented? How do we define value between different countries? 18