POST-LAUNCH DEMANDS: Dr Thomas Lönngren ISPOR Content

Transcription

1 POST-LAUNCH DEMANDS: HOW CAN A COMPANY BEST ADDRESS THE CHALLENGES AND OPPORTUNITIES PRESENTED BY THE NEW EU PHARMACOVIGILANCE REQUIREMENTS OF THE REGULATORS, THE ASSESSOR S NEED FOR REAL WORLD EVALUATION, AND THE PAYERS NEEDS TO DEMONSTRATE VALUE? Dr Thomas Lönngren ISPOR 2013 Content The evolution of evidence generation Why is there an increased demand to demonstrate value post-launch? Regulatory post launch studies Payers demand on follow up and real world data On-going initiatives Type of post launch studies Conclusion 2

2 The evolution of evidence generation Knowledge, investment Regulatory approval Regulatory approval and market access Market access and regulatory approval Real world data RMP/REMS 1980 s time Why is there an increased demand to demonstrate value post-launch? New focus: more focus on benefit risk and cost effectiveness Why is it important to conduct post launch studies? Because it is part of an RMP/REMS Because it will increasingly be requested by HTA-payers bodies Because of new pharmacovigilance legislation focus on benefit risk Because the industry needs to protect its assets Because we need a cost-effective health care system

3 Positive Benefit /Risk Trial population Label Inappropriate population prescription Avandia Negativ e Lack of ext. validity Lack of communication Efficacy-Effectiveness Gap How to maintain value post launch You don t maintain the value by performing post launch studies - you need to manage your product in real life use. Existing methods Regulatory: Risk minimisation activities Payers: Risk sharing, pay for performance There is a need for more innovative interventions to ensure optimal use of medicines

4 Regulatory post launch studies Post-authorisation safety study (PASS) aims to obtain further information on a medicine's safety, or to measure the effectiveness of risk-management measures Post-authorisation efficacy study (PAES) aims to clarify the benefits of a medicine on the market including efficacy in everyday medical practice PRAC assessment Alemtuzumab Studies proposed by MA Applicant Observational PASS: Assess RMP issues and effectiveness of risk minimisation measures. PRAC/CHMP modified study proposal to increase coverage of sites to support efficient recruitment and also asked for yearly updates of progress and data for 5 years. Pregnancy Registry Paediatric study: Controlled head to head active comparative study in children with MS aged 10 to 18 years Effectiveness request for risk minimisation: 100% of cases of ITP and Goodpasture s to be detected! Off-label issue raised as a concern (common issue for PRAC) PRAC and CHMP outcomes: Both Positive Conclusion: Balanced assessment and outcome

5 Payers demand on follow up and getting real world data Data for assessing cost-effectiveness will increasingly come from post launch studies Uncertainty at launch will increasingly be accepted Cost-effectiveness is not only related to the product, but in high degree on an efficient disease management Need to follow up treatment Restricted reimbursement With evidence development With risk sharing schemes Experiences so far Not so many schemes so far Numbers increasing over time Majority in EU Most are covered by evidence development TLV in Sweden, NIHDI in Belgium, NICE in UK, CVZ in Netherlands Some progress But problems with data collection And the validity of the results

6 Requests for follow up studies from TLV in Sweden - some examples Follow up to secure that the drug is used for the right patients (reimbursed indication) Xenical, Reductil (weight loss) Champis (smoke cessation) Follow up to verify predicted effectiveness and/or cost-effectiveness Fosteo (osteoporosis) Elidel (excema) Ongoing initiative IMI PROTECT EMA ENCePP EuNetHTA

7 ENCePP An ENCePP initiative HTA assessment Evidence synthesis generation Full HTA assessment Data collection ENCePP PASS/ PAES Drug research & discovery Clinical development Marketing authorisation Pharmacovigilance Regulatory review

8 Post Launch studies? Post-authorisations(launch) studies Interventional Studies Non- Interventional studies Classical phase IV Pragmatic Trials? Observational studies - Study design? - Randomisation? - Confounding? PASS PAES? Other NIS PRO, QOL Case controlled studies Cohort studies Case series Case reports Conclusion New focus on post launch from regulators and payers Will lead to more request for post launch follow up studies Industry needs to focus on managing the use of medicines in real life settings Risk minimisation activities to maintain a high benefit risk ratio Optimising cost effective use All this is difficult since the use of the medicine is in the hand of the prescriber, the health care system and the patient Industry needs to take into consideration requirements from Regulators and HTA bodies early in drug development Also requirement on post launch studies Regulators, HTA bodies and payers need to give guidance on post launch studies Type of studies that will be accepted Design and validity Industry needs to get access to study sites, registries and health care records This is today a major obstacle in order to perform studies Quality and availability are problematic Regulatory and HTA/Payers requested post launch studies need to be coordinated Time and money for all involved