Adaptive pathways intro_ispor_second_plenary_nov14
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This document summarizes discussions around improving early patient access to innovative new drugs in Europe. It discusses the perspectives and needs of various stakeholders in health care systems, including patients, regulators, payers, providers, and drug companies. It also evaluates different approaches that have been tried or proposed, such as adaptive licensing pathways, managed entry agreements between drug companies and health systems, and performance-based risk-sharing arrangements. Finally, it outlines areas where more progress could still be made, such as increased coordination between regulatory agencies and health technology assessment bodies, better use of disease registries and electronic health records to generate real-world evidence, and public-private partnerships to support these efforts.
Adaptive pathways intro_ispor_second_plenary_nov14
- 1. Adrian Towse Director of the Office of Health Economics New Challenges for Improving European Health Care: EARLIER ACCESS TO INNOVATION – IS IT WORTH IT?
- 2. ISPOR Plenary Earlier Access To Innovation. Is It Worth It? 2 What do we need? Trusheim, October 2014* •Patients: Early, appropriate access refined over time and accounting for their preferences •Regulators: Staged benefit / risk improving over time •Payers: Deliver better health while stewarding resources •Providers: More therapeutic options with improving knowledge of which are best for whom •Sponsors: Sustainable innovation chain from science to patient to investor *Presentation http://vitaltransformation.com/wp- content/uploads/2014/10/Trusheim-Stakeholder-Metrics-Overview-FINAL-PRINT.pdf Success Requires Increased Collaboration Supported by Prospectively Planned Evidence & Decisions to Build Trust and Viability
- 3. ISPOR Plenary Earlier Access To Innovation. Is It Worth It? 3 Can it deliver? Baird et al. 2013. Comparison of stakeholder metrics* Drug Scenario eNPV US$ AdaptivenessScore Change in Patient Access years Inappropriately treated patients Comments Zelboraf 1:Traditional $23m NA NA - (skin cancer) 2: Actual $34m 5 9100 - 3: Adaptive $30m 10 9100 - As per fast track but with continuous planned data collection Gilenya 1: Actual $127M NA NA - (relapsing MS) 2: Staged $169M 12 41,000 - Smaller quicker trials for narrower authorisation. Later expansion of label Acomplia (anti- obesity) 1:Actual ($73M) NA NA 0.9M Withdrawn in EU after 2 years, never approved in the US 3: Post Market Surveillance ($43M) 13 11.5M 1.7M Controlled for off label, adherence, maintained over life cycle 5:Staged ($16M) 16 10.1M None Smaller quicker trials for narrower authorisation. Later expansion of label. Controlled for off label, adherence, maintained over life cycle *Baird et al. 2013. Therapeutic Innovation & Regulatory Science 47(4):474-483.
- 4. ISPOR Plenary Earlier Access To Innovation. Is It Worth It? 4 Where are we now? Boon et al. 2014 Escher Report* •CMA is granted on the base of less comprehensive data, but assessment timelines were longer. CMA is perceived as a ‘rescue option’ by regulators and companies, rather than as a prospectively planned pathway to provide early access. •Of the cohort of 47 NASs approved in 2007, at least one PASS was requested at market entry for 22. Yet Post Authorisation Safety Studies (PASS) are not the main source of new safety information listed in the SmPC, but the costs of conducting these studies appear substantial. •‘Adaptive licensing has been discussed for a long time with very little action ..new regulatory pathways will only be useful if they are considered in a holistic approach including health technology assessment (HTA) and payers’. Furthermore, the importance of ‘understanding the business needs for new pathways’ is stressed. *Improving the EU system for the marketing authorisation of medicines. Learning from regulatory practice. Escher Foundation
- 5. ISPOR Plenary Earlier Access To Innovation. Is It Worth It? 5 •MEAs are used to give access to new technologies where traditional reimbursement is deemed inappropriate. •Three different forms of MEAs have been identified: •management of uncertainty relating to clinical and/or cost- effectiveness; •management of utilization to optimize performance. •management of budget impact; •The rationale for using these approaches and their advantages and disadvantages differ. •All forms of MEA should take the form of a formal written agreement among stakeholders, clearly identifying the rationale for the agreement, aspects to be assessed, methods of data collection and review, and the criteria for ending the agreement. Role for Managed Entry Agreements?* *Klemp, Frønsdal and Facey on behalf of the HTAi Policy Forum (2011) IJTAHC 27: 77-83
- 6. Performance-Based Risk-Sharing Arrangements— Good Practices for Design, Implementation, and Evaluation Garrison, L.P., Towse, A., Briggs, A., de Pouvourville, G., Grueger, J., Mohr, P.E., Severens, J.L., Siviero, P. and Sleeper, M. (2013 Report of the ISPOR Good Practices for Performance-based Risk-sharing Task Force. Value in Health. 16(5), 703-719.
- 7. WHERE COULD WE BE? MOVEMENT TOWARD BETTER EVIDENCE GENERATION IN EUROPE Post-authorisation efficacy studies (PAES) implemented Greater HTA body and EMA coordination pre-launch Disease registries in some countries , and progress in EHRs Coordination across HTA bodies in demand for post-launch studies, often linked to CED, P4P schemes Collaborations across large registries Full use of EHRs Good progress in methods Public-private partnerships have a major role Adaptive licensing applied to a variety of drugs Joint HTA and EMA coordination for pre-and post- launch Most Likely Scenario Scenario Most Conducive To Evidence Generation Anticipating the future for CER and RE in the pharmaceutical industry. Research conducted by CMTP and OHE, sponsored by Amgen, GSK, Lilly, Novartis, Sanofi. Publications in progress.
- 8. ISPOR Plenary Earlier Access To Innovation. Is It Worth It? 8 About OHE To enquire about additional information and analyses, please contact Professor Adrian Towse at atowse@ohe.org. To keep up with the latest news and research, subscribe to our blog, OHE News. Follow us on Twitter @OHENews, LinkedIn and SlideShare. The Office of Health Economics is a research and consulting organisation that has been providing specialised research, analysis and expertise on a range of health care and life sciences issues and topics for 50 years. OHE’s publications may be downloaded free of charge for registered users of its website. Office of Health Economics Southside, 7th Floor 105 Victoria Street London SW1E 6QT United Kingdom +44 20 7747 8850 www.ohe.org ©2013 OHE