Optimising the development of ATMPs to meet patient needs Listening to the Stakeholders of Innovative Medicinal Products – Payer Viewpoint Anna Bucsics, Advisor to MoCA (www.eurordis.org/content/moca) Mechanism of Coordinated Access to Medicinal Products This presentation reflects discussions with colleagues from payer institutions, notably Ad Schuurman, Martin van de Graaff (ZIN) and Marc De Casteele (RIZIV/INAMI). Their contributions are gratefully acknowledged. Nevertheless, the opinions presented here and any mistakes are mine. The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London
Presenting the Payers There is no single payers‘ view on the several different approaches to improve access to medicines …nor on the degree of involvement the pre-approval process. The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
Health Care Systems in Europe „Bismarck“ „Beveridge“ syste ms syste ms Organisation Social health National health insurance service Financing Contributions Taxes Administration Independent by government Coverage Compulsory Inhabitants insurance Images from Wikipedia The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
When in the development process should users be involved in risk/benefit discussions, risk level acceptability? For payers, these discussions are relevant in the context of relative effectiveness : The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
HTA and Payers‘ Questions about the Evidence Does the new product have additional, patient-relevant benefit? Better efficacy/effectiveness? Better tolerability/safety? If yes, how great is this benefit? How reliable and valid are the results? Trial design? Comparator? Surrogate endpoints? What can be done to remedy the deficits? Post-approval data collection? What if the data do not deliver? Managed entry agreements? Will they work? Do we have the resources to make them work? The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London
Pre - Launch MoCA Discussion: Is this MoCA Discussion on design MoCA Discussion of the value product of interest to patients & of pivotal trials – patient & proposition (target population) payers? Can it address an payer input important unmet need? Drug Apply for Pre-clinical Early clinical Late clinical Discovery MA PRIME: continuous support and guidance Color Code: Brown: MoCA Scientific Advice & Protocol Assistance Pink: Company Blue: EMA MoCA Discussion about Pale Green: HTA product delivery, centers, Dark Green: Payer Parallel scientific advice/Early dialogue w. registries and post-approval White: More than two stakeholders responsible HTA data collection Establish Registry, if not already established The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
Peri-Launch Populating the TVF Forming a „Coalition of the Willing“ Positive CHMP Apply for MA Regulatory Review MA by EC Patient Access decision HTA Price Initiate HTA review/ HTA (rapid REA) Recommen Negotiations Scoping dation Develop and Negotiate Managed Entry Framework Ensure registries in place and delivery defined The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
Post-Launch MoCA Input for Data Analysis (Modified) „Coalition of the Willing“ Renegotiate Patient Re-evaluate (Modifed) Collect and Analyze Data Reimburse- Access Product Access ment Managed Entry Agreement The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
ATMPs – Blurring the Boundaries Who will pay? Hospital budgets? Health insurance? Special funds? International cooperation/Cross-border issues? For what? Cells, intervention, follow-up? Who will get the money? Company, centers? How much? When? Up front? Over time, based on performance? What will be the role of the European Centers of Expertise? The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London
Effect and cost-effectiveness are not everything Even cost effective treatments may break the bank... The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics
What is a Fair Price? Value based – what the market will bear? Fair return on investment - including public funds (basic research, RWD collection, etc.) Fair reimbursement For each component of the technology Compared to alternatives Savings for the system should be real, not „skimmed-off“ via high prices Sustainable Equitable Considers uncertainty – adaptive pricing …Transparent? The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London
Th Thank you you for for you your attention on! anna.buc ucsic sics@ s@un univie. ie.ac.at The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London
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