Optimising the development of ATMPs to meet patient needs - - PowerPoint PPT Presentation

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“ syste ms „Beveridge“

syste ms

Organisation Social health insurance National health service Financing Contributions Taxes Administration Independent by government Coverage Compulsory insurance Inhabitants

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?

The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics

For payers, these discussions are relevant in the context of relative effectiveness:

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

Color Code: Brown: MoCA Pink: Company Blue: EMA Pale Green: HTA Dark Green: Payer White: More than two stakeholders responsible The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics

Drug Discovery Pre-clinical Early clinical Late clinical Apply for MA

Scientific Advice & Protocol Assistance Parallel scientific advice/Early dialogue w. HTA

PRIME: continuous support and guidance

Establish Registry, if not already established

MoCA Discussion: Is this product of interest to patients & payers? Can it address an important unmet need? MoCA Discussion on design

• f pivotal trials – patient &

payer input MoCA Discussion of the value proposition (target population) MoCA Discussion about product delivery, centers, registries and post-approval data collection

Peri-Launch

Apply for MA Regulatory Review Positive CHMP decision HTA Recommen dation Price Negotiations MA by EC Patient Access HTA (rapid REA) Develop and Negotiate Managed Entry Framework Ensure registries in place and delivery defined Forming a „Coalition of the Willing“ Populating the TVF Initiate HTA review/ Scoping The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London, Dr. Anna Bucsics

Post-Launch

Patient Access Managed Entry Agreement Collect and Analyze Data Re-evaluate Product Renegotiate Reimburse- ment MoCA Input for Data Analysis (Modifed) Access (Modified) „Coalition of the Willing“

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

The fifth annual regulatory conference organised by EBE, 16 Dec. 2016, London

Th Thank you you for for you your attention

• n!

anna.buc ucsic sics@ s@un univie. ie.ac.at