WORKSHOP: EU OMP review – unmet medical need

NOTE: YOU NEED TO REGISTER (BLUE BUTTON)

This workshop will be undertaken under Chatham House Rules, all workshop  participants are free to contribute their observations, views and  use the information received, but cannot attribute these observations to the speakers or other participants at the workshop. We request the spirit of the Chatham House Rules is adhered to by all those attending.

Please note: This session is LIVE and will not be avaliable on demand.

In view of the European Commission’s forthcoming review of the Orphan Regulation, FIPRA’s Special Adviser Sheela Upadhyaya has been invited as a Board member of the World Orphan Drug Congress to host a workshop on the key issues, which will be addressed in the forthcoming review.

The workshop will be designed so that it offers the opportunity for the European Commission and relevant stakeholders to explore what revisions are needed for the legislation to appropriately incentivise the development and availability of products for people living with rare diseases. The Technopolis report evaluating the Regulation has identified many of the complex challenges in ensuring research is appropriately directed to areas of unmet medical need - scientific and societal considerations come in to play around affordability, innovation and market dynamics. The EU Commission is also planning to launch a new EU Pharmaceutical Strategy in Autumn 2020.

The session will be designed to allow an informal discussion with WODC participants in order to have an open consideration of approaches to update and improve the legislation.

The workshop will be chaired by Sheela Upadhyaya, Avisory Board Member of the World Orphan Drug Conference, and will be held under Chatham House Rules.

The workshop will be supported by the Orphan Drug Incentives Expert Group with funding being provided by Takeda.

Proposed Focus:

The OMP and addressing unmet need - Is a new approach needed?

Currently orphan designation is defined using prevalence 5 in 10,000: along with demonstration of significant benefit however, there is a question as to whether it is a good substitute for stimulating the market to address unmet need. Studies show that 95% of people living with a rare disease condition have yet no treatment option. The Technopolis report has flagged the importance of considering how to stimulate the market, looking at areas of unmet need beyond cancer, alimentary conditions and metabolic disorders, which make up a large percentage of orphan medicines.

Should the definition of ‘condition’ be redefined in the orphan regulation to consider future scientific developments and innovative ways to develop products? Is the use of prevalence as a marker of unmet need to determine orphan designation of a product still fit for purpose? How to redirect research and development to addressing the 95% of unmet need? Is there another way to define what an OMP is? How can we explore the challenges and then consider what elements will be useful to better define OMPs? How to ensure the right incentives are in place to stimulate the development of treatments for patients with rare diseases?

Structure & Timing of the Workshop

The workshop will be designed to stimulate the exchange of views in an open and off - the - record discussion. The virtual meeting will be held under Chatham House Rules.

• 2 November 2020 | 14.00h – 16.30h CET | pre-session to World Orphan Drug Congress (2-5 November)

15mins Introduction  & Setting the Scene and key questions for discussion by Sheela Upadhyaya – Board Member WODC & FIPRA Special adviser

7 mins Andrzej Rys - Director, responsible for Health Systems,   Medical Products and Innovation at DG SANTE, European Commission

7 mins Dr Marcel van Raaij – Director of Pharmaceutical Affairs and Medical Technology, Ministry of Health, The Netherlands

7 mins Simone Boselli - Public Affairs Director, EURORDIS

7 mins Dr Erik Tambuyzer - Executive Chair, BioPontis Alliance for Rare  Diseases

7 mins Toon Digneffe - Chair of the Orphan Drug Incentives Expert Group and member EUCOPE’s Executive Board (Head EU Public Affairs and Public Policy, Takeda)

7 mins Introduction to initial study findings - Orphan Drug Incentives Expert Group by Christian Jervelund - Managing Partner, Copenhagen Economics

15min break for Input of questions from audience

80 mins Q&A debate Moderated by Sheela Upadhyaya