Back to Agenda
From discovery to patient access: how can Europe remain at the forefront of innovation in the development of diagnostics and therapies in rare diseases, and ensure patients benefit from the next hundreds of treatments?
Session Chair(s)
Alexander Natz, JD
Secretary General
European Confederation of Pharmaceutical Entrepreneurs, Belgium
At a key moment when a new EU Commission leadership is being introduced, and a strong agenda on health has been defined, it is timely to engage in a discussion on how European rare disease policy on rare diseases should be steered for the next two decades. From scientific discovery to diagnostic and therapeutic development and patient access, we ought to ask ourselves • How can Europe stay at the forefront of rare disease innovation? • How do we drive R&D in areas of unmet need whilst further encouraging competitiveness in areas where there are therapeutic options? • How can we leverage the use of real-world evidence to reduce uncertainties in the evidence generation for rare disease treatments?
Speaker(s)
Panelist
Tidde Goldhoorn
Health and Food Safety (DG SANTE), European Commission, Belgium
B.5 – Medicines: policy, authorization and monitoring
Panelist
Simone Boselli, MA
EURORDIS, Belgium
Public Affairs Director
Panelist
Maciej Gajewski
Alexion Pharmaceuticals, Inc., Belgium
Executive Director, Head of International Government Affairs and Policy
Panelist
Steven Simoens, PhD, MSc
Katholieke Universiteit Leuven, Belgium
Have an account?