2nd Multi-Stakeholder Symposium on Improving Patient Access to Rare Disease Therapies

22 - 23 February 2017. Hotel Le Plaza, Brussels, Belgium

 

This Symposium on improving patient access to rare disease therapies brought together patient advocates, payers, HTA bodies, academics, clinicians, policy makers, investors and industry representatives. It enabled participants to understand the interests and challenges of each stakeholder group, to discuss current and emerging initiatives and to continue to work together to improve patients’ access to rare disease therapies.

See documents referred to during the Symposium here

 

  Symposium Final Programme

Presentations from the Symposium:

1 Setting the scene, Yann le Cam, EURORDIS

2 PrimeMover Slides: Introduction, Charles Barker, PrimeMover Associates

3 The consequences of diverging and inconsistent decisions, Elizabeth Vroom, Dutch Duchenne Parent Project, Netherlands

4 The Importance of multi-stakeholder collaboration, Lucia Monaco, Fondazione Telethon

5 Collaborating for success, Karen Facey, HTAi

6 Prime, Adaptive pathways, Hans-Georg Eichler, EMA

7 A collaborative conversation, Charles Barker, PrimeMover Associates: part 1

8 A collaborative conversation, Charles Barker, PrimeMover Associates: part 2

9 A collaborative conversation, Charles Barker, PrimeMover Associates: part 3

10 MoCA’s (Mechanism of Coordinated Access to Orphan Medicinal Products) state of play, Ana Palma, Sobi

11 Innovative performance based outcome ageements, Karen Facey, HTAi

12 Recommendations from the European  Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), Lieven Annemans, Ghent University

13 Continued., Adam Hutchings, Dolon Ltd.

14 Feedback from Breakout 3, Karen Facey & Adrian Towse

15 Proposals for coordination of HTA across Europe, Wim Goettsch, EUNetHTA

16 Continued., Alicia Granados, efpia

17 Strengthening of the EU cooperation on HTA, Karolina Hanslik, DG SANTE, European Commission

 

 
 
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases