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

 Executive summary

 Proceedings document

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

 

 
 
La voce dei malati rari in EuropaEURORDIS Rare Diseases International, la voce internazionale dei malati rariRare Disease International RareConnect è un'iniziativa di EURORDIS che mette in contatto malati, famiglie ed esperti per condividere esperienze in un forum multilingua e moderato RareConnect Il Programma Rare Barometer è un’iniziativa di EURORDIS volta a condurre indagini con l’obiettivo di trasformare le esperienze delle persone affette da malattie rare in cifre e fatti che possono essere condivisi con i decisori politici.Rare Barometer La Giornata delle Malattie Rare, una campagna di sensibilizzazione internazionale che si svolge l'ultimo giorno di febbraio di ogni anno.Rare Disease Day La Conferenza Europea biennale sulle Malattie Rare è il più grande incontro di soggetti interessati al tema delle Malattie Rare in Europa. L'ECRD è un'iniziativa di EURORDISEuropean Conference on Rare Diseases