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

 

 
 
A voz das pessoas com doenças raras na EuropaEURORDIS A voz internacional das pessoas com doenças raras, Rare Diseases InternacionalRare Disease International Reúne doentes, famílias e especialistas para partilhar experiências num fórum multilinguístico. RareConnect O programa Rare Barometer é uma iniciativa da EURORDIS para a realização de inquéritos para transformar a experiência das pessoas com doenças raras em números e factos que podem ser partilhados com os responsáveis pela tomada de decisões.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Adira ao maior encontro europeu das partes interessadas no âmbito das doenças raras na Conferência Bienal Europeia sobre Doenças Raras e Produtos Órfãos. A ECRD é uma iniciativa da EURORDISEuropean Conference on Rare Diseases