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 voix des patients atteints de maladies rares en EuropeEURORDIS Donnant la voix, à l'international, aux personnes vivant avec une maladie rareRare Disease International Pour rapprocher patients, familles et experts sur un forum modéré multilingue, où partager leurs expériences. RareConnect Le programme Rare Barometer est une initiative d’EURORDIS qui vise à réaliser des sondages pour transformer les expériences des patients de maladie rare en faits et chiffres à communiquer aux décideurs politiques.Rare Barometer Campagne internationale de sensibilisation organisée chaque année le dernier jour de février, la Journée Internationale des Maladies Rares est une initiative d'EURORDISRare Disease Day Participez au plus grand événement réunissant tous les acteurs des maladies rares en Europe lors de Conférence européenne bisannuelle sur les maladies rares et les médicaments orphelins (European Conference on Rare Diseases and Orphan Products, ou ECRD). L'ECRD est une initiative d'EURORDISEuropean Conference on Rare Diseases