1st Multi-stakeholder Symposium on Improving Patient Access to Rare Disease Therapies

Symposium logo

24 - 25 February 2016

Brussels, Belgium

Symposium session The Multi-Stakeholder Symposium on Improving Patient Access to Rare Disease Therapies aimed at addressing a crucial bottleneck in making orphan medicinal products (OMP) accessible across Europe.

A unique combination of nearly 300 patient advocates, academics, policymakers, industry representatives, payers and HTA bodies came together to discuss the current state of play and how to shape a more effective way to address value determination, appraisal, pricing and reimbursement of orphan medicines, all with the aim of improving patients’ access to rare disease therapies throughout Europe.

By bringing together such a varied range of stakeholders, the Symposium created an opportunity for exchange to reach an understanding of the varied perspectives on issues and challenges surrounding patient access. Participants discussed various methods of appraisal, value determination and reimbursement and also took part in pricing simulation exercises.

Speakers included European Commissioner for Health Vytenis Andriukaitis and Members of the European Parliament Philippe De Backer and Françoise Grossetête.


Download Symposium Conclusions

Download final programme (PDF, 475 KB)

Download final concept paper (PDF 404 KB) 

Download the flyer (PDF, 842 KB)


1. Official Partners

2. Programme Committee Members & Advisors

3. Preparatory Documents

4. Preparatory Webinar

5. Contact

4. Official Partners


5. Programme Committee Members & Advisors

Members of the Programme Committee:

Lieven Annemans
Professor of Health Economics
Gent University

Ri De Ridder
Director-General of Healthcare

Karen Facey
Evidence Based Health Policy Consultant
Glasgow University

Ruediger Gatermann
Director, Healthcare Policy and External Affairs Europe
CSL Behring

Josie Godfrey
Public Policy and External Affairs
Sobi - Swedish Orphan Biovitrum AB

Laura Gutierrez
Senior Director European Government Relations and Public Policy

Adam Heathfield
Co-chair EFPIA - EuropaBio Task Force
Senior Director, International Policy

Christian Hill
Managing Director
Map Biopharma

Virginie Hivert
Therapeutic Development Director

Yann Le Cam
Chief Executive Officer

Vinciane  Pirard
co-chair EFPIA - EuropaBio Task Force
Director Public Affairs
Genzyme Europe BV

Advisors to the Programme Committee:

François Houÿez
Treatment Information and Access Director / Health Policy Advisor

Kristina Larsson
Head of Office – Orphan Medicinal Products
European Medicines Agency (EMA)

Solange Rohou
ADAPT SMART consortium (EMA)
Director Regulatory Affairs

Ad Schuurman
Head of the Business Contact Centre & International Affairs
National Health Care Institute (former CVZ)
The Netherlands

Cees Smit
Patient advocate
European Genetic Alliances Network (EGAN)
The Netherlands

Chris Sotirelis
Patient advocate
UK Thalassaemia Society

7. Preparatory documents

8. Preparatory webinar: "An Introduction to HTA

Date: Thursday 4th February 2016, from 15.00 to 16.00 CET
Moderator: Julia Chamova from EUnetHTA
Co-presented by Conor Teljeur from EUnetHTA & François Houÿez, Treatment Information and Access Director at EURORDIS.

Download the presentation (PDF, 4,27 MB)

This webinar covered the following topics:

  • Definition of the principles and concepts of HTA
  • How patients and industry can be part of an HTA process/exercise
  • Costs and economic aspects of HTA

9. Contact

Please address all questions to Anne-Mary Bodin at anne-mary.bodin@eurordis.org

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