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

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An exceptional two-day event not to be missed!

24 - 25 February 2016

Brussels, Belgium

No new on-site registrations will be accepted

EURORDIS and partners will bring together industry, patient leaders, academics, regulators and payers 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. In line with the EURORDIS Round Table of Companies, this symposium aims to improve patients' access to rare disease therapies throughout Europe.

Download final programme (PDF, 475 KB)

Download final concept paper (PDF 404 KB) 

Download the flyer (PDF, 842 KB)


1. Symposium Objectives

2. Register

3. Venue

4. Official Partners

5. Programme Committee Members & Advisors

6. Hotel & Travel Information

7. Documents

8. Webinar

9. Contact

1. Symposium Objectives

This meeting on the topic of HTA is envisaged as a two-stage process, with a second symposium in 2017 on a more consensual approach to the new solutions discussed throughout 2016. For this 1st meeting, the overarching aim is to gain common understanding and agreement on the determinants of value, and of the assessment and appraisal methods for orphan medicinal products, by:

  1. Bringing all participants to a common understanding of issues/challenges of access to rare disease therapies (webinar/pre-reads to be circulated before meeting);
  2. Sharing existing and new processes to recognise which ones work best;
  3. In view of the 2017 symposium,  agreeing on a structured approach forward and a set of solutions to explore.

To read more about this event and the expected outcomes, please find here the concept paper for the Multi-Stakeholder Symposium: Improving Access to Rare Disease Therapies (PDF, 404 KB)

2. Register

Registrations for the Symposium are closed.

3. Venue

Hotel Le Plaza Brussels

Boulevard Adolphe Max 118 - 126, Brussels, Belgium

Tel. + 32 2 278 01 00

Metro Station: Rogier


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

6. Hotel & Travel Information

EURORDIS has booked a block of rooms and has negotiated special rates at the Hotel Le Plaza for the nights of 24 and 25 February 2016. If you wish to make a reservation at the Hotel Le Plaza, please contact the reservation department directly (Tel. +32 2 278 01 00 /

Single occupancy: 195 Euros per night including breakfast*

Double occupancy: 224 Euros per night including breakfast*
*City Tax is not included in the room rate - a supplement of 9.85 € per room per night will be applied

Details of other hotels close to the venue will be provided here soon.

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

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