3rd EURORDIS Multi-Stakeholder Symposium on Improving Patients’ Access to Rare Disease Therapies

Let’s make a pact to ensure patients’ sustainable access to rare disease therapies

13-14 February 2019 
Crowne Plaza Brussels – Le Palace, Rue Gineste 3, 1210 Brussels, Belgium
Metro Station: Rogier

Final Programme

Over 250 people gathered at this event, including rare disease patients, patient advocates, healthcare industry, consultants, investors, ERN clinical representatives, academia, healthcare professionals, researchers, payer bodies, HTA agencies, National Competent Authorities, regulators, policy makers, and government workers.

Some of the important take-home messages include the recognition that multi-stakeholder collaboration is a fundamental condition to achieving patient access to rare disease medicines; that there is a need for a more trusted and transparent cooperation between payers and companies; and that the expectations for the European Reference Networks (ERNs) to be an important facilitator in evidence generation building is also an area of high interest that will be monitored closely over the coming years.

EURORDIS-Rare Diseases Europe is now gathering the input developed in the plenary and breakout sessions and will include them in the outcomes document as a roadmap for all parties to collaborate in a process to improve patients’ access to rare disease medicines. The final version of the roadmap document will be shared in April 2019.

Presentations

Welcome_and_opening_remarks by Yann Le Cam

Keynote_speech by Marius Bradatan

There_is_a_new_treatment_approved_when_can_I_get_it_the_example_of_SMA by Mencia de Lemus

Introduction_to_Breakout_1 by Victoria Hedley 

Introduction_to_Breakout_3, Anna Bucsics by Simone Boselli

Introduction_to_Breakout_4 by Eileen Treacy 

Breakout_1 by Victoria Hedley

Breakout_1 by Hanneke van der Lee

Breakout_1 by Andreas Jung

Breakout_2 by Kristina Larsson

Breakout_2 by Margaret Galbraith

Breakout_2 by Francois Houyez

Summary_Breakout_1

Summary_Breakout_2

Breakout_3, Anna Bucsics by Wills Hughes Wilson

Breakout_3 by Angela McFarlane

Breakout_4 by Eileen Treacy

Breakout_4 by Josie Godfrey

Breakout_4 by Thomas Morel

Breakout_4 by Diego Ardigo

Summary_Breakout 4

 

Recording of the Symposium Plenary Session

Pre-Symposium Webinars

Two webinars have taken place ahead of this Symposium to provide the knowledge needed to effectively participate in discussions at the event.

 

First pre-symposium webinar

Covering topics in breakout sessions 2 and 3. Webinar speakers:

  • Anna Bucsics, University of Vienna; Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
  • Simone Boselli, Public Affairs Director, EURORDIS-Rare Diseases Europe

 

Second pre-symposium webinar

Covering topics in breakout sessions 1 and 4. Webinar speakers:

  • Victoria Hedley, Newcastle University, Rare Disease Policy Manager, Newcastle University John Walton Muscular Dystrophy Research Centre, MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine
  • Simone Boselli, Public Affairs Director, EURORDIS-Rare Diseases Europe

Documents

Official partners

EPF.jpg                       efpia_2.jpg                         eurocope.jpg

europabio_0.jpg                                HOPE_0.jpg                                  PGEU.jpg

eahp_logo_0.jpg                           htai_logo.png         ispor_logo

 

Contact

Questions regarding this event? Please contact Martina Bergna, Events Junior Manager: martina.bergna@eurordis.org

 

 

 
 
Голос пациентов с редкими заболеваниями в Европе The voice of rare disease patients in EuropeEURORDIS Сеть организаций, представляющих интересы пациентов с редкими заболеваниями их разных стран мираRare Disease International Социальная сеть RareConnect предоставляет пациентам, их семьям и специалистам многоязычную платформу для обмена информацией и опытом RareConnect Программа Rare Barometer — это инициатива EURORDIS, направленная на проведение исследований по преобразованию опыта пациентов с редкими заболеваниями в цифры и факты, которыми можно поделиться с ответственными руководителями. Rare Barometer День больных редкими заболеваниями – международная кампания по привлечению внимания к проблеме редких заболеваний, которая по инициативе EURORDIS ежегодно проводится в последний день февраляRare Disease Day Приняв участие в Европейской конференции по редким заболеваниям и орфанным препаратам (EUCERD), которая по инициативе EURORDIS проводится каждые два года, вы получите возможность побывать на самом представительном европейском форуме по проблемам редких заболеваний.European Conference on Rare Diseases