Eurordis - Rare Disease Europe

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

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.

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

• EURORDIS Position Paper — Breaking the Access Deadlock to Leave No One Behind (EURORDIS Position Paper (January 2018)
• 2018 Overview Report on the ‘State of the Art of Rare Disease Activities in Europe’
• Conclusions and Next Steps from the workshop ‘How ERNs can add value to clinical research’
• RD-ACTION Outputs to support ERNs

Official partners

Contact

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