19th Workshop, Barcelona: "Practical aspects of progressive patient access to OMPs, post-marketing evidence generation and related pricing questions"

19th Workshop of the EURORDIS Round Table of Companies

"Practical aspects of progressive patient access to OMPs, post-marketing evidence generation and related pricing questions"
 

 

October 15th, 2013

Barcelona, Spain

19th Workshop of the EURORDIS Round Table of Companies

This workshop focused mainly on aspects of progressive patient access (or adaptive licensing), to improve access to orphan medicinal products (OMPs). The current approach for licensing of medicines needs to be revisited as therapies, for rare diseases in particular, cannot easily be evaluated by the “gold standard” – randomised clinical trials. Adaptive licensing would enable earlier access for patients to the medicinal product with a continuation of data collection and regulatory evaluation. Industry, regulatory and patient representatives were invited to voice their opinions and concerns for this proposal and the attendees contributed via the afternoon break-out sessions.

Participants: 68

 

Programme 

Proceedings

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The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseasesRare Disease International Bringing together patients, families and experts to share experiences in a moderated multi-language forum. RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer 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