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
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.