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

For ERTC Members only, this document is password protected. If you want to become a member, find out more here or contact Anne-Mary Bodin, Operations Assistant.

 

 
 
La voz de los pacientes de enfermedades raras en EuropaEURORDIS La voz internacional de las personas que tienen enfermedades raras, Rare Diseases International es una iniciativa de EURORDISRare Disease International Reuniendo a pacientes, familiares y expertos para compartir experiencias en un foro moderado en distintos idiomas, RareConnect es una iniciativa de EURORDIS RareConnect El Programa Rare Barometer es una iniciativa de EURORDIS que realiza encuestas para transformar las experiencias de los pacientes con enfermedades raras en cifras que puedan compartirse con los responsables políticos.Rare Barometer Una campaña internacional de sensibilización que se celebra todos los años el último día de febrero, El Día de las Enfermedades Raras es una iniciativa de EURORDISRare Disease Day Únete al mayor grupo de partes interesadas en toda Europa, en la Conferencia Europea sobre Enfermedades Raras y Medicamentos Huérfanos que se celebra cada dos años. ECRD es una iniciativa de EURORDISEuropean Conference on Rare Diseases