In the field of rare diseases, it is impossible to reach a critical mass of resources at national level. To achieve a European critical mass of experts and operational partners, all the proposed actions are based on the coordination and development of already existing and active networks and organisations across Europe: Orphanet, Eurordis, EPPOSI, EuropaBio, EBE. The project aims at developing information tools to address in a comprehensive and integrated approach the set of factors that currently affects research on rare diseases and its coordination. The specific objectives are: (1) to develop an information service, freely accessible on Internet, dedicated to research activities in the field of rare diseases and orphan medicinal products, including a database of research projects, funded at MS level and at the EU level, and a database of collections and research networks. (2) to develop services aiming at speeding up the enrolment of patients in clinical research. (3) to develop a database of research projects with development potential, to help scientists and Industry establish the necessary partnerships. (4) to organise a workshop with all stakeholders to discuss known bottlenecks and find solutions. This project is based on input from the following (1) an EU funded information service on rare diseases: Orphanet www.orpha.net (2) a European platform of Patients organization, Science and Industry (EPPOSI) which actively supports partnering activities. (3) an umbrella organization of patient support groups (Eurordis) involved in supporting research and regulatory activities. The project aims at establishing the platform of services in 11 European countries in the pilot phase in order to propose an extension to the 25 European countries in 2 years time. Ultimately, the goal is to convert scientific developments into diagnostic tools and therapies as quickly as possible
Keywords:
innovative therapy, orphan drugs, clinical trial
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