EUCERD/EMA workshop drives forward a public-private partnership model for rare disease registries
On 4 October, stakeholders gathered at the Canary Wharf, London-based European Medicines Agency for a brainstorming session on how to best design, manage and share rare disease registries in a way that will be purposeful and satisfying for all players. Experts from academia, the biopharmaceutical industry, patient organisations, and regulatory agencies all lent their expertise to the event, which culminated in a consensus towards disease-based registries that could ultimately be shared amongst all relevant public and private partners. This consensus, shifting from the drug- or patient-based designs to a larger-encompassing disease-based model, moves forward the challenge of how to coordinate, manage and share the goldmine of data that the disease registries potentially yield.
Well constructed and managed rare disease registries can significantly speed up clinical research in the fields of rare diseases and orphan drugs, further the understanding of many elements including prevalence, natural history, and treatment outcome for rare diseases, provide regulatory bodies – including pricing and reimbursement agencies - with crucial data, serve as a resource for trial recruitment, and help patient organisations to coordinate efforts and share information.
The EUCERD/EMA workshop, organised in the context of the ongoing scientific activities of the EU Committee of Experts on Rare Diseases (EUCERD) dedicated to registries in the field of rare diseases, builds upon the Rare Diseases Task Force (RDTF) report, Patient registries in the field of rare diseases, based on outcomes of the 2008 RDTF workshop on this field, updated in 2011, as well as the report in preparation on Creation of a mechanism for the exchange of knowledge between Member States and European authorities on the clinical added-value of orphan drugs (CAVOD) and the Orphanet Report Series Disease registries in Europe.
Future meetings already being planned will tackle the complex questions of how to protect important privacy rights for industry, academic and patient registry partners, how to manage post-marketing authorisation data for orphan drugs, how to coordinate at an international level, and harmonisation between registries. The EpiRare project (European Platform for Rare Disease Registries) launched in April 2011, seeks to gather more information on the needs of stakeholders, while the International Rare Disease Research Consortium brings to the table the international perspective, expectations and experience. Yes, there is much to be done, but the EUCERD/EMA workshop took a big step forward on 4 October.