A model for collaborative clinical research in rare diseases: experience from the Rare Disease Clinical Research Network program

Abstract

To overcome a lack of interest in research of rare diseases and an insufficient number of patients who can be recruited into clinical studies of rare diseases and potential diagnostics and treatments, the Rare Diseases Clinical Research Network (RDCRN) was established with the services of a centralized Data Management and Coordinating Center. The RDCRN has been very successful at recruiting patients, initiating clinical studies and trials, meeting patient recruitment milestones, training the next generation of research investigators and developing an active role for patient advocacy groups as research patnerships. The RDCRN has fostered international collaborations at multiple research sites that have led to regulatory approval of several drug products and expanded the knowledgebase for diseases included in the RDCRN. Rare disease research continues to evolve and considerable emphasis is placed on the translational research of discoveries from basic and clinical research activities, leading to the possibl...