The Rare Disease Act of 2002 (P.L. 107-280) directed the NIH Office of Rare Diseases Research (ORDR) to establish a Rare Disease Clinical Research Network—a collaborative, cooperative network of investigators and patient groups in partnership with technology leaders that focuses on studying rare diseases to enhance communication and resource sharing via a multidisciplinary approach.
The resulting RDCRC program collects clinical information to develop biomarkers and new approaches to diagnosis, prevention, and treatment; provides content for an Internet-based resource site about rare diseases; trains new clinical investigators in rare diseases research; supports a comprehensive and integrated approach to data collection, storage, and management, and integrates clinical data with other unique data, including genetic, imaging, pathologic, and laboratory data through the Data Management Coordination Center (DMCC).
The first competition for the RDCRC in 2003 established eight funded consortia using the U54 mechanism, and the program subsequently expanded to 10 centers. During the past eight years, the IDDB provided scientific management for two Consortia (Angelman, Rett, and Prader-Willi Syndromes Consortium and Urea Cycle Disorders) and their affiliate sites that have made significant progress and research contributions within the field of rare diseases research.
Recompetition of the entire program, including the DMCC, occurred in 2009 with a total of 19 consortia funded. The IDDB-supported Consortia successfully recompeted, with the addition of consortia to study Sterol and Isoprenoid Research Consortium as well as mitochondrial disorders (with the National Institute of Neurological Diseases and Stroke (NINDS).