September 29, 2014
In order to enhance the quantity and quality of research on rare diseases, the NIH’s Office of Rare Diseases Research (ORDR) established the Rare Diseases Clinical Research Network (RDCRN) in 2003. The network is composed of 17 consortia, a Data Management and Coordinating Centre (DMCC) and over 90 patient advocacy groups. In an article published in the Journal of General Internal Medicine, Krischer et al. describe the network’s achievements and contribution to clinical research on rare diseases.
The RDCRN trains investigators conducting longitudinal and clinical studies on groups of rare diseases, publishes peer-reviewed articles and books, and gives conference presentations. Through its DMCC, the network operates as a platform to design clinical studies on rare diseases and collect, manage and improve access to clinical data. Besides also providing information concerning the consortia, the 200 or more studied diseases and ongoing clinical studies, the DMCC manages the RDCRN patient Contact Registry’s (CR) 14,776 subscriptions.
During the RDCRN’s first five years, ten of its consortia initiated 40 studies. The following five years saw the launch of 88 studies by 17 consortia. A number of RDCRN projects have incorporated patient-reported outcomes and have resulted in demonstrated efficacy of new treatments and diagnostics for rare diseases. The authors suggest the RDCRN has contributed significantly to accelerate research on rare diseases and the network’s use of technologies should continue evolving to maintain that momentum.