November 19, 2014
Earlier last month, the National Institutes of Health (NIH) announced $29 million towards expanding the National Center for Advancing Translational Sciences (NCATS)’ collaborative Rare Diseases Clinical Research Network (RDCRN) which is dedicated to furthering translational research and investigating new treatments for patients with rare diseases. Currently comprising of 2600 researchers, the NIH Office of Rare Diseases established RDCRN in 2003. RDCRN is overseen by NCATS to work towards advancing medical research on rare diseases by facilitating collaboration, study enrollment and data sharing.
RDCRN is made up of 22 distinctive consortia and a Data Management and Coordinating Center that work collaboratively to improve availability of rare disease information, treatment, clinical studies, and general awareness for both patients and the medical community. The RDCRN also aims to provide up-to-date information for patients and to assist in connecting patients with advocacy groups, expert doctors, and clinical research opportunities.
This new NIH funding will establish six new RDCRN consortia, including bone diseases, lung diseases, food allergy disorders, and three separate neurological diseases concentration areas— amyotrophic lateral sclerosis and related disorders, autism and intellectual disabilities, and frontotemporal lobar degeneration. NIH said researchers at the 22 RDCRN consortia will conduct a minimum of two multisite clinical studies, including one longitudinal natural history study for a group of at least three related rare diseases. Each consortium will partner with relevant patient advocacy groups. According to the NIH press release this award will result in “physician scientists at 22 consortia collaborating with representatives of 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases”.