The Rare Diseases Clinical Research Network is an initiative of the Office of Rare Diseases Research. RDCRN is funded by the ORDR, the National Center for Advancing Translational Sciences and collaborating institute centers. The RDCRN is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation. Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and more.
History
The following is a timeline of the Rare Diseases Clinical Research Network:
As a result of the Rare Diseases Act of 2002, on February 27, 2003, the ORDR, the General Clinical Research Consortium requested applications for a Rare Diseases Clinical Research Network.
On November 3, 2003, the NIH established the Rare Diseases Clinical Research Network with a Data Technology Coordinating Center and the first Rare Disease Clinical Research Consortia. The founding members of the RDCRN were:
On February 8, 2009, the ORDR partnered with 10 other NIH Institutes to release two requests for resubmissions for the RDCRN.
On October 8, 2014, the NIH announced additional funding of $29 million.
On October 3, 2019, the NIH announced funding for 20 rare diseases clinical research consortia and a new Data Management and Coordinating Center through the National Center for Advancing Translational Science's Office of Rare Diseases Research, along with the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy ShriverNational Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.
Rare Diseases Clinical Research Consortia
In its fourth funding cycle, the Rare Diseases Clinical Research Network consists of 20 Rare Diseases Clinical Research Consortia and a Data Management and Coordinating Center. The RDCRCs, the DMCC, and their Principal Investigators are located at the following institutions:
Brittle Bone Disorders Consortium, Brendan Lee, M.D., Ph.D., Baylor College of Medicine, Houston, TX.
Clinical Research in Amyotrophic Lateral Sclerosis and Related Disorders for Therapeutic Development, Michael Benatar, M.D., Ph.D., University of Miami Miller School of Medicine, Miami, FL.
Global Leukodystrophy Initiative Clinical Trials Network, Adeline L. Vanderver, M.D., Children's Hospital of Philadelphia; S. Ali Fatemi, M.D., M.B.A., Kennedy Krieger Institute; and Florian S. Eichler, M.D., Massachusetts General Hospital.
Primary Immune Deficiency Treatment Consortium, Jennifer M. Puck, M.D., University of California San Francisco and Donald B. Kohn, M.D., University of California Los Angeles.
Urea Cycle Disorders Consortium, Andrea L. Gropman, M.D., FAAP, FACMG, Children's National Medical Center, Washington, DC.
Vasculitis Clinical Research Consortium, Peter A. Merkel, M.D., M.P.H., University of Pennsylvania, Philadelphia, PA.
Data Management and Coordinating Center, Eileen King, Ph.D., Maurizio Macaluso, M.D., Ph.D., and Peter White, Ph.D, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
The RDCRN’s Data Management and Coordinating Center is hosted by Cincinnati Children's Hospital Medical Center in Cincinnati, OH. The DMCC manages shared resources and data from the RDCRN research studies. The DMCC emphasizes the standardization of data, increased data sharing and broad dissemination of research findings."
