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Rare Diseases Clinical Research Network

Rare Diseases Clinical Research Network (RDCRN)

About the RDCRN

The Rare Diseases Clinical Research Network (RDCRN) program 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 network, scientists from multiple disciplines at hundreds of clinical sites around the world work together with patient advocacy groups to study more than 280 rare diseases.

The purpose of the RDCRN is to facilitate clinical research in rare diseases through support for:

  • Collaborative activities, including multisite longitudinal studies of individuals with rare diseases, and/or clinical trials
  • Training of clinical investigators in rare diseases research
  • Pilot and demonstration projects
  • Uniform data collection protocols
  • Access to information about rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the public

The RDCRN was established by the NIH Office of Rare Diseases in 2003 and now is overseen by NCATS. Since its launch, nearly 40,000 patients have been enrolled in network clinical studies. As of October 2014, the network is composed of about 2,600 researchers, including scientific program staff from NCATS and collaborating NIH components, academic investigators, and members of 130 patient advocacy groups; 91 studies are under way.

Current Consortia

The RDCRN consists of 22 distinct clinical research consortia with a Data Management and Coordinating Center. View descriptions of the current RDCRN consortia.

RDCRN Clinical Trials

RDCRN consortia support a broad range of clinical research, including clinical trials. However, NCATS' authorization (PDF - 408KB) limits specific support for clinical trials through pilot projects and within training programs to trials only through the end of Phase IIA.

Phase II clinical trials are designed to test drugs for efficacy (or effectiveness) and side effects in a limited number of patients. Phase IIA trials provide data for exposure-response in patients, while Phase IIB trials provide data for dose-ranging in patients.

A Collaborative Environment

NCATS manages the network in collaboration with 10 other NIH components: the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institute of Arthritis and Musculoskeletal and Skin Diseases; the National Institute of Dental and Craniofacial Research; the National Institute of Diabetes and Digestive and Kidney Diseases; the National Institute of Mental Health; the National Institute of Neurological Disorders and Stroke; and the Office of the Director. Staff from NCATS and these Institutes and offices provide scientific oversight and funding support. In addition, patient advocacy organizations may contribute funding.

RDCRN Newsletter: Spotlight on Rare Diseases

View the current and archived issues of the RDCRN Consortium's newsletter Spotlight on Rare Diseases.

Last updated: 09-01-2017
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