The Rare Diseases Clinical Research Network (RDCRN) program advances 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 patients and patient advocacy groups to study more than 280 rare diseases.
The RDCRN is designed to promote highly collaborative, multi-site, patient-centric, translational and clinical research. The Rare Diseases Clinical Research Consortia (RDCRCs) focus on unmet clinical trial readiness needs that will move the field of research forward from its current state.
The RDCRN facilitates clinical research in rare diseases through support for:
- Collaborative activities, including multisite longitudinal studies of individuals with rare diseases and/or clinical trials.
- Career enhancement – encouraging the next generation of rare disease researchers.
- Pilot and feasibility projects
- Data management to facilitate high quality data standards, collection, storage and sharing
- Clinical research support
- 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 the program is now coordinated by NCATS. Since its launch, nearly 40,000 patients have been enrolled in network clinical studies. 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.
The RDCRN consists of 20 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 - 133.5KB) 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
Funding and scientific oversight for the RDCRN are provided by NCATS and 9 other NIH entities: the Eunice Kennedy Shriver National Institute of Child Health and Human Development; 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 Dietary Supplements. In addition, patient advocacy organizations may contribute funding.