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Office of Rare Diseases Research

The NCATS Office of Rare Diseases Research (ORDR) supports and coordinates rare diseases research, responds to research opportunities for rare diseases and provides information on rare diseases. ORDR serves the needs of patients who have any one of the thousands of rare diseases known today. Through programs such as the Genetic and Rare Diseases Information Center and the Rare Diseases Clinical Research Network, ORDR staff coordinate and foster relationships with a variety of stakeholders, from patient advocacy groups to academic institutions as well as other NIH Institutes and Centers.

Genetic and Rare Diseases Information Center (GARD)

NCATS, in collaboration with the National Human Genome Research Institute, supports GARD — a center that provides comprehensive information on rare and/or genetic diseases to patients, their families, health care providers, researchers and the public. NCATS maintains a GARD database of rare disease information. Results include information from the National Library of Medicine, scientific conferences, support groups, and clinical trials and research.

Rare Diseases Clinical Research Network (RDCRN)

The RDCRN is designed to advance medical research on rare diseases by facilitating collaboration, study enrollment and data sharing. Through the networks, physician scientists work together with patient advocacy groups on more than 200 rare diseases at sites across the nation. NCATS manages the network, in collaboration with 10 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 the Director. The RDCRN consists of 22 distinct clinical research consortia and one Data Management and Coordinating Center.

Related Links


ORDR Information

Rare Diseases News

brain MRI

On October 8, 2014, NIH announced awards to expand NCATS’ collaborative Rare Diseases Clinical Research Network (RDCRN). Through the network, physician scientists at 22 consortia will work with 98 patient advocacy groups to advance clinical research and investigate new treatments for patients with rare diseases. The collaborations are made possible by $29 million in fiscal 2014 funding from NIH.

NIH news release

NCATS Director’s Message

RDCRN 2014 consortia and data center project page

RDCRN fact sheet

NCATS at Work

NCATS scientists propose a new way to test drugs and develop better treatments for rare disease patients by targeting common molecular mechanisms across multiple diseases.