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Research

Office of Rare Diseases Research

The Office of Rare Diseases Research (ORDR) in the NCATS Office of the Director 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. To accomplish this, ORDR coordinates and fosters 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)

ORDR, in collaboration with the National Human Genome Research Institute, supports GARD, which provides comprehensive information on rare and/or genetic diseases in English and Spanish to patients, their families, health care providers, researchers and the public. In collaboration with GARD, ORDR maintains a 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)

ORDR, in collaboration with eight NIH Institutes (Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Cancer Institute, National Heart, Lung and Blood Institute, National Institute of Allergy and Infectious Diseases, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institute of Dental and Craniofacial Research, National Institute of Diabetes and Digestive and Kidney Diseases, and National Institute of Neurological Disorders and Stroke), manages the Rare Diseases Clinical Research Network (RDCRN) program, which consists of 19 distinct clinical research consortia and one Data Management and Coordinating Center.

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

  • Collaborative clinical research in rare diseases, including longitudinal studies of individuals with rare diseases, clinical studies, and/or Phase I, II and II/III trials.
  • Training of clinical investigators in rare diseases research.
  • Pilot and demonstration projects.
  • Access to information related to rare diseases for basic and clinical researchers, academic and practicing physicians, patients, and the public.

RDCRN serves as a test bed for distributed clinical data management that incorporates novel approaches and technologies for data management, data mining and data sharing across diseases, data types and platforms.

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ORDR Information


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.


Rare Diseases Research Advocate Retires

Stephen C. Groft

Long-time rare diseases research champion Stephen C. Groft, Pharm.D., director of the NCATS Office of Rare Diseases Research, announced his retirement effective Feb. 8, 2014. Learn more about Groft’s legacy.

In a recent PhRMA Catalyst blog, Groft discusses collaborating to speed rare diseases research.


Rare Disease Day at NIH

Rare Disease Day - February 28, 2014

View the webcast.