NIH/Office of Rare Disease Research

The Office of Rare Disease Research was established in 1993 within the Office of the Director (OD), National Institutes of Health (NIH), to respond to the reporting requirements on the Orphan Drug Act; to implement the recommendations of the National Commission on Orphan Diseases; and to respond to requests for information on rare diseases. The Office of Rare Diseases develops and maintains a centralized database on rare disease clinical research supported by the NIH; stimulates rare disease research by supporting scientific workshops and symposia to identify such research opportunities; responds to requests for information on highly technical matters and matters of public policy relative to rare diseases and orphan products; provides information to the Office of the Director, NIH, on matters relating to rare diseases and orphan products; coordinates and serves as a liaison with Federal and non-Federal national and international organizations concerned with rare disease research and orphan products development; and prepares the NIH Director’s annual report to Congress on rare disease and condition research activities sponsored by NIH. The Office of Rare Diseases also provides a web site on the Internet to help meet the need for up-to-date, accurate information frequently requested by patients and their family members or caregivers, health care providers, and research investigators. The site provides information on rare diseases and conditions; ongoing or planned research including clinical trials; and support services provided by voluntary health organizations. This site provides links to disease-specific information; to searchable databases that provide information on clinical studies; and to other government and non-government home pages and other voluntary health organizations that provide information and support services.