Rare Diseases Act of 2001

The Rare Diseases Act of 2001 (S. 1379) aims to expand federal support for the study and treatment of medical conditions affecting fewer than 200,000 people in the United States. The bill formally establishes the Office of Rare Diseases at the National Institutes of Health (NIH) and creates a network of regional centers of excellence to conduct specialized research and provide public education. Additionally, it increases funding for the Food and Drug Administration to provide grants and contracts for the development of "orphan drugs," which are treatments specifically designed for rare diseases that might otherwise be neglected by the private market.