Orphan Drugs

The term "orphan drug" refers to a product that treats a rare disease affecting fewer than 200,000 Americans. The Orphan Drug Act was signed into law on January 4, 1983. Since the Orphan Drug Act passed, over 100 orphan drugs and biological products have been brought to market.

The intent of the Orphan Drug Act is to stimulate the research, development, and approval of products that treat rare diseases. This mission is accomplished through several mechanisms:

• Sponsors are granted seven years of marketing exclusivity after approval of its orphan drug product.
  
• Sponsors also are granted tax incentives for clinical research they have undertaken.
  
• FDA's Office of Orphan Products Development coordinates research study design assistance for sponsors of drugs for rare diseases [Notice: This link will take you outside the CDER web site].
  
• The Office of Orphan Products Development also encourages sponsors to conduct open protocols, allowing patients to be added to ongoing studies.
  
• Grant funding is available to defray costs of qualified clinical testing expenses incurred in connection with the development of orphan products.