Affiliations: Office of Orphan Products Development, Food and Drug
Administration (FDA), Washington DC, USA
Abstract: In 1983, the United State's Congress passed the U.S. Orphan Drug
Act, which established public policy that the Federal Government would assist
in the development of products for the diagnosis, prevention, or treatment of
rare diseases or conditions . The Orphan Drug Act defined an "orphan
product" as one that is intended to treat a rare disease or condition that
affects fewer than 200,000 people in the United States OR as a
product which will not be profitable within seven years of approval by the FDA.
There are over 6,000 conditions that meet the definition of a rare disease. The Orphan Drug Act allows the government to provide a package of financial
incentives to companies who decide to undertake the development of products for
a rare disease or condition. The Office of Orphan Products Development (OOPD)
conducts scientific and regulatory review of orphan product and humanitarian
use device designation requests and administers a scientific grant program to
defray the cost of clinical studies for orphan product development.
Keywords: FDA, office of orphan products development, orphan designation, orphan drug act