Efforts to develop orphan drugs: The FDA experience

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 [1]. 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.