The Current Environment in Orphan Drug Development

Abstract

Pharmaceutical development over the past two decades has shown a substantial increase in the number of drugs available to treat rare (“orphan”) diseases, largely due to the passage of the Orphan Drug Act. This article provides an overview of orphan diseases and conditions; the Orphan Drug Act; the Food and Drug Administration's Office of Orphan Products Development, which administers the Orphan Drug Act; the changing nature of rare disease treatment; and designing clinical trials to investigate rare diseases. The success of the Orphan Drug Act is far greater than anticipated; to date 242 orphan drugs have been approved. The Orphan Drug Act continues to encourage manufacturers to develop treatments to help more than 11 million patients in the United States who suffer from rare diseases.