Orphan Drugs: The United States Experience

Abstract

This paper provides an overview of orphan drug legislation in the United States. Since passage of the Orphan Drug Act in 1983, 182 designated orphan products have received Food and Drug Administration approval for rare disease treatment or diagnosis. Incentives provided in the act—seven years of marketing exclusivity, protocol assistance, tax credits, the orphan products grant program which authorizes grant moneys to support pivotal clinical trials, and user fee waiver—are discussed. Issues, challenges, and successes are outlined. Important international initiatives in orphan drug legislation triggered by the United States Orphan Drug Act are highlighted.