Adopting Orphan Drugs — Two Dozen Years of Treating Rare Diseases

Abstract

In 1982, when the Orphan Drug Act was passed as an amendment to the Federal Food, Drug, and Cosmetic Act,1 few suspected the extent to which this law would alleviate the plight of patients with rare diseases. The law defines an orphan drug as one with efficacy against a disease affecting fewer than 200,000 people in the United States or one that scientists and economists at the Food and Drug Administration (FDA) determine will not be profitable for seven years after FDA approval.2 In the 24 years since this law was passed, 282 such drugs and biologic products, providing treatment . . .