The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022

Background Rare cancers account for approximately 1 in 8 of all cancers diagnosed in the United States (US) every year. Remarkable scientific advances in cancer research over the past 40 years, in addition to financial incentives provided by the Orphan Drug Act (ODA), have led to hundreds of drug approvals for rare cancers.