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

Abstract

ABSTRACT Background Rare cancers account for approximately one in eight 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. Research design and methods Using an internal US Food and Drug Administration (FDA) database, we classified and analyzed all orphan drug designations and approvals specifically designed to prevent, diagnose, or treat rare cancers, from 1983 to 2022 by affected organ system. Results In the past 40 years, more than 180 rare cancers have had at least one product that has been developed and shown promise in its treatment, diagnosis, or prevention resulting in nearly 100 approved products for rare oncologic indications. Rare hematologic cancers were the most frequently designated and approved cancers, but nearly every organ system was represented by an orphan drug-designated product. Conclusions Orphan drug designations and approvals target a wide variety of rare cancer types and disease locations suggesting an expanding base of research activity, discovery, and options for patients with these often-fatal diseases.