Orphan Drug Approval Takeaways from Recent FDA Data

Authors of the May 2021 Fairness in Orphan Drug Exclusivity Act claim that the act will boost competition in the marketplace and drive down the cost of new medications. In “Orphan Drug Approval Takeaways from Recent FDA Data,” Senior Consultant Katherine Jones and co-author Omar Robles use publicly available records of small molecule approved orphan drugs to explore to what extent generic drugs have been delayed by the regulatory process created by the Orphan Drug Act. They find that overall, marketing approvals for orphan drug designations have increased in recent years. Initial orphan drug approvals are not systematically leveraged to extend drugs' exclusivity—in contrast, the majority of initial orphan drug approvals are granted on the same day as a drug's initial marketing approval. This early start to orphan drug approval means that the orphan drug exclusivity, good for seven years, is likely to overlap with other forms of exclusivity.