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

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.

KEYWORDS:

• Rare cancer
• oncology
• orphan drug designation
• drug development
• rare disease
• FDA approval

Declaration of interests

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Disclaimer

This article reflects the views of the authors and should not be construed to represent FDA’s or HHS’s views or policies.

Acknowledgments

The authors would like to thank Sandra Retzky and Aaron Friedman for their helpful comments on the manuscript.

Author contributions

LJF and KLM conceived the study. AVI, LJF, SK, CM, TL, and KLM designed the study. AVI and LJF collected the data. AVI, LJF, SK, CM, and KLM analyzed and interpreted the data. AVI and KLM drafted the manuscript. LJF, SK, CM, and TL provided substantive edits to the manuscript. All authors read and approved the final manuscript.

Additional information

Funding

This manuscript was funded in part by an appointment to the Research Participation Program at the U.S. Food and Drug Administration administered by the Oak Ridge Institute for Science and Education through an interagency agreement between the U.S. Department of Energy and the U.S. Food and Drug Administration.