Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies

ABSTRACT

Background

The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.

Research design and methods

We used a dataset containing all orphan drug designations between 1983 and 2019. To analyze the orphan products, we constructed a variable, ‘unique product,’ that allowed for the standardization of generic names of drugs and biologics. Additional analysis was performed on the most frequently designated unique products and their repositioning strategies.

Results

We found 5,099 orphan drug designations representing 3,269 unique products, of which 508 had an orphan-designated approval from FDA. Unique products with only a single designation represented 2,448 (75%) of the total products and 26 (1%) products had 10 or more designations. Over 60% of these unique products with 10 or more designations were antineoplastics or immunomodulators.

Conclusions

The most designated unique products revealed a continuum of repositioning strategies, from the repurposing of approved drugs to parallel indication development programs for recently developed drugs. The fact that over 3,000 unique products have been studied for rare diseases indicates that future repositioning opportunities may become increasingly available.

KEYWORDS:

• Orphan designation
• orphan drug act
• rare disease
• repositioning
• repurposing
• US food and drug administration

Acknowledgments

The authors would like to thank Jodi Black, Soumya Patel, Sandra Retzky, and Nicole Wolanski for their valuable comments on the manuscript.

Declaration of interest

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.

Disclaimer

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

Reviewer disclosures

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