Drug Repurposing: Legal and Regulatory Issues in the US

In the US, new drugs must demonstrate “substantial evidence of efficacy” and sufficient safety to the FDA before they can be widely approved. The evidentiary requirements needed for new drugs to be approved has evolved in recent decades, as the FDA applies flexibility to both the design and outcomes of clinical trials. Another regulatory option particularly relevant to repurposed drugs is the 505(b)(2) pathway, which requires a link to an approved reference drug, but can permit approval based on existing data without the need for further clinical testing. New drugs approved through the traditional pathway are invariably protected by patents lasting 20 years and earn a minimum of about 5-7.5 years of minimum exclusivity (12 for biologics). While repurposed drugs may not qualify for patents, about half of 505(b)(2) drugs are associated with patents or other statutory exclusivities. While repurposed drugs still should adhere to the same basic principles of rigorous evidence relevant to new drugs, there are special regulatory pathways and considerations related to their intellectual property that should be considered in establishing a framework for their development.

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Publication date (Electronic): 30 August 2022

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DOI: 10.14293/S2199-rexpo22011v1

SO-VID: f8b65af3-cc8e-404f-80aa-429fa54ba6fb

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Published under Creative Commons Attribution 4.0 International ( CC BY 4.0). Users are allowed to share (copy and redistribute the material in any medium or format) and adapt (remix, transform, and build upon the material for any purpose, even commercially), as long as the authors and the publisher are explicitly identified and properly acknowledged as the original source.

Conference name: RExPO22

Conference location: Maastricht, Netherlands

Conference date: 2-3 September, 2022

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Data availability: Data sharing not applicable to this article as no datasets were generated or analysed during the current study.

ScienceOpen disciplines: Law,Pharmacology & Pharmaceutical medicine

Keywords: Food and Drug Administration (FDA),patent,505(b)(2),substantial evidence

References

Krishnamurthy Nithya, Grimshaw Alyssa A., Axson Sydney A., Choe Sung Hee, Miller Jennifer E.. Drug repurposing: a systematic review on root causes, barriers and facilitators. BMC Health Services Research. Vol. 22(1)2022. Springer Science and Business Media LLC. [Cross Ref]
Kesselheim Aaron S., Tan Yongtian Tina, Avorn Jerry. The Roles Of Academia, Rare Diseases, And Repurposing In The Development Of The Most Transformative Drugs. Health Affairs. Vol. 34(2):286–293. 2015. Health Affairs (Project Hope). [Cross Ref]

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[1] Krishnamurthy Nithya, Grimshaw Alyssa A., Axson Sydney A., Choe Sung Hee, Miller Jennifer E.. Drug repurposing: a systematic review on root causes, barriers and facilitators. BMC Health Services Research. Vol. 22(1)2022. Springer Science and Business Media LLC. [Cross Ref]

[2] Kesselheim Aaron S., Tan Yongtian Tina, Avorn Jerry. The Roles Of Academia, Rare Diseases, And Repurposing In The Development Of The Most Transformative Drugs. Health Affairs. Vol. 34(2):286–293. 2015. Health Affairs (Project Hope). [Cross Ref]