Repurposing existing drugs for new uses: a cohort study of the frequency of FDA-granted new indication exclusivities since 1997
Open accessAbstract
Background: Drug repurposing (i.e., finding novel uses for existing drugs) is essential for maximizing medicines’ therapeutic utility, but obtaining regulatory approval for new indications is costly. Policymakers have therefore created temporary indication-specific market exclusivities to incentivize drug innovators to run new clinical investigations. The effectiveness of these exclusivities is poorly understood. Objective: To determine whether generic entry impacts the probability of new indication additions. Methods: For a cohort of all new small-molecule drugs approved by the FDA between July 1997 and May 2020, we tracked new indications added for the subset of drugs that experienced generic entry during the observation period and then analyzed how the probability of a new indication changed with the number of years since/to generic entry. Results: Of the 197 new drugs that subsequently experienced generic entry, only 64 (32%) had at least one new indication added. The probability of a new indication addition peaked above 4% between 7 and 8 years prior to generic entry and then to dropped to near zero 15 years after FDA approval. We show that the limited duration of exclusivity reduces the number of secondary indications significantly. Conclusion: Status quo for most drug innovators is creating novel one-indication products. Despite indication-specific exclusivities, the imminence of generic entry still has a detectable impact on reducing the chances of new indication additions. There is much room for improvement when it comes to incentivizing clinical investigations for new uses and unlocking existing medicines’ full therapeutic potential.
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Most repurposed drugs are the result of serendipity, either observed through off-label prescriptions written by clinicians or discovered through patient experience.
“However, most repurposed drugs are the result of serendipity7,8—either observed through off-label prescriptions written by clinicians, as with gabapentin and bupropion8, or discovered through patient experience, as with sildenafil6.”
From A foundation model for clinician-centered drug repurposing (2024) · cites this paper as [6]
Approximately 30% of FDA-approved drugs are issued at least one new post-approval indication and many drugs have accrued over ten indications over the years.
“Approximately 30% of FDA-approved drugs are issued at least one new post-approval indication and many drugs have accrued over ten indications over the years6.”
From A foundation model for clinician-centered drug repurposing (2024) · cites this paper as [6]
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References 27 · Cited by 52
- Authors
- Sahragardjoonegani, B., Beall, R. F., Kesselheim, A. S., & Hollis, A.
- Affiliations
- University of Calgary (CA)
- Brigham and Women's Hospital (US)
- Corresponding author
- Hollis, Aidan
- Venue
- Journal of Pharmaceutical Policy and Practice
- Publisher
- Informa UK Limited
- Published
- 2021-01-04
- Volume
- 14
- Issue
- 1
- Pages
- 3-3
- ISSN
- 2052-3211 (ISSN-L)
- PMC
- PMC7780607
- OpenAlex
- W3118556611
- Cited by
- 52
- References
- 27
- Citations by year
- 2026: 3 · 2025: 11 · 2024: 7 · 2023: 14 · 2022: 12 · 2021: 5
- FWCI
- 7.25
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Journal of Pharmaceutical Policy and Practice (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, other-oa)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Libraries and Cultural Resources (University of Calgary) (submittedVersion)
- Funders
- Canadian Institutes of Health Research
- Topics
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmaceutical industry and healthcare
- Social Sciences › Business, Management and Accounting › Management of Technology and Innovation › Intellectual Property and Patents
- Keywords
- drug repurposing, generic entry, FDA approval, small molecule drugs, regulatory incentives
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