Overview of FDA's expanded access program for investigational drugs
Open accessAbstract
Expanded access, also called "compassionate use," provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials. The US Food and Drug Administration (FDA) facilitates the expanded access process; however, access to investigational treatments requires not only FDA's review and authorization but also the active involvement and cooperation of other parties, including drug companies and health care providers, in order to be successful.
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EAPs can enable broader access to investigational products across institutions prior to formal market approval.
“EAPs can enable broader access to investigational products across institutions prior to formal market approval (Klopfenstein et al., 2015; Jarow et al., 2017).”
From Isolator-based point-of-care manufacturing: a practical solution for GMP-compliant cell and extracellular vesicles therapy production (2025) · cites this paper as Jarow et al., 2017
The Introduction (page unavailable) says a widespread-treatment expanded access application can provide investigational drugs to a large population and bridge the period before marketing approval. The paper does not specify access across institutions, so that part of the claim is not established by this source. (LLM, conf=0.85)
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Research works printed but not linked: 5
- [4]Mackey TK Schoenfeld VJ Going “social” to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access BMC Med 2016 14 17 26843367 PMC4739083
- [5]Caplan A Ray A The ethical challenges of compassionate use JAMA 2016 315 10 979 980 26868205
- [6]Darrow JJ Sarpatwari JD Avorn J Kesselheim AS Practical, legal, and ethical issues in expanded access to investigational drugs N Engl J Med 2015 372 279 286 25587952
- [7]Zettler PJ Greely HT The strange allure of state “right-to-try” laws JAMA Intern Med 2014 174 12 1885 1886 25264589
- [10]Jarrow J Lemery S Bugin K Khosin S Moscicki R Expanded access of investigational drugs: the experience of the Center of Drug Evaluation and Research over a 10-year period Therapeutic Innovation & Regulatory Science 2016 50 6 705 709 27917324 PMC5135086
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Chiu, Y.-S., Wang, K.-L., & Wu, K.-L. (2025) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2025.1644318 · Cited by 17 · see citing works
“While HE typically focuses on treatments prepared and used within the same hospital, EAPs can enable broader access to investigational products across institutions prior to formal market approval (Klopfenstein et al., 2015; Jarow et al., 2017).”
- Authors
- Jarow, J. P., Lurie, P., Ikenberry, S. C., & Lemery, S.
- Affiliations
- United States Food and Drug Administration (US)
- Corresponding author
- Ikenberry, Sarah Crowley
- Venue
- Therapeutic Innovation & Regulatory Science
- Published
- 2017-03
- Volume
- 51
- Issue
- 2
- Pages
- 177-179
- ISSN
- 2168-4790 (ISSN-L)
- 2168-4804
- PMC
- PMC5443564
- OpenAlex
- W2588162428
- Cited by
- 84
- References
- 0
- Citations by year
- 2026: 6 · 2025: 9 · 2024: 9 · 2023: 9 · 2022: 12 · 2021: 12 · 2020: 8 · 2019: 11 · 2018: 4 · 2017: 4
- FWCI
- 5.47
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Therapeutic Innovation & Regulatory Science (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (FD999999)
- Topics
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Keywords
- compassionate use, investigational drugs, biologics, medical devices, FDA regulatory process, serious illness, pharmaceutical companies, healthcare workers
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