Expanded access programs: Ethical and practical considerations for Biopharmaceutical Sponsors
Open accessAbstract
Expanded access is a regulatory mechanism by which an investigational drug can be made available outside of a clinical trial to treat patients with serious or life-threatening conditions for which there are no satisfactory treatment options. An expanded access program (EAP) is the formal plan under which preapproval access to an investigational drug can be provided to a group of patients. Although an EAP is a regulated program, the decision to authorize an EAP is the responsibility of the biopharmaceutical sponsor. Because of the significant impact an EAP can have on current patients, drug development, and future patients, we propose that a sponsor's decision must be based not only on regulatory criteria but also on ethical and practical considerations regarding implementation of an EAP. Such an approach will help ensure that decisions and plans uphold ethical precepts such as fairness, promoting good, and minimizing risk of harm.
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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 Klopfenstein et al., 2015
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Principles for allocation of scarce medical interventions
Persad, G., Wertheimer, A., & Emanuel, E. J. (2009) · The Lancet · doi:10.1016/s0140-6736(09)60137-9 · Cited by 929 · see citing works
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Research works printed but not linked: 16
- [2]Whitefield K Huemer KH Winter D Compassionate use of interventions: results of a European clinical research infrastructures network survey of ten European countries. Trials. 2010; 11: 104. 21073691 PMC2997627
- [3]DiMasi JA Feldman L Seckler A Wilson A Trends in risks associated with new drug development: success rates for investigational drugs. Clin Pharmacol Ther. 2010; 87(3): 272– 277. 20130567
- [4]Caplan A Is it sound public policy to let the terminally ill access experimental medical innovations? Am J Bioeth. 2007; 7(6): 1– 3. 17558976
- [5]Annas G Cancer and the constitution: choice at life’s end. N Engl J Med. 2007; 357(4): 408– 413. 17652658
- [6]Jacobson PD Parmet WE A new era of unapproved drugs: the case of Abagail Alliance v Von Eschenbach. JAMA. 2007; 97(2): 205– 208. 17213404
- [7]Menikoff J Beyond Abigail Alliance: the reality behind the right to get experimental drugs. Kansas Law Review. 2008; 56: 1045– 1074.
- [8]Leonard EW Right to experimental treatment: FDA new drug approval, constitutional rights, and the public’s health. J Law Med Ethics. 2009; 37(2): 269– 279. 19493072
- [9]Shah S Zettler P From a constitutional right to a policy of exceptions: Abigail Alliance and the future of access to experimental therapy. Yale J Health Policy Law Ethics. 2013; 10(1): 137– 196. 20229846
- [10]Bener S Flicker L Rhodes R Access for the terminally ill to experimental medical innovations: a three-pronged threat. Am J Bioeth. 2007; 7(10): 3– 6. 17926208
- [11]London AJ Kimmelman J Emborg ME Beyond access vs. protection in trials of innovative therapies. Science. 2010; 328: 829– 830. 20466907 PMC4516403
- [12]Schulenk U Lowry C Terminal illness and access to phase I experimental agents, surgeries and devices: reviewing the ethical arguments. Br Med Bull. 2009; 89: 7– 22. 19056723
- [13]Chahal M Off-trial access to experimental cancer agents for the terminally ill: balancing the needs of individuals and society. J Med Ethics. 2010; 36: 367– 370. 20511354
- [14]Tuma RS Expanded-access programs: little heard views from industry. Oncology Times. 2008; 30(15): 19, 22– 23.
- [15]Childress J Beauchamp T Principles of Biomedical Ethics. New York, NY: Oxford University Press; 2009.
- [17]Scheunemann LP White DB The ethics and reality of rationing in medicine. Chest. 2011; 140(6): 1625– 1632. 22147821 PMC3415127
- [18]Edwards SJL Restricted treatments, inducements, and research participation. Bioethics. 2006; 20: 77– 91. 16770878
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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
- Klopfenstein, M., Van Campen, L. E., & Garnett, T.
- Affiliations
- Eli Lilly (United States) (US)
- Corresponding author
- Klopfenstein, Mitchell
- Venue
- Therapeutic Innovation & Regulatory Science
- Published
- 2015-05
- Volume
- 49
- Issue
- 3
- Pages
- 352-358
- ISSN
- 2168-4790 (ISSN-L)
- 2168-4804
- PMC
- PMC5802520
- OpenAlex
- W2156490363
- Cited by
- 11
- References
- 1
- Citations by year
- 2026: 1 · 2025: 3 · 2022: 1 · 2021: 1 · 2020: 2 · 2019: 1 · 2018: 1 · 2017: 1
- FWCI
- 0.49
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Therapeutic Innovation & Regulatory Science (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Funders
- Eli Lilly and Company
- Topics
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics and bioethics in healthcare
- Keywords
- expanded access program, investigational drugs, regulatory decision-making, fairness, risk minimization, drug development, compassionate use
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