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Expanded Access: Keywords, Definitions, and Resources

Expanded Access: Keywords, Definitions, and Resources

Keyword Definition
Expanded access Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Immediately life-threatening disease or condition Immediately life-threatening disease or condition means a stage of disease in which there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment. (21 CFR 312.300)
Industry The company responsible for developing the investigational medical product, and who may or may not be the manufacturer of the investigational medical product. They may be the commercial holder of the investigational new drug application (IND) or investigational device exemption (IDE) application or a noncommercial developer of the investigational medical product.
Informed consent Informed consent means that the purpose of the research is explained to the participant, including what their role would be and how the trial or study will work.
Institutional review board (IRB) Institutional Review Board (IRB) means any board, committee, or other group formally designated by an institution to review, to approve the initiation of, and to conduct periodic review of, biomedical research involving human subjects. The primary purpose of such review is to assure the protection of the rights and welfare of the human subjects. The term has the same meaning as the phrase institutional review committee as used in section 520(g) of the act. (21 CFR 56.102)
Investigational medical product Includes investigational new drugs and biologics, and investigational devices. Investigational new drug means a new drug or biological drug that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes. The terms “investigational drug” and “investigational new drug” are deemed to be synonymous. Investigational device means a device, including a transitional device, that is the object of an investigation. (21 CFR 312.3 and 21 CFR 812.3).
Investigator An individual who actually conducts a clinical investigation (i.e., under whose immediate direction the test article is administered or dispensed to, or used involving, a subject) or, in the event of an investigation conducted by a team of individuals, is the responsible leader of that team. (21 CFR 56.102)
Letter of Authorization (LOA) A letter permitting FDA to refer to the company’s IND or IDE file to provide certain necessary information about the investigational medical product (e.g., chemistry, manufacturing, controls) for the individual patient expanded access IND or IDE submitted by the applying licensed physician. The company should include the IND or IDE number for its investigational medical product in the LOA.
Serious disease or condition Serious disease or condition means a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible, provided it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one. (21 CFR 312.300)
Sponsor A person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. The sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator. A person other than an individual that uses one or more of its own employees to conduct an investigation that it has initiated is a sponsor, not a sponsor-investigator, and the employees are investigators. (21 CFR 312.3)
Sponsor-Investigator Sponsor-investigator means an individual who both initiates and actually conducts, alone or with others, a clinical investigation, i.e., under whose immediate direction the test article is administered or dispensed to, or used involving, a subject. The term does not include any person other than an individual, e.g., it does not include a corporation or agency. The obligations of a sponsor-investigator under this part include both those of a sponsor and those of an investigator. (21 CFR 56.102)

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Additional resources

21 CFR 312 Subpart I
Learn more about FDA’s current expanded access regulations for investigational drugs (including biologics).

21 CFR 812.35 - 21 CFR 812.36
Learn more about FDA's current expanded access regulations for supporting investigational devices expanded access.

Clinical Trial Information
Expanded Access Search Tool
Form and Letter Resources
Guidance Documents
FDA Expanded Access Program Data
Learning Resources
Articles and Reports
  1. Jarow, J. P., Lemery, S., Bugin, K., Khozin, S., & Moscicki, R. (2016). Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period. Therapeutic Innovation & Regulatory Science, 50(6), 705–709. Retrieved from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5135086/
  2. Jarow, J. P., Lemery, S., Bugin, K., & Lowy, N. (2017). Ten-Year Experience for the Center for Drug Evaluation and Research, Part 2: FDA’s Role in Ensuring Patient Safety. Therapeutic Innovation & Regulatory Science, 51(2), 246–249. Retrieved from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443559/
  3. Jarow, J. P. & Moscicki, R. (2017). Impact of Expanded Access on FDA Regulatory Action and Product Labeling. Therapeutic Innovation & Regulatory Science, 51(6), 787-789. Retrieved from: https://journals.sagepub.com/doi/pdf/10.1177/2168479017707800/
  4. Report from the 2018 external assessment of the Expanded Access program