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FDA-Regulated Drugs and Biologics Used in Research


SOP 022: Research Involving Investigational Drugs and Biologics

Frequently Asked Questions

What is a drug or biologic?

Drug: A substance intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease. A substance (other than food) intended to affect the structure or any function of the body.

Biologic: Biologics include a wide range of products such as vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins. A biologic can be composed of sugars, proteins, or nucleic acids or complex combinations of these substances, or may be living entities such as cells and tissues.

When is a drug or biologic used in a research project subject to FDA regulations?

If your study involves a drug or biologic that is not being used for its approved use and during the course of medical practice (i.e., standard of care), then your study is subject to FDA regulations.

Do I need to get IRB approval if I am using a marketed drug off-label in my clinic?

If physicians use a product for an indication not in the approved labeling, they have the responsibility to be well-informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain records of the product's use and effects. Use of a marketed product in this manner when the intent is the "practice of medicine" does not require the submission of an Investigational New Drug Application (IND). See FDA Guidance on "Off-Label" and Investigational Use Of Marketed Drugs, Biologics, and Medical Devices.

What is a clinical investigation?

Clinical investigation means any experiment that involves a test article and one or more human subjects (21 CFR 56.102c). A project is not a clinical investigation if the drug/biologic is being used during medical practice and is not being used to diagnose, treat, cure, prevent, or mitigate a disease or condition.

What is meant by IND exempt?

Not all clinical studies need to operate under an IND. The FDA has outlined specific situations where the use of a drug or biologic in research is exempt from IND requirements. See WVU OHRP’s Drugs and Biologics Decision Tool (LINK) for more information.

A drug/biologic may be IND exempt if it meets all of the following criteria:

  • Study is not intended to be reported to the FDA as a well-controlled study in support of a new indication or a change in labeling.
  • Study is not intended to support a change in advertising for the drug.
  • Study does not involve changing the route of administration, dosing, population, or other factors that significantly increase or decrease the acceptability of subject risk.
  • Study is conducted in compliance with FDA requirements for review by an IRB and informed consent requirements.
  • Study is not intended to promote or commercialize the drug.

Or if it falls into one of the following categories:

  • Testing of selected in vitro diagnostic biological products (21 CFR 312.2)
  • Some bioavailability or bioequivalence studies
  • Some cold isotopes

Is a dietary supplement a drug? Do I need an IND to use a dietary supplement in a study?

According to FDA’s 2013 FDA IND Determination Guidance (page 12), whether an IND is needed for a study evaluating a dietary supplement is determined by the intent of the clinical investigation.

  • An IND is not required if the clinical investigation is intended only to evaluate the dietary supplement’s effect on the structure or function of the body or reduce the risk of a disease, intended to support a new or expanded health claim and conducted in healthy individuals over 12 months of age.
  • However, an IND is required if the clinical investigation is intended to evaluate the dietary supplement’s ability to diagnose, cure, mitigate, treat, or prevent a disease. An IND may also be needed if clinical investigations is intended to evaluate whether a food substance/supplement may reduce the risk of a disease in individuals less than 12 months of age, those with altered immune systems, and those with serious or life-threatening medical conditions.

Please contact WVU OHRP with any questions if you are unsure or seek clarification from the FDA at

What is expanded access? Is it the same thing as compassionate use?

Expanded access refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient’s disease or condition. The terms expanded access, access, and treatment use are used interchangeably. Expanded access allows for drugs that are investigational (i.e., not FDA approved) to be used for treatment use outside of a clinical trial. This is also sometimes referred to as compassionate use. Treatment use is not considered a clinical investigation, however, FDA submission and IRB review are necessary. The criteria for Expanded Access are largely determined by (1) the seriousness of a patient's condition, (2) other available FDA-approved treatment options, (3) patient population size needing treatment, and (4) drug status. This webpage is a summarized guide to the FDA regulations on Expanded Access to Investigational Drugs for Treatment Use, found in [21 CFR 312.300]. It does not provide full guidance for Expanded Access to Investigational Drugs; as such, FDA regulatory materials must also be consulted for full information.

Under FDA’s current regulations, there are three categories of expanded access:

  • Expanded access for individual patients, including for emergency use (21 CFR 312.310).
  • Expanded access for intermediate-size patient populations (generally smaller than those typical of a treatment IND or treatment protocol — a treatment protocol is submitted as a protocol to an existing IND by the sponsor of the existing IND) (21 CFR 312.315).
  • Expanded access for widespread treatment use through a treatment IND or treatment protocol (designed for use in larger patient populations) (21 CFR 312.320).

Do I need to obtain consent from a subject who receives an investigational drug through expanded access?

Yes. Expanded access to an investigational drug for treatment use, including emergency use, requires informed consent as described in 21 CFR part 50. Investigators treating a patient(s) with an investigational drug under expanded access are responsible for ensuring that the informed consent requirements of part 50 are met (21 CFR 312.305(c)(4)). One of the purposes of informed consent is to ensure that the patient is informed that he/she will be treated with an investigational product and that there may be uncertainty about the safety and effectiveness of the product. See FDA Guidance on Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers.

What is emergency use?

Emergency use is defined as the use of an investigational drug or biological product with a human subject in a life-threatening situation in which no standard acceptable treatment is available and in which there is not sufficient time to obtain IRB approval [21 CFR 56.102(d)]. The emergency use provision in the FDA regulations [21 CFR 56.104(c)] is an exemption from prior review and approval by the IRB. The exemption, which may not be used unless all of the conditions described in 21 CFR 56.102(d) exist, allows for one emergency use of a test article without prospective IRB review. FDA regulations require that any subsequent use of the investigational product at the institution have prospective IRB review and approval. FDA acknowledges, however, that it would be inappropriate to deny emergency treatment to a second individual if the only obstacle is that the IRB has not had sufficient time to convene a meeting to review the issue.

Life-threatening, for the purposes of section 56.102(d), includes the scope of both life-threatening and severely debilitating, as defined below.

  • Life-threatening means diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted and diseases or conditions with potentially fatal outcomes, where the end point of clinical trial analysis is survival. The criteria for life-threatening do not require the condition to be immediately life-threatening or to immediately result in death. Rather, the subjects must be in a life-threatening situation requiring intervention before review at a convened meeting of the IRB is feasible.
  • Severely debilitating means diseases or conditions that cause major irreversible morbidity. Examples of severely debilitating conditions include blindness, loss of arm, leg, hand or foot, loss of hearing, paralysis or stroke.

If you believe you need to submit a request to administer a test article in an emergent situation in which there is no have time to obtain prior IRB approval, (see additional information below to be sure your situation qualifies), please complete and submit the Emergency Use Request Form.

The physician reviews the FDA definition of life threatening [21 CFR 56.102(d)] and can verify that the proposed use of an investigational drug or device meets that definition. The physician should contact the WVU OHRP or IRB for consultation regarding the potential emergency use. If the definition of life threatening is not met, emergency use procedures must not be followed. For additional options, consult the IRB guidance on Expanded Access for Investigational Drugs and Expanded Access for Unapproved Devices.

Visit WVU OHRP’s Emergency Use of a Test Article website for further guidance.