Index: Investigational Drugs & Biologics

Investigational Drug Classification

Applicable Regulations

Guidance

Clinical Investigations Requiring an IND

An IND is required when

1. A drug/biologic has not been FDA approved for marketing; or
2. A marketed drug/biologic does not meet the IND exemption criteria.

IND Regulations:
21 CFR 312

Consent and IRB Review:
21 CFR 50 & 56

Investigational New Drug Applications (INDs) - Determining Whether Human Research Studies Can Be Conducted Without an IND (FDA Guidance)

Charging for Investigational Products (FDA Guidance)

Drug-Device Combination Products (FDA Guidance)

Off-Label and Investigational Use of Marketed Drugs, Biologics, and Medical Devices (FDA Information Sheet)

FAQ - Statement of Investigator, Form FDA 1572 (FDA Information Sheet)

UUHSC Investigational Pharmacy Policies (UU Policy)

Preparation Packet for Investigator-Initiated Drug and Device Studies (UU Guidance)

Investigator Responsibilities for Drug Studies (UU Guidance)

IRB SOP 502: Clinical Research Involving Investigational Drugs and Devices (UU Policy)

Clinical Investigations Under an IND Exemption

A clinical investigation of a marketed drug is exempt from the IND requirements if all of the criteria for an exemption in 21 CFR 312.2(b) are met:

1. The drug product is lawfully marketed in the United States.
2. The investigation is not intended to be reported to FDA as a well-controlled study in support of a new indication and there is no intent to use it to support a significant change in the labeling of the drug.
3. In the case of a prescription drug, the investigation is not intended to support a significant change in the advertising for the drug.
4. The investigational does not involve a route of administration, dose, patient population, or other factor that significantly increases the risk (or decreased the acceptability of the risk) associated with the use of the drug product.
5. The investigation is conducted in compliance with the requirements for review by an IRB and with the requirements for informed consent.
   1. Planned emergency research may not be conducted under an IND exemption. 
6. The investigation is conducted in compliance with the requirements of 21 CFR 312.7 (i.e., the investigation is not intended to promote or commercialize the drug product).

(Investigational New Drug Applications (INDs) - Determining Whether Human Research Studies Can Be Conducted Without an IND (FDA Guidance))

Consent and IRB Review:
21 CFR 50 & 56

Promotion of Investigational Drugs:
21 CFR 312.7

Expanded Access to Investigational Drugs

The Food and Drug Administration's Expanded Access Program allows for drugs that are investigational (i.e., not FDA approved) to be used for treatment use outside of a clinical trial. This includes Emergency Use of an Investigational Drug. 

Expanded Access to Investigational Drugs (UU Guidance)

Expanded Access to Investigational Drugs for Treatment Use (FDA Regulation)