Is your Study IND Exempt?

Check the DailyMed database external site (opens in a new window) for approved indications of the drug/biologic

The FDA regulations can be reviewed at 21 CFR 312.2(b) external site (opens in a new window) and are summarized below.

A drug that is lawfully marketed in the United States is exempt from the requirements for an IND if all of the following apply:

1. The investigation is not intended to be reported to the FDA in support of a new indication for use or any other significant change in the labeling for the drug.
2. For prescription drugs, the investigation is not intended to support a significant change in the advertising for a prescription drug product.
3. The investigation does not involve a change in route of administration, dosage level, or patient population, or other factor that significantly increases the risks (or decreases the acceptability of the risks) associated with use of the drug product.
4. The investigation is conducted in compliance with the requirements for IRB review (21 CFR 56 external site (opens in a new window) ) and informed consent (21 CFR 50 external site (opens in a new window) ).
5. The drug may not be represented as safe or effective for the purposes for which it is under investigation, nor may it be commercially distributed or sold (i.e., the investigation is not intended to promote or commercialize the drug product).

If there is uncertainty about whether the exemption criteria are met, the potential sponsor-investigator can seek advice from FDA on the applicability of the IND regulations (§ 312.2(e)).

The potential sponsor-investigator of a clinical investigation using a marketed drug is responsible for determining whether the investigation meets the criteria for an IND exemption.  

• Answer the questions in the IND Decision Tool external site (opens in a new window) , with particular attention to requirement #3.
• For assistance, see the FDA Guidance Investigational New Drug Applications (INDs) – Determining Whether Human Research Studies Can Be Conducted without an IND external site (opens in a new window) (especially pages 5-7).

Your IRB submission should include the following:

• IND Decision Tool external site (opens in a new window)
• Email documenting guidance provided by UCSF Regulatory Support staff regarding IND exemption, if applicable
• Documentation of any official guidance provided by FDA indicating that an IND application is not required (less common)

If the IRB does not agree with the sponsor-investigator's rationale for IND exemption, an IND application must be submitted to FDA’s CDER (Center for Drug Evaluation and Research) or CBER (Center for Biologics Evaluation and Research).