Expanded Access Program
Overview
Expanded access, sometimes referred to as "compassionate use", is a way to provide access to an investigational medical product (drug/biologic or medical device) to patients with a serious or immediately life-threatening disease or condition outside of a clinical trial when there are no satisfactory alternative therapy options available.
- Serious means that the disease or condition is associated with morbidity that has substantial impact on day-to-day functioning.
- Immediately life-threatening means a stage of disease in which there is reasonable likelihood that death will occur without early treatment.
The FDA provides several expanded access treatment pathways, each having specific criteria that must be met. The designated expanded access pathway depends on the type of medical product used (drug/biologic or medical device) and the number of individuals to be treated. See FDA Guidance on Expanded Access for additional details.
Criteria for All Expanded Access Programs
To qualify for expanded access use, the FDA must determine the following conditions have been met (21 CFR 312.305(a)):
- The patient(s) to be treated has a serious or immediately life-threatening disease or condition;
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
- The potential patient benefit justifies the potential risks of the treatment, and those potential risks are not unreasonable in the context of the disease or condition to be treated; and
- Providing the investigational product for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.
Expanded Access Requirements
Expanded Access Pathway | Is Prior IRB Approval Required? | Is Prior FDA Approval Required? | Is Consent Required from patient (or LAR)? | How Many Individuals Can Be Treated? |
|---|---|---|---|---|
Emergency Use IND (drugs/biologics) | NO | YES | YES* | 1 |
Single Patient IND (drugs/biologics) | YES | YES | YES | 1 |
Intermediate Size IND (drugs/biologics) | YES | YES | YES | Small Group |
Emergency Use pathway (devices) | NO | NO | YES* | 1 |
Compassionate Use provision (devices) | YES | YES | YES | 1 or Small Group |
Treatment IND/IDE (drugs/biologics/devices | YES | YES | YES | Widespread Use/No Limit |
*See detailed information on exception to informed consent requirements for Emergency Use.
Types of Expanded Access
Select one of the options below to review the different Expanded Access pathways, including specific criteria and process requirements.
Emergency Use
Expanded Access to Drugs/Biologics
Expanded Access to Medical Devices
Page updated October, 2025