Expanded Access, occasionally referred to as “compassionate use”, 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 there is no comparable or satisfactory alternative therapy options available. In most circumstances, approval by the IRB and FDA is required prior to enrolling the participant.
Contrary to common usage, the terms “emergency use” and “compassionate use/expanded access” are not the same. FDA’s definition of “emergency use” exemption allows the use of a test article on 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 FDA and IRB approval. See Emergency Use guidance for additional information.
In This Section
First, determine if expanded access is appropriate. Expanded access may be appropriate when all the following apply:
- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
If expanded access is appropriate, IRB approval is required.
Submit a New Project for IRB review. Include the following in your application:
- Initial Review Application
- HRP-506 Consent Form Template
- HRP-503 General Template Protocol or Sponsor Protocol
- Ancillary Approvals if applicable (CCSRC Approval is not required for Single Patient IND trials)
- HRP-226 Administrative Approval Form
- Investigator Brochure or Package Insert, if applicable (Drugs/Biologics)
- Instructions for Use, if applicable (Devices)
- Evidence of FDA IND or IDE