Expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
There are two types of expanded access programs available for individual patients. Click on the links below for a definition of each expanded access type.
Expanded Access – Compassionate Use
Access to an investigational medical product for use by a single patient meeting all expanded access criteria in a non-emergency situation, which is a situation in which there is sufficient time for a written submission to be made, there is time to use existing procedures to obtain FDA and IRB approval prior to use of the product.
Expanded Access – Emergency Use
Access to an investigational medical product for use by a single patient meeting all expanded access criteria in an emergency situation, which is a situation that requires a patient to be treated before a written submission can be made and there is no time to use existing procedures to obtain FDA approval prior to use of the product. The regulations do not require prior IRB review and approval for emergency use prior to patient treatment.
Criteria for all Expanded Access Uses:
- Patient(s) have 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;
- Patient enrollment in a clinical trial is not possible (i.e., eligibility, location);
- The potential patient benefit justifies the potential risks of the treatment and the potential risks are not unreasonable in the context of the disease of condition to be treated; and
- Providing the investigational medical product (drug/device) will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
Which Expanded Access Program Option Fits My Patient’s Situation?
Click here to use the flowchart to determine the appropriate Expanded Access pathway for your patient.
Scenario 1:
My patient meets all the criteria of Expanded Access and there is enough time to submit IRB approval before treatment.
Consider Compassionate Use. See the checklist for details.
➡️ Compassionate Use Tools and Resources
Scenario 2:
My patient meets all the criteria of Expanded Access but there is not enough time to submit IRB approval before treatment.
Consider Emergency Use. See the checklist for details.
➡️ Emergency Use Tools and Resources
Scenario 3:
My patient does not meet all the Expanded Access criteria. The patient is not eligible for Expanded Access.