Expanded Access Use of Investigational Products
Sometimes called "compassionate use", expanded access is a potential pathway for a patient with an immediately life-threatening or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy options are available.
Expanded access may be appropriate when all of the following apply:
- The patient to be treated has a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor or treat the disease or condition;
- The potential benefit to the patient justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated; and
- Providing the investigational medical 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.
An "investigational" drug, biologic, or medical device is one that the FDA has not yet approved or cleared for sale or has not deemed it safe and effective for its specific use. Furthermore, the investigational medical product may or may not be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.
In all cases of expanded access, the product manufacturer must agree to provide the product for the specific expanded access use.
Non-emergency expanded access submissions to the FDA are appropriate when:
- The patient has a serious or immediately life-threatening disease and/or condition;
- No generally acceptable alternative treatment for the condition exists;
- There is enough time to wait for prospective IRB approval or Chair concurrence*
- There is enough time to wait the full 30-days for traditional FDA approval.
*Box 10b must be checked off on the Form FDA 3926 (drugs only) in order to allow for alternative IRB procedures in lieu of traditional IRB Board approval.
Expanded access requirements for drugs and biologics follow 21 CFR 312 and follow the requirements of Investigational New Drug (IND) applications.
Expanded access requirements for medical devices are different from Investigational Device Exemption (IDE) requirements and may occur before an IDE is approved and/or when a device is not being studied under an IDE.
The following criteria must be met for emergency use of an investigational product:
- The patient has a life-threatening or serious disease or condition that requires immediate treatment;
- No generally acceptable alternative treatment for the condition exists;
- There is not sufficient time to obtain prospective IRB approval;
- There is not sufficient time to wait the full 30-days for FDA's approval timeline of non-emergency requests.
Emergency use requests also require concurrence from a non-treating physician and other requirements.