The Food and Drug Administration's Expanded Access Program allows for medical devices that are investigational (i.e., not FDA approved) to be used for treatment use outside of a clinical trial. Sometimes this is referred to as compassionate use. Such use is not considered a clinical investigation, however, FDA submission and IRB review are necessary. The criteria for Expanded Access are largely determined by (1) the seriousness of a patient's condition, (2) other available FDA-approved treatment options, (3) patient population size needing treatment, and (4) device status.
This webpage is a summarized guide to the FDA regulations on Expanded Access to Investigational Devices for Treatment Use, found in FDA guidance on Expanded Access for Medical Devices. It does not provide full guidance for Expanded Access to Investigational Devices; as such, FDA regulatory materials must also be consulted for full information.
The following mechanisms may be employed to use unapproved medical devices:
Emergency Use of an Unapproved Device
The FDA defines "emergency use" as the use of a test article on a human subject in a life-threatening situation in which no stan dare acceptable treatment is available, and in which there is not sufficient time to obtain IRB approval [21 CFR 56.102 (d)] [PDF].
For more information about emergency use and for instructions on submitting an emergency use application, please see the IRB guidance, Emergency Use of a Test Article.
Compassionate Use of an Unapproved Device
The FDA defines "compassionate use" as the use of a test article on a human subject with a serious disease or condition for which there is no acceptable treatment available. FDA and IRB approval for compassionate use is required. The IRB will not approve an application for compassionate use until it has been approved by the FDA.
Compassionate use may only be employed during the clinical trial for which the device is being tested. Compassionate use approval may be given for an individual patient or a small group of patients.
Treatment Investigational Device Exemption
A device that is not approved for marketing may be under clinical investigation for a serious or immediately life-threatening disease or condition in patients for whom no comparable or satisfactory alternative device or other therapy is available. During the clinical trial or prior to final action on the marketing application, it may be appropriate to use the device in the treatment of patients not in the trial under the provisions of a treatment investigations device exemption [21 CFR 812.36].
The FDA will consider approval of an investigational device under a treatment of IDE if the following criteria are met:
-
- The device is intended to treat or diagnose a serious or immediately life-threatening disease or condition;
- There is no comparable or satisfactory alternative device or other therapy available to treat or diagnose that stage of the disease or condition in the intended patient population;
- The device is under investigation in a controlled clinical trial for the same use under an approved IDE, or such clinical trials have been completed; and
- The sponsor of the investigation is actively pursuing marketing approval/clearance of the investigational device with due diligence.
IRB approval of a treatment use IDE is also required before use may begin. The IRB will not approve an application for a treatment IDE until it has been approved by the FDA.
Continued Access to Investigational Devices
The FDA will consider allowing an investigational device may be made available during the preparation or review of a marketing application if the following criteria are met;
-
- There is a public health need for the device, and
- There is preliminary evidence that the device is likely to be effective and not significant safety concerns have been identified for the proposed indication.
For additional information regarding continued access, please see the FDA guidance, Continued Access to Investigational Devices During PMA Preparation and Review."
IRB approval of continued access to an investigational device is also required before use may begin. The IRB will not approve an application for continued access until it has been approved by the FDA.