Expanded Access Use of Investigational Medical Devices
The use of an investigational device (one that has not been approved or cleared by the FDA) outside of a clinical trial for treatment of a patient is also called expanded access. Normally, significant risk (SR) devices may only be used in human subjects through a FDA-approved clinical trial conducted under an investigational device exemption (IDE). However, when a patient has a serious or life-threatening condition that is not addressed by current approved treatments and enrollment in an existing clinical trial protocol is not possible, patients and physicians have the potential to receive expanded access to investigational devices under one of three mechanisms:
- Emergency Use
- Individual-Patient/Small Group (commonly referred to as compassionate use)
- Treatment Use
The following is a quick guide to the criteria and requirements for expanded access use of medical devices:
Criteria and Requirements for Expanded Access Submissions of Medical Devices
- Criteria
- Individual (Single Patient) and Small Group
- The device is intended to treat or diagnose a serious disease or condition; and
- There is no comparable or satisfactory alternative device or therapy available; and
- The medical device manufacturer agrees to provide the device for compassionate use.
- Treatment
- 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.
- FDA Approval
- Individual (Single Patient) and Small Group
- Required prior to use
- Treatment
- Required prior to use, which may begin 30 days after the FDA receives a treatment IDE submission or earlier if the FDA provides notification that the expanded access use may begin.
- FDA Submission (contact CTO IAP)
- Individual (Single Patient) and Small Group
- If an IDE already exists for the device, the IDE sponsor should submit an IDE supplement requesting approval to treat the patient.
- If no IDE exists for the device, the physician submits a request to the FDA, along with a description of the device provided by the manufacturer. Contact [email protected] for assistance.
- Treatment
- Treatment Use IDE is submitted by the applicant (i.e., IDE sponsor). The sponsor of a treatment IDE must submit progress reports on a semi-annual basis to all reviewing IRBs and the FDA until the filing of marketing application, as well as other reports required under 21 CFR 812.150.
- Contractual Requirements (contact CTO Contracts)
- Individual (Single Patient) and Small Group
- CDA/NDA
- Agreement (may not be needed)
- Review of ICF (confidentiality, subject injury and compensation)
- Treatment
- CDA/NDA
- Agreement
- Review of ICF (confidentiality, subject injury and compensation)
- IRB Review
- Individual (Single Patient) and Small Group
- Required prior to use. In some cases, convened IRB review may be required.
- Treatment
- Required prior to use. The FDA will issue a treatment use IDE to the applicant (i.e., IDE sponsor).
- IRB Submission
- Individual (Single Patient) and Small Group
- The physician should include the following in the Rascal IRB submission:
- A description of the patient's or patients’ condition and the circumstances necessitating treatment;
- a discussion of why alternative therapies are unsatisfactory and why the probable risk of using the investigational device is no greater than the probable risk from the disease or condition;
- identification of any deviations in the approved clinical protocol that may be needed in order to treat the patient(s);
- a description of the patient protection measures that will be followed, including a copy of the consent document that will be used;
- a monitoring schedule; and
- an independent assessment from an uninvolved physician.
- Treatment
- The physician must submit for convened IRB review.
Updated October 2023