For Medical Devices: Small Group Access and Treatment Use IDEs

Medical devices are regulated differently from drugs and biologics. However, as with drugs and biologics, FDA offers pathways for expanded access to investigational medical devices by allowing physicians and product manufacturers to apply for investigational device exemptions (IDEs). (Note: For devices, FDA refers to expanded access by the term “compassionate use.”)

Compassionate use can be requested for medical devices that are being studied in a clinical trial under an IDE for patients who do not meet the requirements for inclusion in the clinical trial, but for whom the treating physician believes the device may provide a benefit in treating and/or diagnosing their disease or condition. Compassionate use can also be requested for devices that are not being studied in a clinical investigation (i.e., an IDE for the device does not exist).

FDA cannot require a company to provide an investigational device for compassionate use. If the device manufacturer agrees to provide the device under compassionate use, there are two different processes to follow to obtain FDA approval, depending on whether there is an existing IDE for a clinical trial for that device.

Although compassionate use is typically approved for individual patients, it may be approved to treat multiple patients. More information on how to request individual (single) patient access to an investigational device is available under Resources for Expanded Access. Information on compassionate use for small groups of patients and for treatment uses is provided below.

Small Group Access

 If there is an IDE for the medical device:

The sponsor of the IDE (usually the device manufacturer or a physician who has submitted the IDE to conduct the clinical study for the device) should submit an IDE supplement requesting approval for a compassionate use under 21 CFR 812.35(a) in order to treat the patients. The IDE supplement should include:

  • A description of the patients’ conditions and the circumstances necessitating treatment.
  • The number of patients to be treated.
  • 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.
  • An identification of any deviations in the approved clinical protocol that may be needed in order to treat the patients.
  • The patient protection measures that will be followed:
    • A draft of the informed consent document that will be used.
    • Clearance from the institution as specified by their policies.
    • Concurrence of the IRB chairperson. (Full board review is not required.)
    • An independent assessment from an uninvolved physician.
    • Authorization from the device manufacturer for use of the device.

If there is no IDE for the medical device:

The physician or manufacturer should submit the above information to FDA, along with a description of the device provided by the manufacturer. Physicians and manufacturers should contact for assistance.

The review process

 After a compassionate use request is received, FDA will approve, approve with conditions, or disapprove the request. When there is an IDE for the device, compassionate use request IDE supplements have the same statutory 30-day waiting period as other IDE submissions. However, the patient need is considered when reviewing these requests, and they are often expedited when necessary. IDE requests can be reviewed in as little as 1 day; on average decisions are made in 18 days. Compassionate use requests received without an IDE are sometimes reviewed on the same day as receipt; average review time is 10 days.

FDA considers the above information and whether the preliminary evidence of safety and effectiveness justifies such use and whether such use would interfere with the conduct of a clinical trial to support marketing approval.

The physician should not treat the patients identified in the request until FDA approves use of the device under the proposed circumstances.

Treatment IDE 

The treatment IDE is used during the clinical trial(s) or after completion of the trial(s), but prior to final action on the marketing application, when it may be appropriate to use the device in the treatment of patients not in the trial(s). An approved IDE specifies the maximum number of clinical sites and the maximum number of patients that may be enrolled in the clinical trial. During the course of the trial, if the data suggest that the device is effective, then the trial may be expanded to include additional patients with life-threatening or serious diseases. This is called treatment use. The purpose of this type of use is to facilitate the availability of promising new devices to desperately ill patients as early in the device development process as possible, before general marketing begins, and to obtain additional data on the device’s safety and effectiveness.

In the case of a serious disease—defined as a disease or condition associated with morbidity that has substantial impact on day-to-day functioning—or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one, a device ordinarily may be made available for treatment use under this section after all clinical trials have been completed.

In the case of an immediately life-threatening disease—defined as a stage of a disease in which there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment—a device may be made available for treatment use under this section prior to the completion of all clinical trials. “Treatment use” of a device includes the use of a device for diagnostic purposes.

Criteria for treatment use include:

  • 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 available to treat or diagnose the disease or condition in the intended patient or patient population;
  • The device is under investigation in a controlled clinical trial for the same use under an approved IDE, or all clinical trials have been completed; and
  • The sponsor of the controlled clinical trial is pursuing marketing approval/clearance of the investigational device with due diligence.

FDA regulations describing the requirements for treatment use of an investigational device can be found under 21 CFR 812.36.

Sponsor-Investigator Responsibilities 

A physician who receives an investigational device for treatment use under a Treatment IDE is considered an “investigator” under the IDE and is responsible for meeting all applicable investigator responsibilities, including:

  • Applying to and obtaining approval from FDA and an IRB prior to administering the investigational device.
  • Seeking IRB approval for continuing review if the treatment use extends longer than one year or a second course of treatment is needed.
  • Obtaining and documenting appropriate informed consent from the patient or legally authorized representative prior to treatment. More information about the required content of the informed consent documents is found at 21 CFR 50.
  • Maintaining accurate case history records and observations related to provision of the device, including adverse events.
  • Reporting serious and unexpected adverse reactions that are believed to be related to the use of the investigational device, as required by FDA.
  • Maintaining accurate documentation of the disposition of the investigational device, including date and use.
  • Adhering to reporting obligations of the IRB, FDA, and product manufacturer.
  • Maintaining confidentiality of the information both about the patient and the condition.
  • Complying with applicable local laws and institutional policies.

In addition, the sponsor of a treatment IDE is required to submit progress reports on a semi-annual basis (based on the time since initial approval of the treatment IDE) to all reviewing IRBs and FDA until the filing of a marketing application. These reports must include:

  • The number of patients treated with the device under the treatment IDE.
  • The names of the investigators participating in the treatment IDE.
  • A brief description of the sponsor’s efforts to pursue marketing approval/clearance of the device (e.g., clinical trial progress).

After all compassionate use patients have been treated, a follow-up report should be submitted by whoever submitted the original compassionate use request to FDA. The report should present summary information regarding patient outcome. If any problems occurred as a result of device use, these should be discussed in the follow-up report and reported to the reviewing IRB as soon as possible.

Additional Resources

From Clinical Research Pathways

FDA Expanded Access