Expanded Access of Investigational Medical Product Requests (Non-emergency)

Version Date: March 2020

Under FDA regulations, an investigational medical product may be used to treat a single patient or a group of patients with an immediately life-threatening condition or serious disease or condition outside a clinical trial when no comparable or satisfactory alternative therapy options are available. The is called expanded access or compassionate use. This document addresses the use of an investigational medical product in one patient or a group of patients under the expanded access regulations when the use is not an emergency (i.e. there is enough time to obtain prospective IRB approval for the use). If you have any questions about this guidance or a potential emergency use, please contact emergencyuse@hsirb.wisc.edu.

For emergency expanded access requests, see Emergency, Expanded Access for an Investigational Medical Product (One Patient).


When is an expanded access request appropriate?

FDA guidance states that using an investigational medical product under expanded access regulations (including emergency uses) may be appropriate when all the following apply:

  • Patient has a serious disease or condition, or whose life is immediately threatened by their 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.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.


Expanded Access for Drugs/Biologics:

There are three different types of expanded access for drugs/biologics:

  • Expanded access for individual patients, including for emergency use*
  • Expanded access for intermediate-size patient groups

    • For use by more than one patient, but generally fewer patients than are treated under a typical treatment IND or protocol
  • Expanded access for widespread treatment use

    • Access to an investigational drug (including a biologic) for treatment use by a large (widespread) population. The investigational product must be under active development for marketing.


Expanded Access for Devices:

There are also three different mechanisms for expanded access of an unapproved device:

  • Emergency Use* for an individual patient.
  • Compassionate Use (or Individual Patient/Small Group Access)

    • Used for 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 investigation but for whom the treating physician believes the device may provide a benefit in treating or diagnosing their disease or condition. It can also be used for devices that are not being studied in a clinical investigation. This provision is typically approved for individual patients but may be approved to treat a small group, if the small group request is under an IDE.
  • Treatment Investigational Device Exemption (IDE)

    • An approved IDE specifies the maximum number of clinical sites and the maximum number of human subjects that may be enrolled in the study. During the course of the clinical trial, if the data suggest that the device is effective, then the trial may be expanded under a new IDE to include additional patients with life-threatening or serious diseases. This is called a treatment IDE.

*If there is not time to obtain IRB approval for the treatment use of an investigational product (drug/biologic, or device), this constitutes an emergency use, and treating teams should follow this guidance: Emergency, Expanded Access for an Investigational Medical Product (One Patient).

The following steps should be completed to obtain approval for Expanded Access requests:

  1. Contact the Manufacturer

    For all types of investigational medical products, the FDA requires that the clinician contact the manufacturer to see if they will provide the investigational medical product for expanded access use. Investigational medical products may be provided through a supplement to a manufacturer’s existing IND or IDE, or through an IND or IDE specific to the use (e.g., under a Treatment IND or Treatment IDE).

  2. Submit Request to the FDA

    For drugs, biologics OR devices, clinicians MUST contact the FDA prior to use of the investigational product.

  3. Obtain IRB approval and informed consent from the patient(s)

    Once the appropriate expanded access route has been identified and the FDA and drug/biologic/device manufacturer contacted, treating teams will need to submit an application through ARROW for prospective IRB approval, just as they would for research studies. This application will be reviewed by the convened IRB and approval granted before the use can proceed.


Reporting to the FDA

Additional reporting to the FDA may be required in the case of individual and patient group expanded access uses. Please contact the FDA for guidance on what additional reporting may be required.


Resources

See Also:




Keywords:FDA, drug, biologic, device   Doc ID:98966
Owner:Monica E.Group:Health Sciences IRBs
Created:2020-03-17 11:09 CDTUpdated:2020-03-18 05:58 CDT
Sites:Health Sciences IRBs
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