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:

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Expanded Access for Drugs/Biologics:

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

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Expanded Access for Devices:

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

*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.

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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.

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See Also: