Emergency, Expanded Access for an Investigational Medical Product (One Patient)

Version Date: March 2020

Under FDA regulations, an investigational medical product may be used to treat a patient 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 only emergency uses of an investigational medical product in one patient under the expanded access regulations. If you have any questions about this guidance or a potential emergency use, please contact emergencyuse@hsirb.wisc.edu.

For expanded access for a single patient or group of patients in a non-emergency setting (I.e. there is time to obtain prospective IRB approval), see Expanded Access of Investigational Medical Product Requests (Non-emergency).


What Is Expanded Access

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

The treating clinician should confirm that all the above criteria have been met before proceeding with an emergency use request.

Back to top


Criteria for Emergency Use

To qualify as an emergency under FDA regulations, the proposed use must meet the following criteria, which differ for drugs/biologics and devices.

For a drug or biologic:

For a device:

The FDA defines life-threatening as diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted, and diseases or conditions with potentially fatal outcomes where the end point of clinical trial analysis is survival. The criteria for life-threatening do not require the condition to be immediately life-threatening or to immediately result in death. Rather, the subjects must be in a life-threatening situation requiring intervention before review at a convened meeting of the IRB is feasible.

The FDA defines severely debilitating as diseases or conditions that cause major irreversible morbidity. Examples of severely debilitating conditions include blindness; loss of arm, leg, hand or foot; loss of hearing; paralysis or stroke.

Back to top


Contacting 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. The manufacturer may provide the investigational medical product for use under its own IND or IDE.

If no IND for use of a drug or biologic exists, the clinician may need to obtain their own IND; however, the FDA may authorize shipment of a drug or biologic in advance of an IND submission in an emergency. For devices, the FDA does not require the clinician to obtain an IDE in emergency use situations if one does not already exist. See “Reporting to the FDA” below for contact information for the FDA.

Back to top


Contacting the Pharmaceutical Research Center (PRC)

For emergency use of a drug or biologic only, the clinician must contact the PRC for assistance (608.263.8902).

Back to top


Initial Notification to the FDA

To contact the FDA for approval of or guidance regarding a potential emergency use, please see the contact information found at the bottom of this page: https://www.fda.gov/news-events/public-health-focus/expanded-access.

Back to top


Initial Notification to the IRB

For use of investigational medical products, clinicians should notify the IRB of the proposed use prior to the actual use or, if not feasible, within five days of the use. Please notify the IRB as follows:

Back to top


IRB Chair Concurrence Process

Back to top


Obtaining Informed Consent

For all emergency uses, informed consent must be obtained from the patient or their legally authorized representative prior to treatment unless the criteria in 21 CFR 50.23(a) are met.

Back to top


Reporting to the IRB

If not completed before use, a treatment use application must be submitted in ARROW within 5 days of the emergency use. This application will be reviewed by the convened IRB to determine whether the convened board agreed with the IRB Chair’s assessment or additional information should be provided to the patient (or their representatives).

Back to top


Reporting to the FDA

Additional reporting to the FDA may be required following the emergency use of the test. Please contact the FDA for guidance on what additional reporting may be required.

Back to top


Resources

Back to top

See Also: