Emergency Use of Investigational Drugs

Physicians may need to submit a request the use of an investigational agent for the treatment of a patient in an emergency situation when there is not sufficient time to obtain IRB approval. This guidance outlines the necessary steps to use these investigational drugs and the requirements after treatment has started. Please note that Emergency use situations are limited to life-threatening or severely debilitating situations as outlined in 21CFR56.102(d):

• "Life-threatening" means 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.
• "Severely debilitating" means 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

The basic steps for obtaining IRB approval for an emergency use of an investigational drug or biologic are as follows:

1. Confirm the willingness of the holder of the drug/biologic to provide the product.
2. Obtain an IND number or other permission from FDA
3. Consult with the KUMC Investigational Pharmacy about any requirements related to the shipment or receipt of the drug.
4. Obtain written acknowledgment from the KUMC Institutional Review Board (IRB) prior to the use. Acknowledgment is contingent on submission of the following documents:
   • Submit a letter to the IRB chair, discussing the six justifications for the emergency use, as outlined in the guidance document.
   • Submit information about the source, nature, and use of the drug (drug label or Investigational Brochure)
   • Submit an informed consent document to be signed by the patient or legally authorized representative.
   • Within 5 days after the emergency use, file a formal notification to the IRB.
   • Adhere to the federal rules on use of the data and subsequent emergency uses

These documents should be submitted to:

• IRB office
• HRPP Director: Kyle Stephens (913) 588-0942

Guidance and additional forms:

• Emergency Use of an Investigational Drug or Biologic
• Emergency Use Notification Form