Investigational Drugs: Expanded Access
FDA uses the term "Expanded Access" for the use of investigational drugs for treatment of patients who are not enrolled as subjects in studies under the IND that covers the drug being studied. Expanded access, sometimes called "compassionate use", permits the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition for whom there is no comparable or satisfactory alternative treatment options.
FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.
For specific information on procedures at Emory see:
EMERGENCY USE: When a patient needs to be treated with an investigational drug before a written IND submission can be completed, the PI must obtain authorization for individual use from FDA by telephone or electronic communication with subsequent submission of IND paperwork and IRB notification. FDA provides a Physician Fact Sheet which covers the steps required and a link to contact information. In this situation, Emory physicians should reference the steps outlined in IRB P&P 70 Investigational New Drugs.
NON-EMERGENCY USE: In this situation, a written Expanded Access IND must be submitted for review and approval by the FDA prior to use of an investigational drug. The protocol must also be approved by the Emory Institutional Review Board (IRB) prior to treating a patient.
- The Checklist for Individual Patient Expanded Access non-Emergency IND Submission provides a list of items needed and includes form links for your use in preparing an Expanded Access IND submission.
A licensed practitioner who holds the expanded access IND and receives an investigational new drug for treatment use under an expanded access IND is a "sponsor-investigator" and is responsible for meeting all applicable sponsor responsibilities as well as investigator responsibilities. FDA reporting requirements include submission of IND safety reports and annual reports. Expanded Access regulations for drugs are contained in Title 21, Code of Federal Regulations, Part 312.300.
For additional information see:
- FDA Draft Guidance: Expanded Access to Investigational Drugs for Treatment Use - Q and A
- Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use
- IRB P & P 70 Investigational New Drugs