27-29 February 2024
The FDA published a draft guidance for institutional review boards (IRBs) and clinical investigators regarding the key factors and procedures IRBs should consider when reviewing individual patient expanded access submissions (IPEAS), including reviews conducted by a single member of the IRB.
Expanded access refers to the use of an investigational medicinal product (IMP) when the primary purpose is to diagnose, monitor, or treat a patient’s disease or condition rather than to obtain the kind of information about the drug that is generally derived from clinical trials (see also FDA's draft guidance on Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers). This pathway is sometimes called "compassionate use" and allows a patient with a serious or immediately life-threatening disease or condition to gain access to an IMP when there is no comparable or satisfactory alternative therapy. In this case, the potential patient benefit justifies the potential risks to the patient. An individual patient expanded access request can be submitted to FDA by a licensed physician as a new investigational new drug application (IND) or by a sponsor of an existing IND as a protocol amendment, either on an emergency (e.g., a situation that requires a patient to be treated before a written submission to the IRB can be made) or non-emergency use basis. For non-emergency IPEAS, prior IRB review and approval is required before the treatment begins.
FDA recommends that IRBs:
For more information please see FDA's guidance Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products.