12-14 December 2023
The FDA published a revised draft guidance on frequently asked questions about the agency´s regulation of expanded access to investigational medicinal products (IMPs). The agency is providing the revised guidance in a question-and-answer format, to address the most recently asked questions and sharing recommendations to fulfill new statutory requirements. Amongst others, the document contains Q&As related to expanded access submission, individual (or single) patient expanded access, and general questions. Moreover, the guidance provides an appendix with an informed consent template for individual patient expanded access.
The terms expanded access and treatment use are used interchangeably to refer to the use of an 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 (i.e., safety / effectiveness). The terms compassionate use and preapproval access are also occasionally used in the context of the use of IMPs to treat a patient. However, although the terms compassionate use and preapproval access have been used informally in the U.S. and are also used outside the U.S., they are not defined or described in FDA regulations. This has led to some confusion or lack of clarity about the meaning of the terms. For this reason, the terms compassionate use and preapproval access will not be used in the FDA document.
When finalized, the revised guidance "Expanded Access to Investigational Drugs for Treatment Use - Questions and Answers" will replace the June 2016 (updated in October 2017) final guidance. In a separate guidance, the FDA provides Q&As concerning the implementation of the regulation on charging for IMPs.