Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers; Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers." This guidance finalizes the revised draft guidance issued on November 2, 2022, and replaces the final guidance of the same title issued in June 2016 and updated in October 2017. The guidance addresses frequently asked questions, including those related to the implementation of FDA's regulations on expanded access to investigational drugs and other topics related to expanded access that are promulgated through the 21st Century Cures Act (Cures Act) and the FDA Reauthorization Act of 2017 (FDARA). Upon publication of this guidance, FDA is withdrawing the 1998 information sheet guidance, entitled "Emergency Use of an Investigational Drug or Biologic," for institutional review boards and clinical investigators because the information provided in the 1998 guidance is included in this final guidance.

Abstract as published in the Federal Register.

The complete document — every provision, the docket, and the comment instructions — is published in the Federal Register. SPEQ surfaces and indexes it for reference; the authoritative text lives on federalregister.gov.