Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers October 2025

GUIDANCE DOCUMENT

• Docket Number: FDA-2013-D-0446
• Issued by: Center for Drug Evaluation and Research

FDA is posting this document to provide advance notice to the public. After the lapse in appropriations ends, a notice of availability for the guidance will be published in the Federal Register, which will detail how to submit comments on this document.

This guidance provides information for industry, researchers, physicians, institutional review boards, and patients about the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (21 CFR part 312, subpart I), which went into effect on October 13, 2009. FDA received numerous questions concerning implementation of the regulatory requirements for expanded access. As a result, FDA issued the guidance for industry Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers (June 2016, updated October 2017), providing recommendations in a question-and-answer format, addressing the most frequently asked questions. Since 2017, FDA has received additional questions concerning implementation of the regulatory and statutory requirements of expanded access to investigational drugs, including those added by the 21st Century Cures Act and the FDA Reauthorization Act of 2017.

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All written comments should be identified with this document's docket number: FDA-2013-D-0446.