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GUIDANCE DOCUMENT

Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers November 2022

Download the Draft Guidance Document Read the Federal Register Notice
Draft

Not for implementation. Contains non-binding recommendations.

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

This guidance provides information for industry, researchers, physicians, institutional review boards (IRBs), and patients about the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND) (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) (the 2017 guidance), 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 (Cures Act) and the FDA Reauthorization Act of 2017 (FDARA). When finalized, this guidance will replace the 2017 guidance. Significant changes from the 2017 version include additional recommendations related to IRB review, informed consent, and new requirements established by the Cures Act and FDARA related to sponsors making their policies for evaluating and responding to expanded access requests (i.e., expanded access policy) public and readily available).

In a separate guidance, FDA provides answers to questions concerning the implementation of the regulation on charging for investigational drugs under an IND (21 CFR 312.8). Also, in a separate guidance, FDA describes Form FDA 3926 (Individual Patient Expanded Access—Investigational New Drug Application (IND) and the process for submitting expanded access requests for individual patient INDs.

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

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