The U.S. Food and Drug Administration (FDA) recently issued a revised version of the draft guidance “Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers,” which includes recommendations for fulfilling new statutory requirements related to expanded access, and which if finalized would update the October 2017 guidance of the same name. As summarized below, the updated guidance includes additional insights into expanded access requirements and best practices and incorporates new requirements established by the Cures Act and FDA Reauthorization Act of 2017 related to sponsors making their expanded access policies public and readily available.

FDA is seeking comments on the revised draft guidance through January 3.

Since issuing the “Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers” final guidance in June 2016 (revised in October 2017), FDA said it has received questions related to the implementation of the regulatory requirements of the expanded access program. As compared to the 2017 guidance, in the updated draft guidance the agency:

  • Further emphasizes sponsor’s obligation to post an expanded access policy on its website and expanded access records on ClinicalTrials.gov;

  • Confirms that posting an expanded access policy will not be viewed as pre-approval promotion, so long as the policy is not promotional in nature (i.e., does not make claims that the product is safe or effective for an unapproved use);

  • Outlines key obligations for both sponsors and investigators, including:

    • Investigators’ obligations to report adverse events to the sponsor, obtain required IRB review and approval, obtain informed consent, and maintain accurate case histories and drug disposition records.

    • Sponsors’ obligations to comply with expedited IND safety reporting requirements, submit to FDA annual reports (when the IND or protocol continues for 1 year or longer), ensure that investigators are qualified to administer the investigational drug for the expanded access use and are provided with all necessary information, maintain an effective IND for the expanded access use, and maintain adequate drug disposition records.

  • Provides an extremely helpful template informed consent form for expanded access use; and

  • Advises that expanded access use has a higher probability of interfering with clinical trials and drug development for rare disease programs.

The publication of this guidance comes on the heels of FDA’s August 2022 revised draft guidance on charging for investigational drugs under an IND, which we summarized online here, and it further demonstrates FDA’s increasing role in regulating these programs. We are seeing FDA putting pressure on companies to have clear expanded access policies in place to enhance compliance with the Cures Act, as well as an increased number of expanded access requests to pharmaceutical and biotechnology companies.

Comments on the draft guidance are due by January 3, 2023.