Expanded Access Q&A Draft Guidance Includes Informed Consent, IRB Review, and Public Access to Policy

11/15/2022

The U.S. Food and Drug Administration (FDA) released a draft guidance “Expanded Access to Investigational Drugs for Treatment Use Questions and Answers” in November 2022. The regulation governing expanded access went into effect in October 2009, and due to a large volume of questions on how to comply with the regulations, released a guidance documents in June 2016 which was updated in October 2017. After the 2017 guidance, the Agency continued to receive additional questions and changes occurred to the regulation relating to the FDA Reauthorization Act of 2017 (FDARA) and the 21st Century Cures Act (Cures Act). When finalized, this guidance will replace the October 2017 guidance.

Expanded access for treatment use, also known as compassionate use, is a treatment option for patients who have an “immediately life threatening condition or serious disease” when there is no viable treatment option. The investigational product is provided outside the clinical trial as treatment although it has not yet been found to be safe or efficacious. The sponsor must agree to provide the investigational drug for expanded access and may charge for the investigational drug under certain circumstances. Read our related blog “Charging for Investigational Drugs Draft Guidance” for more information.

There are three categories of expanded access:

  • Individual patients, including for emergency use (21 CFR 312.310)

  • Intermediate-size patient populations (21 CFR 312.315)

  • Widespread treatment use through a treatment IND or treatment protocol (21 CFR 71 312.320)

The Cures Act added the requirement for sponsors who are providing their investigational product for expanded access to include additional information in their expanded access policy and to make the policy openly available to the public. The expanded access policy should include how sponsors evaluate and respond to expanded access requests, their contact information, and a link to their ClinicalTrials.gov listing.

Included are 38 questions with answers and an appendix with an informed consent for expanded access template. New questions cover topics related to the changes for informed consent, Institutional Review Board (IRB) review, and the requirements for the publicly available policy under the Cures Act previously mentioned. Other topics with new questions included safeguards for expanded use and emergency and non-emergency individual patient expanded access.

The FDA maintains it is appropriate to obtain informed consent for expanded access since the investigational drug “involves research” and considers the physician administering it an investigator. This also means the sponsor must ensure the physician is qualified and provide to them information to guide safe administration, and that IRB review, IND safety reporting, and maintaining and retaining source and drug disposition records are required. A Q&A on emergency and nonemergency use clarifies that a waiver of IRB review is permissible for individual expanded access, but not for either intermediate or widespread use.

You may be interested in a related guidance “Individual Patient Expanded Access Applications: Form FDA 3926” from June 2016, which was updated in June 2017.

Comment now through January 03, 2023 on the draft guidance HERE.

 

- The Clinical Pathways Team

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