Introduction
The U.S. Food and Drug Administration (FDA) is critical in ensuring patient access to investigational drugs and biological products through expanded access programs. Let’s explore the recently released FDA guidance titled “Institutional Review Board (IRB) Review of Individual Patient Expanded Access Submissions for Investigational Drugs and Biological Products.” This guidance clarifies to Institutional Review Boards (IRBs) and clinical investigators the review process for individual patient expanded access submissions, mainly when conducted by a single IRB member.
The Need for Clarity
Although the FDA previously issued guidance on expanded access requests, including those for individual patients, it acknowledges the ongoing need for greater clarity in this area. Expanded access is categorized into three types: individual patients, intermediate-size patient populations, and treatment for larger populations. This guidance exclusively addresses IRB review of individual patient expanded access submissions, as outlined in 21 CFR 312.310. The goal is to streamline and enhance the efficiency of these reviews.
COVID-19 Context
In June 2020, the FDA introduced the “Institutional Review Board (IRB) Review of Individual Patient Expanded Access Requests for Investigational Drugs and Biological Products During the COVID–19 Public Health Emergency” guidance. This guidance addresses the COVID-19 public health emergency and will serve as a valuable resource beyond that context. As of March 13, 2023, the FDA listed the 2020 COVID–19 guidance as one of the documents to be revised and continued for 180 days after the COVID-19 public health emergency declaration expired on May 11, 2023.
Revised Guidance
In light of the ongoing need for clear guidance in individual patient expanded access submissions and to remove references to the COVID-19 public health emergency, the FDA has issued a revised final guidance. This new guidance supersedes the 2020 COVID–19 guidance. It incorporates lessons from the pandemic, addresses relevant recommendations, and clarifies IRB procedures and factors to consider.
Immediate Implementation
The FDA has chosen to implement this guidance immediately without seeking prior public comment due to the unpredictable nature of public health emergencies. They recognize that situations may arise without notice, hindering physicians’ ability to treat patients promptly. This implementation underscores the importance of providing clear recommendations for IRBs in such cases. While immediate implementation is in place, the guidance remains subject to comment, adhering to the FDA’s good guidance practices regulation.
Conclusion
The FDA’s latest guidance on the IRB review of individual patient expanded access submissions for investigational drugs and biological products represents a significant step toward ensuring efficient and consistent review processes. This guidance is a testament to the FDA’s commitment to facilitating patient access to investigational treatments while maintaining the highest standard of safety and ethics.
IRBs, clinical investigators, and healthcare professionals should stay informed about these regulatory updates to effectively navigate the complex landscape of expanded access programs. As the FDA continues to evolve its guidance to meet the needs of patients and healthcare providers, the academic community should remain engaged and provide valuable feedback to support these critical initiatives.
