P314: Single-Patient Expanded Access Programs: Data and Best Practices from a Central IRB
Poster Presenter
Sharad Adekar
Senior Medical Chair
WCG United States
Objectives
Single patient Expanded Access Programs (EA) can pose unique challenges and responsibilities to healthcare professionals and Institutional Review Boards. IRBs needs to review EA requests in line with FDA guidance. Here we outline our experience and best practices based on real-word experience at IRB.
Method
EA requests from Jan-Dec 2025 received at a central IRB were reviewed. Documents like FDA Form 3926, IND approval, protocol, investigator brochure, consent form, and IRB submission forms were analyzed, and requests were categorized by therapeutic area utilizing Excel-based algorithms.
Results
In 2025, our IRB received 259 Single Patient Expanded Access requests. As authorized by FDA, these requests received an IRB Chairperson concurrence in lieu of review and approval at a convened IRB meeting. Of these EA requests, 173 (67%) were for drugs/biologics and 86 (33%) for medical devices. Of the 173 drug/biologic requests, 129 (75%) were related to oncology. Twenty-one (12%) focused on infectious diseases, including eight (38%) for antibiotic to treat mycobacteria infections—a drug not marketed in the US and available only through expanded access. Six requests (3%) addressed neurologic conditions, and seventeen (10%) involved other therapeutic areas. Additionally, we observed that among the 129 oncology-related requests, 83 (64%) involved gene therapy products: 59 (71%) used genetically engineered Natural Killer cells with chimeric antigen receptor (CAR-Natural Killer cells) with Program Death-Ligand1, and 16 (19%) utilized adeno-virus-based immunotherapy. The remaining 46 (36%) requests were for non-gene therapy products, with 25 (54%) focusing on small molecule kinase inhibitors and 10 (22%) on human monoclonal antibodies. Health care professionals and staff were guided through the expanded access submission process, including requiring FDA Form 3926, investigator brochures, fulfilling IND requirements, preparing consent forms, and completing IRB documentation. These findings illustrate both the complexity of range of indications of EA submissions and the crucial role played by IRBs standard practices in facilitating safe and ethical access to investigational therapies for patients with serious conditions.
Conclusion
Our 2025 annual review of Single Patient Expanded Access requests highlights the diverse therapeutic needs of patients and the evolving landscape of investigational treatments requests for EA. With most requests centered on oncology, and a significant portion involving advanced therapies like gene and immunotherapy products, patients with serious conditions are seeking increasingly complex interventions through EA. Notably, there were also requests for EA in situations where off-label drug use such as antibiotics, which was only accessible via expanded access programs. The active engagement of healthcare professionals and Institutional Review Boards (IRBs) is critical throughout the process, from regulatory documentation to rigorous ethical oversight, ensuring both patient safety and compliance. These findings reinforce the importance of rigorous review processes and collaborative practices that adapt to new trends, such as emerging therapies. By maintaining high standards and fostering clear communication, IRBs and clinical teams play a pivotal role in facilitating responsible access to potentially life-saving treatments, while safeguarding patient well-being and trusting in expanded access programs. As a best practice, we recommend a. Ensuring all required forms (FDA Form 3926, IND documentation, consent forms, investigator brochures) are accurately prepared and submitted to IRBs, b. Provide training and step-by-step support to healthcare professionals navigating the expanded access process, c. Adhere strictly to FDA guidance and all relevant regulations, updating processes as guidance evolves. By implementing these best practices, healthcare professionals and IRBs can ensure responsible, ethical, and efficient review of single patient expanded access submissions, supporting patient access to innovative therapies while safeguarding well-being and public trust.
