International Society for Cell & Gene Therapy Expanded Access Working Group position paper: key considerations to...

This position paper reviews the Expanded Access pathway for cell and gene therapies, examining its critical role at the nexus of patient need, regulatory frameworks, and scientific advancement. Spearheaded by the International Society for Cell & Gene Therapy's Expanded Access Working Group, it explores how investigational therapies are accessed outside of clinical trials for patients with serious or life-threatening conditions when no approved alternatives exist. Access to cell and gene therapy products are of specific interest to patients because many times the products are bespoke, being used to treat serious and/or incurable conditions, and are potentially curative. As the field of cell and gene therapy rapidly progresses, healthcare professionals face mounting challenges in navigating the balance between access and oversight. Key considerations include transparent communication with patients, robust data reporting, and a discussion of cost recovery models and their implications for long-term commercialization strategies. Equity and inclusivity are central themes, highlighting the need to design pathways that are accessible to diverse patient populations while upholding high scientific and ethical standards. This position paper is presented as a resource for clinicians, researchers, and policymakers navigating the evolving landscape of investigational cell and gene therapies. It emphasizes the importance of ethical frameworks and equitable practices in delivering transformative treatments to patients in need.