Cell therapies: are we doing enough to protect patients and address the barriers to patient access?

This article is intended for cell therapy developers. It examines how unproven, unregulated cell therapies continue to pose significant risks to patients while simultaneously creating tangible obstacles for legitimate cell therapy developers. Specifically, direct-to-consumer clinics offering unproven and unregulated cell therapies expose vulnerable individuals to harm, erode public trust, and complicate the cell therapy clinical development landscape by competing unfairly with rigorously regulated cell therapies. This article examines these challenges and emphasizes the importance of distinguishing evidence-based cell therapy development from the unproven and unregulated practices of direct-to-consumer clinics. In the context of cell therapy, we provide an overview of regulated non-trial access pathways available to patients with serious or life-threatening conditions in the United States of America and European Union. These pathways, most notably the US Food and Drug Administration's Expanded Access Program and the European Union's Hospital Exemption, enable patient access to cell therapies outside of standard regulatory approval pathways when appropriate alternative therapies are unavailable or unsuitable. By operating within established regulatory frameworks, these pathways aim to balance patient access with safety oversight. Additionally, we address some of the practical challenges associated with conducting cell therapy clinical trials, which include trial design, endpoint selection, and patient expectation management. The viewpoints presented in the article aim to provide cell therapy developers with the framing and context needed to advocate for stronger regulatory coherence, protect legitimate cell therapy development pipelines, and ensure that patients access only safe and effective cell therapies.