CAR-T Therapies: Access Considerations May Influence US FDA's Efficacy Evaluation

CBER's Lola Fashoyin-Aje offers some thoughts on when and how the FDA might consider the accessibility of a product in its approval decisions at BIO 2024.

3d illustration of a T cell attacking a cancer cell
US FDA discusses efficacy and access trade-offs for CAR-T therapies. • Source: Shutterstock

The definition of accessibility may influence whether the US Food and Drug Administration would consider efficacy concessions on a CAR-T treatment if the product were more easily available to patients than current options.

Key Takeaways
  • FDA considers access in the context of unmet need in its regulatory decisions.

  • The reach of CAR-T therapy currently is limited by a range of access challenges, including high cost, geographical limitations for administration, and manufacturing complexities

Access is not necessarily a part of FDA’s regulatory framework per se, but the agency does consider unmet need, Lola Fashoyin-Aje, director of the Office of Clinical Evaluation for cellular, tissue and gene therapies in the Center for Biologics Evaluation and

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