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# FDA Raises Approval Standards for CAR-T Cancer Therapies
- URL: https://www.fdaweb.com/fda-raises-approval-standards-for-car-t-cancer-therapies/
- Published: 2025-12-09T12:00:00.000Z
- Updated: 2026-09-14T15:28:45.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160348

FDA is signaling a major policy shift for cancer CAR T-cell therapies, moving away from reliance on single-arm studies toward randomized controlled trials (RCTs) that demonstrate superiority over existing treatments. Writing in a new *JAMA* Perspective [piece](https://jamanetwork.com/journals/jama/article-abstract/2842439?ref=fdaweb.com), CBER director **Vinay Prasad** and other Center officials say that many new CAR-T therapies will now be expected to show that their products outperform (superiority) standard-of-care treatments. This marks a departure from the approach used since the agency approved Novartis’ Kymriah in 2017, which largely relied on single-arm trials.

Writing in *JAMA*, Prasad *et al* say the agency’s goal is to maintain high evidentiary standards while using regulatory flexibilities when appropriate. They recommend that initial approvals for CAR-Ts be based on RCTs with clinically meaningful endpoints such as overall survival or acceptable time-to-event measures.

“Evidence should generally demonstrate superiority of the investigational CAR T-cell therapy compared with control,” the authors write. Plans to seek approval based on equivalence or noninferiority will require FDA discussion and justification.

The choice of control groups is critical, their guidance notes. Sponsors must carefully assess standard-of-care options and ethical considerations to ensure trial results reliably reflect the therapy’s effects rather than other factors. Single-arm trials may still be acceptable in rare cancers or in heavily pretreated populations where RCTs are not feasible. Accelerated approvals may still be granted based on high, durable response rates in these contexts, but traditional approval will require evidence from RCTs showing clinical benefit.