Autologous Chimeric antigen receptor (CAR) T-cell therapies have demonstrated substantial efficacy in patients with relapsed or refractory hematologic malignancies; however, their implementation has been constrained by regulatory barriers. Risk Evaluation and Mitigation Strategies (REMS), mandated by the U.S. Food and Drug Administration (FDA), were initially implemented to mitigate risks associated with cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and other treatment-related toxicities. On 27 June 2025, the FDA removed REMS requirements for all approved B-cell maturation antigen (BCMA) and CD19-directed autologous CAR T-cell therapies, citing that current product labeling sufficiently communicates safety information. Key regulatory changes include the elimination of site certification and tocilizumab stocking requirements, a reduction in the recommended post-infusion proximity period from four weeks to two weeks, increased flexibility regarding monitoring locations, and a shortened driving restriction from eight weeks to two weeks. This review examines the rationale for the REMS requirements for CAR T-cell therapies, synthesizes contemporary safety data from clinical trials and real-world practice, and explores the implications of this regulatory shift for access to care, particularly in rural and underserved populations. The removal of REMS requirements may facilitate broader implementation of CAR T-cell therapies and alleviate logistical and institutional barriers, offering the potential to expand access while preserving patient safety.
自体嵌合抗原受体（CAR）T细胞疗法在复发或难治性血液系统恶性肿瘤患者中已显示出显著疗效，但其临床应用长期受限于监管壁垒。美国食品药品监督管理局（FDA）最初通过强制实施风险评估与缓解策略（REMS），以控制细胞因子释放综合征（CRS）、免疫效应细胞相关神经毒性综合征（ICANS）及其他治疗相关毒性的风险。2025年6月27日，FDA宣布取消所有已获批的B细胞成熟抗原（BCMA）及CD19靶向自体CAR T细胞疗法的REMS要求，理由是现有产品说明书已能充分传达安全信息。核心监管变更包括：取消治疗机构资质认证及托珠单抗备货要求，将建议的输注后就近观察期从四周缩短至两周，放宽监测地点限制，并将驾驶限制期从八周缩减至两周。本文通过梳理CAR T细胞疗法REMS要求的设立依据，整合临床试验与真实世界实践中的最新安全性数据，探讨此次监管调整对治疗可及性（特别是农村及医疗服务不足人群）的影响。REMS要求的取消有望促进CAR T细胞疗法的更广泛实施，缓解物流与机构层面的障碍，在保障患者安全的同时为拓展治疗可及性创造可能。
Eliminating REMS for CAR T-Cell Therapies: An Opportunity to Improve Access
肿瘤(癌症)患者之家