嵌合抗原受体
医学
食品药品监督管理局
重要事件
临床试验
许可证
药品审批
监管科学
药物开发
肿瘤科
药品
癌症
内科学
药理学
免疫疗法
病理
考古
法学
历史
政治学
作者
Xue Lin,Shiowjen Lee,Poornima Sharma,Bindu George,John Scott
摘要
The approval of tisagenlecleucel and axicabtagene ciloleucel in 2017 marked a milestone in the development of oncology therapies. Since 2017, the breakthrough in treatment or even cure of previously intractable diseases represented by this new class of cancer treatments has continued with subsequent chimeric antigen receptor T (CAR T)-cell approvals. To date, the US Food and Drug Administration has approved five autologous CAR T-cell products for seven indications. A feature of autologous CAR T-cell products that differentiates them from traditional oncology drugs is that they need to be manufactured specifically for each patient. This feature has implications in study design, statistical analyses, and interpretation of study results. In this article, we share our experiences in the statistical review of CAR T-cell products and provide considerations for the design and statistical analyses of CAR T-cell trials. We also describe how the newly adopted estimand framework for clinical trials can help clarify nuanced issues in CAR T-cell trial design.
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