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作用机制,效力和疗效:对细胞疗法的考虑

Carl G Simon¹, Erich H Bozenhardt², Christina M Celluzzi³

¹Biosystems and Biomaterials Division, National Institute of Standards and Technology (NIST), Gaithersburg, MD, USA. carl.simon@nist.gov.

Journal of translational medicine

|May 2, 2024

概括

开发先进的细胞疗法产品 (CTP) 需要明确的作用机制 (MOA),效力和疗效的定义. 这种观点提供了一个以计量学为基础的框架,以澄清这些概念并改善CTP的开发.

科学领域:

生物技术是生物技术.
细胞疗法细胞疗法
监管科学监管科学

背景情况:

定义作用机制 (MOA),效力和疗效对于先进细胞疗法产品 (CTP) 开发至关重要.
现有的监管指南以及美国FDA批准的27个CTP的经验教训为这一观点提供了信息.
对这些概念的误解可能会使开发和临床试验复杂化.

研究的目的:

在CTP开发中检查和澄清MOA,功效和疗效的概念.
为理解这些关键的产品属性提供基于计量学的框架.
改善沟通,并为CTP开发人员设置现实的期望.

主要方法:

对27个FDA批准的CTP的MOA,功效和疗效数据的审查.
引入功效和疗效过程图表,以说明关键概念关系.
分析强度测试与临床结果之间的潜在脱节.

主要成果:

提出了一个逻辑框架,以使MOA,功效和疗效与监管期望保持一致.
功效和疗效过程图表澄清了MOA,功效,测试,疗效,终点和测试之间的关系.
这些例子说明了诸如"强效但不有效"和"不强效但有效"之类的场景.

结论:

在人类中确定MOA存在重大挑战,正如不同研究模型的比较评估所表明的那样.
仔细考虑MOA,功效和疗效的定义有助于将功效测试结果与临床结果相关联.
拟议的框架有助于CTP开发人员设定适当的期望,避免假设,并加强团队沟通.

关键词:

细胞治疗产品细胞治疗产品有效性有效性.疗效终点是有效性的终点.有效性终点测试试验的测试结果.作用机制作用机制强度强度强度强度强度测试试验强度测试试验

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