临床试验中疗效指标观察与测量相关问题的考虑  

Considerations on observation and measurement of efficacy variables in clinical trials

作　　者：刘炳林[1] 薛斐然 LIU Bing-lin;XUE Fei-ran(Center for Drug Evaluation,National Medical Products Administration,Beijing 100022,China)

机构地区：[1]国家药品监督管理局药品审评中心

出　　处：《中国新药杂志》2019年第24期2939-2947,共9页Chinese Journal of New Drugs

摘　　要：疗效指标评价是临床试验最重要的研究内容之一。而疗效指标的认真观察和准确测量则是保证临床试验有效性数据真实、可靠、准确、完整的重要因素。本文对药物临床试验中疗效指标观察和测量相关问题进行了讨论。其中包括临床试验设计中需要关注的疗效终点指标定义、疗效指标访视点和访视时间框架的设计以及疗效指标观测时间周期设计考虑的因素,访视点间隔与临床试验疗程的设计要求,每次访视的访视窗的设计和要求,每个观测时间周期内观测次数的设计和要求等。本文还讨论了生存状态及相关临床事件类、量表类、实验室类、病理组织学类、影像学类等指标等观察和测量需要注意的问题,疗效指标的观测和收集方式方法、与疗效指标相关的基线取值以及疗效指标观测中的盲法要求等内容,以期对新药临床疗效指标的观察和测量设计和实施有所帮助。Efficacy evaluation is one of the most important components in clinical trials of drugs.And careful observation and accurate measurement of efficacy variables are important factors to ensure the validity,reliability,accuracy and completeness of efficacy data in clinical trials.This paper discussed some questions about the observation and measurement of efficacy variables in clinical trials,including the definition of efficacy endpoints,design of interview points and time frame,considerations on design of observation period of efficacy variables,requirements on design of the interval between interview points and the course of clinical trials,interview time windows and design,and requirement of observation frequency in each observation period.The paper also introduced the problems needing attention in observation and measurement of survival status and related clinical events,scales,laboratories,pathological histology,imaging and other efficacy variables.The observation and collection methods of efficacy variables,measurement of baseline data of efficacy variables,and requirement on blinding method of observation and measurement of efficacy variables were also discussed.It is hoped that the paper will be helpful to researchers who design and implement observation and measurement of efficacy variables in clinical trials of new drugs.

关 键 词：药物 临床试验 方案 疗效指标 终点 疗效评价 观察与测量 

分 类 号：R95[医药卫生—药学]