药物临床试验中疗效指标的选择  

Selection of endpoints for efficacy in clinical trials of new drugs

作　　者：刘炳林[1] 

机构地区：[1]国家食品药品监督管理总局药品审评中心,北京100038

出　　处：《中国新药杂志》2017年第18期2113-2120,共8页Chinese Journal of New Drugs

摘　　要：本文参考国内外药品注册相关法规、技术要求和指导原则,根据20多年新药审评经验和对技术标准及指导原则的深入理解,对新药临床试验中疗效指标概念和与疗效指标选择密切相关的几种疗效指标类型进行了分析和说明。对药物临床试验中主要疗效指标的特点以及主要疗效指标和次要疗效指标选择的基本原则进行了梳理和归纳。对疗效指标选择时需要考虑的问题进行了详细的概括和总结。对使用几种常见类型疗效指标进行疗效评价时需要注意的事项和问题进行了认真的分析和讨论。Referring to domestic and international drug registration regulations,technical standards and guidelines,this paper analyzes and explains the concepts and types of efficacy endpoints related to selection for endpoints in clinical trial for drugs based on twenty years of experience in drug evaluation and in-depth understanding of technical standards and guidelines of new drug registration. The characteristics of the primary efficacy endpoints and the basic principles of selecting the primary and second endpoints for efficacy in clinical trials for drugs are also discussed and summarized. The problems that need to be considered when selecting the endpoints for efficacy in the clinical trials are explained. This paper illustrates the matters needing attention and problems in the use of several common types of endpoints in clinical trials.

关 键 词：新药 临床试验 疗效指标 疗效指标的选择 主要疗效指标 考虑的问题 

分 类 号：R95[医药卫生—药学]