新药临床试验中药物临床价值的考虑  

Consideration of Clinical Value of Drugs in Clinical Trials of New Drugs

作　　者：刘炳林[1] 杨娜 吴艳 侯晨晨 LIU Bing-lin;YANG Na;WU Yan;HOU Chen-chen(Center for Drug Evaluation,National Medical Products Administration)

机构地区：[1]国家药品监督管理局药品审评中心

出　　处：《中国食品药品监管》2021年第4期24-31,共8页China Food & Drug Administration Magazine

摘　　要：新药上市的目的是为满足临床需求、实现临床价值。疗效是药物临床价值和临床意义的核心体现。因此,在新药研发临床试验的设计与疗效评价中,要充分重视临床价值评估。本文从药物临床试验目的和临床定位的确定、临床试验比较类型选择及相关界值的确定、临床疗效指标的选择和使用等多方面进行了分析和讨论,强调在新药临床试验中,需要重视药物临床价值和临床意义的评价相关的指标、概念和问题。期望通过对抽象概念具体化,为新药临床试验中药物临床价值和临床意义评价提供参考。The goal of new drug authorization is to meet clinical needs and achieve its clinical value.Clinically meaningfulefficacyis the core embodiment of clinical value and clinical significance of drugs.Therefore,more attention should be paid to the clinical value in the design and evaluation of clinical trials during new drug development.This paper analyzes and discusses several aspects related to clinical value,including the determination of the objectives and indications of clinical trials,the selection of clinical trial comparison types and the determination of relevant cut-off margins,and the selection and use of clinical efficacy endpoints.The authors stress that special attention should be paid to endpoints,concepts and problems relevant to the evaluation of clinical value and clinical significance of new drugs in clinical trials,and hope that discussion of the efficacy endpoints will be helpful for the evaluation of clinical value and clinically meaningful benefits of drugs in new drug clinical trials.

关 键 词：临床试验 新药 临床价值 有临床意义终点 终点指标 界值 

分 类 号：R95[医药卫生—药学]