药物临床试验中有效性指标的分类

Classification of efficacy endpoints in a clinical trial of the new drug

本文参考相关法规、指导性文件和指南,经过认真梳理,针对新药临床试验中疗效指标的多种分类及其与之密切相关的疾病临床结局分类,详细说明了各种分类中不同疗效指标和疾病临床结局的定义、概念、特点和在药物临床试验有效性设计、观测和评价等应用中应该注意的问题,特别是重点阐述了与新药临床试验密切相关的临床结局指标、临床终点、替代终点、主要疗效指标、次要疗效指标等概念和特点,以及不同指标的选择对新药临床试验设计、实施和评价的影响。

According to regulations and guidance documents,this paper introduces mainly classification of efficacy endpoints in a clinical trial of the new drug,and classification of clinical outcomes of a disease and health condition. The definition,concept,characteristics of different clinical efficacy endpoints and clinical outcomes of diseases are also introduced,such as clinical outcomes,clinical endpoints,surrogate endpoints,intermediate endpoints,primary endpoints,secondary endpoints,biomarker,PRO,ClinRO,ObsRO,PerfO and so on.