临床试验研究方案设计中的统计学要素

Statistical elements in the design of clinical trial research protocol

临床试验方案设计是临床研究的首要环节。一份撰写规范的临床研究计划方案可以明确研究目的、指导临床试验研究的规范开展。临床研究方案的设计是一项科学、严谨的工作,在满足临床诊疗需求的前提下,须遵从统计学要求规范。参考随机对照试验报告统一标准(CONSORT)声明、PICO原则和我国《药物临床研究质量管理规范》,本文从临床研究设计类型入手,介绍随机化方案、对照设置、重复、盲法、终点指标、分析集、数据监察管理委员会、缺失数据及数据填补方法、统计分析和亚组分析等临床研究方案设计中的统计学要素,以期为研究者开展临床研究方案撰写提供参考。

The design of a clinical trial protocol is the primary aspect of clinical research.A well-written clinical research protocol can clarify the purpose of the research and guide the standardization of clinical trial research.The design of a clinical research protocol is also a scientific and rigorous task and must comply with the statistical requirements of the specifications while meeting the clinical treatment needs.With reference to the Uniform Standards for Reporting of Randomized Controlled Trials(CONSORT)statement,PICO principles and China’s Good Clinical Practice,the clinical study design types were introduced,including statistical elements in clinical study protocol design such as randomization protocols,control settings,replication,blinding,endpoint indicators,analysis sets,data monitoring and management committees,missing data and data filling methods,statistical analysis and subgroup analysis,so as to provide reference for researchers to write clinical study protocols.