统计分析计划(SAP)扩展版报告指南的解读及其对新药早期临床研究设计的启示

Interpretation of the statistical analysis plan(SAP)extended reporting guidelines and their enlightenment for the design of early stage clinical studies of new drugs

统计分析计划(statistical analysis plan,SAP)能够增加临床研究的可信度、透明度和减少统计分析过程的偏倚。SAP报告指南在开发时,主要针对后期(late phase)临床研究,即Ⅱ期和Ⅲ期的随机对照试验。目前,针对早期(early phase)临床研究,即I期临床研究和Ⅱ期非随机对照试验的SAP扩展版报告指南,主要从试验目的、设计、贝叶斯统计、数据模拟、样本量和ICH E9(R1)的应用6个角度在原报告指南基础上进行了扩展。扩展版报告指南能够使早期临床试验的SAP规范化,提高早期临床研究的透明性、可重复性,从而提高早期临床研究的质量,对后期临床研究亦起到关键作用。

The utilisation of statistical analysis plan(SAP)has the potential to enhance the reliability,transparency,and impartiality of statistical analysis procedures in the context of clinical studies.These plans are primarily designed for late phase clinical studies,namely phaseⅡand phaseⅢrandomised controlled trials.The extended SAP reporting guidelines for early phase clinical studies,i.e.,phaseⅠclinical studies and phaseⅡnon-randomised controlled trials,have been expanded from the original reporting guidelines in six key areas:trial purpose,design,Bayesian statistics,data simulation,sample size,and the application of ICH E9(R1).The expanded reporting guidelines facilitate the standardisation of SAP for early phase clinical trials,enhance the transparency and reproducibility of early phase clinical studies,and thereby improve the quality of early phase clinical studies.This,in turn,plays a pivotal role in later phase clinical studies.