药物流行病学研究的统计学分析技术规范

Statistical Analysis Guideline for Pharmacoepidemiology Research

药物研究领域的飞速发展给医学带来积极推动的同时,药物应用可能造成的负面效应也不断被公众所关注,从流行病学延伸出旨在研究药物在人群中应用的有效性及安全性的药物流行病学也随之不断壮大。中国药学会于2019年发布《药物流行病学研究方法学指南》,为我国规范性地开展药物流行病学研究提供了实践标准。本文为该指南统计学分析技术规范的进一步解读。首先,阐述了药物流行病学研究开始前制定统计分析计划的重要性及具体内容,并讨论了针对不同研究设计类型如何计算研究所需样本量。其次,详细叙述了统计资料的各种变量类型及多变量统计分析方法和注意事项、针对混杂因素使用倾向性评分等统计学方法来控制组间效应、缺失数据的处理以及使用敏感性分析评价研究结果的稳定性等。最后,阐明了药物流行病学研究的统计分析报告的内容及统计图表的撰写规范。

The rapid development of drug research has brought positive impetus to medicine,at the same time,the possible adverse effects of drug application have also been constantly concerned by the public.Extending from epidemiology,pharmacoepidemiology,which aims to study the effectiveness and safety of drug application in the population,is also growing.In 2019,the Chinese Pharmaceutical Association issued the Guide on Methodological Standards in Pharmacoepidemiology,which provides a practical standard for the normative development of pharmacoepidemiology research in China.This article is the further interpretation of the statistical analysis technical specifications of this guideline.First of all,it explains the importance and specific content of the statistical analysis plan before the beginning of the pharmacoepidemiology study,and how to estimate the sample size required for different study design types was also discussed.Secondly,it states in detail the statistical analysis methods and precautions for different variable types,the multivariable analysis methods,the use of propensity scores for control confounding,the processing of missing data,and the use of sensitivity analysis to evaluate the stability of the study results.Finally,it clarified the content of the statistical analysis report of the pharmacoepidemiology study and the writing standard of statistical tables and charts.