《中国药物流行病学研究方法学指南(第2版)》及其系列解读(4):药物流行病学研究的问题来源与方案制定  

Guide on Methodological Standards in Pharmacoepidemiology in China(2nd edition)and their series interpretation(4):sources of research question and study protocol development in pharmacoepidemiological research

作　　者：程吟楚 何娜 闫盈盈[1,2] 詹思延 孙凤[4,5,7,8,9,10] CHENG Yinchu;HE Na;YAN Yingying;ZHAN Siyan;SUN Feng(Department of Pharmacy,Peking University Third Hospital,Beijing 100191,China;Institute for Drug Evaluation,Peking University Health Science Center,Beijing 100191,China;Department of Population Medicine,Harvard Medical School and Harvard Pilgrim Healthcare Institute,Boston,Massachusetts 02215,United States;Department of Epidemiology and Biostatistics,School of Public Health,Peking University,Beijing 100191,China;Key Laboratory of Epidemiology of Major Diseases(Peking University),Ministry of Education,Beijing 100191,China;Research Center of Clinical Epidemiology,Peking University Third Hospital,Beijing 100191,China;Center of Postmarketing Safety Evaluation PUHSC,Beijing 100191,China;Department of Ophthalmology,Peking University Third Hospital,Beijing 100191,China;School of Traditional Chinese Medicine,Xinjiang Medical University,Urumqi 830017,China;School of Medicine,Shihezi University,Shihezi 832000,Xinjiang Uygur Autonomous Region,China)

机构地区：[1]北京大学第三医院药学部,北京100191 [2]北京大学医学部药物评价中心,北京100191 [3]美国哈佛大学医学院与哈佛朝圣者医疗保健研究所人群医学系,美国波士顿02215 [4]北京大学公共卫生学院流行病与卫生统计学系,北京100191 [5]重大疾病流行病学教育部重点实验室(北京大学),北京100191 [6]北京大学第三医院临床流行病学研究中心,北京100191 [7]北京大学医学部药品上市后安全性研究中心,北京100191 [8]北京大学第三医院眼科,北京100191 [9]新疆医科大学中医学院,乌鲁木齐830017 [10]新疆石河子大学医学院,新疆石河子832000

出　　处：《药物流行病学杂志》2025年第4期365-372,共8页Chinese Journal of Pharmacoepidemiology

基　　金：国家自然科学基金青年科学基金项目(71904002、72304010);国家自然科学基金面上项目(72474008);国家自然科学基金国际(地区)合作与交流项目(72361127500);北京大学第三医院临床重点项目(BYSYZD2021028);海南省科学技术厅重点研发专项(ZDYF2024LCLH002)。

摘　　要：在药物流行病学研究中,提出合理的研究问题并制定科学的研究方案是确保研究质量的关键。本文基于《中国药物流行病学研究方法学指南(第2版)》(以下简称“指南第2版”),对其中关于研究问题来源与研究方案制定的相关内容进行解读。首先,明确了研究问题的来源和范畴,从提出问题和定义问题两个步骤阐述了构建研究问题的方法,并介绍了相应的评价工具和定义框架。其次,对研究方案的构成要素进行了逐条解读,并重点分析了研究方法的内容和关键点。最后,强调了可行性评估的重要性,指出研究问题和方案的确定是一个动态迭代的过程。指南第2版相较于前版,针对研究问题构建和方案制定进行了细化和更新,新增了“方案的存档或注册”等三项方案构成要素及“研究目标”等三个内容条目,扩充了研究方法的内涵并强化了可行性评估等内容,进一步提升了指导实践时的全面性和严谨性。本文结合指南内容和实践经验,解读其对药物流行病学研究的指导意义,为相关研究人员提供参考。Formulating a well-defined research question and developing a scientifically sound study protocol is important for ensuring the quality of pharmacoepidemiological research.Based on the Guide on Methodological Standards in Pharmacoepidemiology in China(2nd edition),this research provided an interpretation of the sections related to the formulation of research questions and the development of study protocols.First,it clarified the sources and scope of research questions,outlining a two-step approach—question generation and definition—and introduces relevant evaluation tools and defining frameworks.Furthermore,it systematically explained the key elements of a study protocol and provides an in-depth explanation of the research methodology and its critical aspects.Finally,the paper highlighted the importance of feasibility assessment,emphasizing that the process of defining research questions and study protocols is dynamic and iterative.Compared with the previous edition,the 2nd edition of the guidelines offers more detailed and updated guidance on question formulation and protocol development.It added three new elements—such as protocol archiving or registration—and three new content items,including study objectives.The 2nd edition also expanded the methodological framework and strengthens feasibility assessments,thereby enhancing the comprehensiveness and rigor of its practical guidance.By integrating guideline content with practical experience,this paper provided insights into its implications for pharmacoepidemiological research and serves as a reference for researchers in the field.

关 键 词：药物流行病学 方法学 指南 研究问题 研究方案 

分 类 号：R181.35[医药卫生—流行病学]