Schneeweiss Sebastian, Schneeweiss Maria C
Dermato-Pharmacoepidemiology Work Group, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
JID Innov. 2023 Aug 29;3(6):100226. doi: 10.1016/j.xjidi.2023.100226. eCollection 2023 Nov.
The U.S. Food and Drug Administration and clinical guidelines use evidence from pharmacoepidemiology studies to inform prescribing decisions and fill evidence gaps left by randomized controlled trials (RCTs). The long-term safety and infrequent adverse reactions are not well-understood when RCTs are short and involve few patients, as is the case for most systemic immunomodulating drugs in dermatology. A better understanding of the design and implementation of pharmacoepidemiology studies will help practitioners assess the accuracy of etiologic findings and use them with confidence in clinical practice. Conducting pharmacoepidemiology studies follows a structured approach, which we discuss in this article: (i) a design layer connects the research question with the appropriate study design, and considering which hypothetical RCT one ideally would want to conduct reduces inadvertent investigator errors; (ii) a measurement layer transforms longitudinal patient-level data into variables that identify the study population, patient characteristics, treatment, and outcomes; and (iii) the analysis focuses on the causal treatment effect estimation. The review and interpretation of pharmacoepidemiology studies should consider issues beyond a typical review of RCTs, chiefly the lack of baseline randomization and the use of secondary data. Well-designed and well-conducted pharmacoepidemiologic studies complement dermatology practice with critical information on prescribing systemic medications.
美国食品药品监督管理局及临床指南利用药物流行病学研究的证据来指导处方决策，并填补随机对照试验（RCT）留下的证据空白。当RCT时间较短且纳入患者较少时，其长期安全性和罕见不良反应就难以充分了解，皮肤科的大多数全身免疫调节药物便是如此。更好地理解药物流行病学研究的设计与实施，将有助于从业者评估病因学研究结果的准确性，并在临床实践中放心使用这些结果。开展药物流行病学研究需遵循结构化方法，我们将在本文中进行讨论：（i）设计层面将研究问题与适当的研究设计相联系，考虑理想情况下想要开展的假设性RCT可减少研究者的无意错误；（ii）测量层面将纵向患者层面的数据转化为识别研究人群、患者特征、治疗及结局的变量；（iii）分析聚焦于因果治疗效果估计。对药物流行病学研究的审查和解读应考虑超出RCT典型审查范围的问题，主要是缺乏基线随机化和使用二手数据的问题。设计良好且实施得当的药物流行病学研究可为皮肤科实践提供有关全身用药处方的关键信息，起到补充作用。
