Department of Dermatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Department of Dermatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA; Biostatistics, Epidemiology and Informatics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
J Invest Dermatol. 2018 Feb;138(2):e13-e18. doi: 10.1016/j.jid.2017.10.026.
Clinical trials have several important limitations for evaluating the safety of new medications, leading to many adverse events not being identified until the postmarketing period. Descriptive studies, including case reports, case series, cross-sectional, and ecologic studies, help identify potential safety signals and generate hypotheses. Further research using analytic study methods, including case-control studies and cohort studies, are necessary to determine if an association truly exists and to better understand the potential for causation. Pharmacoepidemiology research examines the use and effects of drugs when used in large populations of patients, using a variety of study designs and biostatistical techniques to reduce the confounding and systematic error associated with observational research. Understanding the strengths and limitations of pharmacoepidemiology research techniques is necessary to interpret the validity of drug safety studies, guiding both individual patient decisions and broader public health decisions.
临床试验在评估新药安全性方面存在若干重要限制,导致许多不良事件直到上市后才被发现。描述性研究,包括病例报告、病例系列、横断面和生态学研究,有助于识别潜在的安全信号并提出假说。需要使用分析性研究方法(包括病例对照研究和队列研究)进一步研究,以确定是否存在真正的关联,并更好地了解因果关系的可能性。药物流行病学研究在使用各种研究设计和生物统计学技术来减少与观察性研究相关的混杂和系统误差的情况下,研究药物在大量患者中的使用和效果,以确定药物的安全性。了解药物流行病学研究技术的优缺点对于解释药物安全性研究的有效性至关重要,这既指导了个体患者的决策,也指导了更广泛的公共卫生决策。