Empirical Performance of the Case-Control Method: Lessons for Developing a Risk Identification and Analysis System
Empirical Performance of the Case-Control Method: Lessons for Developing a Risk Identification and Analysis System
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DOI：
10.1007/s40264-013-0105-z
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发表时间：
2013-01-01
期刊：
影响因子：
4.2
通讯作者：
Ryan, Patrick B.
中科院分区：
文献类型：
--
作者：
Madigan, David;Schuemie, Martijn J.;Ryan, Patrick B.
背景：目前，大量的注意力集中在使用大规模观察性医疗数据来了解药物安全性。在这种情况下，分析人员利用各种统计和流行病学方法，如病例控制，队列和自我控制方法。人们对这些方法的操作特性了解甚少。目的建立病例对照法在药品安全性大规模观察分析中的操作特点。研究设计我们在5个真实的观察性医疗数据库和6个模拟数据集中对病例对照方法进行了实证评估。我们回顾性地研究了该方法在4种结局（急性肝损伤、急性心肌梗死、急性肾损伤和上消化道出血）中对165例阳性对照和234例阴性对照的预测准确性。结果病例对照法对阳性对照和阴性对照的区分较弱。此外，该方法产生的正偏估计和置信区间具有较差的覆盖特性。结论对于我们检查的四种结果，病例对照法可能不是评估药物潜在有害影响的选择方法。
Background Considerable attention now focuses on the use of large-scale observational healthcare data for understanding drug safety. In this context, analysts utilize a variety of statistical and epidemiological approaches such as case control, cohort, and self-controlled methods. The operating characteristics of these methods are poorly understood.Objective Establish the operating characteristics of the case control method for large scale observational analysis in drug safety.Research Design We empirically evaluated the case control approach in 5 real observational healthcare databases and 6 simulated datasets. We retrospectively studied the predictive accuracy of the method when applied to a collection of 165 positive controls and 234 negative controls across 4 outcomes: acute liver injury, acute myocardial infarction, acute kidney injury, and upper gastrointestinal bleeding.Results In our experiment, the case control method provided weak discrimination between positive and negative controls. Furthermore, the method yielded positively biased estimates and confidence intervals that had poor coverage properties.Conclusions For the four outcomes we examined, the case control method may not be the method of choice for estimating potentially harmful effects of drugs.