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.
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.
Authors: Martijn J Schuemie; M Soledad Cepeda; Marc A Suchard; Jianxiao Yang; Yuxi Tian; Alejandro Schuler; Patrick B Ryan; David Madigan; George Hripcsak Journal: Harv Data Sci Rev Date: 2020-01-31
Authors: Martijn J Schuemie; Rosa Gini; Preciosa M Coloma; Huub Straatman; Ron M C Herings; Lars Pedersen; Francesco Innocenti; Giampiero Mazzaglia; Gino Picelli; Johan van der Lei; Miriam C J M Sturkenboom Journal: Drug Saf Date: 2013-10 Impact factor: 5.606
Authors: Paul E Stang; Patrick B Ryan; J Marc Overhage; Martijn J Schuemie; Abraham G Hartzema; Emily Welebob Journal: Drug Saf Date: 2013-10 Impact factor: 5.606
Authors: Patrick B Ryan; Paul E Stang; J Marc Overhage; Marc A Suchard; Abraham G Hartzema; William DuMouchel; Christian G Reich; Martijn J Schuemie; David Madigan Journal: Drug Saf Date: 2013-10 Impact factor: 5.606
Authors: Patrick B Ryan; Martijn J Schuemie; Emily Welebob; Jon Duke; Sarah Valentine; Abraham G Hartzema Journal: Drug Saf Date: 2013-10 Impact factor: 5.606