Zhang Di, Kim Jessica
Department of Biostatistics, Graduate School of Public Health, University of Pittsburgh, Pittsburgh, PA, USA.
Division of Biometrics VIII/Office of Biostatistics/Center for Drug Evaluation and Research, FDA, Silver Spring, MD, USA.
J Biopharm Stat. 2019;29(6):1103-1115. doi: 10.1080/10543406.2019.1584205. Epub 2019 Mar 4.
Propensity score (PS) and disease risk score (DRS) are often used in pharmacoepidemiologic safety studies. Methods of applying these two balancing scores are extensively studied in binary treatment settings. However, the use of PS and DRS is not well understood in the case of non-ordinal multiple treatments. Some PS methods of multiple treatments have been implemented since the theoretical establishment. Nevertheless, most of the work applies to continuous or binary outcomes. Little work has been done for time-to-event outcomes. In this study, we extend the application of the PS and DRS methods to time-to-event outcomes in multiple treatment settings. The analytical approaches include weighing, matching, stratification, and regression. Simulation studies with rare event rates are conducted to evaluate the performances of different methods. Different treatment-covariates and outcome-covariates strength of associations are considered. Additionally, the impacts of imbalanced designs and large or limited PS overlaps are investigated on various analytical approaches. We found that the inverse probability treatment weighting with bootstrap variance estimator, the generalized PS matching, and the Cox regression estimated DRS in full cohort generally performed well in multiple treatment settings. This study aims to provide additional guidance for researchers on PS and DRS analyses in pharmacoepidemiologic observational studies.
倾向评分(PS)和疾病风险评分(DRS)常用于药物流行病学安全性研究。在二元治疗环境中,对应用这两种平衡评分的方法进行了广泛研究。然而,在非有序多治疗情况下,PS和DRS的使用尚不清楚。自理论确立以来,已经实施了一些多治疗的PS方法。尽管如此,大多数工作适用于连续或二元结局。对于事件发生时间结局,相关研究较少。在本研究中,我们将PS和DRS方法的应用扩展到多治疗环境中的事件发生时间结局。分析方法包括加权、匹配、分层和回归。进行了具有罕见事件率的模拟研究,以评估不同方法的性能。考虑了不同的治疗协变量和结局协变量关联强度。此外,还研究了不平衡设计以及PS重叠大或有限对各种分析方法的影响。我们发现,使用自助方差估计器的逆概率治疗加权、广义PS匹配以及在全队列中使用Cox回归估计DRS在多治疗环境中通常表现良好。本研究旨在为药物流行病学观察性研究中PS和DRS分析的研究人员提供更多指导。