Oganisian Arman, Girard Anthony, Steingrimsson Jon A, Moyo Patience
Department of Biostatistics, Brown University, Providence, RI 02903, United States.
Leonard Davis Institute for Health Economics, University of Pennsylvania, Philadelphia, PA 19104, United States.
Biometrics. 2024 Oct 3;80(4). doi: 10.1093/biomtc/ujae145.
Observational studies of recurrent event rates are common in biomedical statistics. Broadly, the goal is to estimate differences in event rates under 2 treatments within a defined target population over a specified follow-up window. Estimation with observational data is challenging because, while membership in the target population is defined in terms of eligibility criteria, treatment is rarely observed exactly at the time of eligibility. Ad hoc solutions to this timing misalignment can induce bias by incorrectly attributing prior event counts and person-time to treatment. Even if eligibility and treatment are aligned, a terminal event process (eg, death) often stops the recurrent event process of interest. In practice, both processes can be censored so that events are not observed over the entire follow-up window. Our approach addresses misalignment by casting it as a time-varying treatment problem: some patients are on treatment at eligibility while others are off treatment but may switch to treatment at a specified time-if they survive long enough. We define and identify an average causal effect estimand under right-censoring. Estimation is done using a g-computation procedure with a joint semiparametric Bayesian model for the death and recurrent event processes. We apply the method to contrast hospitalization rates among patients with different opioid treatments using Medicare insurance claims data.
在生物医学统计学中,对复发事件率进行观察性研究很常见。大致而言,目标是估计在特定随访期内,定义的目标人群中两种治疗方法下事件率的差异。使用观察性数据进行估计具有挑战性,因为虽然目标人群的成员资格是根据资格标准定义的,但治疗很少恰好在符合资格时被观察到。针对这种时间错位的临时解决方案可能会因错误地将先前的事件计数和人时归因于治疗而导致偏差。即使资格和治疗是一致的,终末事件过程(例如死亡)通常也会终止感兴趣的复发事件过程。在实践中,两个过程都可能被截尾,因此在整个随访期内都无法观察到事件。我们的方法将时间错位视为一个随时间变化的治疗问题来解决:一些患者在符合资格时接受治疗,而另一些患者未接受治疗,但如果存活时间足够长,可能会在特定时间转为接受治疗。我们定义并识别了右删失情况下的平均因果效应估计量。估计使用g计算程序和用于死亡和复发事件过程的联合半参数贝叶斯模型进行。我们应用该方法,利用医疗保险索赔数据对比不同阿片类药物治疗患者的住院率。