Observational studies of recurrent event rates are common in biomedical statistics. Broadly, the goal is to estimate differences in event rates under two treatments within a defined target population over a specified followup window. Estimation with observational claims data is challenging because while membership in the target population is defined in terms of eligibility criteria, treatment is rarely assigned exactly at the time of eligibility. Ad-hoc solutions to this timing misalignment, such as assigning treatment at eligibility based on subsequent assignment, incorrectly attribute prior event rates to treatment - resulting in immortal risk bias. Even if eligibility and treatment are aligned, a terminal event process (e.g. death) often stops the recurrent event process of interest. Both processes are also censored so that events are not observed over the entire followup window. Our approach addresses misalignment by casting it as a treatment switching 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 of switching under specified causal assumptions. Estimation is done using a g-computation framework with a joint semiparametric Bayesian model for the death and recurrent event processes. Computing the estimand for various switching times allows us to assess the impact of treatment timing. We apply the method to contrast hospitalization rates under different opioid treatment strategies among patients with chronic back pain using Medicare claims data.
翻译:在生物医学统计中,对复发事件的观察性研究很常见。总体上,目的是在指定的随访时间内估计目标人群中两种治疗方法的事件率差异。使用观察性索赔数据进行估计是具有挑战性的,因为虽然目标人群的成员资格是以资格标准为基础定义的,但治疗很少会在资格的确切时间被准确分配。此时,应对不当的时间对齐进行处理,例如根据后续分配在资格时间点上分配治疗,错误地将先前的事件率归因于治疗 - 这将导致不朽风险偏倚。即使资格和治疗是一致的,末端事件进程(例如死亡)常常会停止我们感兴趣的复发事件进程。这两个过程也因未观察到在整个随访时间内的事件而被截断。我们的方法通过将其视为治疗转换问题来解决时间对齐问题。有些患者在资格时接受治疗,而其他人则没有接受治疗,但在特定时间可能会转换到治疗 - 如果他们存活时间足够长的话。在指定因果假设下,我们定义并确定了开关的平均因果效应。利用联合半参数贝叶斯模型对死亡和复发事件进程进行估计。通过对各种开关时间的估量来计算估计量,以评估治疗时机的影响。我们使用医疗保险索赔数据将该方法应用于比较治疗慢性腰痛患者的不同阿片类药物策略下的住院率。