A General Framework for Treatment Effect Estimators Considering Patient Adherence
A General Framework for Treatment Effect Estimators Considering Patient Adherence
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DOI：
10.1080/19466315.2019.1700157
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发表时间：
2020-01-21
影响因子：
1.8
通讯作者：
Ruberg, Stephen J.
中科院分区：
文献类型：
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作者：
Qu, Yongming;Fu, Haoda;Ruberg, Stephen J.
随机对照试验仍然是评估新疗法疗效和安全性的金标准。理想情况下，患者在研究期间坚持治疗，并且可以明确分析所得数据的有效性和安全性结果。然而，一些患者可能由于并发事件而停止研究治疗，这会导致观察结果缺失或观察结果不能反映随机分配的治疗。通常，进行意向治疗分析（或其修改），以估计所有随机化患者的治疗效果，而不考虑并发事件的发生。或者，临床医生可能更感兴趣的是了解能够坚持研究治疗的患者的疗效和安全性。由于观察到的依从性人群在两种治疗之间可能不具有可比性，因此，朴素符合方案分析可能提供治疗差异的偏倚估计值。在这篇文章中，我们提出了两种方法来估计那些谁可以坚持一个或两个治疗的基础上的反事实框架的治疗差异。理论推导和模拟研究表明，所提出的方法提供了一致的估计的治疗差异的粘附人口的利益。一个真实的数据的例子，比较两种基础胰岛素的1型糖尿病患者使用所提出的方法。可以在网上找到。
Randomized controlled trials remain a gold standard in evaluating the efficacy and safety of a new treatment. Ideally, patients adhere to their treatments for the duration of the study, and the resulting data can be analyzed unambiguously for efficacy and safety outcomes. However, some patients may discontinue the study treatment due to intercurrent events, which leaves missing observations or observations that do not reflect the randomly assigned treatment. Frequently, an intent-to-treat analysis (or a modification thereof) is done to estimate the treatment effect for all randomized patients regardless of the occurrence of intercurrent events. Alternatively, clinicians may be more interested in understanding the efficacy and safety for those who can adhere to the study treatment. The naive per-protocol analysis may provide a biased estimate for the treatment difference because the observed adherence populations may not be comparable between two treatments. In this article, we propose two methods for estimation of the treatment difference for those who can adhere to one or both treatments based on the counterfactual framework. Theoretical derivations and a simulation study show the proposed methods provide consistent estimators for the treatment difference for the adherent population of interest. A real data example comparing two basal insulins for patients with type-1 diabetes is provided using the proposed methods. for this article are available online.