论文标题

结构性因果模型的务实临床试验

Pragmatic Clinical Trials in the Rubric of Structural Causal Models

论文作者

Adib, Riddhiman, Ahamed, Sheikh Iqbal, Adibuzzaman, Mohammad

论文摘要

解释性研究(例如随机对照试验)的目标是提取干预措施对结果的真正因果作用,并通过随机分组对协变量进行调整。相反,观察性研究是无需干预的事件的代表。两者都可以使用结构性因果模型(SCM)进行说明,并且可以使用DO-Calculus来估计因果效应。务实的临床试验(PCT)属于试验设计光谱的这两个末端,因此很难定义。由于其务实的性质,尚未确定PCT对PCT的标准化表示。在本文中,我们通过在结构因果模型(SCM)的标题下提出PCT的广义表示来解决此问题。我们讨论了使用所提出的图形模型(例如意向性处理,经过处理和每项协议分析)中常用的PCT中常用的分析技术。为了显示我们提出的方法的应用,我们利用了实用临床试验的实验数据集。我们通过PCT对SCM的主张为利用临床数据集的DO-Calculus和相关的数学操作创造了途径。

Explanatory studies, such as randomized controlled trials, are targeted to extract the true causal effect of interventions on outcomes and are by design adjusted for covariates through randomization. On the contrary, observational studies are a representation of events that occurred without intervention. Both can be illustrated using the Structural Causal Model (SCM), and do-calculus can be employed to estimate the causal effects. Pragmatic clinical trials (PCT) fall between these two ends of the trial design spectra and are thus hard to define. Due to its pragmatic nature, no standardized representation of PCT through SCM has been yet established. In this paper, we approach this problem by proposing a generalized representation of PCT under the rubric of structural causal models (SCM). We discuss different analysis techniques commonly employed in PCT using the proposed graphical model, such as intention-to-treat, as-treated, and per-protocol analysis. To show the application of our proposed approach, we leverage an experimental dataset from a pragmatic clinical trial. Our proposition of SCM through PCT creates a pathway to leveraging do-calculus and related mathematical operations on clinical datasets.

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