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监管试验协变量调整的良好实践：从固定到数据自适应方法AI 解读

While randomization justifies the use of unadjusted effect estimators in randomized trials, there is growing interest in covariate adjustment to improve precision. Adjusting for baseline variables that are prognostic of the outcome can reduce estimator variance, resulting in narrower confidence intervals and increased statistical power. Recent guidance by the U.S. Food and Drug Administration supports fixed adjustment for prognostic covariates using parametric regression…

评估扩大筛查与改善结局策略总效应的因果框架AI 解读

For many health conditions, there are highly efficacious treatment and prevention products. Maximizing their impact requires strategies that improve the reach of health screening in order to establish who could benefit. For example, HIV prevention strategies aim to expand risk screening and to improve uptake of pre-exposure prophylaxis (PrEP) among those experiencing risk. Often, these strategies induce changes at the group-level (e.g., health clinics or communities) and are…

暴露与结局均存在缺失时的因果推断AI 解读

Missing data are ubiquitous in public health research. When estimating causal effects, there are well-established methods to address bias to due missing outcomes. Commonly, causal estimands are defined under hypothetical interventions to "set" the exposure and to prevent missingness. We demonstrate how this framework can be extended to missing exposures. We further extend this framework to incorporate missingness on the baseline outcome, which induces missingness on the…

以因果路线图和模拟提高真实数据应用的严谨性与可复现性AI 解读

The Causal Roadmap outlines a systematic approach to asking and answering questions of cause-and-effect: define the quantity of interest, evaluate needed assumptions, conduct statistical estimation, and carefully interpret results. To protect research integrity, it is essential that the algorithm for statistical estimation and inference be pre-specified prior to conducting any effectiveness analyses. However, it is often unclear which algorithm will perform optimally for the…

当暴露影响子群成员资格：围产期流行病学及其他情境中的因果问题AI 解读

Perinatal epidemiology often aims to evaluate exposures on infant outcomes. When the exposure affects the composition of people who give birth to live infants (e.g., by affecting fertility, behavior, or birth outcomes), this "live birth process" mediates the exposure effect on infant outcomes. Causal estimands previously proposed for this setting include the total exposure effect on composite birth and infant outcomes, controlled direct effects (e.g., enforcing birth), and…

整群随机试验中的效应定义与估计：方法比较AI 解读

Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs, several challenges remain. First, there are many possible ways to specify the causal effect of interest (e.g., at the individual-level or at the cluster-level). Second, the theoretical and practical performance of common methods for CRT…