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随机试验的比较：试验参与效应与效应迁移AI 解读

Disagreements between randomized trials that address the same causal question pose challenges for the interpretation and synthesis of evidence. We sketch a causal framework for comparing the results of randomized trials that explicitly accommodates the possibility of trial engagement effects. Using counterfactual outcomes defined under joint interventions on treatment assignment and trial-related actions, we compare trial-native treatment effects with the effects transported…

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…

利用汇总数据开展具有因果解释的元分析AI 解读

Evidence syntheses and meta-analyses are used to inform clinical practice guidelines and health economic evaluations. However, heterogeneity of treatment effects poses a significant challenge. Conventional meta-analysis addresses heterogeneity through random-effect assumptions, which are not supported by design and lead to estimates that may not apply to any real-world population. Causally-interpretable meta-analysis (CIMA) offers a rigorous framework for specification,…

利用目标试验框架整合信息：外部对照分析及其他应用AI 解读

We describe how the target trial framework can be used to plan and report analyses that attempt to answer causal questions by combining information from multiple, diverse sources. Such analyses may involve comparisons of treatments evaluated in different populations, for example when an index trial is combined with other data sources in external comparator analyses, or when extending causal inferences from a randomized trial to a new target population in generalizability and…

将嵌套随机试验的条件平均处理效应推广至全部符合资格者AI 解读

Randomized controlled trials often enroll participants whose characteristics differ from those of a target population, which can limit the generalizability of the estimated treatment effects when effect modifiers differ across populations. While existing generalizability methods primarily focus on estimating the average treatment effect (ATE) in the target population, such summaries may obscure important heterogeneity that is relevant for clinical and policy decision-making.…

通过均值或效应尺度可迁移性构造外部对照组AI 解读

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data from an external comparator, which may facilitate head-to-head comparisons of treatments initially studied in different populations. We delineate external comparator analyses under two distinct, but related, identification strategies. The…

借助外部数据稳健估计随机试验中的异质性处理效应AI 解读

Randomized trials are typically designed to detect average treatment effects but often lack the statistical power to uncover individual-level treatment effect heterogeneity, limiting their value for personalized decision-making. To address this, we propose the QR-learner, a model-agnostic learner that estimates conditional average treatment effects (CATE) within the trial population by leveraging external data from other trials or observational studies. The proposed method…

随机试验中分配对因果效应定义与识别的作用AI 解读

In randomized trials, the per-protocol effect, that is, the effect of being assigned a treatment strategy and receiving treatment according to the assigned strategy, is sometimes thought to reflect the effect of the treatment strategy itself, without intervention on assignment. Here, we argue by example that this is not necessarily the case. We examine a causal structure for a randomized trial where these two causal estimands -- the per-protocol effect and the effect of the…

随机试验中外部对照数据的稳健整合AI 解读

One approach for increasing the efficiency of randomized trials is the use of "external controls" -- individuals who received the control treatment studied in the trial during routine practice or in prior experimental studies. Existing external control methods, however, can be biased if the populations underlying the trial and the external control data are not exchangeable. Here, we characterize a randomization-aware class of treatment effect estimators in the population…

支持可推广推断的整群随机试验设计AI 解读

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with certain characteristics to improve trial economy or to support inference about subgroups of clusters, may preclude simple random sampling from the cohort into the trial, and thus interfere with the goal of producing generalizable…

存在试验参与效应时随机试验因果推断的推广与迁移AI 解读

Trial engagement effects are effects of trial participation on the outcome that are not mediated by treatment assignment. Most work on extending (generalizing or transporting) causal inferences from a randomized trial to a target population has, explicitly or implicitly, assumed that trial engagement effects are absent, allowing evidence about the effects of the treatments examined in trials to be applied to non-experimental settings. Here, we define novel causal estimands…

部分嵌套试验设计中的可推广性分析：坏死性小肠结肠炎外科试验AI 解读

We discuss generalizability analyses under a partially nested trial design, where part of the trial is nested within a cohort of trial-eligible individuals, while the rest of the trial is not nested. This design arises, for example, when only some centers participating in a trial are able to collect data on non-randomized individuals, or when data on non-randomized individuals cannot be collected for the full duration of the trial. Our work is motivated by the Necrotizing…

存在不依从时治疗分配效应推断的推广与迁移AI 解读

We discuss the identifiability of causal estimands for generalizability and transportability analyses, both under perfect and imperfect adherence to treatment assignment. We consider a setting where the trial data contain information on baseline covariates, assignment at baseline, intervention at baseline (point treatment), and outcomes; and where the data from non-randomized individuals only contain information on baseline covariates. In this setting, we review…

电子健康记录中的树式亚组发现：含 DTG 疗法的治疗效应异质性AI 解读

The rich longitudinal individual level data available from electronic health records (EHRs) can be used to examine treatment effect heterogeneity. However, estimating treatment effects using EHR data poses several challenges, including time-varying confounding, repeated and temporally non-aligned measurements of covariates, treatment assignments and outcomes, and loss-to-follow-up due to dropout. Here, we develop the Subgroup Discovery for Longitudinal Data (SDLD) algorithm,…

将随机试验推断推广至目标总体的全局敏感性分析AI 解读

When individuals participating in a randomized trial differ with respect to the distribution of effect modifiers compared compared with the target population where the trial results will be used, treatment effect estimates from the trial may not directly apply to target population. Methods for extending -- generalizing or transporting -- causal inferences from the trial to the target population rely on conditional exchangeability assumptions between randomized and…

因果可解释元分析中的系统性数据缺失AI 解读

Causally interpretable meta-analysis combines information from a collection of randomized controlled trials to estimate treatment effects in a target population in which experimentation may not be possible but covariate information can be collected from a simple random sample. In such analyses, a key practical challenge is systematically missing data when some baseline covariates are not collected in all trials. Here, we provide identification results for potential…

将整群随机试验的因果推断推广至目标总体AI 解读

We describe methods that extend (generalize or transport) causal inferences from cluster randomized trials to a target population of clusters, under a general nonparametric model that allows for arbitrary within-cluster dependence. We propose doubly robust estimators of potential outcome means in the target population that exploit individual-level data on covariates and outcomes to improve efficiency and are appropriate for use with machine learning methods. We illustrate…

在相对效应可迁移假设下识别新目标人群的处理效应AI 解读

Epidemiologists and applied statisticians often believe that relative effect measures conditional on covariates, such as risk ratios and mean ratios, are ``transportable'' across populations. Here, we examine the identification of causal effects in a target population using an assumption that conditional relative effect measures (e.g., conditional risk ratios or mean ratios) are transportable from a trial to the target population. We show that transportability for relative…

推广与迁移分析中的亚组效应估计AI 解读

Methods for extending -- generalizing or transporting -- inferences from a randomized trial to a target population involve conditioning on a large set of covariates that is sufficient for rendering the randomized and non-randomized groups exchangeable. Yet, decision-makers are often interested in examining treatment effects in subgroups of the target population defined in terms of only a few discrete covariates. Here, we propose methods for estimating subgroup-specific…

多中心试验中的中心特定因果推断AI 解读

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers. Here, we describe methods for reinterpreting the evidence produced by a multicenter trial in the context of the population underlying each center. We describe how to identify center-specific effects under identifiability conditions that…