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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…

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…

统计方法学 · 统计学 2022-11-10 Issa J. Dahabreh , Sarah E. Robertson , Miguel A. Hernán

Generalizing treatment effects from a randomized trial to a target population requires the assumption that potential outcome distributions are invariant across populations after conditioning on observed covariates. This assumption fails…

统计方法学 · 统计学 2026-04-16 Amir Asiaee , Samhita Pal , Cole Beck , Jared D. Huling

Background: Randomized controlled trials are often used to inform policy and practice for broad populations. The average treatment effect (ATE) for a target population, however, may be different from the ATE observed in a trial if there are…

统计方法学 · 统计学 2023-01-19 Trang Quynh Nguyen , Benjamin Ackerman , Ian Schmid , Stephen R. Cole , Elizabeth A. Stuart

Enhancing the external validity of trial results is essential for their applicability to real-world populations. However, violations of the positivity assumption can limit both the generalizability and transportability of findings. To…

统计方法学 · 统计学 2025-04-22 Jun Lu , Sanjib Basu

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

统计方法学 · 统计学 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

Generalizability and transportability methods have been proposed to address the external validity bias of randomized clinical trials that results from differences in the distribution of treatment effect modifiers between trial and target…

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…

统计方法学 · 统计学 2022-02-24 Issa J. Dahabreh , Sarah E. Robertson , Jon A. Steingrimsson

When treatment effect modifiers influence the decision to participate in a randomized trial, the average treatment effect in the population represented by the randomized individuals will differ from the effect in other populations. In this…

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

统计方法学 · 统计学 2022-02-08 Melody Huang

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…

统计方法学 · 统计学 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh

In the presence of treatment effect heterogeneity, the average treatment effect (ATE) in a randomized controlled trial (RCT) may differ from the average effect of the same treatment if applied to a target population of interest. If all…

统计方法学 · 统计学 2017-05-02 Trang Quynh Nguyen , Cyrus Ebnesajjad , Stephen R. Cole , Elizabeth A. Stuart

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…

统计方法学 · 统计学 2021-10-04 Sarah E Robertson , Jon A Steingrimsson , Issa J Dahabreh

Estimation of local average treatment effects in randomized trials typically requires an assumption known as the exclusion restriction in cases where we are unwilling to rule out unmeasured confounding. Under this assumption, any benefit…

统计方法学 · 统计学 2020-08-17 Andrew J. Spieker , Robert A. Greevy , Lyndsay A. Nelson , Lindsay S. Mayberry

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

统计方法学 · 统计学 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

Extending (generalizing or transporting) causal inferences from a randomized trial to a target population requires ``generalizability'' or ``transportability'' assumptions, which state that randomized and non-randomized individuals are…

When engagement with a randomized trial is driven by factors that affect the outcome or when trial engagement directly affects the outcome independent of treatment, the average treatment effect among trial participants is unlikely to…

统计方法学 · 统计学 2019-06-27 Issa J. Dahabreh , James M. Robins , Sebastien J-P. A. Haneuse , Miguel A. Hernán

We present methods for causally interpretable meta-analyses that combine information from multiple randomized trials to estimate potential (counterfactual) outcome means and average treatment effects in a target population. We consider…

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…

When assessing causal effects, determining the target population to which the results are intended to generalize is a critical decision. Randomized and observational studies each have strengths and limitations for estimating causal effects…

统计方法学 · 统计学 2022-10-21 Irina Degtiar , Sherri Rose
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