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Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

统计方法学 · 统计学 2026-05-01 Elizabeth Tipton , Michalis Mamakos

Investigators are increasingly using novel methods for extending (generalizing or transporting) causal inferences from a trial to a target population. In many generalizability and transportability analyses, the trial and the observational…

统计方法学 · 统计学 2022-09-20 Yu-Han Chiu , Issa J. Dahabreh

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

We study variants of the average treatment effect on the treated with population parameters replaced by their sample counterparts. For each estimand, we derive the limiting distribution with respect to a semiparametric efficient estimator…

统计方法学 · 统计学 2024-02-12 Andrew Yiu

We provide sufficient conditions for the identification of the heterogeneous treatment effects, defined as the conditional expectation for the differences of potential outcomes given the untreated outcome, under the nonignorable treatment…

统计方法学 · 统计学 2019-01-15 Keisuke Takahata , Takahiro Hoshino

Kernel matching is a widely used technique for estimating treatment effects, particularly valuable in observational studies where randomized controlled trials are not feasible. While kernel-matching approaches have demonstrated practical…

统计方法学 · 统计学 2025-12-11 Chong Ding , Zheng Li , Hon Keung Tony Ng , Wei Gao

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with…

统计方法学 · 统计学 2022-03-22 Siyi Liu , Yilong Zhang , Gregory T Golm , Guanghan , Liu , Shu Yang

In this paper the estimation of the distribution function for potential outcomes to receiving or not receiving a treatment is studied. The approach is based on weighting observed data on the basis on estimated propensity score. A weighted…

统计方法学 · 统计学 2019-04-30 Pier Luigi Conti , Livia De Giovanni

Outcome-dependent sampling designs are extensively utilized in various scientific disciplines, including epidemiology, ecology, and economics, with retrospective case-control studies being specific examples of such designs. Additionally, if…

统计方法学 · 统计学 2023-09-22 Min Zeng , Zeyang Jia , Zijian Sui , Jinfeng Xu , Hong Zhang

For observational studies, we study the sensitivity of causal inference when treatment assignments may depend on unobserved confounders. We develop a loss minimization approach for estimating bounds on the conditional average treatment…

统计方法学 · 统计学 2022-03-11 Steve Yadlowsky , Hongseok Namkoong , Sanjay Basu , John Duchi , Lu Tian

Most of the widely used estimators of the average treatment effect (ATE) in causal inference rely on the assumptions of unconfoundedness and overlap. Unconfoundedness requires that the observed covariates account for all correlations…

统计理论 · 数学 2025-07-01 Yang Cai , Alkis Kalavasis , Katerina Mamali , Anay Mehrotra , Manolis Zampetakis

Matching and weighting methods for observational studies involve the choice of an estimand, the causal effect with reference to a specific target population. Commonly used estimands include the average treatment effect in the treated (ATT),…

统计方法学 · 统计学 2023-07-12 Noah Greifer , Elizabeth A. Stuart

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

统计方法学 · 统计学 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

While a randomized control trial is considered the gold standard for estimating causal treatment effects, there are many research settings in which randomization is infeasible or unethical. In such cases, researchers rely on analytical…

统计方法学 · 统计学 2024-02-21 Julia C. Thome , Peter F. Rebeiro , Andrew J. Spieker , Bryan E. Shepherd

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In…

统计方法学 · 统计学 2025-08-27 Dane Isenberg , Nandita Mitra , Steven C. Marcus , Rinad S. Beidas , Kristin A. Linn

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

统计方法学 · 统计学 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

统计方法学 · 统计学 2025-05-26 Huan Wang , Fei Wu , Yeh-Fong Chen

This study introduces a new approach to power analysis in the context of estimating a local average treatment effect (LATE), where the study subjects exhibit noncompliance with treatment assignment. As a result of distributional…

统计方法学 · 统计学 2020-04-10 Kirk Bansak

We consider the estimation of measures of model performance in a target population when covariate and outcome data are available on a sample from some source population and covariate data, but not outcome data, are available on a simple…

统计方法学 · 统计学 2023-06-16 Jon A. Steingrimsson , Sarah E. Robertson , Issa J. Dahabreh

Attrition is a common and potentially important threat to internal validity in treatment effect studies. We extend the changes-in-changes approach to identify the average treatment effect for respondents and the entire study population in…

计量经济学 · 经济学 2024-03-29 Dalia Ghanem , Sarojini Hirshleifer , Désiré Kédagni , Karen Ortiz-Becerra