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In paired randomized experiments individuals in a given matched pair may differ on prognostically important covariates despite the best efforts of practitioners. We examine the use of regression adjustment as a way to correct for persistent…

统计方法学 · 统计学 2017-11-27 Colin B. Fogarty

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to…

统计方法学 · 统计学 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

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…

Randomized controlled trials (RCTs) are the gold standard for evaluating causal effects but are often costly and difficult to scale; consequently, they are frequently augmented with auxiliary external controls in many applications. Prior…

统计方法学 · 统计学 2026-05-28 Jiawei Shan , Yiteng Tu , Guanbo Wang , Chao Ying , Jiwei Zhao

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

The gold standard for identifying causal relationships is a randomized controlled experiment. In many applications in the social sciences and medicine, the researcher does not control the assignment mechanism and instead may rely upon…

应用统计 · 统计学 2016-11-22 Johann Gagnon-Bartsch , Yotam Shem-Tov

In cluster randomized experiments, individuals are often recruited after the cluster treatment assignment, and data are typically only available for the recruited sample. Post-randomization recruitment can lead to selection bias, inducing…

统计方法学 · 统计学 2024-10-11 Georgia Papadogeorgou , Bo Liu , Fan Li , Fan Li

Randomized experiments are the "gold standard" for estimating causal effects, yet often in practice, chance imbalances exist in covariate distributions between treatment groups. If covariate data are available before units are exposed to…

统计理论 · 数学 2012-07-25 Kari Lock Morgan , Donald B. Rubin

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

We consider estimation of the target population average treatment effect (TATE) when outcome information is unavailable. Instead, we observe the outcome in multiple source populations and wish to combine the treatment effects therein to…

统计方法学 · 统计学 2025-05-16 Zehao Su , Helene Charlotte Rytgaard , Henrik Ravn , Frank Eriksson

The ICH E9(R1) addendum provides guidelines on accounting for intercurrent events in clinical trials using the estimands framework. However, there has been limited attention to the estimands framework for meta-analysis. Using treatment…

Participants enrolled into randomized controlled trials (RCTs) often do not reflect real-world populations. Previous research in how best to translate RCT results to target populations has focused on weighting RCT data to look like the…

Randomized experiments are widely used to estimate the causal effects of a proposed treatment in many areas of science, from medicine and healthcare to the physical and biological sciences, from the social sciences to engineering, to public…

统计方法学 · 统计学 2022-11-30 Christina Lee Yu , Edoardo M Airoldi , Christian Borgs , Jennifer T Chayes

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

应用统计 · 统计学 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

In this study, we focus on estimating the heterogeneous treatment effect (HTE) for survival outcome. The outcome is subject to censoring and the number of covariates is high-dimensional. We utilize data from both the randomized controlled…

统计方法学 · 统计学 2025-03-21 Xin Ye , Shu Yang , Xiaofei Wang , Yanyan Liu

The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

信号处理 · 电气工程与系统科学 2023-05-10 Sayeri Lala , Niraj K. Jha

Matching is a widely used causal inference design that aims to approximate a randomized experiment using observational data by forming matched sets of treated and control units based on similarities in their covariates. Ideally, treated…

统计方法学 · 统计学 2026-04-06 Jianan Zhu , Jeffrey Zhang , Zijian Guo , Siyu Heng

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

统计方法学 · 统计学 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Randomised Controlled Trials (RCTs) are the gold standard for estimating treatment effects across many fields of science. Technology companies have adopted A/B-testing methods as a modern RCT counterpart, where end-users are randomly…

社会与信息网络 · 计算机科学 2024-09-20 Olivier Jeunen