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Although randomized controlled trials have long been regarded as the ``gold standard'' for evaluating treatment effects, there is no natural prevention from post-treatment events. For example, non-compliance makes the actual treatment…

Methodology · Statistics 2025-04-25 Qinqing Liu , Xiang Peng , Tao Zhang , Yuhao Deng

In clinical trials, a covariate-adjusted response-adaptive (CARA) design allows a subject newly entering a trial a better chance of being allocated to a superior treatment regimen based on cumulative information from previous subjects, and…

Applications · Statistics 2011-06-21 Yuan-chin Ivan Chang , Eunsik Park

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

Attrition is a common occurrence in cluster randomised trials (CRTs) which leads to missing outcome data. Two approaches for analysing such trials are cluster-level analysis and individual-level analysis. This paper compares the performance…

Methodology · Statistics 2016-03-15 Anower Hossain , Karla Diaz-Ordaz , Jonathan W. Bartlett

Randomized controlled trials are not only the golden standard in medicine and vaccine trials but have spread to many other disciplines like behavioral economics, making it an important interdisciplinary tool for scientists. When designing…

Methodology · Statistics 2021-11-30 Tassilo Schwarz

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.…

Linear mixed models are commonly used in analyzing stepped-wedge cluster randomized trials (SW-CRTs). A key consideration for analyzing a SW-CRT is accounting for the potentially complex correlation structure, which can be achieved by…

Methodology · Statistics 2024-08-21 Yongdong Ouyang , Monica Taljaard , Andrew B Forbes , Fan Li

Consider a panel data setting where repeated observations on individuals are available. Often it is reasonable to assume that there exist groups of individuals that share similar effects of observed characteristics, but the grouping is…

Methodology · Statistics 2024-02-09 Lu Yu , Jiaying Gu , Stanislav Volgushev

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A…

When evaluating the efficacy of social programs and medical treatments using randomized experiments, the estimated overall average causal effect alone is often of limited value and the researchers must investigate when the treatments do and…

Applications · Statistics 2013-05-27 Kosuke Imai , Marc Ratkovic

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

In this paper we revisit some common recommendations regarding the analysis of matched-pair and stratified experimental designs in the presence of attrition. Our main objective is to clarify a number of well-known claims about the practice…

Econometrics · Economics 2023-10-20 Yuehao Bai , Meng Hsuan Hsieh , Jizhou Liu , Max Tabord-Meehan

Evaluations often inform future program implementation decisions. However, the implementation context may differ, sometimes substantially, from the evaluation study context. This difference leads to uncertainty regarding the relevance of…

Methodology · Statistics 2023-12-14 Irina Degtiar , Mariel Finucane

A/B testing is a standard approach for evaluating the effect of online experiments; the goal is to estimate the `average treatment effect' of a new feature or condition by exposing a sample of the overall population to it. A drawback with…

Social and Information Networks · Computer Science 2013-05-31 Johan Ugander , Brian Karrer , Lars Backstrom , Jon Kleinberg

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

Methodology · Statistics 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

In matched-pairs experiments in which one cluster per pair of clusters is assigned to treatment, to estimate treatment effects, researchers often regress their outcome on a treatment indicator and pair fixed effects, clustering standard…

Econometrics · Economics 2023-06-29 Clément de Chaisemartin , Jaime Ramirez-Cuellar

Random-effects models are central to meta-analysis, yet the between-study variance is often underestimated when the number of studies is small. In such settings, confidence intervals become unduly narrow and fail to attain the nominal…

Methodology · Statistics 2025-11-18 Keisuke Hanada , Tomoyuki Sugimoto

Instrumental variable methods are widely used in medical and social science research to draw causal conclusions when the treatment and outcome are confounded by unmeasured confounding variables. One important feature of such studies is that…

Methodology · Statistics 2021-05-25 Bo Zhang , Siyu Heng , Emily J. MacKay , Ting Ye
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