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Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one…

Methodology · Statistics 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

For randomized controlled trials to be conclusive, it is important to set the target sample size accurately at the design stage. Comparing two normal populations, the sample size calculation requires specification of the variance other than…

Methodology · Statistics 2026-02-04 Hirotada Maeda , Satoshi Hattori , Tim Friede

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Multi-arm randomization has increasingly widespread applications recently and it is also crucial to ensure that the distributions of important observed covariates as well as the potential unobserved covariates are similar and comparable…

Applications · Statistics 2024-12-20 Xingjian Ma , Yang Liu

Mahalanobis distance between treatment group and control group covariate means is often adopted as a balance criterion when implementing a rerandomization strategy. However, this criterion may not work well for high-dimensional cases…

Methodology · Statistics 2021-02-25 Hengtao Zhang , Guosheng Yin , Donald B. Rubin

Randomized experiments are considered the gold standard for estimating causal effects. However, out of the set of possible randomized assignments, some may be likely to produce poor effect estimates and misleading conclusions. Restricted…

Methodology · Statistics 2025-08-28 Maggie Wang , René F. Kizilcec , Michael Baiocchi

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Although randomized experiments are widely regarded as the gold standard for estimating causal effects, missing data of the pretreatment covariates makes it challenging to estimate the subgroup causal effects. When the missing data…

Statistics Theory · Mathematics 2014-01-08 Peng Ding , Zhi Geng

Split-plot designs find wide applicability in multifactor experiments with randomization restrictions. Practical considerations often warrant the use of unbalanced designs. This paper investigates randomization based causal inference in…

Methodology · Statistics 2019-06-21 Rahul Mukerjee , Tirthankar Dasgupta

In this paper, we address the issue of estimating and inferring distributional treatment effects in randomized experiments. The distributional treatment effect provides a more comprehensive understanding of treatment heterogeneity compared…

Econometrics · Economics 2025-01-15 Tatsushi Oka , Shota Yasui , Yuta Hayakawa , Undral Byambadalai

We present a new experimental design procedure that divides a set of experimental units into two groups so that the two groups are balanced on a prespecified set of covariates and being almost as random as complete randomization. Under…

Statistics Theory · Mathematics 2016-12-08 Abba M. Krieger , David Azriel , Adam Kapelner

Batch effects are pervasive in biomedical studies. One approach to address the batch effects is repeatedly measuring a subset of samples in each batch. These remeasured samples are used to estimate and correct the batch effects. However,…

Methodology · Statistics 2023-11-07 Hanxuan Ye , Xianyang Zhang , Chen Wang , Ellen L. Goode , Jun Chen

A data set sampled from a certain population is biased if the subgroups of the population are sampled at proportions that are significantly different from their underlying proportions. Training machine learning models on biased data sets…

Machine Learning · Computer Science 2021-08-30 Jing An , Lexing Ying , Yuhua Zhu

A randomized trial and an analysis of observational data designed to emulate the trial sample observations separately, but have the same eligibility criteria, collect information on some shared baseline covariates, and compare the effects…

Methodology · Statistics 2022-03-29 Issa J. Dahabreh , Jon A. Steingrimsson , James M. Robins , Miguel A. Hernán

Randomized experiments are the gold standard for estimating the causal effects of an intervention. In the simplest setting, each experimental unit is randomly assigned to receive treatment or control, and then the outcomes in each treatment…

Methodology · Statistics 2020-06-05 Guillaume Basse , Yi Ding , Panos Toulis

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

Propensity score (PS) methods have been increasingly used in recent years when assessing treatment effects in nonrandomized studies. In terms of statistical methods, a number of new PS weighting methods were developed, and it was shown that…

Methodology · Statistics 2022-12-20 Tim Filla , Holger Schwender , Oliver Kuß

A practical limitation of cluster randomized controlled trials (cRCTs) is that the number of available clusters may be small, resulting in an increased risk of baseline imbalance under simple randomization. Constrained randomization…

Methodology · Statistics 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

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…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…