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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

Measurement error is a common challenge for causal inference studies using electronic health record (EHR) data, where clinical outcomes and treatments are frequently mismeasured. Researchers often address measurement error by conducting…

Sample size calculations play a central role in study design because sample size affects study interpretability, costs, hospital resources, and staff time. For most veterinary orthopaedic risk-factor studies, either the sample size…

Applications · Statistics 2023-10-10 Richard Evans , Antonio Pozzi

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

Machine Learning · Statistics 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

Small sample sizes in clinical studies arises from factors such as reduced costs, limited subject availability, and the rarity of studied conditions. This creates challenges for accurately calculating confidence intervals (CIs) using the…

Methodology · Statistics 2025-11-11 Mulan Wu , Mengyu Xu , Dongyun Kim

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

Methodology · Statistics 2021-08-06 Fangzhou Su , Peng Ding

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

We study estimation of and inference for the average causal effect of treating every member of a population, as opposed to none, using an experiment that treats only some. Considering settings where spillovers can occur between any pair of…

Econometrics · Economics 2026-01-29 Stefan Faridani , Paul Niehaus

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

There are a number of available methods for selecting whom to prioritize for treatment, including ones based on treatment effect estimation, risk scoring, and hand-crafted rules. We propose rank-weighted average treatment effect (RATE)…

Methodology · Statistics 2023-11-30 Steve Yadlowsky , Scott Fleming , Nigam Shah , Emma Brunskill , Stefan Wager

Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, providing an ethical advantage by ensuring all clusters eventually receive the intervention. The staggered rollout of treatment…

Methodology · Statistics 2026-04-03 Xi Fang , Xueqi Wang , Patrick J. Heagerty , Bingkai Wang , Fan Li

Pragmatic trials evaluating health care interventions often adopt cluster randomization due to scientific or logistical considerations. Previous reviews have shown that co-primary endpoints are common in pragmatic trials but infrequently…

Methodology · Statistics 2022-05-03 Siyun Yang , Mirjam Moerbeek , Monica Taljaard , Fan Li

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

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…

Methodology · Statistics 2026-05-01 Elizabeth Tipton , Michalis Mamakos

We introduce a new multiple type I error criterion for clinical trials with multiple populations. Such trials are of interest in precision medicine where the goal is to develop treatments that are targeted to specific sub-populations…

Methodology · Statistics 2021-02-05 Werner Brannath , Charlie Hillner , Kornelius Rohmeyer

The focus of this paper is on quantifying the capacity of covariates in devising efficient treatment rules when data from a randomized trial are available. Conventional one-variable-at-a-time subgroup analysis based on statistical…

Applications · Statistics 2020-02-04 Mohsen Sadatsafavi , Mohammad Mansournia , Paul Gustafson

Alcohol misuse is a key target of public health strategies aimed at reducing cardiovascular risk. The effect of excessive alcohol consumption on blood pressure may vary systematically with individuals' unobserved propensity to engage in…

Methodology · Statistics 2026-03-11 Ashish Patel , Francis J DiTraglia , Stephen Burgess

Recent advances have shown that statistical tests for the rank of cross-covariance matrices play an important role in causal discovery. These rank tests include partial correlation tests as special cases and provide further graphical…

Machine Learning · Computer Science 2025-06-13 Xinshuai Dong , Ignavier Ng , Boyang Sun , Haoyue Dai , Guang-Yuan Hao , Shunxing Fan , Peter Spirtes , Yumou Qiu , Kun Zhang

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…