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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…

Methodology · Statistics 2026-05-28 Jiawei Shan , Yiteng Tu , Guanbo Wang , Chao Ying , Jiwei Zhao

In biomedical science, analyzing treatment effect heterogeneity plays an essential role in assisting personalized medicine. The main goals of analyzing treatment effect heterogeneity include estimating treatment effects in clinically…

Methodology · Statistics 2022-12-06 Waverly Wei , Maya Petersen , Mark J van der Laan , Zeyu Zheng , Chong Wu , Jingshen Wang

We consider the problem of selecting the optimal subgroup to treat when data on covariates is available from a randomized trial or observational study. We distinguish between four different settings including (i) treatment selection when…

Methodology · Statistics 2018-02-28 Tyler J. VanderWeele , Alex R. Luedtke , Mark J. van der Laan , Ronald C. Kessler

The standard paradigm for confirmatory clinical trials is to compare experimental treatments with a control, for example the standard of care or a placebo. However, it is not always the case that a suitable control exists. Efficient…

Methodology · Statistics 2024-10-29 Thomas Burnett , Thomas Jaki

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

We study treatment effect modifiers for causal analysis in a social network, where neighbors' characteristics or network structure may affect the outcome of a unit, and the goal is to identify sub-populations with varying treatment effects…

Social and Information Networks · Computer Science 2021-11-09 Amir Gilad , Harsh Parikh , Sudeepa Roy , Babak Salimi

Randomized clinical trials are considered the gold standard for informing treatment guidelines, but results may not generalize to real-world populations. Generalizability is hindered by distributional differences in baseline covariates and…

Methodology · Statistics 2025-06-03 Rachael K. Ross , Ivan Diaz , Amy J. Pitts , Elizabeth A. Stuart , Kara E. Rudolph

Algorithms and technologies are essential tools that pervade all aspects of our daily lives. In the last decades, health care research benefited from new computer-based recruiting methods, the use of federated architectures for data…

Computers and Society · Computer Science 2023-01-26 Chiara Criscuolo , Tommaso Dolci , Mattia Salnitri

In this paper we review recent advances in statistical methods for the evaluation of the heterogeneity of treatment effects (HTE), including subgroup identification and estimation of individualized treatment regimens, from randomized…

Methodology · Statistics 2024-10-22 Ilya Lipkovich , David Svensson , Bohdana Ratitch , Alex Dmitrienko

In many psychometric applications, the relationship between the mean of an outcome and a quantitative covariate is too complex to be described by simple parametric functions; instead, flexible nonlinear relationships can be incorporated…

Methodology · Statistics 2023-02-28 Boyu Ren , Stuart R. Lipsitz , Garrett M. Fitzmaurice , Roger D. Weiss

Combining observational and experimental data for causal inference can improve treatment effect estimation. However, many observational data sets cannot be released due to data privacy considerations, so one researcher may not have access…

Methodology · Statistics 2024-08-26 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

Statistics Theory · Mathematics 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

Econometrics · Economics 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

Permutation testing in linear models, where the number of nuisance coefficients is smaller than the sample size, is a well-studied topic. The common approach of such tests is to permute residuals after regressing on the nuisance covariates.…

Methodology · Statistics 2020-10-09 Jesse Hemerik , Magne Thoresen , Livio Finos

The notion of testing for equivalence of two treatments is widely used in clinical trials, pharmaceutical experiments,bioequivalence and quality control. It is essentially approached within the intersection-union (IU) principle. According…

Applications · Statistics 2018-02-07 R. Arboretti , E. Carrozzo , F. Pesarin , L. Salmaso

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

Policy decisions often depend on evidence generated elsewhere. We take a Bayesian decision-theoretic approach to choosing where to experiment to optimize external validity. We frame external validity through a policy lens, developing a…

Exploration of treatment effect heterogeneity (TEH) is an increasingly important aspect of modern statistical analysis for stratified medicine in randomised controlled trials (RCTs) as we start to gather more information on trial…

Methodology · Statistics 2019-01-14 Chieh-Hsi Wu , Chris C. Holmes

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

Applications · Statistics 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

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…