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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 extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

Understanding how treatment effects vary across patient characteristics is essential for personalized medicine, yet randomized controlled trials (RCTs) are often underpowered to detect heterogeneous treatment effects (HTEs). We propose a…

Methodology · Statistics 2025-07-22 Amir Asiaee , Chiara Di Gravio , Cole Beck , Yuting Mei , Samhita Pal , Jared D. Huling

In a novel approach to the multiple testing problem, Efron (2004; 2007) formulated estimators of the distribution of test statistics or nominal p-values under a null distribution suitable for modeling the data of thousands of unaffected…

Methodology · Statistics 2012-10-30 David R. Bickel

Utilizing established risk factors and prognostic models can often improve the construction of a newer risk model that uses novel biomarkers in a smaller, internal study. However, directly borrowing information from an established…

Methodology · Statistics 2026-03-12 Nicholas C. Henderson

For some variants of regression models, including partial, measurement error or error-in-variables, latent effects, semi-parametric and otherwise corrupted linear models, the classical parametric tests generally do not perform well. Various…

Statistics Theory · Mathematics 2015-03-25 Pranab K. Sen , Jana Jureckova , Jan Picek

In randomized clinical trials (RCTs), the accurate estimation of marginal treatment effects is crucial for determining the efficacy of interventions. Enhancing the statistical power of these analyses is a key objective for statisticians.…

Methodology · Statistics 2026-01-30 Emilie Højbjerre-Frandsen , Mark J. van der Laan , Alejandro Schuler

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…

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 observational studies of treatment effects, matched samples are created so treated and control groups are similar in terms of observable covariates. Traditionally such matched samples consist of matched pairs. If a pair match fails to…

Methodology · Statistics 2014-10-22 Luke Keele , Sam Pimentel , Frank Yoon

Estimating heterogeneous treatment effects has become increasingly important in many fields and life and death decisions are now based on these estimates: for example, selecting a personalized course of medical treatment. Recently, a…

Methodology · Statistics 2019-04-01 Sören R. Künzel , Simon J. S. Walter , Jasjeet S. Sekhon

Rerandomization enforces covariate balance across treatment groups in the design stage of experiments. Despite its intuitive appeal, its theoretical justification remains unsatisfying because its benefits of improving efficiency for…

Statistics Theory · Mathematics 2025-05-05 Xin Lu , Peng Ding

Covariate adjustment is a general method for improving precision when estimating treatment effects in randomized trials and is recommended by the FDA in its 2023 guidance when baseline variables are prognostic for the primary outcome. We…

This paper provides a design-based framework for variance (bound) estimation in experimental analysis. Results are applicable to virtually any combination of experimental design, linear estimator (e.g., difference-in-means, OLS, WLS) and…

Methodology · Statistics 2021-09-21 Joel A. Middleton

Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

Online experiments such as Randomised Controlled Trials (RCTs) or A/B-tests are the bread and butter of modern platforms on the web. They are conducted continuously to allow platforms to estimate the causal effect of replacing system…

Machine Learning · Computer Science 2023-04-24 Olivier Jeunen

Randomization is a basis for the statistical inference of treatment effects without strong assumptions on the outcome-generating process. Appropriately using covariates further yields more precise estimators in randomized experiments. R. A.…

Statistics Theory · Mathematics 2020-01-03 Xinran Li , Peng Ding

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods…

Methodology · Statistics 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

While the evaluation of explanations is an important step towards trustworthy models, it needs to be done carefully, and the employed metrics need to be well-understood. Specifically model randomization testing is often overestimated and…

We propose generalized additive partial linear models for complex data which allow one to capture nonlinear patterns of some covariates, in the presence of linear components. The proposed method improves estimation efficiency and increases…

Statistics Theory · Mathematics 2014-05-26 Li Wang , Lan Xue , Annie Qu , Hua Liang