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When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate…

Methodology · Statistics 2023-07-04 Chia-Rui Chang , Yue Song , Fan Li , Rui Wang

Mediation analysis is an important statistical tool in many research fields, where the joint significance test is widely utilized for examining mediation effects. Nevertheless, the limitation of this mediation testing method stems from its…

Methodology · Statistics 2024-09-06 Haixiang Zhang

Adaptive interventions (AIs) are increasingly becoming popular in medical and behavioral sciences. An AI is a sequence of individualized intervention options that specify for whom and under what conditions different intervention options…

Applications · Statistics 2018-12-18 Palash Ghosh , Inbal Nahum-Shani , Bonnie Spring , Bibhas Chakraborty

Beyond conditional average treatment effects, treatments may impact the entire outcome distribution in covariate-dependent ways, for example, by altering the variance or tail risks for specific subpopulations. We propose a novel estimand to…

Machine Learning · Statistics 2026-03-18 Saksham Jain , Alex Luedtke

The comparison of different medical treatments from observational studies or across different clinical studies is often biased by confounding factors such as systematic differences in patient demographics or in the inclusion criteria for…

Methodology · Statistics 2025-05-15 Ekkehard Glimm , Lillian Yau

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

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

We present a general framework for using existing data to estimate the efficiency gain from using a covariate-adjusted estimator of a marginal treatment effect in a future randomized trial. We describe conditions under which it is possible…

Methodology · Statistics 2021-05-03 Xiudi Li , Sijia Li , Alex Luedtke

This paper studies inference on the average treatment effect in experiments in which treatment status is determined according to "matched pairs" and it is additionally desired to adjust for observed, baseline covariates to gain further…

Econometrics · Economics 2023-10-20 Yuehao Bai , Liang Jiang , Joseph P. Romano , Azeem M. Shaikh , Yichong Zhang

Two-sample tests are important in statistics and machine learning, both as tools for scientific discovery as well as to detect distribution shifts. This led to the development of many sophisticated test procedures going beyond the standard…

Machine Learning · Computer Science 2023-01-18 Jonas M. Kübler , Vincent Stimper , Simon Buchholz , Krikamol Muandet , Bernhard Schölkopf

While multiple testing procedures have been the focus of much statistical research, an important facet of the problem is how to deal with possible confounding. Procedures have been developed by authors in genetics and statistics. In this…

Methodology · Statistics 2008-12-18 Debashis Ghosh

We present a new way of testing ordered hypotheses against all alternatives which overpowers the classical approach both in simplicity and statistical power. Our new method tests the constrained likelihood ratio statistic against the…

Methodology · Statistics 2018-06-26 Diaa Al Mohamad , Jelle J. Goeman , Erik W. van Zwet , Eric A. Cator

A {\it pure significance test} (PST) tests a simple null hypothesis $H_f:Y\sim f$ {\it without specifying an alternative hypothesis} by rejecting $H_f$ for {\it small} values of $f(Y)$. When the sample space supports a proper uniform pmf…

Statistics Theory · Mathematics 2024-04-23 Michael D. Perlman

A number of biomedical problems require performing many hypothesis tests, with an attendant need to apply stringent thresholds. Often the data take the form of a series of predictor vectors, each of which must be compared with a single…

Methodology · Statistics 2014-05-13 Yi-Hui Zhou , Fred Wright

In clinical studies upon which decisions are based there are two types of errors that can be made: a type I error arises when the decision is taken to declare a positive outcome when the truth is in fact negative, and a type II error arises…

Methodology · Statistics 2024-09-19 Andrew P Grieve

Some classical uncertainty quantification problems require the estimation of multiple expectations. Estimating all of them accurately is crucial and can have a major impact on the analysis to perform, and standard existing Monte Carlo…

Methodology · Statistics 2022-12-02 Julien Demange-Chryst , François Bachoc , Jérôme Morio

We often seek to estimate the causal effect of an exposure on a particular outcome in both randomized and observational settings. One such estimation method is the covariate-adjusted residuals estimator, which was designed for individually…

Methodology · Statistics 2019-10-28 Stephen A. Lauer , Nicholas G. Reich , Laura B. Balzer

Time-to-event endpoints show an increasing popularity in phase II cancer trials. The standard statistical tool for such one-armed survival trials is the one-sample log-rank test. Its distributional properties are commonly derived in the…

Methodology · Statistics 2026-03-02 Moritz Fabian Danzer , Andreas Faldum , Rene Schmidt

This paper considers the optimal modification of the likelihood ratio test (LRT) for the equality of two high-dimensional covariance matrices. The classical LRT is not well defined when the dimensions are larger than or equal to one of the…

Statistics Theory · Mathematics 2018-04-06 Qiuyan Zhang , Jiang Hu , Zhidong Bai

Consider two random variables contaminated by two unknown transformations. The aim of this paper is to test the equality of those transformations. Two cases are distinguished: first, the two random variables have known distributions.…

Methodology · Statistics 2011-11-01 Mohamed Boutahar , Denys Pommeret