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Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

Monte Carlo (MC) permutation test is considered the gold standard for statistical hypothesis testing, especially when standard parametric assumptions are not clear or likely to fail. However, in modern data science settings where a large…

Methodology · Statistics 2019-05-21 Martin J. Zhang , James Zou , David Tse

This paper takes a look at omnibus tests of goodness of fit in the context of reweighted Anderson-Darling tests and makes threefold contributions. The first contribution is to provide a geometric understanding. It is argued that the test…

Methodology · Statistics 2023-05-11 Chuanhai Liu

Primarily motivated by the drug development process, several publications have now presented methodology for the design of multi-arm multi-stage experiments with normally distributed outcome variables of known variance. Here, we extend…

Methodology · Statistics 2017-12-04 Michael Grayling , James Wason , Adrian Mander

While randomized trials may be the gold standard for evaluating the effectiveness of the treatment intervention, in some special circumstances, single-arm clinical trials utilizing external control may be considered. The causal treatment…

Methodology · Statistics 2025-05-26 Huan Wang , Fei Wu , Yeh-Fong Chen

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

Randomized controlled trials are susceptible to imbalance on covariates predictive of the outcome. Rerandomization and deterministic treatment assignment are two proposed solutions. This paper explores the relationship between…

Methodology · Statistics 2023-10-03 Connor T. Jerzak , Rebecca Goldstein

Two-sample tests for multivariate data and non-Euclidean data are widely used in many fields. Parametric tests are mostly restrained to certain types of data that meets the assumptions of the parametric models. In this paper, we study a…

Methodology · Statistics 2018-05-01 Hao Chen , Xu Chen , Yi Su

Method comparison studies are essential for development in medical and clinical fields. These studies often compare a cheaper, faster, or less invasive measuring method with a widely used one to see if they have sufficient agreement for…

Methodology · Statistics 2019-06-27 Wei Wang , Nan Lin , Jordan D. Oberhaus , Michael S. Avidan

Causal analyses for observational studies are often complicated by covariate imbalances among treatment groups, and matching methodologies alleviate this complication by finding subsets of treatment groups that exhibit covariate balance. It…

Methodology · Statistics 2021-04-26 Zach Branson

In observational studies, contingency tables provide a simple and intuitive approach to study associations between categorical variables. However, any test of association in contingency tables may be biased due to unmeasured confounders.…

Methodology · Statistics 2025-10-10 Elaine K. Chiu , Hyunseung Kang

For testing the statistical significance of a treatment effect, we usually compare between two parts of a population, one is exposed to the treatment, and the other is not exposed to it. Standard parametric and nonparametric two-sample…

Computation · Statistics 2012-11-02 Bikram Karmakar , Kumaresh Dhara , Kushal Kumar Dey , Analabha Basu , Anil Ghosh

Seamless phase II/III trials have become a cornerstone of modern drug development, offering a means to accelerate evaluation while maintaining statistical rigor. However, most existing inference procedures are model-based, designed…

Methodology · Statistics 2025-12-17 Kun Yi , Lucy Xia

We focus on the problem of best-arm identification in a stochastic multi-arm bandit with temporally decreasing variances for the arms' rewards. We model arm rewards as Gaussian random variables with fixed means and variances that decrease…

Machine Learning · Computer Science 2025-02-12 Tamojeet Roychowdhury , Kota Srinivas Reddy , Krishna P Jagannathan , Sharayu Moharir

In this paper, we study sequential testing problems with \emph{overlapping} hypotheses. We first focus on the simple problem of assessing if the mean $\mu$ of a Gaussian distribution is smaller or larger than a fixed $\epsilon>0$; if…

Statistics Theory · Mathematics 2021-11-19 Aurélien Garivier , Emilie Kaufmann

Negative binomial (NB) regression is a popular method for identifying differentially expressed genes in genomics data, such as bulk and single-cell RNA sequencing data. However, NB regression makes stringent parametric and asymptotic…

Methodology · Statistics 2025-01-08 Timothy Barry , Ziang Niu , Eugene Katsevich , Xihong Lin

A benefit of randomized experiments is that covariate distributions of treatment and control groups are balanced on average, resulting in simple unbiased estimators for treatment effects. However, it is possible that a particular…

Methodology · Statistics 2019-02-01 Zach Branson , Luke Miratrix

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

Tie-breaker experimental designs are hybrids of Randomized Controlled Trials (RCTs) and Regression Discontinuity Designs (RDDs) in which subjects with moderate scores are placed in an RCT while subjects with extreme scores are…

Methodology · Statistics 2023-01-24 Dan M. Kluger , Art B. Owen

Given a predictor of outcome derived from a high-dimensional dataset, pre-validation is a useful technique for comparing it to competing predictors on the same dataset. For microarray data, it allows one to compare a newly derived predictor…

Applications · Statistics 2008-07-28 Holger Höfling , Robert Tibshirani