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A common goal in clinical trials is to conduct tests on estimated treatment effects adjusted for covariates such as age or sex. Analysis of Covariance (ANCOVA) is often used in these scenarios to test the global null hypothesis of no…

Methodology · Statistics 2024-04-24 Matthias Becher , Ludwig A. Hothorn , Frank Konietschke

Considerable interest has recently been focused on studying multiple phenotypes simultaneously in both epidemiological and genomic studies, either to capture the multidimensionality of complex disorders or to understand shared etiology of…

Methodology · Statistics 2015-11-26 Denis Agniel , Katherine P. Liao , Tianxi Cai

Functional data analysis is becoming increasingly popular to study data from real-valued random functions. Nevertheless, there is a lack of multiple testing procedures for such data. These are particularly important in factorial designs to…

Methodology · Statistics 2024-06-04 Merle Munko , Marc Ditzhaus , Markus Pauly , Łukasz Smaga

One fundamental statistical question for research areas such as precision medicine and health disparity is about discovering effect modification of treatment or exposure by observed covariates. We propose a semiparametric framework for…

Methodology · Statistics 2020-08-04 Muxuan Liang , Menggang Yu

Clinical trials often involve the assessment of multiple endpoints to comprehensively evaluate the efficacy and safety of interventions. In the work, we consider a global nonparametric testing procedure based on multivariate rank for the…

Methodology · Statistics 2023-06-29 Kexuan Li , Lingli Yang , Shaofei Zhao , Susie Sinks , Luan Lin , Peng Sun

The causal effects of continuous treatments are often characterized through the average dose response function, which is challenging to estimate from observational data due to confounding and positivity violations. Modified treatment…

Methodology · Statistics 2025-01-22 Ziren Jiang , Jared D. Huling

The analysis of multiple time-to-event outcomes in a randomised controlled clinical trial can be accomplished with exisiting methods. However, depending on the characteristics of the disease under investigation and the circumstances in…

Methodology · Statistics 2026-03-02 Moritz Fabian Danzer , Andreas Faldum , Thorsten Simon , Barbara Hero , Rene Schmidt

In randomized trials with continuous-valued outcomes the goal is often to estimate the difference in average outcomes between two treatment groups. However, the outcome in some trials is longitudinal, meaning that multiple measurements of…

Methodology · Statistics 2021-09-21 Alejandro Schuler

Multivariate analysis of variance (MANOVA) is a powerful and versatile method to infer and quantify main and interaction effects in metric multivariate multi-factor data. It is, however, neither robust against change in units nor a…

Statistics Theory · Mathematics 2018-02-13 Dennis Dobler , Sarah Friedrich , Markus Pauly

Multivariate (average) equivalence testing is widely used to assess whether the means of two conditions of interest are `equivalent' for different outcomes simultaneously. The multivariate Two One-Sided Tests (TOST) procedure is typically…

Multivariate analysis-of-variance (MANOVA) is a well established tool to examine multivariate endpoints. While classical approaches depend on restrictive assumptions like normality and homogeneity, there is a recent trend to more general…

Statistics Theory · Mathematics 2022-11-29 Marléne Baumeister , Marc Ditzhaus , Markus Pauly

A common problem in clinical trials is to test whether the effect of an explanatory variable on a response of interest is similar between two groups, e.g. patient or treatment groups. In this regard, similarity is defined as equivalence up…

Methodology · Statistics 2024-01-12 Niklas Hagemann , Giampiero Marra , Frank Bretz , Kathrin Möllenhoff

The multivariate coefficient of variation (MCV) is an attractive and easy-to-interpret effect size for the dispersion in multivariate data. Recently, the first inference methods for the MCV were proposed by Ditzhaus and Smaga (2022) for…

Methodology · Statistics 2023-01-31 Marc Ditzhaus , Łukasz Smaga

Comparing the mean vectors across different groups is a cornerstone in the realm of multivariate statistics, with quadratic forms commonly serving as test statistics. However, when the overall hypothesis is rejected, identifying specific…

Methodology · Statistics 2025-06-04 Paavo Sattler , Markus Pauly , Merle Munko

In learning-phase clinical trials in drug development, adaptive designs can be efficient and highly informative when used appropriately. In this article, we extend the multiple comparison procedures with modeling techniques (MCP-Mod)…

Methodology · Statistics 2021-08-02 Shiyang Ma , Michael P. McDermott

In applied research, it is often sensible to account for one or several covariates when testing for differences between multivariate means of several groups. However, the "classical" parametric multivariate analysis of covariance (MANCOVA)…

Methodology · Statistics 2020-04-28 Georg Zimmermann , Markus Pauly , Arne C. Bathke

Multivariate outcomes are common in pragmatic cluster randomized trials. While sample size calculation procedures for multivariate outcomes exist under parallel assignment, none have been developed for a stepped wedge design. In this…

Methodology · Statistics 2022-12-05 Kendra Davis-Plourde , Monica Taljaard , Fan Li

In the evaluation of treatment effects, it is of major policy interest to know if the treatment is beneficial for some and harmful for others, a phenomenon known as qualitative interaction. We formulate this question as a multiple testing…

Methodology · Statistics 2017-08-29 Qingyuan Zhao , Dylan S. Small , Weijie Su

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

As a common step in refining their scientific inquiry, investigators are often interested in performing some screening of a collection of given statistical hypotheses. For example, they may wish to determine whether any one of several…

Methodology · Statistics 2022-03-04 Adam Elder , Marco Carone , Peter Gilbert , Alex Luedtke
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