Purely nonparametric methods are developed for general two-sample problems in which each experimental unit may have an individual number of possibly correlated replicates. In particular, equality of the variances, or higher moments, of the distributions of the data is not assumed, even under the null hypothesis of no treatment effect. Thus, a solution for the so-called nonparametric Behrens-Fisher problem is proposed for such models. The methods are valid for metric, count, ordered categorical, and even dichotomous data in a unified way. Point estimators of the treatment effects as well as their asymptotic distributions will be studied in detail. For small sample sizes, the distributions of the proposed test statistics are approximated using Satterthwaite-Welch-type t-approximations. Extensive simulation studies show favorable performance of the new methods, in particular, in small sample size situations. A real data set illustrates the application of the proposed methods.
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http://dx.doi.org/10.1002/sim.8343 | DOI Listing |
Stat Med
December 2024
Institute of Biometry and Clinical Epidemiology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
In many trials and experiments, subjects are not only observed once but multiple times, resulting in a cluster of possibly correlated observations (e.g., brain regions per patient).
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September 2024
Department of Epidemiology and Biostatistics, Western University, London, Ontario, Canada.
A nonparametric method proposed by DeLong et al in 1988 for comparing areas under correlated receiver operating characteristic curves is used widely in practice. However, the DeLong method as implemented in popular software quietly deletes individuals with any missing values, yielding potentially invalid and/or inefficient results. We simplify the DeLong algorithm using ranks and extend it to accommodate missing data by using a mixed model approach for multivariate data.
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October 2022
Charité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Berlin, Germany.
Late phase clinical trials are occasionally planned with one or more interim analyses to allow for early termination or adaptation of the study. While extensive theory has been developed for the analysis of ordered categorical data in terms of the Wilcoxon-Mann-Whitney test, there has been comparatively little discussion in the group sequential literature on how to provide repeated confidence intervals and simple power formulas to ease sample size determination. Dealing more broadly with the nonparametric Behrens-Fisher problem, we focus on the comparison of two parallel treatment arms and show that the Wilcoxon-Mann-Whitney test, the Brunner-Munzel test, as well as a test procedure based on the log win odds, a modification of the win ratio, asymptotically follow the canonical joint distribution.
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January 2022
14903Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, Berlin, Germany.
We develop purely nonparametric methods for the analysis of repeated measures designs with missing values. Hypotheses are formulated in terms of purely nonparametric treatment effects. In particular, data can have different shapes even under the null hypothesis and therefore, a solution to the nonparametric Behrens-Fisher problem in repeated measures designs will be presented.
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July 2021
School of Statistics, Capital University of Economics and Business, Beijing, China.
For a nonparametric Behrens-Fisher problem, a directional-sum test is proposed based on division-combination strategy. A one-layer wild bootstrap procedure is given to calculate its statistical significance. We conduct simulation studies with data generated from lognormal, and Laplace distributions to show that the proposed test can control the type I error rates properly and is more powerful than the existing rank-sum and maximum-type tests under most of the considered scenarios.
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