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Computer simulations have become an important tool across the biomedical sciences and beyond. For many important problems several different models or hypotheses exist and choosing which one best describes reality or observed data is not…

Quantitative Methods · Quantitative Biology 2010-01-20 Tina Toni , Michael P. H. Stumpf

Determination of sample size is critical, however not easy to do. Sample size defined as the number of observations in a sample should be big enough to have a high likelihood of detecting a true difference between groups. Practical…

Methodology · Statistics 2025-02-28 Hoi-Jeong Lim

Biomedical data are widely accepted in developing prediction models for identifying a specific tumor, drug discovery and classification of human cancers. However, previous studies usually focused on different classifiers, and overlook the…

Quantitative Methods · Quantitative Biology 2019-11-05 Shigang Liu , Jun Zhang , Yang Xiang , Wanlei Zhou , Dongxi Xiang

The increasing interest in subpopulation analysis has led to the development of various new trial designs and analysis methods in the fields of personalized medicine and targeted therapies. In this paper, subpopulations are defined in terms…

Methodology · Statistics 2020-12-01 Roland Gerard Gera , Tim Friede

Missing data is a common issue in many biomedical studies. Under a paired design, some subjects may have missing values in either one or both of the conditions due to loss of follow-up, insufficient biological samples, etc. Such partially…

A Bayesian design is given by maximising an expected utility over a design space. The utility is chosen to represent the aim of the experiment and its expectation is taken with respect to all unknowns: responses, parameters and/or models.…

Methodology · Statistics 2019-01-16 Antony M. Overstall , James M. McGree

There is a growing trend in molecular and synthetic biology of using mechanistic (non machine learning) models to design biomolecular networks. Once designed, these networks need to be validated by experimental results to ensure the…

Quantitative Methods · Quantitative Biology 2020-11-26 Ruby Sedgwick , John Goertz , Molly Stevens , Ruth Misener , Mark van der Wilk

Two semimetrics on probability distributions are proposed, given as the sum of differences of expectations of analytic functions evaluated at spatial or frequency locations (i.e, features). The features are chosen so as to maximize the…

Machine Learning · Statistics 2016-10-31 Wittawat Jitkrittum , Zoltan Szabo , Kacper Chwialkowski , Arthur Gretton

Modern studies increasingly leverage outcomes predicted by machine learning and artificial intelligence (AI/ML) models, and recent work, such as prediction-powered inference (PPI), has developed valid downstream statistical inference…

Methodology · Statistics 2026-03-18 Yiqun T. Chen , Moran Guo , Shengy Li

Experimental designs with hierarchically-structured errors are pervasive in many biomedical areas; it is important to take into account this hierarchical architecture in order to account for the dispersion and make reliable inferences from…

Applications · Statistics 2023-05-05 Elyas Mouhou , Vincent Audigier , Josselin Noirel

The ratio of two densities provides a direct characterization of their differences. We consider the two-sample comparison problem by estimating this ratio given i.i.d. observations from two distributions. To this end, we propose additive…

Methodology · Statistics 2026-04-23 Naoki Awaya , Yuliang Xu , Li Ma

This paper proposes a specification test for the conventional distributional assumptions of error terms in binary choice models, focusing on its tail properties. Based on extreme value theory, we first establish that the tail index of the…

Econometrics · Economics 2026-03-31 Ting Ji , Laura Liu , Yulong Wang , Jiahe Xing

Accurate diagnostic tests are essential for effective screening and treatment. However, individual biomarkers often fail to provide sufficient diagnostic accuracy, as they typically capture only one aspect of the complex disease process.…

Methodology · Statistics 2025-07-08 Ainesh Sewak , Sandra Siegfried , Torsten Hothorn

Statistical samples, in order to be representative, have to be drawn from a population in a random and unbiased way. Nevertheless, it is common practice in the field of model-based diagnosis to make estimations from (biased) best-first…

Artificial Intelligence · Computer Science 2022-08-05 Patrick Rodler , Fatima Elichanova

We address the asymptotic and approximate distributions of a large class of test statistics with quadratic forms used in association studies. The statistics of interest do not necessarily follow a chi-square distribution and take the…

Applications · Statistics 2009-09-24 Liping Tong , Jie Yang , Richard S. Cooper

In basket trials a treatment is investigated in several subgroups. They are primarily used in oncology in early clinical phases as single-arm trials with a binary endpoint. For their analysis primarily Bayesian methods have been suggested,…

Methodology · Statistics 2024-02-16 Lukas Baumann , Lukas Sauer , Meinhard Kieser

Data-driven most powerful tests are statistical hypothesis decision-making tools that deliver the greatest power against a fixed null hypothesis among all corresponding data-based tests of a given size. When the underlying data…

Statistics Theory · Mathematics 2023-03-15 Albert Vexler , Alan D. Hutson

The choice of sample size in the context of co-primary endpoints for a randomised trial is discussed. Current guidance can leave endpoints with unequal marginal power. A method is provided to achieve equal marginal power by using the…

Methodology · Statistics 2026-02-23 Simon Bond

Accurate power and sample size (PSS) calculations are essential for designing studies that use quasi-likelihood (QL) models, which extend generalized linear models (GLMs) to settings where the full distribution of the outcome is not…

Methodology · Statistics 2026-03-03 Shijie Yuan , Amy Cochran , Paul Rathouz

Replication studies are essential for assessing the credibility of claims from original studies. A critical aspect of designing replication studies is determining their sample size; a too small sample size may lead to inconclusive studies…

Methodology · Statistics 2023-08-14 Samuel Pawel , Guido Consonni , Leonhard Held
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