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Analyses of randomised trials are often based on regression models which adjust for baseline covariates, in addition to randomised group. Based on such models, one can obtain estimates of the marginal mean outcome for the population under…

Methodology · Statistics 2017-07-17 Jonathan W. Bartlett

Background: Mendelian randomization (MR) has been widely applied to causal inference in medical research. It uses genetic variants as instrumental variables (IVs) to investigate putative causal relationship between an exposure and an…

Methodology · Statistics 2020-11-04 Linyi Zou , Hui Guo , Carlo Berzuini

Next-generation sequencing technologies now constitute a method of choice to measure gene expression. Data to analyze are read counts, commonly modeled using Negative Binomial distributions. A relevant issue associated with this…

Methodology · Statistics 2014-11-10 Elisabetta Bonafede , Franck Picard , Stéphane Robin , Cinzia Viroli

Dose-finding studies in oncology often include an up-and-down dose transition rule that assigns a dose to each cohort of patients based on accumulating data on dose-limiting toxicity (DLT) events. In making a dose transition decision, a key…

Methodology · Statistics 2025-01-30 Zhiwei Zhang

We consider the general performance of the difference-in-means estimator in an equally-allocated two-arm randomized experiment under common experimental endpoints such as continuous (regression), incidence, proportion, count and uncensored…

Methodology · Statistics 2024-11-07 David Azriel , Abba M. Krieger , Adam Kapelner

In many situations we are interested in modeling real data where the response distribution, even conditionally on the covariates, presents asymmetry and/or heavy/light tails. In these situations, it is more suitable to consider models based…

Methodology · Statistics 2024-06-06 João Victor B. de Freitas , Caio L. N. Azevedo

We propose a novel regression adjustment method designed for estimating distributional treatment effect parameters in randomized experiments. Randomized experiments have been extensively used to estimate treatment effects in various…

Econometrics · Economics 2024-07-24 Undral Byambadalai , Tatsushi Oka , Shota Yasui

Finite mixtures of multivariate normal distributions have been widely used in empirical applications in diverse fields such as statistical genetics and statistical finance. Testing the number of components in multivariate normal mixture…

Statistics Theory · Mathematics 2019-02-11 Hiroyuki Kasahara , Katsumi Shimotsu

Random-effects models are central to meta-analysis, yet the between-study variance is often underestimated when the number of studies is small. In such settings, confidence intervals become unduly narrow and fail to attain the nominal…

Methodology · Statistics 2025-11-18 Keisuke Hanada , Tomoyuki Sugimoto

Complex biological processes are usually experimented along time among a collection of individuals. Longitudinal data are then available and the statistical challenge is to better understand the underlying biological mechanisms. The…

Statistics Theory · Mathematics 2015-06-11 Pierre Barbillon , Célia Barthélémy , Adeline Samson

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

We present a unified three-state model (TSM) framework for evaluating treatment effects in clinical trials in the presence of treatment crossover. Researchers have proposed diverse methodologies to estimate the treatment effect that would…

Methodology · Statistics 2024-09-18 Zile Zhao , Ye Li , Xiaodong Luo , Ray Bai

Medical data often exhibit characteristics that make cluster analysis particularly challenging, such as missing values, outliers, and cluster features like skewness. Typically, such data would need to be preprocessed -- by cleaning outliers…

Methodology · Statistics 2025-12-16 Jason Pillay , Cristina Tortora , Antonio Punzo , Andriette Bekker

The mixed effects model for repeated measures (MMRM) has been widely used for the analysis of longitudinal clinical data collected at a number of fixed time points. We propose a robust extension of the MMRM for skewed and heavy-tailed data…

Methodology · Statistics 2019-08-12 Yongqiang Tang

Three-way data can be conveniently modelled by using matrix variate distributions. Although there has been a lot of work for the matrix variate normal distribution, there is little work in the area of matrix skew distributions. Three matrix…

Methodology · Statistics 2018-08-15 Michael P. B. Gallaugher , Paul D. McNicholas

The density ratio model (DRM) is a semiparametric model that relates the distributions from multiple samples to a nonparametrically defined reference distribution via exponential tilting, with finite-dimensional parameters governing their…

Methodology · Statistics 2025-11-13 James Hugh McVittie , Archer Gong Zhang

In randomized clinical trials, adjustments for baseline covariates at both design and analysis stages are highly encouraged by regulatory agencies. A recent trend is to use a model-assisted approach for covariate adjustment to gain…

Methodology · Statistics 2021-07-14 Ting Ye , Jun Shao , Yanyao Yi , Qingyuan Zhao

This paper focuses on the multivariate linear mixed-effects model, including all the correlations between the random effects when the marginal residual terms are assumed uncorrelated and homoscedastic with possibly different standard…

Methodology · Statistics 2017-05-04 Eric Adjakossa , Grégory Nuel

This paper describes an algorithm for fitting finite mixtures of unrestricted Multivariate Skew t (FM-uMST) distributions. The package EMMIX-uskew implements a closed-form expectation-maximization (EM) algorithm for computing the maximum…

Computation · Statistics 2013-03-29 Sharon X. Lee , Geoffrey J. McLachlan

We study targeted maximum likelihood estimation (TMLE) of the average treatment effect in a semiparametric regression model whose mean function is indexed by a finite-dimensional parameter, while the additive error distribution is left…

Methodology · Statistics 2026-04-20 Mijeong Kim
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