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Misclassification in binary outcomes is not uncommon and statistical methods to investigate its impact on policy-driving study results are lacking. While misclassifying binary outcomes is a statistically ubiquitous phenomena, we focus on…

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical…

Methodology · Statistics 2026-03-25 Yuki Ohnishi , Michael J. Daniels , Lei Yang , Fan Li

A mixture of multivariate contaminated normal (MCN) distributions is a useful model-based clustering technique to accommodate data sets with mild outliers. However, this model only works when fitted to complete data sets, which is often not…

Methodology · Statistics 2020-12-11 Hung Tong , Cristina Tortora

Randomization, as a key technique in clinical trials, can eliminate sources of bias and produce comparable treatment groups. In randomized experiments, the treatment effect is a parameter of general interest. Researchers have explored the…

Methodology · Statistics 2023-12-05 Fuyi Tu , Wei Ma , Hanzhong Liu

Multiple imputation (MI) is a popular method for dealing with missing values. One main advantage of MI is to separate the imputation phase and the analysis one. However, both are related since they are based on distribution assumptions that…

Methodology · Statistics 2021-06-09 Vincent Audigier , Ndèye Niang , Matthieu Resche-Rigon

In Randomised Controlled Trials (RCT) with treatment non-compliance, instrumental variable approaches are used to estimate complier average causal effects. We extend these approaches to cost-effectiveness analyses, where methods need to…

Methodology · Statistics 2016-12-02 Karla DiazOrdaz , Angelo Franchini , Richard Grieve

Gaussian Mixture models (GMMs) are a powerful tool for clustering, classification and density estimation when clustering structures are embedded in the data. The presence of missing values can largely impact the GMMs estimation process,…

Machine Learning · Statistics 2020-06-05 Alessio Serafini , Thomas Brendan Murphy , Luca Scrucca

When the difference between treatments in a clinical trial is estimated by a difference in means, then it is well known that randomization ensures unbiassed estimation, even if no account is taken of important baseline covariates. However,…

Statistics Theory · Mathematics 2014-07-22 J. N. S. Matthews , Nuri H. Badi

We propose a novel approach for modeling multivariate longitudinal data in the presence of unobserved heterogeneity for the analysis of the Health and Retirement Study (HRS) data. Our proposal can be cast within the framework of linear…

Methodology · Statistics 2015-09-17 Laura Anderlucci , Cinzia Viroli

The primary analysis for longitudinal randomized controlled trials (RCTs) often compares treatment groups at the last timepoint, referred to as the landmark time. Assuming data are normally distributed and missing at random, the mixed model…

Methodology · Statistics 2026-03-18 Guangyong Zou , Shi-Fang Qui , Joshua Zou , Emma Davies Smith , Yun-Hee Choi , Yuhan Bi

A model based clustering procedure for data of mixed type, clustMD, is developed using a latent variable model. It is proposed that a latent variable, following a mixture of Gaussian distributions, generates the observed data of mixed type.…

Methodology · Statistics 2015-11-06 Damien McParland , Isobel Claire Gormley

Background: Stepped wedge cluster randomized trials (SW-CRTs) involve sequential measurements within clusters over time. Initially, all clusters start in the control condition before crossing over to the intervention on a staggered…

Methodology · Statistics 2026-01-21 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Covariate-adaptive randomization is widely used in clinical trials to balance prognostic factors, and regression adjustments are often adopted to further enhance the estimation and inference efficiency. In practice, the covariates may…

Methodology · Statistics 2025-08-15 Wanjia Fu , Yingying Ma , Hanzhong Liu

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent…

Methodology · Statistics 2025-09-19 Fan Li , Jiaqi Tong , Xi Fang , Chao Cheng , Brennan C. Kahan , Bingkai Wang

Design-based causal inference, also known as randomization-based or finite-population causal inference, is one of the most widely used causal inference frameworks, largely due to the merit that its validity can be guaranteed by study design…

Methodology · Statistics 2025-05-27 Siyu Heng , Jiawei Zhang , Yang Feng

Identifying causal treatment (or exposure) effects in observational studies requires the data to satisfy the unconfoundedness assumption which is not testable using the observed data. With sensitivity analysis, one can determine how the…

Methodology · Statistics 2023-01-31 Yang Ou , Lu Tang , Chung-Chou H. Chang

This paper tackles the problem of robust covariance matrix estimation when the data is incomplete. Classical statistical estimation methodologies are usually built upon the Gaussian assumption, whereas existing robust estimation ones assume…

Multivariate longitudinal data of mixed-type are increasingly collected in many science domains. However, algorithms to cluster this kind of data remain scarce, due to the challenge to simultaneously model the within- and between-time…

Machine Learning · Statistics 2025-09-16 Francesco Amato , Julien Jacques

We propose two approaches for selecting variables in latent class analysis (i.e.,mixture model assuming within component independence), which is the common model-based clustering method for mixed data. The first approach consists in…

Computation · Statistics 2017-03-08 Matthieu Marbac , Mohammed Sedki

The parallel cluster randomized trial with baseline (PB-CRT) is a common variant of the standard parallel cluster randomized trial (P-CRT). We define two natural estimands in the context of PB-CRTs with informative cluster sizes, the…

Methodology · Statistics 2025-05-01 Kenneth Menglin Lee , Fan Li