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Mixed outcome endpoints that combine multiple continuous and discrete components to form co-primary, multiple primary or composite endpoints are often employed as primary outcome measures in clinical trials. There are many advantages to…

Methodology · Statistics 2019-12-12 Martina McMenamin , Jessica K. Barrett , Anna Berglind , James M. S. Wason

Structured Latent Attribute Models (SLAMs) are a family of discrete latent variable models widely used in education, psychology, and epidemiology to model multivariate categorical data. A SLAM assumes that multiple discrete latent…

Methodology · Statistics 2021-07-12 Yuqi Gu , Gongjun Xu

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

We propose a method for obtaining maximum likelihood estimates in a model with continuous and binary outcomes. Combinations of left and right censored observations are also naturally modeled in this framework. The model and estimation…

Methodology · Statistics 2015-07-07 Klaus K. Holst , Esben Budtz-Jørgensen , Gitte Moos Knudsen

We consider a high-dimensional multi-outcome regression in which $q,$ possibly dependent, binary and continuous outcomes are regressed onto $p$ covariates. We model the observed outcome vector as a partially observed latent realization from…

Methodology · Statistics 2025-11-05 Soham Ghosh , Sameer K. Deshpande

Existing approaches to modelling antibody concentration data are mostly based on finite mixture models that rely on the assumption that individuals can be divided into two distinct groups: seronegative and seropositive. Here, we challenge…

Methodology · Statistics 2026-04-20 Emanuele Giorgi , Jonas Wallin

Many empirical applications estimate causal effects of a continuous endogenous variable (treatment) using a binary instrument. Estimation is typically done through linear 2SLS. This approach requires a mean treatment change and causal…

Econometrics · Economics 2024-02-28 Yingying Dong , Ying-Ying Lee

Summary points: - This article considers the combination of two binary or two time-to-event endpoints to form the primary composite endpoint for leading a trial. - It discusses the relative efficiency of choosing a composite endpoint over…

Applications · Statistics 2020-01-13 Marta Bofill Roig , Jordi Cortés Martínez , Guadalupe Gómez Melis

Latent variable models provide a powerful framework for incorporating and inferring unobserved factors in observational data. In causal inference, they help account for hidden factors influencing treatment or outcome, thereby addressing…

Machine Learning · Computer Science 2025-08-29 Tetsuro Morimura , Tatsushi Oka , Yugo Suzuki , Daisuke Moriwaki

Recurrent binary outcomes within individuals, such as hospital readmissions, often reflect latent risk processes that evolve over time. Conventional methods like generalized linear mixed models and generalized estimating equations estimate…

Methodology · Statistics 2026-02-24 Niloofar Ramezani , Lori P. Selby , Pascal Nitiema , Jeffrey R. Wilson

Response times collected in computerised assessments provide information about the underlying response process and may exhibit within-person variation over the course of a test. We propose a latent variable model for log response times that…

Methodology · Statistics 2026-05-29 Gabriel Wallin , Nivedita Bhaktha

Non-adherence to assigned treatment is common in randomised controlled trials (RCTs). Recently, there has been an increased interest in estimating causal effects of treatment received, for example the so-called local average treatment…

Methodology · Statistics 2018-12-05 Karla DiazOrdaz , James Carpenter

Latent class models have been successfully used to handle complex datasets in different disciplines. For longitudinal outcomes, we often get a trajectory of the outcome for each individual, and on that basis, we cluster them for a powerful…

Methodology · Statistics 2025-09-08 Chitradipa Chakraborty , Kiranmoy Das

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

In longitudinal studies, time-varying covariates are often endogenous, meaning their values depend on both their own history and that of the outcome variable. This violates key assumptions of Generalized Linear Mixed Effects Models (GLMMs),…

Many estimators of the average effect of a treatment on an outcome require estimation of the propensity score, the outcome regression, or both. It is often beneficial to utilize flexible techniques such as semiparametric regression or…

Methodology · Statistics 2019-05-14 Cheng Ju , David Benkeser , Mark J. van der Laan

Non-terminal events can represent a meaningful change in a patient's life. Thus, better understanding and predicting their occurrence can bring valuable information to individuals. In a context where longitudinal markers could inform these…

Methodology · Statistics 2025-01-16 Juliette Ortholand , Stanley Durrleman , Sophie Tezenas du Montcel

Finite mixture models are widely used in econometric analyses to capture unobserved heterogeneity. This paper shows that maximum likelihood estimation of finite mixtures of parametric densities can suffer from substantial finite-sample bias…

Methodology · Statistics 2026-02-04 Raphaël Langevin

Joint models for longitudinal and survival data have gained a lot of attention in recent years, with the development of myriad extensions to the basic model, including those which allow for multivariate longitudinal data, competing risks…

Methodology · Statistics 2020-03-09 Katya Mauff , Ewout Steyerberg , Isabella Kardys , Eric Boersma , Dimitris Rizopoulos

Composite endpoints, which combine two or more distinct outcomes, are frequently used in clinical trials to enhance the event rate and improve the statistical power. In the recent literature, the while-alive cumulative frequency measure…

Methodology · Statistics 2025-11-14 Xi Fang , Hajime Uno , Fan Li
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