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A randomized trial and an analysis of observational data designed to emulate the trial sample observations separately, but have the same eligibility criteria, collect information on some shared baseline covariates, and compare the effects…

Methodology · Statistics 2022-03-29 Issa J. Dahabreh , Jon A. Steingrimsson , James M. Robins , Miguel A. Hernán

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…

Methodology · Statistics 2023-01-23 Lars Lau Raket

Adaptive enrichment allows for pre-defined patient subgroups of interest to be investigated throughout the course of a clinical trial. Many trials which measure a long-term time-to-event endpoint often also routinely collect repeated…

Methodology · Statistics 2024-02-26 Abigail J. Burdon , Richard D. Baird , Thomas Jaki

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

Methodology · Statistics 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

Missing data arise in most applied settings and are ubiquitous in electronic health records (EHR). When data are missing not at random (MNAR) with respect to measured covariates, sensitivity analyses are often considered. These post-hoc…

Methodology · Statistics 2023-07-11 Alexander W. Levis , Rajarshi Mukherjee , Rui Wang , Heidi Fischer , Sebastien Haneuse

This article aims to study efficient/trace optimal designs for crossover trials with multiple responses recorded from each subject in the time periods. A multivariate fixed effects model is proposed with direct and carryover effects…

Methodology · Statistics 2024-06-04 Shubham Niphadkar , Siuli Mukhopadhyay

This paper introduces an overidentification test of two alternative assumptions to identify the average treatment effect on the treated in a two-period panel data setting: unconfoundedness and common trends. Under the unconfoundedness…

Econometrics · Economics 2024-06-25 Martin Huber , Eva-Maria Oeß

We study causal inference in randomized experiments (or quasi-experiments) following a $2\times 2$ factorial design. There are two treatments, denoted $A$ and $B$, and units are randomly assigned to one of four categories: treatment $A$…

Econometrics · Economics 2024-12-12 Mate Kormos , Robert P. Lieli , Martin Huber

Nonlinear mixed effects models represent a powerful tool to simultaneously analyze data from several individuals. In this study a compartmental model of leucine kinetics is examined and extended with a stochastic differential equation to…

Quantitative Methods · Quantitative Biology 2011-01-05 Martin Berglund , Mikael Sunnåker , Martin Adiels , Mats Jirstrand , Bernt Wennberg

When estimating treatment effects, the golden standard is to conduct a randomized experiment and then contrast outcomes associated with the treatment group and the control group. However, in many cases, randomized experiments are either…

Methodology · Statistics 2023-06-08 Kevin Han

This paper addresses one of the most prevalent problems encountered by political scientists working with difference-in-differences (DID) design: missingness in panel data. A common practice for handling missing data, known as complete case…

Methodology · Statistics 2024-12-02 Sooahn Shin

Multivariate bounded discrete data arises in many fields. In the setting of dementia studies, such data is collected when individuals complete neuropsychological tests. We outline a modeling and inference procedure that can model the joint…

Methodology · Statistics 2026-02-10 Daniel Suen , Yen-Chi Chen

When estimating heterogeneous treatment effects, missing outcome data can complicate treatment effect estimation, causing certain subgroups of the population to be poorly represented. In this work, we discuss this commonly overlooked…

Machine Learning · Statistics 2025-04-15 Matthew Pryce , Karla Diaz-Ordaz , Ruth H. Keogh , Stijn Vansteelandt

Ecological Momentary Assessments (EMA) capture real-time thoughts and behaviors in natural settings, producing rich longitudinal data for statistical and physiological analyses. However, the robustness of these analyses can be compromised…

Methodology · Statistics 2023-11-21 Yiheng Wei , Donald Hedeker

Bivariate linear mixed models are useful when analyzing longitudinal data of two associated markers. In this paper, we present a bivariate linear mixed model including random effects or first-order auto-regressive process and independent…

How should researchers analyze randomized experiments in which the main outcome is latent and measured in multiple ways but each measure contains some degree of error? We first identify a critical study-specific noncomparability problem in…

Econometrics · Economics 2026-01-13 Jiawei Fu , Donald P. Green

Joint modelling of longitudinal observations and event times continues to remain a topic of considerable interest in biomedical research. For example, in HIV studies, the longitudinal bio-marker such as CD4 cell count in a patient's blood…

Methodology · Statistics 2024-07-19 Srimanti Dutta , Arindom Chakraborty , Dipankar Bandyopadhyay

Longitudinal electronic health record (EHR) data offer opportunities to study biomarker trajectories; however, association estimates-the primary inferential target-from standard models designed for regular observation times may be biased by…

Methodology · Statistics 2026-02-18 Cheng-Han Yang , Xu Shi , Bhramar Mukherjee

We propose a Bayesian nonparametric (BNP) approach to causal inference using observational data consisting of outcome, treatment, and a set of confounders. The conditional distribution of the outcome given treatment and confounders is…

Methodology · Statistics 2025-12-01 Yongseok Hur , Joonhyuk Jung , Juhee Lee
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