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Linear mixed effects are considered excellent predictors of cluster-level parameters in various domains. However, previous work has shown that their performance can be seriously affected by departures from modelling assumptions. Since the…

Methodology · Statistics 2022-07-27 Katarzyna Reluga , Stefan Sperlich

Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

Applications · Statistics 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

Meta-analyses frequently include trials that report multiple effect sizes based on a common set of study participants. These effect sizes will generally be correlated. Cluster-robust variance-covariance estimators are a fruitful approach…

Methodology · Statistics 2022-03-07 Thilo Welz , Wolfgang Viechtbauer , Markus Pauly

Selective inference aims at providing valid inference after a data-driven selection of models or hypotheses. It is essential to avoid overconfident results and replicability issues. While significant advances have been made in this area for…

Methodology · Statistics 2025-03-14 Matteo D'Alessandro , Magne Thoresen

Accurately estimating treatment effects over time is crucial in fields such as precision medicine, epidemiology, economics, and marketing. Many current methods for estimating treatment effects over time assume that all confounders are…

Machine Learning · Statistics 2025-11-11 Mouad El Bouchattaoui , Myriam Tami , Benoit Lepetit , Paul-Henry Cournède

Background: Randomized controlled trials are often used to inform policy and practice for broad populations. The average treatment effect (ATE) for a target population, however, may be different from the ATE observed in a trial if there are…

We propose simple inferential approaches for the fixed effects in complex functional mixed effects models. We estimate the fixed effects under the independence of functional residuals assumption and then bootstrap independent units (e.g.…

Methodology · Statistics 2016-07-06 So Young Park , Ana-Maria Staicu , Luo Xiao , Ciprian Crainiceanu

This paper proposes a framework that incorporates the two-way fixed effects model as a special case to conduct causal inference with a continuous treatment. Treatments are allowed to change over time and potential outcomes are dependent on…

Methodology · Statistics 2025-07-01 Zhiguo Xiao , Peikai Wu

Cluster randomized trials (CRTs) are popular in public health and in the social sciences to evaluate a new treatment or policy where the new policy is randomly allocated to clusters of units rather than individual units. CRTs often feature…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

In this article, we aim to provide a general and complete understanding of semi-supervised (SS) causal inference for treatment effects. Specifically, we consider two such estimands: (a) the average treatment effect and (b) the quantile…

Methodology · Statistics 2024-08-15 Abhishek Chakrabortty , Guorong Dai

In causal inference about two treatments, Conditional Average Treatment Effects (CATEs) play an important role as a quantity representing an individualized causal effect, defined as a difference between the expected outcomes of the two…

Econometrics · Economics 2023-10-26 Masahiro Kato , Masaaki Imaizumi

This paper considers how to classify the effects of interventions in causal models for outcomes and exposures observed over time. First, we demonstrate the limitations of the most common uses of potential outcomes and causal directed…

Methodology · Statistics 2026-05-29 Russell Steele , Naftali Weinberger , Tess Baker , Ian Shrier

Recently, there has been great interest in estimating the conditional average treatment effect using flexible machine learning methods. However, in practice, investigators often have working hypotheses about effect heterogeneity across…

Methodology · Statistics 2023-09-13 Chan Park , Hyunseung Kang

Non-linear mixed effects modeling and simulation (NLME M&S) is evaluated to be used for standardization with longitudinal data in presence of confounders. Standardization is a well-known method in causal inference to correct for confounding…

Bridging the gap between internal and external validity is crucial for heterogeneous treatment effect estimation. Randomised controlled trials (RCTs), favoured for their internal validity due to randomisation, often encounter challenges in…

Cluster-randomized trials (CRTs) are a well-established class of designs for evaluating community-based interventions. An essential task in planning these trials is determining the number of clusters and cluster sizes needed to achieve…

Clustered observations are ubiquitous in controlled and observational studies and arise naturally in multi-centre trials or longitudinal surveys. We present a novel model for the analysis of clustered observations where the marginal…

Methodology · Statistics 2022-11-03 Luisa Barbanti , Torsten Hothorn

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

We consider the problem of estimating the effects of a binary treatment on a continuous outcome of interest from observational data in the absence of confounding by unmeasured factors. We provide a new estimator of the population average…

Methodology · Statistics 2020-08-04 James Robins , Mariela Sued , Quanhong Lei-Gomez , Andrea Rotnitzky

Beyond conditional average treatment effects, treatments may impact the entire outcome distribution in covariate-dependent ways, for example, by altering the variance or tail risks for specific subpopulations. We propose a novel estimand to…

Machine Learning · Statistics 2026-03-18 Saksham Jain , Alex Luedtke