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Attrition is a common occurrence in cluster randomised trials (CRTs) which leads to missing outcome data. Two approaches for analysing such trials are cluster-level analysis and individual-level analysis. This paper compares the performance…

Methodology · Statistics 2016-03-15 Anower Hossain , Karla Diaz-Ordaz , Jonathan W. Bartlett

Obtaining reliable inferences with traditional difference-in-differences (DiD) methods can be difficult. Problems can arise when both outcomes and errors are serially correlated, when there are few clusters or few treated clusters, when…

Econometrics · Economics 2026-02-13 Sunny R. Karim , Morten Ørregaard Nielsen , James G. MacKinnon , Matthew D. Webb

Machine learning is increasingly used to select which individuals receive limited-resource interventions in domains such as human services, education, development, and more. However, it is often not apparent what the right quantity is for…

Machine Learning · Computer Science 2025-03-20 Vibhhu Sharma , Bryan Wilder

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Staggered treatment adoption arises in the evaluation of policy impact and implementation in many settings, including both randomized stepped-wedge trials and non-randomized quasi-experiments with panel data. In both settings, getting an…

Methodology · Statistics 2024-10-14 Lee Kennedy-Shaffer

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

Methodology · Statistics 2026-05-01 Elizabeth Tipton , Michalis Mamakos

There is tremendous interest in precision medicine as a means to improve patient outcomes by tailoring treatment to individual characteristics. An individualized treatment rule formalizes precision medicine as a map from patient information…

Machine Learning · Statistics 2020-05-28 Daniel J. Luckett , Eric B. Laber , Michael R. Kosorok

In this study, we present a novel clinical decision support system and discuss its interpretability-related properties. It combines a decision set of rules with a machine learning scheme to offer global and local interpretability. More…

Methodology · Statistics 2021-07-16 Francisco Valente , Simão Paredes , Jorge Henriques

Therapeutic advancements in oncology have shifted towards targeted therapy based on genomic aberrations. This necessitates innovative statistical approaches in clinical trials, particularly in master protocol studies. Basket trials, a type…

Applications · Statistics 2025-02-12 Antonios Daletzakis , Rutger van den Bor , Vincent van der Noort , Kit CB Roes

Defining a causal estimand for a longitudinal outcome truncated by death is challenging, because the outcome may be undefined at the end of follow-up. Although a range of estimands and several estimators have been proposed, guidance on the…

Methodology · Statistics 2026-04-30 Juliette Ortholand , Young Lee , Marie-Abele C Bind

Cluster algorithms are increasingly popular in biomedical research due to their compelling ability to identify discrete subgroups in data, and their increasing accessibility in mainstream software. While guidelines exist for algorithm…

Machine Learning · Statistics 2021-05-26 E. S. Dalmaijer , C. L. Nord , D. E. Astle

Millions of drivers worldwide have enjoyed financial benefits and work schedule flexibility through a ride-sharing economy, but meanwhile they have suffered from the lack of a sense of identity and career achievement. Equipped with social…

Computers and Society · Computer Science 2020-08-18 Teng Ye , Wei Ai , Lingyu Zhang , Ning Luo , Lulu Zhang , Jieping Ye , Qiaozhu Mei

In randomized experiments, treatment and control groups should be roughly the same--balanced--in their distributions of pretreatment variables. But how nearly so? Can descriptive comparisons meaningfully be paired with significance tests?…

Methodology · Statistics 2008-08-29 Ben B. Hansen , Jake Bowers

In causal inference with binary outcomes, there is a growing interest in estimation of treatment harm rate (THR), which is a measure of treatment risk and reveals treatment effect heterogeneity in a subpopulation. The THR is generally…

Methodology · Statistics 2025-05-20 Wei Liang , Changbao Wu

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

This study considers various semiparametric difference-in-differences models under different assumptions on the relation between the treatment group identifier, time and covariates for cross-sectional and panel data. The variance lower…

Econometrics · Economics 2020-08-17 Michael Zimmert

Longitudinal studies are often conducted to explore the cohort and age effects in many scientific areas. The within cluster correlation structure plays a very important role in longitudinal data analysis. This is because not only can an…

Statistics Theory · Mathematics 2008-12-18 Yan Sun , Wenyang Zhang , Howell Tong

I introduce a generic method for inference about a scalar parameter in research designs with a finite number of heterogeneous clusters where only a single cluster received treatment. This situation is commonplace in…

Econometrics · Economics 2020-10-09 Andreas Hagemann

This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs)…

Methodology · Statistics 2026-04-10 Kenneth M. Lee , Fan Li

The randomization inference literature studying randomized controlled trials (RCTs) assumes that units' potential outcomes are deterministic. This assumption is unlikely to hold, as stochastic shocks may take place during the experiment. In…

Econometrics · Economics 2022-12-15 Antoine Deeb , Clément de Chaisemartin
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