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Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Cluster randomized trials with measurements at baseline can improve power over post-test only designs by using difference in difference designs. However, subjects may be lost to follow-up between the baseline and follow-up periods. While…

Methodology · Statistics 2019-03-26 Jonathan Moyer , Ken Kleinman

Sequential Multiple-Assignment Randomized Trials (SMARTs) play an increasingly important role in psychological and behavioral health research. This experimental approach enables researchers to answer scientific questions about how to…

Methodology · Statistics 2023-06-21 John J. Dziak , Daniel Almirall , Walter Dempsey , Catherine Stanger , Inbal Nahum-Shani

Multiple outcomes, both continuous and discrete, are routinely gathered on subjects in longitudinal studies and during routine clinical follow-up in general. To motivate our work, we consider a longitudinal study on patients with primary…

Applications · Statistics 2013-04-17 Arnošt Komárek , Lenka Komárková

Mendelian randomization is the use of genetic variants to assess the existence of a causal relationship between a risk factor and an outcome of interest. Here, we focus on two-sample summary-data Mendelian randomization analyses with many…

Quantitative Methods · Quantitative Biology 2022-09-16 Apostolos Gkatzionis , Stephen Burgess , Paul J. Newcombe

The intra-cluster correlation coefficient (ICC) plays an important role while designing the cluster randomized trials (CRTs). Often optimal CRTs are designed assuming that the magnitude of the ICC is constant across the clusters. However,…

Computation · Statistics 2019-02-21 Satya Prakash Singh , Pradeep Yadav

We investigate the estimation of multivariate extreme models with a discrete spectral measure using spherical clustering techniques. The primary contribution involves devising a method for selecting the order, that is, the number of…

Methodology · Statistics 2025-02-20 Shiyuan Deng , He Tang , Shuyang Bai

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

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

The effect of space distribution of randomly-placed particles in a representative composite volume on the thermoelastic effective properties and local stress and strain distribution is analyzed. Quantitative assessment is performed using…

Soft Condensed Matter · Physics 2026-05-13 Pawel Holobut , Michal Majewski , Katarzyna Kowalczyk-Gajewska

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

Methodology · Statistics 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

Clustering is a central approach for unsupervised learning. After clustering is applied, the most fundamental analysis is to quantitatively compare clusterings. Such comparisons are crucial for the evaluation of clustering methods as well…

Machine Learning · Statistics 2017-10-03 Alexander J Gates , Yong-Yeol Ahn

Adaptive designs are commonly used in clinical and drug development studies for optimum utilization of available resources. In this article, we consider the problem of estimating the effect of the selected (better) treatment using a…

Statistics Theory · Mathematics 2023-01-24 Masihuddin , Neeraj Misra

Clinical trials usually involve sequential patient entry. When designing a clinical trial, it is often desirable to include a provision for interim analyses of accumulating data with the potential for stopping the trial early. We review…

Methodology · Statistics 2023-03-13 Tianjian Zhou , Yuan Ji

To estimate the causal effect of an endogenous treatment using clustered data, the canonical two-stage least squares (2sls) estimates a linear regression of the outcome on treatment status using an instrumental variable (IV) and conducts…

Methodology · Statistics 2026-04-03 Anqi Zhao , Peng Ding , Fan Li

The aim of this paper is to review methods of designing screening experiments, ranging from designs originally developed for physical experiments to those especially tailored to experiments on numerical models. The strengths and weaknesses…

Methodology · Statistics 2015-10-20 David C. Woods , Susan M. Lewis

A combinatorial intervention, consisting of multiple treatments applied to a single unit with potentially interactive effects, has substantial applications in fields such as biomedicine, engineering, and beyond. Given $p$ possible…

Machine Learning · Computer Science 2025-06-05 Divya Shyamal , Jiaqi Zhang , Caroline Uhler

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Understanding causality should be a core requirement of any attempt to build real impact through AI. Due to the inherent unobservability of counterfactuals, large randomised trials (RCTs) are the standard for causal inference. But large…

There is growing interest in platform trials that allow for adding of new treatment arms as the trial progresses as well as being able to stop treatments part way through the trial for either lack of benefit/futility or for superiority. In…

Methodology · Statistics 2021-12-14 Peter Greenstreet , Thomas Jaki , Alun Bedding , Chris Harbron , Pavel Mozgunov