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Generalized linear mixed models (GLMM) are commonly used to analyze clustered data, but when the number of clusters is small to moderate, standard statistical tests may produce elevated type I error rates. Small-sample corrections have been…

Methodology · Statistics 2023-11-07 Hongxiang Qiu , Andrea J. Cook , Jennifer F. Bobb

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

Econometrics · Economics 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

The optimal strategy for deploying a treatment in a population may recommend giving all in the population that treatment. Such a strategy may not be feasible, especially in resource-limited settings. One approach for determining how to…

Applications · Statistics 2025-02-21 Lina M. Montoya , Elvin H. Geng , Harriet F. Adhiambo , Maya L. Petersen

Personalized medicine, a paradigm of medicine tailored to a patient's characteristics, is an increasingly attractive field in health care. An important goal of personalized medicine is to identify a subgroup of patients, based on baseline…

Machine Learning · Statistics 2023-01-31 Hengrui Cai , Wenbin Lu , Rachel Marceau West , Devan V. Mehrotra , Lingkang Huang

We propose a novel adaptive design for clinical trials with time-to-event outcomes and covariates (which may consist of or include biomarkers). Our method is based on the expected entropy of the posterior distribution of a proportional…

Applications · Statistics 2016-03-29 James E. Barrett

Practitioners conducting adaptive experiments often encounter two competing priorities: maximizing total welfare (or `reward') through effective treatment assignment and swiftly concluding experiments to implement population-wide…

Machine Learning · Computer Science 2024-07-31 Chao Qin , Daniel Russo

Heart failure (HF) is a leading cause of morbidity, mortality, and health care costs. Prolonged conduction through the myocardium can occur with HF, and a device-driven approach, termed cardiac resynchronization therapy (CRT), can improve…

Machine Learning · Computer Science 2021-09-14 Brendan E. Odigwe , Francis G. Spinale , Homayoun Valafar

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

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

Adaptive trials are now mainstream science. Recently, researchers have taken the adaptive trial concept to its natural conclusion, proposing what we call "Global Cumulative Treatment Analysis" (GCTA). Similar to the adaptive trial, decision…

Applications · Statistics 2013-08-06 Jeff Shrager

Public service programs often allocate limited resources under uncertainty about their benefits, creating a need for randomization to support credible evaluation. In practice, however, applicants commonly enter waitlists where resources are…

Machine Learning · Computer Science 2026-05-26 JungHo Lee , Johnna Sundberg , Pim Welle , Bryan Wilder

Randomization, as a key technique in clinical trials, can eliminate sources of bias and produce comparable treatment groups. In randomized experiments, the treatment effect is a parameter of general interest. Researchers have explored the…

Methodology · Statistics 2023-12-05 Fuyi Tu , Wei Ma , Hanzhong Liu

Modern precision medicine aims to utilize real-world data to provide the best treatment for an individual patient. An individualized treatment rule (ITR) maps each patient's characteristics to a recommended treatment scheme that maximizes…

Applications · Statistics 2025-01-07 Andong Wang , Kelly Wentzlof , Johnny Rajala , Miontranese Green , Yunshu Zhang , Shu Yang

There is a growing literature on design-based methods to estimate average treatment effects (ATEs) for randomized controlled trials (RCTs) for full sample analyses. This article extends these methods to estimate ATEs for discrete subgroups…

Methodology · Statistics 2023-10-16 Peter Z. Schochet

Robot allocation plays an essential role in facilitating robotic service provision across various domains. Yet the increasing number of users and the uncertainties regarding the users' true service requirements have posed challenges for the…

Robotics · Computer Science 2024-03-19 Yuhan Zhao , Quanyan Zhu

Randomized trials balance all covariates on average and provide the gold standard for estimating treatment effects. Chance imbalances nevertheless exist more or less in realized treatment allocations and intrigue an important question: what…

Methodology · Statistics 2023-07-18 Anqi Zhao , Peng Ding

Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in…

Methodology · Statistics 2021-05-27 Siyun Yang , Fan Li , Laine E. Thomas , Fan Li

A treatment regime is a function that maps individual patient information to a recommended treatment, hence explicitly incorporating the heterogeneity in need for treatment across individuals. Patient responses are dichotomous and can be…

Machine Learning · Statistics 2016-07-07 Yingfei Wang , Warren Powell

When resources are scarce, an allocation policy is needed to decide who receives a resource. This problem occurs, for instance, when allocating scarce medical resources and is often solved using modern ML methods. This paper introduces…

Machine Learning · Computer Science 2024-02-20 Niclas Boehmer , Yash Nair , Sanket Shah , Lucas Janson , Aparna Taneja , Milind Tambe

This paper presents the foundations of a computer oriented approach for preparing a list of random treatment assignments to be adopted in randomised controlled trials. Software is presented which can be applied in the earliest stage of…

Applications · Statistics 2015-02-12 N. S. Santos-Magalhaes , H. M. de Oliveira , A. J. Alves