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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

We consider the design of a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome. We detail Bayesian inference for the analysis of the trial using a linear mixed-effects model. The treatment is compared to…

Methodology · Statistics 2022-08-29 Kevin J Wilson

Suppose a researcher observes individuals within a county within a state. Given concerns about correlation across individuals, it is common to group observations into clusters and conduct inference treating observations across clusters as…

Econometrics · Economics 2022-01-24 Yong Cai

Public policy-makers use cost-effectiveness analyses (CEA) to decide which health and social care interventions to provide. Appropriate methods have not been developed for handling missing data in complex settings, such as for CEA that use…

Methodology · Statistics 2012-06-27 Karla Diaz-Ordaz , Michael G. Kenward , Richard Grieve

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

Methodology · Statistics 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

Randomized trials are often conducted with separate randomizations across multiple sites such as schools, voting districts, or hospitals. These sites can differ in important ways, including the site's implementation, local conditions, and…

Methodology · Statistics 2018-03-19 Lo-Hua Yuan , Avi Feller , Luke W. Miratrix

The parallel cluster randomized trial with baseline (PB-CRT) is a common variant of the standard parallel cluster randomized trial (P-CRT). We define two natural estimands in the context of PB-CRTs with informative cluster sizes, the…

Methodology · Statistics 2025-05-01 Kenneth Menglin Lee , Fan Li

Conventional survival analysis approaches estimate risk scores or individualized time-to-event distributions conditioned on covariates. In practice, there is often great population-level phenotypic heterogeneity, resulting from (unknown)…

Machine Learning · Statistics 2020-03-03 Paidamoyo Chapfuwa , Chunyuan Li , Nikhil Mehta , Lawrence Carin , Ricardo Henao

Before embarking on data collection, researchers typically compute how many individual observations they should do. This is vital for doing studies with sufficient statistical power, and often a cornerstone in study pre-registrations and…

Methodology · Statistics 2023-09-06 Edwin S Dalmaijer

Missing outcomes are a commonly occurring problem for cluster randomised trials, which can lead to biased and inefficient inference if ignored or handled inappropriately. Two approaches for analysing such trials are cluster-level analysis…

Methodology · Statistics 2016-08-19 Anower Hossain , Karla Diaz-Ordaz , Jonathan W. Bartlett

Clustering is widely used in unsupervised learning to find homogeneous groups of observations within a dataset. However, clustering mixed-type data remains a challenge, as few existing approaches are suited for this task. This study…

Machine Learning · Statistics 2025-11-26 Badih Ghattas , Alvaro Sanchez San-Benito

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

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

We propose a novel nonparametric Bayesian IRT model in this paper by introducing the clustering effect at question level and further assume heterogeneity at examinee level under each question cluster, characterized by the mixture of…

Methodology · Statistics 2022-11-23 Tianyu Pan , Weining Shen , Clintin P. Davis-Stober , Guanyu Hu

Cluster analysis has proved to be an invaluable tool for the exploratory and unsupervised analysis of high dimensional datasets. Among methods for clustering, hierarchical approaches have enjoyed substantial popularity in genomics and other…

Methodology · Statistics 2014-11-20 Patrick K. Kimes , Yufeng Liu , D. Neil Hayes , J. S. Marron

In cancer research, clustering techniques are widely used for exploratory analyses and dimensionality reduction, playing a critical role in the identification of novel cancer subtypes, often with direct implications for patient management.…

In many practical tasks it is needed to estimate an effect of treatment on individual level. For example, in medicine it is essential to determine the patients that would benefit from a certain medicament. In marketing, knowing the persons…

Methodology · Statistics 2019-02-28 Aleksey Buzmakov

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Estimating causal effects from randomized experiments is only possible if participants are willing to disclose their potentially sensitive responses. Differential privacy, a widely used framework for ensuring an algorithms privacy…

Machine Learning · Statistics 2025-05-29 Adel Javanmard , Vahab Mirrokni , Jean Pouget-Abadie