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We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Phase I dose-finding trials are increasingly challenging as the relationship between efficacy and toxicity of new compounds (or combination of them) becomes more complex. Despite this, most commonly used methods in practice focus on…

Machine Learning · Computer Science 2020-06-16 Cong Shen , Zhiyang Wang , Sofia S. Villar , Mihaela van der Schaar

Supervised learning under measurement constraints is a common challenge in statistical and machine learning. In many applications, despite extensive design points, acquiring responses for all points is often impractical due to resource…

Methodology · Statistics 2025-03-19 Lin Wang

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

The goal of uplift modeling is to recommend actions that optimize specific outcomes by determining which entities should receive treatment. One common approach involves two steps: first, an inference step that estimates conditional average…

Machine Learning · Computer Science 2025-05-21 Simon De Vos , Christopher Bockel-Rickermann , Stefan Lessmann , Wouter Verbeke

Patients in clinical studies often exhibit heterogeneous treatment effect (HTE). Classical subgroup analyses provide inferential tools to test for effect modification, while modern machine learning methods estimate the Conditional Average…

Applications · Statistics 2026-01-05 Nan Miles Xi , Xin Huang , Lin Wang

Identification of optimal dose combinations in early phase dose-finding trials is challenging, due to the trade-off between precisely estimating the many parameters required to flexibly model the possibly non-monotonic dose-response…

Methodology · Statistics 2024-02-13 James Willard , Shirin Golchi , Erica E. M. Moodie , Bruno Boulanger , Bradley P. Carlin

In recent years, with the rapid development of science and technology, heterogeneous treatment effects have emerged as a focal research topic in statistics, econometrics, and sociology. This paper investigates HTE through semiparametric…

Methodology · Statistics 2025-07-21 Jichang Yu , Wenjing Chang , Peichao Yu , Lijun Chen , Yuanshan Wu

Consider an experiment, where a new drug is tested for the first time on human subjects - healthy volunteers. Such experiments are often performed as dose-escalation studies: a set of increasing doses is pre-selected, individuals are…

Statistics Theory · Mathematics 2016-08-17 Samuel Rosa , Radoslav Harman

Binary classification rules based on covariates typically depend on simple loss functions such as zero-one misclassification. Some cases may require more complex loss functions. For example, individual-level monitoring of HIV-infected…

Machine Learning · Statistics 2019-05-14 Yizhen Xu , Tao Liu , Michael J. Daniels , Rami Kantor , Ann Mwangi , Joseph W. Hogan

Motivated by two case studies using primary care records from the Clinical Practice Research Datalink, we describe statistical methods that facilitate the analysis of tall data, with very large numbers of observations. Our focus is on…

Methodology · Statistics 2018-05-14 Kirsty Rhodes , Rebecca Turner , Rupert Payne , Ian White

Zero-shot text classifiers based on label descriptions embed an input text and a set of labels into the same space: measures such as cosine similarity can then be used to select the most similar label description to the input text as the…

Computation and Language · Computer Science 2022-05-25 Angelo Basile , Marc Franco-Salvador , Paolo Rosso

Identifying optimal medical treatments to improve survival has long been a critical goal of pharmacoepidemiology. Traditionally, we use an average treatment effect measure to compare outcomes between treatment plans. However, new methods…

Semi-supervised image classification has shown substantial progress in learning from limited labeled data, but recent advances remain largely untested for clinical applications. Motivated by the urgent need to improve timely diagnosis of…

Computer Vision and Pattern Recognition · Computer Science 2021-08-03 Zhe Huang , Gary Long , Benjamin Wessler , Michael C. Hughes

Matching is a popular nonparametric covariate adjustment strategy in empirical health services research. Matching helps construct two groups comparable in many baseline covariates but different in some key aspects under investigation. In…

Applications · Statistics 2023-08-17 Chang Chen , Zhiyu Qian , Bo Zhang

Given the prominence of targeted therapy and immunotherapy in cancer treatment, it becomes imperative to consider heterogeneity in patients' responses to treatments, which contributes greatly to the widely used proportional hazard…

Methodology · Statistics 2024-09-01 Powei Chen , Siying Hu , Haojin Zhou

We consider the optimal design problem for identifying effective dose combinations within drug combination studies where the effect of the combination of two drugs is investigated. Drug combination studies are becoming increasingly…

Subclassification estimators are one of the methods used to estimate causal effects of interest using the propensity score. This method is more stable compared to other weighting methods, such as inverse probability weighting estimators, in…

Methodology · Statistics 2024-10-22 Shunichiro Orihara , Tomotaka Momozaki

In this paper, we develop topological data analysis methods for classification tasks on univariate time series. As an application, we perform binary and ternary classification tasks on two public datasets that consist of physiological…

Machine Learning · Statistics 2021-06-15 Alperen Karan , Atabey Kaygun

In observational studies of treatment effects, matched samples are created so treated and control groups are similar in terms of observable covariates. Traditionally such matched samples consist of matched pairs. If a pair match fails to…

Methodology · Statistics 2014-10-22 Luke Keele , Sam Pimentel , Frank Yoon