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Estimating heterogeneous treatment effects (HTEs) is crucial for precision medicine. While multiple studies can improve the generalizability of results, leveraging them for estimation is statistically challenging. Existing approaches often…

Methodology · Statistics 2025-12-22 Cathy Shyr , Boyu Ren , Prasad Patil , Giovanni Parmigiani

Digital technologies (e.g., mobile phones) can be used to obtain objective, frequent, and real-world digital phenotypes from individuals. However, modeling these data poses substantial challenges since observational data are subject to…

Applications · Statistics 2026-05-12 Tianchen Xu , Yuan Chen , Donglin Zeng , Yuanjia Wang

Wearable sensor technologies and deep learning are transforming healthcare management. Yet, most health sensing studies focus narrowly on physical chronic diseases. This overlooks the critical need for joint assessment of comorbid physical…

Machine Learning · Computer Science 2025-11-21 Yidong Chai , Haoxin Liu , Jiaheng Xie , Chaopeng Wang , Xiao Fang

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

Developing new drugs for target diseases is a time-consuming and expensive task, drug repurposing has become a popular topic in the drug development field. As much health claim data become available, many studies have been conducted on the…

Machine Learning · Computer Science 2023-02-22 Yaobin Ling , Pulakesh Upadhyaya , Luyao Chen , Xiaoqian Jiang , Yejin Kim

Uncovering causal mediation effects is of significant value to practitioners seeking to isolate the direct treatment effect from the potential mediated effect. We propose a double machine learning (DML) algorithm for mediation analysis that…

Machine Learning · Statistics 2025-03-11 Houssam Zenati , Judith Abécassis , Julie Josse , Bertrand Thirion

Researchers are increasingly turning to machine learning (ML) algorithms to investigate causal heterogeneity in randomized experiments. Despite their promise, ML algorithms may fail to accurately ascertain heterogeneous treatment effects…

Methodology · Statistics 2024-04-23 Kosuke Imai , Michael Lingzhi Li

We propose a novel multi-task neural network approach for estimating distributional treatment effects (DTE) in randomized experiments. While DTE provides more granular insights into the experiment outcomes over conventional methods focusing…

Machine Learning · Computer Science 2025-07-11 Tomu Hirata , Undral Byambadalai , Tatsushi Oka , Shota Yasui , Shingo Uto

The advent of the Internet era has led to an explosive growth in the Electronic Health Records (EHR) in the past decades. The EHR data can be regarded as a collection of clinical events, including laboratory results, medication records,…

Machine Learning · Computer Science 2019-11-14 Zichang Wang , Haoran Li , Luchen Liu , Haoxian Wu , Ming Zhang

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…

Methodology · Statistics 2023-01-23 Lars Lau Raket

In many clinical trials studying neurodegenerative diseases such as Parkinson's disease (PD), multiple longitudinal outcomes are collected to fully explore the multidimensional impairment caused by this disease. If the outcomes deteriorate…

Applications · Statistics 2017-05-18 Jue Wang , Sheng Luo , Liang Li

Disease progression modeling (DPM) involves using mathematical frameworks to quantitatively measure the severity of how certain disease progresses. DPM is useful in many ways such as predicting health state, categorizing disease stages, and…

Machine Learning · Computer Science 2021-10-12 Zicong Zhang , Changchang Yin , Ping Zhang

We propose a doubly robust inference method for causal effects of continuous treatment variables, under unconfoundedness and with nonparametric or high-dimensional nuisance functions. Our double debiased machine learning (DML) estimators…

Econometrics · Economics 2023-10-02 Kyle Colangelo , Ying-Ying Lee

We introduce a new instrumental variable (IV) estimator for heterogeneous treatment effects in the presence of endogeneity. Our estimator is based on double/debiased machine learning (DML) and uses efficient machine learning instruments…

Methodology · Statistics 2026-02-06 Cyrill Scheidegger , Zijian Guo , Peter Bühlmann

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

This paper explores the use of unstructured, multimodal data, namely text and images, in causal inference and treatment effect estimation. We propose a neural network architecture that is adapted to the double machine learning (DML)…

Machine Learning · Computer Science 2024-02-06 Sven Klaassen , Jan Teichert-Kluge , Philipp Bach , Victor Chernozhukov , Martin Spindler , Suhas Vijaykumar

Causal inference literature has extensively focused on binary treatments, with relatively fewer methods developed for multi-valued treatments. In particular, methods for multiple simultaneously assigned treatments remain understudied…

Modern medicine requires generalised approaches to the synthesis and integration of multimodal data, often at different biological scales, that can be applied to a variety of evidence structures, such as complex disease analyses and…

Quantitative Methods · Quantitative Biology 2019-11-11 Devin Taylor , Simeon Spasov , Pietro Liò

Smartphone sensing offers an unobtrusive and scalable way to track daily behaviors linked to mental health, capturing changes in sleep, mobility, and phone use that often precede symptoms of stress, anxiety, or depression. While most prior…

Machine Learning · Computer Science 2026-01-14 Kaidong Feng , Zhu Sun , Roy Ka-Wei Lee , Xun Jiang , Yin-Leng Theng , Yi Ding
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