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In pharmaceutical R&D, predicting the efficacy of a pharmaceutical in treating a particular disease prior to clinical testing or any real-world use has been challenging. In this paper, we propose a flexible and modular machine…

Machine Learning · Computer Science 2025-09-10 Adrien Couetoux , Thomas Devenyns , Lise Diagne , David Champagne , Pierre-Yves Mousset , Chris Anagnostopoulos

Machine learning models used in medical applications often face challenges due to the covariate shift, which occurs when there are discrepancies between the distributions of training and target data. This can lead to decreased predictive…

Machine Learning · Computer Science 2024-12-24 Mingyang Cai , Thomas Klausch , Mark A. van de Wiel

Healthcare decision-making requires not only accurate predictions but also insights into how factors influence patient outcomes. While traditional Machine Learning (ML) models excel at predicting outcomes, such as identifying high risk…

Machine Learning · Computer Science 2025-01-28 Sheresh Zahoor , Pietro Liò , Gaël Dias , Mohammed Hasanuzzaman

Breast cancer remains a critical global health challenge, necessitating early and accurate detection for effective treatment. This paper introduces a methodology that combines automated image augmentation selection (RandAugment) with search…

Image and Video Processing · Electrical Eng. & Systems 2023-11-21 Leon Hamnett , Mary Adewunmi , Modinat Abayomi , Kayode Raheem , Fahad Ahmed

The concept of personalised medicine in cancer therapy is becoming increasingly important. There already exist drugs administered specifically for patients with tumours presenting well-defined mutations. However, the field is still in its…

Biomolecules · Quantitative Biology 2024-08-26 Abbi Abdel-Rehim , Oghenejokpeme Orhobor , Gareth Griffiths , Larisa Soldatova , Ross D. King

A platform trial with a master protocol provides an infrastructure to ethically and efficiently evaluate multiple treatment options in multiple diseases. Given that certain study drugs can enter or exit a platform trial, the randomization…

Methodology · Statistics 2025-07-15 Tianyu Zhan , Jane Zhang , Lei Shu , Yihua Gu

We present Collaborative Trees, a novel tree model designed for regression prediction, along with its bagging version, which aims to analyze complex statistical associations between features and uncover potential patterns inherent in the…

Methodology · Statistics 2024-05-21 Chien-Ming Chi

Uncertainty in the estimation of the causal effect in observational studies is often due to unmeasured confounding, i.e., the presence of unobserved covariates linking treatments and outcomes. Instrumental Variables (IV) are commonly used…

Methodology · Statistics 2019-07-30 M. Usaid Awan , Yameng Liu , Marco Morucci , Sudeepa Roy , Cynthia Rudin , Alexander Volfovsky

Estimating a causal effect from observational data can be biased if we do not control for self-selection. This selection is based on confounding variables that affect the treatment assignment and the outcome. Propensity score methods aim to…

Econometrics · Economics 2021-09-10 Daniel Jacob

Precision medicine is becoming a focus in medical research recently, as its implementation brings values to all stakeholders in the healthcare system. Various statistical methodologies have been developed tackling problems in different…

Quantitative Methods · Quantitative Biology 2019-10-07 Zhen Zeng , Yuefeng Lu , Judong Shen , Wei Zheng , Peter Shaw , Mary Beth Dorr

While the inverse probability of treatment weighting (IPTW) is a commonly used approach for treatment comparisons in observational data, the resulting estimates may be subject to bias and excessively large variance when there is lack of…

Methodology · Statistics 2024-02-13 Zhiqiang Cao , Lama Ghazi , Claudia Mastrogiacomo , Laura Forastiere , F. Perry Wilson , Fan Li

Our work was motivated by a recent study on birth defects of infants born to pregnant women exposed to a certain medication for treating chronic diseases. Outcomes such as birth defects are rare events in the general population, which often…

Applications · Statistics 2017-02-24 Ronghui Xu , Jue Hou , Christina D. Chambers

Identification of treatment effects in the presence of unmeasured confounding is a persistent problem in the social, biological, and medical sciences. The problem of unmeasured confounding in settings with multiple treatments is most common…

Methodology · Statistics 2022-07-12 Wang Miao , Wenjie Hu , Elizabeth L. Ogburn , Xiaohua Zhou

Decision-making plays a pivotal role in shaping outcomes in various disciplines, such as medicine, economics, and business. This paper provides guidance to practitioners on how to implement a decision tree designed to address treatment…

Econometrics · Economics 2024-06-05 Hugo Bodory , Federica Mascolo , Michael Lechner

Estimating conditional average treatment effects (CATE) from randomized controlled trials (RCTs) and generalizing them to broader populations is essential for personalizing treatment rules but is complicated by selection bias due to trial…

Methodology · Statistics 2026-05-15 Rikuta Hamaya , Etsuji Suzuki , Konan Hara

A treatment benefit predictor (TBP) is a function that maps patient characteristics to an estimate of the treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to…

Methodology · Statistics 2025-09-30 Yuan Xia , Mohsen Sadatsafavi , Paul Gustafson

Uncovering the heterogeneity of causal effects of policies and business decisions at various levels of granularity provides substantial value to decision makers. This paper develops estimation and inference procedures for multiple treatment…

Econometrics · Economics 2022-09-09 Michael Lechner , Jana Mareckova

Unmeasured confounding presents a common challenge in observational studies, potentially making standard causal parameters unidentifiable without additional assumptions. Given the increasing availability of diverse data sources, exploiting…

Methodology · Statistics 2023-09-18 Shanshan Luo , Yechi Zhang , Wei Li

Propensity score (PS) methods are widely used in observational studies to reduce confounding and estimate causal treatment effects. However, the validity of PS-based causal estimators depends heavily on correct model specification, and…

Prediction methods for time-to-event outcomes often utilize survival models that rely on strong assumptions about noninformative censoring or on how individual-level covariates and survival functions are related. When the main interest is…

Methodology · Statistics 2024-02-29 Mahsa Ashouri , Nicholas C. Henderson
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