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Health economic evaluations face the issues of non-compliance and missing data. Here, non-compliance is defined as non-adherence to a specific treatment, and occurs within randomised controlled trials (RCTs) when participants depart from…

Applications · Statistics 2019-02-26 Karla DiazOrdaz , Richard Grieve

A central obstacle in the objective assessment of treatment effect (TE) estimators in randomized control trials (RCTs) is the lack of ground truth (or validation set) to test their performance. In this paper, we propose a novel…

Clinical decision requires reasoning in the presence of imperfect data. DTs are a well-known decision support tool, owing to their interpretability, fundamental in safety-critical contexts such as medical diagnosis. However, learning DTs…

Cost-Effectiveness Analyses (CEAs) alongside randomised controlled trials (RCTs) are increasingly often designed to collect resource use and preference-based health status data for the purpose of healthcare technology assessment. However,…

Applications · Statistics 2016-07-22 Andrea Gabrio , Alexina Mason , Gianluca Baio

Observational studies provide the only evidence on the effectiveness of interventions when randomized controlled trials (RCTs) are impractical due to cost, ethical concerns, or time constraints. While many methodologies aim to draw causal…

Sound event detection (SED), as a core module of acoustic environmental analysis, suffers from the problem of data deficiency. The integration of semi-supervised learning (SSL) largely mitigates such problem while bringing no extra…

Audio and Speech Processing · Electrical Eng. & Systems 2024-01-01 Nian Shao , Erfan Loweimi , Xiaofei Li

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Semi-supervised text classification-based paradigms (SSTC) typically employ the spirit of self-training. The key idea is to train a deep classifier on limited labeled texts and then iteratively predict the unlabeled texts as their…

Computation and Language · Computer Science 2023-06-14 Ahmed Murtadha , Shengfeng Pan , Wen Bo , Jianlin Su , Xinxin Cao , Wenze Zhang , Yunfeng Liu

Randomized Controlled Trials (RCT)s are relied upon to assess new treatments, but suffer from limited power to guide personalized treatment decisions. On the other hand, observational (i.e., non-experimental) studies have large and diverse…

Methodology · Statistics 2023-03-07 Zeshan Hussain , Ming-Chieh Shih , Michael Oberst , Ilker Demirel , David Sontag

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the…

Methodology · Statistics 2025-11-06 Bingkai Wang , Fan Li , Rui Wang

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

An individualized treatment rule (ITR) tailors treatments to a patient's specific characteristics. However, randomized controlled trials (RCTs) are often underpowered to detect the treatment effect heterogeneity needed for reliable ITR…

Methodology · Statistics 2026-04-14 Yuan Bian , Donglin Zeng , Hyun-Joon Yang , Leanne M. Williams , Yuanjia Wang

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Machine learning is increasingly used to select which individuals receive limited-resource interventions in domains such as human services, education, development, and more. However, it is often not apparent what the right quantity is for…

Machine Learning · Computer Science 2025-03-20 Vibhhu Sharma , Bryan Wilder

Causal inference is vital for informed decision-making across fields such as biomedical research and social sciences. Randomized controlled trials (RCTs) are considered the gold standard for internal validity of inferences, whereas…

Methodology · Statistics 2025-12-02 Ruoqi Yu , Bikram Karmakar , Jessica Vandeleest , Eleanor Bimla Schwarz

Differential diagnosis of dementia is challenging due to overlapping symptoms, with structural magnetic resonance imaging (MRI) being the primary method for diagnosis. Despite the clinical value of computer-aided differential diagnosis,…

Computer Vision and Pattern Recognition · Computer Science 2024-04-10 Yitong Li , Tom Nuno Wolf , Sebastian Pölsterl , Igor Yakushev , Dennis M. Hedderich , Christian Wachinger

Most practical data science problems encounter missing data. A wide variety of solutions exist, each with strengths and weaknesses that depend upon the missingness-generating process. Here we develop a theoretical framework for training and…

Machine Learning · Computer Science 2022-11-15 Jahan C. Penny-Dimri , Christoph Bergmeir , Julian Smith

Reinforcement learning (RL) has garnered increasing recognition for its potential to optimise dynamic treatment regimes (DTRs) in personalised medicine, particularly for drug dosage prescriptions and medication recommendations. However, a…

Machine Learning · Computer Science 2024-05-30 Zhiyao Luo , Mingcheng Zhu , Fenglin Liu , Jiali Li , Yangchen Pan , Jiandong Zhou , Tingting Zhu
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