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Bayesian dynamic borrowing has become an increasingly important tool for evaluating the consistency of regional treatment effects which is a key requirement for local regulatory approval of a new drug. It helps increase the precision of…

Methodology · Statistics 2026-01-06 Xin Zhang , Hui Zhang , Satrajit Roychoudhury

Incorporating preclinical animal data, which can be regarded as a special kind of historical data, into phase I clinical trials can improve decision making when very little about human toxicity is known. In this paper, we develop a robust…

Methodology · Statistics 2021-01-29 Haiyan Zheng , Lisa V. Hampson , Thomas Jaki

Accurate forecast of a clinical trial enrollment timeline at the planning phase is of great importance to both corporate strategic planning and trial operational excellence. While predictions of key milestones such as last subject first…

Applications · Statistics 2023-01-05 Sheng Zhong , Yunzhao Xing , Mengjia Yu , Li Wang

Background: Advanced adaptive randomised clinical trials are increasingly used. Compared to their conventional counterparts, their flexibility may make them more efficient, increase the probability of obtaining conclusive results without…

Understanding treatment heterogeneity is essential to the development of precision medicine, which seeks to tailor medical treatments to subgroups of patients with similar characteristics. One of the challenges to achieve this goal is that…

Methodology · Statistics 2019-08-21 Shujie Ma , Jian Huang , Zhiwei Zhang , Mingming Liu

Low attendance levels in medical appointments have been associated with poor health outcomes and efficiency problems for service providers. To address this problem, healthcare managers could aim at improving attendance levels or minimizing…

Machine Learning · Computer Science 2020-12-11 David Barrera Ferro , Sally Brailsford , Cristián Bravo , Honora Smith

Opioids are an effective analgesic for acute and chronic pain, but also carry a considerable risk of addiction leading to millions of opioid use disorder (OUD) cases and tens of thousands of premature deaths in the United States yearly.…

Quantitative Methods · Quantitative Biology 2024-03-27 Sybille Légitime , Kaustubh Prabhu , Devin McConnell , Bing Wang , Dipak K. Dey , Derek Aguiar

Aims: Combinations of treatments can offer additional benefit over the treatments individually. However, trials of these combinations are lower priority than the development of novel therapies, which can restrict funding, timelines and…

We consider Bayesian logistic regression models with group-structured covariates. In high-dimensional settings, it is often assumed that only small portion of groups are significant, thus consistent group selection is of significant…

Methodology · Statistics 2019-12-05 Kyoungjae Lee , Xuan Cao

Most clinical trials involve the comparison of a new treatment to a control arm (e.g., the standard of care) and the estimation of a treatment effect. External data, including historical clinical trial data and real-world observational…

Methodology · Statistics 2021-03-17 Tianjian Zhou , Yuan Ji

Randomized experiments have been the gold standard for assessing the effectiveness of a treatment or policy. The classical complete randomization approach assigns treatments based on a prespecified probability and may lead to inefficient…

Methodology · Statistics 2023-10-26 Waverly Wei , Xinwei Ma , Jingshen Wang

Practitioners in diverse fields such as healthcare, economics and education are eager to apply machine learning to improve decision making. The cost and impracticality of performing experiments and a recent monumental increase in electronic…

Machine Learning · Computer Science 2023-08-01 Fredrik D. Johansson , Uri Shalit , Nathan Kallus , David Sontag

Mental health disorders are rising worldwide. However, the availability of trained clinicians has not scaled proportionally, leaving many people without adequate or timely support. To bridge this gap, recent studies have shown the promise…

Computation and Language · Computer Science 2025-08-19 Aishik Mandal , Prottay Kumar Adhikary , Hiba Arnaout , Iryna Gurevych , Tanmoy Chakraborty

Estimating intervention effects in dynamical systems is crucial for outcome optimization. In medicine, such interventions arise in physiological regulation (e.g., cardiovascular system under fluid administration) and pharmacokinetics, among…

Machine Learning · Computer Science 2026-02-13 Tomer Meir , Ori Linial , Danny Eytan , Uri Shalit

Predicting the presence of major depressive disorder (MDD) using behavioural and cognitive signals is a highly non-trivial task. The heterogeneous clinical profile of MDD means that any given speech, facial expression and/or observed…

Machine Learning · Computer Science 2023-06-26 Salvatore Fara , Orlaith Hickey , Alexandra Georgescu , Stefano Goria , Emilia Molimpakis , Nicholas Cummins

Adverse childhood experiences (ACEs) have been linked to a wide range of negative health outcomes in adulthood. However, few studies have investigated what specific combinations of ACEs most substantially impact mental health. In this…

Applications · Statistics 2025-11-25 Ruizhe Zhang , Jooyoung Kong , Dylan S. Small , William Bekerman

It is recognised that treatment-related clustering should be allowed for in the sample size and analyses of individually-randomised parallel-group trials that evaluate therapist-delivered interventions such as psychotherapy. Here,…

Methodology · Statistics 2026-02-03 Rebecca EA Walwyn , Rosemary A Bailey , Arpan Singh , Neil Corrigan , Steven G Gilmour

To analyze the impacts of certain types of public health interventions we need to estimate the treatment effects and outcomes as these apply to heterogeneous open populations. Dynamically modifying populations containing risk groups that…

Methodology · Statistics 2019-10-14 Aditi Shenvi , Jim Q. Smith

In pre- and non-clinical toxicology, the reduction of animal use is highly desireable. Although approaches for possible sample size reduction in the concurrent control group were suggested previously under the virtual control groups…

Subgroup identification (clustering) is an important problem in biomedical research. Gene expression profiles are commonly utilized to define subgroups. Longitudinal gene expression profiles might provide additional information on disease…

Methodology · Statistics 2016-09-27 Jiehuan Sun , Jose D. Herazo-Maya , Naftali Kaminski , Hongyu Zhao , Joshua L. Warren