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An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

Dose-response models express the effect of different dose or exposure levels on a specific outcome. In meta-analysis, where aggregated-level data is available, dose-response evidence is synthesized using either one-stage or two-stage models…

Basket trials have gained increasing attention for their efficiency, as multiple patient subgroups are evaluated simultaneously. Conducted basket trials focus primarily on establishing the early efficacy of a treatment, yet continued…

Applications · Statistics 2025-05-16 Zhi Cao , Pavel Mozgunov , Haiyan Zheng

Randomized controlled trials estimate average treatment effects, but treatment response heterogeneity motivates personalized approaches. A critical question is whether statistically detectable heterogeneity translates into improved…

Machine Learning · Computer Science 2026-02-10 Cristian Minoccheri , Sophia Tesic , Kayvan Najarian , Ryan Stidham

Tuberculosis (TB) is a major global health challenge, and is compounded by co-morbidities such as HIV, diabetes, and anemia, which complicate treatment outcomes and contribute to heterogeneous patient responses. Traditional models of TB…

Machine Learning · Computer Science 2024-11-19 Ethan Wu , Caleb Ellington , Ben Lengerich , Eric P. Xing

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

Botulinum toxin (Botox) injections are the gold standard for managing facial asymmetry and aesthetic rejuvenation, yet determining the optimal dosage remains largely intuitive, often leading to suboptimal outcomes. We propose a localized…

Computer Vision and Pattern Recognition · Computer Science 2026-01-28 Estèphe Arnaud , Mohamed Daoudi , Pierre Guerreschi

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…

Accurate models of clinical actions and their impacts on disease progression are critical for estimating personalized optimal dynamic treatment regimes (DTRs) in medical/health research, especially in managing chronic conditions.…

Methodology · Statistics 2021-02-19 William Hua , Hongyuan Mei , Sarah Zohar , Magali Giral , Yanxun Xu

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

We introduce Targeted Smooth Bayesian Causal Forests (tsBCF), a nonparametric Bayesian approach for estimating heterogeneous treatment effects which vary smoothly over a single covariate in the observational data setting. The tsBCF method…

Estimating varying treatment effects in randomized trials with noncompliance is inherently challenging since variation comes from two separate sources: variation in the impact itself and variation in the compliance rate. In this setting,…

Applications · Statistics 2024-08-28 Jared D. Fisher , David W. Puelz , Sameer K. Deshpande

Imaging in clinical oncology trials provides a wealth of information that contributes to the drug development process, especially in early phase studies. This paper focuses on kinetic modeling in DCE-MRI, inspired by mixed-effects models…

Applications · Statistics 2020-04-22 Volker J. Schmid , Brandon Whitcher , Anwar R. Padhani , N. Jane Taylor , Guang-Zhong Yang

Early phase, personalized dose-finding trials for combination therapies seek to identify patient-specific optimal biological dose (OBD) combinations, which are defined as safe dose combinations which maximize therapeutic benefit for a…

Methodology · Statistics 2024-04-18 James Willard , Shirin Golchi , Erica EM Moodie

A dynamic treatment regime is a sequence of medical decisions that adapts to the evolving clinical status of a patient over time. To facilitate personalized care, it is crucial to assess the probability of each available treatment option…

Methodology · Statistics 2024-11-05 Jiefeng Bi , Matteo Borrotti , Bernardo Nipoti

Phase I dose-finding trials in oncology seek to find the maximum tolerated dose (MTD) of a drug under a specific schedule. Evaluating drug-schedules aims at improving treatment safety while maintaining efficacy. However, while we can…

Most conventional risk analysis methods rely on a single best estimate of exposure per person which does not allow for adjustment for exposure-related uncertainty. Here, we propose a Bayesian model averaging method to properly quantify the…

Applications · Statistics 2020-04-07 Deukwoo Kwon , F. Owen Hoffman , Brian E. Moroz , Steven L. Simon

Radiation therapy treatment planning can be viewed as an iterative hyperparameter tuning process to balance conflicting clinical goals. In this work, we investigated the performance of modern Bayesian Optimization (BO) methods on automated…

Medical Physics · Physics 2023-02-15 Qingying Wang , Ruoxi Wang , Jiacheng Liu , Fan Jiang , Haizhen Yue , Yi Du , Hao Wu

Background: Depression in people with bipolar disorder is a major cause of long-term disability, possibly leading to early mortality and currently, limited safe and effective therapies exist. A double-blinded randomized placebo-controlled…

Quantitative Methods · Quantitative Biology 2020-11-12 Antoine Nzeyimana , Kate EA Saunders , John R Geddes , Patrick E McSharry
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