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In medical treatment and elsewhere, it has become standard to base treatment intensity (dosage) on evidence in randomized trials. Yet it has been rare to study how outcomes vary with dosage. In trials to obtain drug approval, the norm has…

Econometrics · Economics 2023-05-30 Charles F. Manski

Pulse-modulated feedback is utilized in drug dosing to mimic sustained over a longer period of time manual discrete dose administration, the latter is in contrast with continuous drug infusion. The intermittent mode of dosing calls for a…

Dynamical Systems · Mathematics 2025-02-28 Alexander Medvedev , Anton V. Proskurnikov , Zhanybai T. Zhusubaliyev

Patient-derived xenografts (PDXs) are an appealing platform for preclinical drug studies because the in vivo environment of PDXs helps preserve tumor heterogeneity and usually better mimics drug response of patients with cancer compared to…

This paper deals with a skew-normal model of the relation between a dose $d > 0$ and a quantitative measure $E(d)$ of an effect of the administered drug. Precisely, $E(d)$ is a measure of the therapeutic response or a measure of a…

Methodology · Statistics 2015-04-17 Francis Lavergne , Nicolas Marie

The identification of sets of co-regulated genes that share a common function is a key question of modern genomics. Bayesian profile regression is a semi-supervised mixture modelling approach that makes use of a response to guide inference…

Hierarchical Bayesian models are increasingly used in large, inhomogeneous complex network dynamical systems by modeling parameters as draws from a hyperparameter-governed distribution. However, theoretical guarantees for these estimates as…

Statistics Theory · Mathematics 2026-01-23 Yi Yu , Yubo Hou , Yinchong Wang , Nan Zhang , Jianfeng Feng , Wenlian Lu

Oncology drug development starts with a dose escalation phase to find the maximal tolerable dose (MTD). Dose limiting toxicity (DLT) is the primary endpoint for dose escalation phase. Traditionally, model-based dose escalation trial designs…

Applications · Statistics 2024-04-18 Arnab Kumar Maity , Satrajit Roy Chowdhury , Ray Li , Lada Markovtsova , Roberto Bugarini

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

The Binary Emax model is widely employed in dose-response analysis during drug development, where missing data often pose significant challenges. Addressing nonignorable missing binary responses, where the likelihood of missing data is…

Methodology · Statistics 2026-01-01 Jiangshan Zhang , Vivek Pradhan , Yuxi Zhao

Basket trials are increasingly used for the simultaneous evaluation of a new treatment in various patient subgroups under one overarching protocol. We propose a Bayesian approach to sample size determination in basket trials that permit…

Methodology · Statistics 2022-09-02 Haiyan Zheng , Michael J. Grayling , Pavel Mozgunov , Thomas Jaki , James M. S. Wason

In preclinical investigations, e.g. in in vitro, in vivo and in silico studies, the pharmacokinetic, pharmacodynamic and toxicological characteristics of a drug are evaluated before advancing to first-in-man trial. Usually, each study is…

Bayesian hierarchical models fit to complex survey data require variance correction for the sampling design, yet applying this correction uniformly harms parameters already protected by the hierarchical structure. We propose the Design…

Methodology · Statistics 2026-03-10 JoonHo Lee

Meta-analysis is a powerful tool for assessing drug safety by combining treatment-related toxicological findings across multiple studies, as clinical trials are typically underpowered for detecting adverse drug effects. However, incomplete…

The causal dose response curve is commonly selected as the statistical parameter of interest in studies where the goal is to understand the effect of a continuous exposure on an outcome.Most of the available methodology for statistical…

Substances of abuse are known to activate and disrupt neuronal circuits in the brain reward system. We propose a simple and easily interpretable dynamical systems model to describe the neurobiology of drug addiction that incorporates the…

Neurons and Cognition · Quantitative Biology 2026-05-12 Tom Chou , Maria D'Orsogna

The purpose of a phase I dose-finding clinical trial is to investigate the toxicity profiles of various doses for a new drug and identify the maximum tolerated dose. Over the past three decades, various dose-finding designs have been…

Methodology · Statistics 2021-11-25 Yunshan Duan , Shijie Yuan , Yuan Ji , Peter Mueller

Studies of the effects of medical interventions increasingly take place in distributed research settings using data from multiple clinical data sources including electronic health records and administrative claims. In such settings, privacy…

Methodology · Statistics 2021-01-06 Martijn J. Schuemie , Yong Chen , David Madigan , Marc A. Suchard

We present a bayesassurance R package that computes the Bayesian assurance under various settings characterized by different assumptions and objectives. The package offers a constructive set of simulation-based functions suitable for…

Methodology · Statistics 2022-03-30 Jane Pan , Sudipto Banerjee

Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…

This paper builds on recent research that focuses on regression modeling of continuous bounded data, such as proportions measured on a continuous scale. Specifically, it deals with beta regression models with mixed effects from a Bayesian…