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Healthcare companies must submit pharmaceutical drugs or medical devices to regulatory bodies before marketing new technology. Regulatory bodies frequently require transparent and interpretable computational modelling to justify a new…

应用统计 · 统计学 2019-01-15 Simon Olofsson , Marc Peter Deisenroth , Ruth Misener

We consider a dose-optimization design for first-in-human oncology trial that aims to identify a suitable dose for late-phase drug development. The proposed approach, called the Pharmacometrics-Enabled DOse OPtimization (PEDOOP) design,…

应用统计 · 统计学 2024-06-19 Shijie Yuan , Zhanbo Huang , Jiaxin Liu , Yuan Ji

The ICH E9(R1) guideline presents a framework of estimand for clinical trials, proposes five strategies for handling intercurrent events (ICEs), and provides a comprehensive discussion and many real-life clinical examples for quantitative…

统计方法学 · 统计学 2025-10-20 Yixin Fang , Man Jin

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

Contextual sensing and delivery of digital interventions to improve health outcomes have gained significant traction in behavioral and psychiatric studies. Micro-randomized trials (MRTs) are a common experimental design for obtaining…

统计方法学 · 统计学 2025-04-01 Jieru Shi , Zhenke Wu , Walter Dempsey

Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expected treatment effects or group differences among all units of…

应用统计 · 统计学 2021-04-07 Maryna Prus , Norbert Benda , Rainer Schwabe

Online mental health treatment has the premise to meet the increasing demand for mental health treatment at a lower cost than traditional treatment. However, online treatment suffers from high drop-out rates, which might negate their cost…

统计计算 · 统计学 2018-09-13 Dennis Becker

We propose a rule-based statistical design for combination dose-finding trials with two agents. The Ci3+3 design is an extension of the i3+3 design with simple decision rules comparing the observed toxicity rates and equivalence intervals…

应用统计 · 统计学 2023-03-29 Shijie Yuan , Tianjian Zhou , Yawen Lin , Yuan Ji

We consider the sequential experimental design problem in the predict-then-optimize paradigm. In this paradigm, the outputs of the prediction model are used as coefficient vectors in a downstream linear optimization problem. Traditional…

机器学习 · 统计学 2026-02-06 Beichen Wan , Mo Liu , Paul Grigas , Zuo-Jun Max Shen

AI-enabled decision-support systems aim to help medical providers rapidly make decisions with limited information during medical emergencies. A critical challenge in developing these systems is supporting providers in interpreting the…

Model-based experimental design is attracting increasing attention in chemical process engineering. Typically, an iterative procedure is pursued: an approximate model is devised, prescribed experiments are then performed and the resulting…

Computational methods in drug repositioning can help to conserve resources. In particular, methods based on biological networks are showing promise. Considering only the network topology and knowledge on drug target genes is not sufficient…

分子网络 · 定量生物学 2025-04-02 Atte Aalto , La Mi , Diego A. Blanco-Mora , Jorge Goncalves

Leveraging preclinical animal data for a phase I first-in-man trial is appealing yet challenging. A prior based on animal data may place large probability mass on values of the dose-toxicity model parameter(s), which appear infeasible in…

应用统计 · 统计学 2020-04-15 Haiyan Zheng , Lisa V. Hampson

Covariate-adjusted response-adaptive (CARA) designs have gained widespread adoption for their clear benefits in enhancing experimental efficiency and participant welfare. These designs dynamically adjust treatment allocations during interim…

统计方法学 · 统计学 2025-12-10 Xinwei Ma , Jingshen Wang , Waverly Wei

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

计量经济学 · 经济学 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

We consider the problem of how to assign treatment in a randomized experiment, in which the correlation among the outcomes is informed by a network available pre-intervention. Working within the potential outcome causal framework, we…

统计方法学 · 统计学 2017-05-19 Guillaume W. Basse , Edoardo M. Airoldi

Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

统计方法学 · 统计学 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

统计方法学 · 统计学 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

Prior probabilities of clinical hypotheses are not systematically used for clinical trial design yet, due to a concern that poor priors may lead to poor decisions. To address this concern, a conservative approach to Bayesian trial design is…

统计方法学 · 统计学 2023-09-21 Fabio Rigat

Strategic test allocation plays a major role in the control of both emerging and existing pandemics (e.g., COVID-19, HIV). Widespread testing supports effective epidemic control by (1) reducing transmission via identifying cases, and (2)…

统计方法学 · 统计学 2022-12-06 Ivana Malenica , Jeremy R. Coyle , Mark J. van der Laan , Maya L. Petersen