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Related papers: A Bayesian dose-response meta-analysis model: simu…

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In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

Modeling dose-response relationships of drugs is essential to understanding their effect on patient outcomes under realistic circumstances. While intention-to-treat analyses of clinical trials provide the effect of assignment to a…

Applications · Statistics 2018-02-15 Jacob Spertus , Marcela Horvitz-Lennon , Sharon-Lise Normand

We develop a nonparametric Bayesian modeling framework for clustered ordinal responses in developmental toxicity studies, which typically exhibit extensive heterogeneity. The primary focus of these studies is to examine the dose-response…

Methodology · Statistics 2024-08-22 Jizhou Kang , Athanasios Kottas

Commonly, clinical trials report effects not only for the full study population but also for patient subgroups. Meta-analyses of subgroup-specific effects and treatment-by-subgroup interactions may be inconsistent, especially when trials…

Methodology · Statistics 2025-12-23 Renato Panaro , Christian Röver , Tim Friede

We consider the problem of estimating a dose-response curve. Continuous treatments arise often in practice, e.g. in the form of time spent on an operation, distance traveled to a location or dosage of a drug. Letting $A$ denote a continuous…

Methodology · Statistics 2026-04-14 Matteo Bonvini , Edward H. Kennedy

Evidence from animal models and epidemiological studies has linked prenatal alcohol exposure (PAE) to a broad range of long-term cognitive and behavioral deficits. However, there is virtually no information in the scientific literature…

Personalized cancer treatments based on the molecular profile of a patient's tumor are an emerging and exciting class of treatments in oncology. As genomic tumor profiling is becoming more common, targeted treatments to specific molecular…

In Oncology, trials evaluating drug combinations are becoming more common. While combination therapies bring the potential for greater efficacy, they also create unique challenges for ensuring drug safety. In Phase-I dose escalation trials…

Applications · Statistics 2023-02-23 Lukas A. Widmer , Andrew Bean , David Ohlssen , Sebastian Weber

An important goal of precision medicine is to personalize medical treatment by identifying individuals who are most likely to benefit from a specific treatment. The Likely Responder (LR) framework, which identifies a subpopulation where…

Methodology · Statistics 2026-03-13 Annan Deng , Carole Siegel , Hyung G. Park

Biphasic, non-sigmoidal dose-response relationships are frequently observed in biochemistry and pharmacology, but they are not always analyzed with appropriate statistical methods. Here, we examine curve fitting methods for "hormetic"…

Quantitative Methods · Quantitative Biology 2023-08-21 Venkat D. Abbaraju , Tamaraty L. Robinson , Brian P. Weiser

Standard random-effects meta-analysis methods perform poorly when applied to few studies only. Such settings however are commonly encountered in practice. It is unclear, whether or to what extent small-sample-size behaviour can be improved…

Methodology · Statistics 2019-01-15 Svenja E. Seide , Christian Röver , Tim Friede

In randomized dose-finding trials, although drug exposure data form a part of key information for dose selection, the evaluation of the dose-response (DR) relationship often mainly uses DR data. We examine the benefit of…

Methodology · Statistics 2025-08-07 Jixian Wang , Zhiwei Zhang , Ram Tiwari

Background: Pairwise and network meta-analyses using fixed effect and random effects models are commonly applied to synthesise evidence from randomised controlled trials. The models differ in their assumptions and the interpretation of the…

Methodology · Statistics 2017-08-04 Shijie Ren , Jeremy E. Oakley , John W. Stevens

Linear mixed-effects models are a central analytical tool for modeling hierarchical and longitudinal data, as they allow simultaneous representation of fixed and random sources of variation. In practice, inference for such models is most…

Methodology · Statistics 2026-02-12 Hilde Vinje , Lars Erik Gangsei

An important objective in biomedical risk assessment is estimation of minimum exposure levels that induce a pre-specified adverse response in a target population. The exposure/dose points in such settings are known as Benchmark Doses…

Methodology · Statistics 2014-02-18 Qijun Fang , Walter W. Piegorsch , Susan J. Simmons , Xiaosong Li , Cuixian Chen , Yishi Wang

Meta-analysis is widely used to integrate results from multiple experiments to obtain generalized insights. Since meta-analysis datasets are often heteroscedastic due to varying subgroups and temporal heterogeneity arising from experiments…

Methodology · Statistics 2026-01-19 Kohsuke Kubota , Shonosuke Sugasawa , Keiichi Ochiai , Takahiro Hoshino

Approving and assessing new drugs is complex because multiple criteria must be considered simultaneously. A common approach is benefit-risk analysis, often conducted within a Bayesian framework to account for uncertainty and combine data…

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

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

Methodology · Statistics 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki

In a meta-analysis, it is important to specify a model that adequately describes the effect-size distribution of the underlying population of studies. The conventional normal fixed-effect and normal random-effects models assume a normal…

Methodology · Statistics 2013-10-21 George Karabatsos , Elizabeth Talbott , Stephen G. Walker