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Phase I early-phase clinical studies aim at investigating the safety and the underlying dose-toxicity relationship of a drug or combination. While little may still be known about the compound's properties, it is crucial to consider…

Methodology · Statistics 2022-09-13 Christian Röver , Moreno Ursino , Tim Friede , Sarah Zohar

Opportunistic pharmacokinetic (PK) studies have sparse and imbalanced clinical measurement data, and the impact of sample time errors is an important concern when seeking accurate estimates of treatment response. We evaluated an approximate…

Applications · Statistics 2023-10-25 Hannah L Weeks , Matthew S Shotwell

Flexible estimation of heterogeneous treatment effects lies at the heart of many statistical challenges, such as personalized medicine and optimal resource allocation. In this paper, we develop a general class of two-step algorithms for…

Machine Learning · Statistics 2020-08-07 Xinkun Nie , Stefan Wager

Benchmark dose analysis aims to estimate the level of exposure to a toxin that results in a clinically-significant adverse outcome and quantifies uncertainty using the lower limit of a confidence interval for this level. We develop a novel…

This paper describes several approaches for estimating the benchmark dose (BMD) in a risk assessment study with quantal dose-response data and when there are competing model classes for the dose-response function. Strategies involving a…

Statistics Theory · Mathematics 2019-11-19 Edsel A. Pena , Wensong Wu , Walter Piegorsch , Ronald W. West , Lingling An

Signal denoising---also known as non-parametric regression---is often performed through shrinkage estimation in a transformed (e.g., wavelet) domain; shrinkage in the transformed domain corresponds to smoothing in the original domain. A key…

Methodology · Statistics 2020-09-09 Zhengrong Xing , Peter Carbonetto , Matthew Stephens

Purpose: We present a framework for robust automated treatment planning using machine learning, comprising scenario-specific dose prediction and robust dose mimicking. Methods: The scenario dose prediction pipeline is divided into the…

Medical Physics · Physics 2022-10-12 Oskar Eriksson , Tianfang Zhang

At present, most surface-quality prediction methods can only perform single-task prediction which results in under-utilised datasets, repetitive work and increased experimental costs. To counter this, the authors propose a Bayesian…

Assessing treatment effect moderation is critical in biomedical research and many other fields, as it guides personalized intervention strategies to improve participant's outcomes. Individual participant-level data meta-analysis (IPD-MA)…

Methodology · Statistics 2024-11-01 Qiao Wang , Hwanhee Hong

Graphical models are commonly used to discover associations within gene or protein networks for complex diseases such as cancer. Most existing methods estimate a single graph for a population, while in many cases, researchers are interested…

This study presents a Bayesian hierarchical model for analyzing spatially correlated functional data and handling irregularly spaced observations. The model uses Bernstein polynomial (BP) bases combined with autoregressive random effects,…

Methodology · Statistics 2024-12-02 Alvaro Alexander Burbano Moreno , Ronaldo Dias

This paper addresses patient heterogeneity associated with prediction problems in biomedical applications. We propose a systematic hypothesis testing approach to determine the existence of patient subgroup structure and the number of…

Methodology · Statistics 2021-01-08 Xu Gao , Weining Shen , Jing Ning , Ziding Feng , Jianhua Hu

Medication adherence is a well-known problem for pharmaceutical treatment of chronic diseases. Understanding how nonadherence affects treatment efficacy is made difficult by the ethics of clinical trials that force patients to skip doses of…

Quantitative Methods · Quantitative Biology 2021-12-30 Elijah D Counterman , Sean D Lawley

Heterogeneity in gene expression across isogenic cell populations can give rise to phenotypic diversity, even when cells are in homogenous environments. This diversity arises from the discrete, stochastic nature of biochemical reactions,…

Quantitative Methods · Quantitative Biology 2017-08-31 Zachary Fox , Brian Munsky

In observational studies, potential confounders may distort the causal relationship between an exposure and an outcome. However, under some conditions, a causal dose-response curve can be recovered using the G-computation formula. Most…

Methodology · Statistics 2019-12-18 Ted Westling , Peter Gilbert , Marco Carone

Both cluster randomized trials and quasi-experimental designs are used to evaluate the impact of health and social policies and interventions. Stepped-wedge cluster randomized trials randomize a staggered adoption approach, while recent…

Methodology · Statistics 2026-04-15 Haidong Lu , Gregg S. Gonsalves , Fan Li , Guanyu Tong , Lee Kennedy-Shaffer

We develop a Bayesian methodology aimed at simultaneously estimating low-rank and row-sparse matrices in a high-dimensional multiple-response linear regression model. We consider a carefully devised shrinkage prior on the matrix of…

Methodology · Statistics 2019-04-10 Antik Chakraborty , Anirban Bhattacharya , Bani K. Mallick

Statistical inference about the average effect in random-effects meta-analysis has been considered insufficient in the presence of substantial between-study heterogeneity. Predictive distributions are well-suited for quantifying…

Methodology · Statistics 2025-10-16 David Kronthaler , Leonhard Held

The known connection between shrinkage estimation, empirical Bayes, and mixed effects models is explored and applied to balanced and unbalanced designs in which the responses are correlated. As an illustration, a mixed model is proposed for…

Methodology · Statistics 2022-01-04 Yihan Bao , James G. Booth

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall
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