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Successful pharmaceutical drug development requires finding correct doses that provide an optimum balance between efficacy and toxicity. Competing responses to dose such as efficacy and toxicity often will increase with dose, and it is…

Applications · Statistics 2024-01-26 A. Lawrence Gould

Reaction-diffusion models are widely used to study spatially-extended chemical reaction systems. In order to understand how the dynamics of a reaction-diffusion model are affected by changes in its input parameters, efficient methods for…

Quantitative Methods · Quantitative Biology 2017-03-08 Christopher Lester , Christian A. Yates , Ruth E. Baker

Plausible identification of conditional average treatment effects (CATEs) may rely on controlling for a large number of variables to account for confounding factors. In these high-dimensional settings, estimation of the CATE requires…

Econometrics · Economics 2023-01-18 Adam Baybutt , Manu Navjeevan

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

Applications · Statistics 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips

Semiparametric regression offers a flexible framework for modeling non-linear relationships between a response and covariates. A prime example are generalized additive models where splines (say) are used to approximate non-linear functional…

Statistics Theory · Mathematics 2018-10-05 Francis K. C. Hui , Chong You , Han Lin Shang , Samuel Müller

Increasingly, medical research is dependent on data collected for non-research purposes, such as electronic health records data (EHR). EHR data and other large databases can be prone to measurement error in key exposures, and unadjusted…

Methodology · Statistics 2020-05-13 Kyunghee Han , Thomas Lumley , Bryan E. Shepherd , Pamela A. Shaw

An adaptive design adjusts dynamically as information is accrued and a consequence of applying an adaptive design is the potential for inducing small-sample bias in estimates. In psychometrics and psychophysics, a common class of studies…

Methodology · Statistics 2025-02-17 Simon Bang Kristensen , Katrine Bødkergaard , Bo Martin Bibby

A key challenge in estimating causal effects from observational data is handling confounding and is commonly achieved through weighting methods that balance distribution of covariates between treatment and control groups. Weighting…

Methodology · Statistics 2025-12-23 Simion De , Jared D. Huling

Random-effects meta-analyses are very commonly used in medical statistics. Recent methodological developments include multivariate (multiple outcomes) and network (multiple treatments) meta-analysis. Here we provide a new model and…

Methodology · Statistics 2017-08-16 Dan Jackson , Sylwia Bujkiewicz , Martin Law , Richard D Riley , Ian White

Identifying effects of actions (treatments) on outcome variables from observational data and causal assumptions is a fundamental problem in causal inference. This identification is made difficult by the presence of confounders which can be…

Methodology · Statistics 2012-03-19 Ilya Shpitser , Tyler VanderWeele , James M. Robins

In epidemiology, identifying subpopulations that are particularly vulnerable to exposures and those who may benefit differently from exposure-reducing interventions is essential. Factors such as age, gender-specific vulnerabilities, and…

Methodology · Statistics 2024-12-11 David McCoy , Wenxin Zhang , Alan Hubbard , Mark van der Laan , Alejandro Schuler

We consider a setting in which we have a treatment and a large number of covariates for a set of observations, and wish to model their relationship with an outcome of interest. We propose a simple method for modeling interactions between…

Methodology · Statistics 2012-12-14 Lu Tian , Ash Alizadeh , Andrew Gentles , Robert Tibshirani

Global QCD analyses provide the primary framework for extracting hadron structure from experimental data, yet the mechanisms by which data constrain non-perturbative functions remain difficult to interpret due to the high dimensionality and…

High Energy Physics - Phenomenology · Physics 2026-05-01 Richard Whitehill

Interval identification of parameters such as average treatment effects, average partial effects and welfare is particularly common when using observational data and experimental data with imperfect compliance due to the endogeneity of…

Econometrics · Economics 2025-04-09 Sukjin Han , Adam McCloskey

For exchangeable data, mixture models are an extremely useful tool for density estimation due to their attractive balance between smoothness and flexibility. When additional covariate information is present, mixture models can be extended…

Methodology · Statistics 2023-08-01 Sara Wade , Vanda Inacio , Sonia Petrone

Estimating causal effects from nonexperimental data is a fundamental problem in many fields of science. A key component of this task is selecting an appropriate set of covariates for confounding adjustment to avoid bias. Most existing…

Machine Learning · Computer Science 2025-10-28 Zheng Li , Xichen Guo , Feng Xie , Yan Zeng , Hao Zhang , Zhi Geng

Every design choice will have different effects on different units. However traditional A/B tests are often underpowered to identify these heterogeneous effects. This is especially true when the set of unit-level attributes is…

Artificial Intelligence · Computer Science 2016-11-09 Alexander Peysakhovich , Akos Lada

Traditionally, spline or kernel approaches in combination with parametric estimation are used to infer the linear coefficient (fixed effects) in a partially linear mixed-effects model for repeated measurements. Using machine learning…

Methodology · Statistics 2023-04-03 Corinne Emmenegger , Peter Bühlmann

A common problem in clinical trials is to test whether the effect of an explanatory variable on a response of interest is similar between two groups, e.g. patient or treatment groups. In this regard, similarity is defined as equivalence up…

Methodology · Statistics 2024-01-12 Niklas Hagemann , Giampiero Marra , Frank Bretz , Kathrin Möllenhoff

Pharmacological challenge imaging has mapped, but rarely quantified, the sensitivity of a biological system to a given drug. We describe a novel method called rapid quantitative pharmacodynamic imaging. This method combines…

Quantitative Methods · Quantitative Biology 2014-05-28 Kevin J. Black , Jonathan M. Koller , Brad D. Miller