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Evaluating treatment effect heterogeneity across patient subgroups is a fundamental aspect of clinical trial analysis. Yet, these analyses have inherent limitations due to small sample sizes and the substantial number of subgroups…

统计方法学 · 统计学 2026-03-24 Marcel Wolbers , Miriam Pedrera Gómez , Alex Ocampo , Isaac Gravestock

This paper develops a sparsity-inducing version of Bayesian Causal Forests, a recently proposed nonparametric causal regression model that employs Bayesian Additive Regression Trees and is specifically designed to estimate heterogeneous…

统计方法学 · 统计学 2021-11-17 Alberto Caron , Gianluca Baio , Ioanna Manolopoulou

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

统计计算 · 统计学 2016-06-28 Marius Thomas , Björn Bornkamp

The inclusion of the propensity score as a covariate in Bayesian regression trees for causal inference can reduce the bias in treatment effect estimations, which occurs due to the regularization-induced confounding phenomenon. This study…

统计方法学 · 统计学 2018-08-30 Pedro Henrique Filipini dos Santos , Hedibert Freitas Lopes

Recently, phase II trials with multiple schedules (frequency of administrations) have become more popular, for instance in the development of treatments for atopic dermatitis. If the relationship of the dose and response is described by a…

应用统计 · 统计学 2020-11-17 Burak Kürsad Günhan , Paul Meyvisch , Tim Friede

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

统计方法学 · 统计学 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

An important task in clinical medicine is the construction of risk prediction models for specific subgroups of patients based on high-dimensional molecular measurements such as gene expression data. Major objectives in modeling…

统计方法学 · 统计学 2020-03-23 Katrin Madjar , Jörg Rahnenführer

We develop and analyze empirical Bayes Stein-type estimators for use in the estimation of causal effects in large-scale online experiments. While online experiments are generally thought to be distinguished by their large sample size, we…

统计方法学 · 统计学 2019-11-15 Drew Dimmery , Eytan Bakshy , Jasjeet Sekhon

Motivated by the increasing use of and rapid changes in array technologies, we consider the prediction problem of fitting a linear regression relating a continuous outcome $Y$ to a large number of covariates $\mathbf {X}$, for example,…

应用统计 · 统计学 2014-01-13 Philip S. Boonstra , Bhramar Mukherjee , Jeremy M. G. Taylor

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

应用统计 · 统计学 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

统计方法学 · 统计学 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

Many scientific and engineering challenges -- ranging from personalized medicine to customized marketing recommendations -- require an understanding of treatment effect heterogeneity. In this paper, we develop a non-parametric causal forest…

统计方法学 · 统计学 2017-07-11 Stefan Wager , Susan Athey

Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

统计方法学 · 统计学 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

Important objectives in cancer research are the prediction of a patient's risk based on molecular measurements such as gene expression data and the identification of new prognostic biomarkers (e.g. genes). In clinical practice, this is…

应用统计 · 统计学 2020-04-17 Katrin Madjar , Manuela Zucknick , Katja Ickstadt , Jörg Rahnenführer

Randomized controlled trials play an important role in how Internet companies predict the impact of policy decisions and product changes. In these `digital experiments', different units (people, devices, products) respond differently to the…

应用统计 · 统计学 2015-12-21 Matt Taddy , Matt Gardner , Liyun Chen , David Draper

Most estimates for penalised linear regression can be viewed as posterior modes for an appropriate choice of prior distribution. Bayesian shrinkage methods, particularly the horseshoe estimator, have recently attracted a great deal of…

统计方法学 · 统计学 2017-11-06 Zemei Xu , Daniel F. Schmidt , Enes Makalic , Guoqi Qian , John L. Hopper

We consider random forests and LASSO methods for model-based small area estimation when the number of areas with sampled data is a small fraction of the total areas for which estimates are required. Abundant auxiliary information is…

We develop a Bayesian tree ensemble model to estimate heterogeneous treatment effects in censored survival data with high-dimensional covariates. Instead of imposing sparsity through the tree structure, we place a horseshoe prior directly…

统计方法学 · 统计学 2026-05-08 Tijn Jacobs , Wessel N. van Wieringen , Stéphanie L. van der Pas

A new matching method is proposed for the estimation of the average treatment effect of social policy interventions (e.g., training programs or health care measures). Given an outcome variable, a treatment and a set of pre-treatment…

统计理论 · 数学 2007-06-13 Stefano Iacus , Giuseppe Porro

Analysing subgroups defined by biomarkers is of increasing importance in clinical research. In some situations the biomarker is subject to misclassification error, meaning the true subgroups are identified with imperfect sensitivity and…

统计方法学 · 统计学 2018-01-16 Fang Wan , Andrew C. Titman , Thomas F. Jaki
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