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A central goal in designing clinical trials is to find the test that maximizes power (or equivalently minimizes required sample size) for finding a false null hypothesis subject to the constraint of type I error. When there is more than one…

统计方法学 · 统计学 2022-09-21 Ruth Heller , Abba Krieger , Saharon Rosset

Treatment effects can be estimated from observational data as the difference in potential outcomes. In this paper, we address the challenge of estimating the potential outcome when treatment-dose levels can vary continuously over time.…

机器学习 · 统计学 2017-11-07 Hossein Soleimani , Adarsh Subbaswamy , Suchi Saria

It is shown how by not losing information on higher order interactions, optimal paired comparison designs involving alternatives of either full or partial profiles to reduce information overload as frequently encountered in applications can…

统计方法学 · 统计学 2019-12-04 Eric Nyarko

Identifying heterogeneity in a population's response to a health or policy intervention is crucial for evaluating and informing policy decisions. We propose a novel heterogeneous treatment effect estimator in the difference-in-differences…

统计方法学 · 统计学 2021-08-24 Xinkun Nie , Chen Lu , Stefan Wager

Traditional methods for covariate adjustment of treatment means in designed experiments are inherently conditional on the observed covariate values. In order to develop a coherent general methodology for analysis of covariance, we propose a…

统计方法学 · 统计学 2010-01-19 James G. Booth , Walter T. Federer , Martin T. Wells , Russell D. Wolfinger

We consider in this paper the problem of optimal experiment design where a decision maker can choose which points to sample to obtain an estimate $\hat{\beta}$ of the hidden parameter $\beta^{\star}$ of an underlying linear model. The key…

机器学习 · 统计学 2021-01-01 Xavier Fontaine , Pierre Perrault , Michal Valko , Vianney Perchet

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

统计方法学 · 统计学 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

Consider an experiment consisting of a set of independent trials for comparing a set of treatments. In each trial, one treatment is chosen and the mean response of the trial is equal to the effect of the chosen treatment. We examine the…

统计理论 · 数学 2016-12-21 Samuel Rosa

Adaptive designs are increasingly used in clinical trials and online experiments to improve participant outcomes by dynamically updating treatment allocation as data accumulate. In practice, experimenters often consider multiple candidate…

统计方法学 · 统计学 2026-04-08 Wenxin Zhang , Aaron Hudson , Maya Petersen , Mark van der Laan

Evaluating the influence of continuous covariates, like exposure time or dose, on a response variable is a pivotal objective in the assessment of a compound's effect, particularly when determining toxicity in pre-clinical research or…

统计方法学 · 统计学 2026-04-16 Lucia Ameis , Niklas Hagemann , Kathrin Möllenhoff

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

A multiplicative effects model is introduced for the identification of the factors that are influential to the performance of highly-trained endurance runners. The model extends the established power-law relationship between performance…

应用统计 · 统计学 2015-07-02 Ioannis Kosmidis , Louis Passfield

We consider the problem of designing experiments for the comparison of two regression curves describing the relation between a predictor and a response in two groups, where the data between and within the group may be dependent. In order to…

统计理论 · 数学 2021-01-15 Kirsten Schorning , Holger Dette

Crossover designs are an extremely useful tool to investigators, whilst group sequential methods have proven highly proficient at improving the efficiency of parallel group trials. Yet, group sequential methods and crossover designs have…

统计方法学 · 统计学 2017-10-11 Michael Grayling , James Wason , Adrian Mander

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

Loss of power and clear description of treatment differences are key issues in designing and analyzing a clinical trial where non-proportional hazard is a possibility. A log-rank test may be very inefficient and interpretation of the hazard…

应用统计 · 统计学 2021-01-13 Satrajit Roychoudhury , Keaven M Anderson , Jiabu Ye , Pralay Mukhopadhyay

We consider the following comparative effectiveness scenario. There are two treatments for a particular medical condition: a randomized experiment has demonstrated mediocre effectiveness for the first treatment, while a non-randomized study…

统计方法学 · 统计学 2024-08-23 Brian Knaeble , Erich Kummerfeld

We propose a flexible design for the identification of optimal dose combinations in dual-agent dose-finding clinical trials. The design is called AAA, standing for three adaptations: adaptive model selection, adaptive dose insertion, and…

统计方法学 · 统计学 2017-06-13 Jiaying Lyu , Yuan Ji , Naiqing Zhao , Daniel V. T. Catenacci

We propose an approach to better inform treatment decisions at an individual level by adapting recent advances in average treatment effect estimation to conditional average treatment effect estimation. Our work is based on doubly robust…

统计方法学 · 统计学 2023-06-13 Aaron Fisher , Virginia Fisher

Matched-pair experimental designs aim to detect treatment effects by pairing participants and comparing within-pair outcome differences. In many situations, the overall effect size across the entire population is small. Then, the focus…

机器学习 · 计算机科学 2025-09-26 Weizhi Li , Gautam Dasarathy , Visar Berisha