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相关论文: Digital N-of-1 Trials and their Application in Exp…

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N-of-1 trials, single participant trials in which multiple treatments are sequentially randomized over the study period, can give direct estimates of individual-specific treatment effects. Combining n-of-1 trials gives extra information for…

统计方法学 · 统计学 2021-10-19 Jiabei Yang , Jon A. Steingrimsson , Christopher H. Schmid

N-of-1 trials are within-person crossover trials allowing both personalized and population-level inference on the effect of health interventions. Using the full potential of modern technologies, multimodal N-of-1 trials can integrate…

应用统计 · 统计学 2025-10-31 Juliana Schneider , Thomas Gärtner , Stefan Konigorski

N-of-1 trials allow inference between two treatments given to a single individual. Most often, clinical investigators analyze an individual's N-of-1 trial data with usual t-tests or simple nonparametric methods. These simple methods do not…

统计方法学 · 统计学 2020-07-01 Jillian Tang , Reid D. Landes

N-of-1 trials aim to estimate treatment effects on the individual level and can be applied to personalize a wide range of physical and digital interventions in mHealth. In this study, we propose and apply a framework for multimodal N-of-1…

N-of-1 trials are the gold standard study design to evaluate individual treatment effects and derive personalized treatment strategies. Digital tools have the potential to initiate a new era of N-of-1 trials in terms of scale and scope, but…

N-of-1 experiments, where a unit serves as its own control and treatment in different time windows, have been used in certain medical contexts for decades. However, due to effects that accumulate over long time windows and interventions…

统计方法学 · 统计学 2025-02-25 Tengyuan Liang , Benjamin Recht

App-based N-of-1 trials offer a scalable experimental design for assessing the effects of health interventions at an individual level. Their practical success depends on the strong motivation of participants, which, in turn, translates into…

统计方法学 · 统计学 2023-09-15 Ivana Malenica , Yongyi Guo , Kyra Gan , Stefan Konigorski

Studying individual causal effects of health interventions is of interest whenever intervention effects are heterogeneous between study participants. Conducting N-of-1 trials, which are single-person randomized controlled trials, is the…

应用统计 · 统计学 2024-03-11 Lin Zhou , Juliana Schneider , Bert Arnrich , Stefan Konigorski

This article proposes a novel adaptive design algorithm that can be used to find optimal treatment allocations in N-of-1 clinical trials. This new methodology uses two Laplace approximations to provide a computationally efficient estimate…

统计计算 · 统计学 2020-07-30 S. G. Jagath Senarathne , Antony M. Overstall , James M. McGree

An N-of-1 trial is a multi-period crossover trial performed in a single individual, with a primary goal to estimate treatment effect on the individual instead of population-level mean responses. As in a conventional crossover trial, it is…

应用统计 · 统计学 2021-12-30 Ziwei Liao , Min Qian , Ian M. Kronish , Ying Kuen Cheung

The aim of personalized medicine is to tailor treatment decisions to individuals' characteristics. N-of-1 trials are within-person crossover trials that hold the promise of targeting individual-specific effects. While the idea behind N-of-1…

统计方法学 · 统计学 2025-12-03 Marco Piccininni , Mats J. Stensrud , Zachary Shahn , Stefan Konigorski

An N-of-1 trial is a multiple crossover trial conducted in a single individual to provide evidence to directly inform personalized treatment decisions. Advancements in wearable devices greatly improved the feasibility of adopting these…

统计方法学 · 统计学 2024-10-15 Kexin Qu , Christopher H. Schmid , Tao Liu

Time-series experiments, also called switchback experiments or N-of-1 trials, play increasingly important roles in modern applications in medical and industrial areas. Under the potential outcomes framework, recent research has studied…

统计方法学 · 统计学 2025-10-28 Zhexiao Lin , Peng Ding

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…

统计方法学 · 统计学 2010-12-01 Peter F. Thall

Personalized adaptive interventions offer the opportunity to increase patient benefits, however, there are challenges in their planning and implementation. Once implemented, it is an important question whether personalized adaptive…

机器学习 · 计算机科学 2023-11-27 Dominik Meier , Ipek Ensari , Stefan Konigorski

Clinical trials are an indispensable part of the drug development process, bridging the gap between basic research and clinical application. During the development of new drugs, clinical trials are used not only to evaluate the safety and…

Efficacy testing is a cornerstone of clinical trials, ensuring that medical interventions achieve their intended therapeutic effects. Over the decades, a wide range of statistical methodologies have been developed to address the…

其他统计学 · 统计学 2026-04-13 Dhrubajyoti Ghosh , Samhita Pal

Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…

统计方法学 · 统计学 2011-05-18 Jay Bartroff , Tze Leung Lai

Artificial intelligence in medicine is built to serve the average patient. By minimizing error across large datasets, most systems deliver strong aggregate accuracy yet falter at the margins: patients with rare variants, multimorbidity, or…

人工智能 · 计算机科学 2025-10-29 Pedram Fard , Alaleh Azhir , Neguine Rezaii , Jiazi Tian , Hossein Estiri

There is strong interest in estimating how the magnitude of treatment effects of an intervention vary across sub-groups of the population of interest. In our paper, we propose a two-study approach to first propose and then test…

统计方法学 · 统计学 2020-06-23 Rahul Ladhania , Amelia Haviland , Neeraj Sood , Edward Kennedy , Ateev Mehrotra
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