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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

This article discusses D-optimal Bayesian crossover designs for generalized linear models. Crossover trials with t treatments and p periods, for $t <= p$, are considered. The designs proposed in this paper minimize the log determinant of…

统计计算 · 统计学 2018-08-16 Satya Prakash Singh , Siuli Mukhopadhyay

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 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…

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

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

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 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

A crossover trial is an efficient trial design when there is no carry-over effect. To reduce the impact of the biological carry-over effect, a washout period is often designed. However, the carry-over effect remains an outstanding concern…

应用统计 · 统计学 2024-03-06 Danni Shi , Ting Ye

Traditionally, studies in experimental physiology have been conducted in small groups of human participants, animal models or cell lines. Identifying optimal study designs that achieve sufficient power for drawing proper statistical…

应用统计 · 统计学 2026-05-21 Stefan Konigorski , Mathias Ried-Larsen , Christopher H Schmid

We propose a novel Bayesian model selection technique on linear mixed-effects models to compare multiple treatments with a control. A fully Bayesian approach is implemented to estimate the marginal inclusion probabilities that provide a…

应用统计 · 统计学 2015-09-28 Lei Gong , James M. Flegal , Stephen R. Spindler , Patricia L. Mote

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

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

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

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

计量经济学 · 经济学 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

It has been argued for many years that models used to analyze data from crossover designs are not appropriate when simple carryover effects are assumed. Furthermore, a statistical model that could estimate complex carry-over effects in…

统计方法学 · 统计学 2025-08-22 N. A. Cruz , K. Mylona , O. O. Melo

Distributed lag models (DLMs) express the cumulative and delayed dependence between pairs of time-indexed response and explanatory variables. In practical application, users of DLMs examine the estimated influence of a series of lagged…

应用统计 · 统计学 2018-01-23 Alastair Rushworth

In empirical studies with time-to-event outcomes, investigators often leverage observational data to conduct causal inference on the effect of exposure when randomized controlled trial data is unavailable. Model misspecification and lack of…

统计方法学 · 统计学 2023-05-05 Shenbo Xu , Bang Zheng , Bowen Su , Stan Finkelstein , Roy Welsch , Kenney Ng , Ioanna Tzoulaki , Zach Shahn

We conducted a systematic comparison of statistical methods used for the analysis of time-to-event outcomes under various proportional and nonproportional hazard (NPH) scenarios. Our study used data from recently published oncology trials…

应用统计 · 统计学 2025-02-12 Xinyu Zhang , Erich J. Greene , Ondrej Blaha , Wei Wei

The effectiveness of personalized oncology treatments ultimately depends on whether outcomes can be causally attributed to the treatment. Advances in precision oncology have improved molecular profiling of individuals, and tailored…

应用统计 · 统计学 2025-09-29 Lydia Jang , Stefan Konigorski
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