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相关论文: Evaluating virtual-control-augmented trials for re…

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Randomized controlled trials (RCTs) have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically…

统计方法学 · 统计学 2025-11-26 Shu Yang , Margaret Gamalo , Haoda Fu

The paper considers two different designs; a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome, and a twoarm superiority cluster RCT with a binary outcome. From a Bayesian perspective, for the analysis…

统计方法学 · 统计学 2025-11-11 Abdullah Aloufi , Kevin Wilson , Nina Wilson , Lisa Shaw , Christopher Price

Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

应用统计 · 统计学 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

统计方法学 · 统计学 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Cluster-randomized trials (CRTs) are experimental designs where groups or clusters of participants, rather than the individual participants themselves, are randomized to intervention groups. Analyzing CRT requires distinguishing between…

统计方法学 · 统计学 2025-10-10 Xi Fang , Bingkai Wang , Liangyuan Hu , Fan Li

Randomized experiments play a major role in data-driven decision making across many different fields and disciplines. In medicine, for example, randomized controlled trials (RCTs) are the backbone of clinical trial methodology for testing…

应用统计 · 统计学 2016-08-30 Andrew W. Correia

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Response-adaptive clinical trial designs allow targeting a given objective by skewing the allocation of participants to treatments based on observed outcomes. Response-adaptive designs face greater regulatory scrutiny due to potential type…

统计方法学 · 统计学 2025-03-19 Stef Baas , Peter Jacko , Sofía S. Villar

Shared controls in platform trials comprise concurrent and non-concurrent controls. For a given experimental arm, non-concurrent controls refer to data from patients allocated to the control arm before the arm enters the trial. The use of…

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

定量方法 · 定量生物学 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

A central obstacle in the objective assessment of treatment effect (TE) estimators in randomized control trials (RCTs) is the lack of ground truth (or validation set) to test their performance. In this paper, we propose a novel…

Hybrid randomized controlled trials (hybrid RCTs) integrate external control data, such as historical or concurrent data, with data from randomized trials. While numerous frequentist and Bayesian methods, such as the test-then-pool and…

统计方法学 · 统计学 2025-10-07 Han Chang Chiam , Franz König , Martin Posch

An individualized treatment rule (ITR) tailors treatments to a patient's specific characteristics. However, randomized controlled trials (RCTs) are often underpowered to detect the treatment effect heterogeneity needed for reliable ITR…

统计方法学 · 统计学 2026-04-14 Yuan Bian , Donglin Zeng , Hyun-Joon Yang , Leanne M. Williams , Yuanjia Wang

In a randomized control trial, the precision of an average treatment effect estimator can be improved either by collecting data on additional individuals, or by collecting additional covariates that predict the outcome variable. We propose…

统计方法学 · 统计学 2017-09-27 Pedro Carneiro , Sokbae Lee , Daniel Wilhelm

Clinical data usually cannot be freely distributed due to their highly confidential nature and this hampers the development of machine learning in the healthcare domain. One way to mitigate this problem is by generating realistic synthetic…

Augmenting a randomized controlled trial (RCT) with external data may increase power at the risk of introducing bias. To select and analyze the experiment (RCT alone or combined with external data) with the optimal bias-variance tradeoff,…

Learning from visual observations is a fundamental yet challenging problem in Reinforcement Learning (RL). Although algorithmic advances combined with convolutional neural networks have proved to be a recipe for success, current methods are…

机器学习 · 计算机科学 2020-11-06 Michael Laskin , Kimin Lee , Adam Stooke , Lerrel Pinto , Pieter Abbeel , Aravind Srinivas

Objective: We aimed to develop a meta-analytic model for evaluation of predictive biomarkers and targeted therapies, utilising data from digital sources when individual participant data (IPD) from randomised controlled trials (RCTs) are…

Purpose: To assess the feasibility of deep learning-based high resolution synthetic CT generation from MRI scans of the lower arm for orthopedic applications. Methods: A conditional Generative Adversarial Network was trained to synthesize…

This study leverages synthetic data as a validation set to reduce overfitting and ease the selection of the best model in AI development. While synthetic data have been used for augmenting the training set, we find that synthetic data can…

计算机视觉与模式识别 · 计算机科学 2023-10-25 Qixin Hu , Alan Yuille , Zongwei Zhou