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Incorporating historical information into the design and analysis of a new clinical trial has been the subject of much recent discussion. For example, in the context of clinical trials of antibiotics for drug resistant infections, where…

统计方法学 · 统计学 2018-06-08 Isaac Gravestock , Leonhard Held

Deep Neural Networks (DNN) have achieved human level performance in many image analytics tasks but DNNs are mostly deployed to GPU platforms that consume a considerable amount of power. Brain-inspired spiking neuromorphic chips consume low…

神经与进化计算 · 计算机科学 2016-05-26 Antonio Jimeno Yepes , Jianbin Tang

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

统计方法学 · 统计学 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Two common concerns raised in analyses of randomized experiments are (i) appropriately handling issues of non-compliance, and (ii) appropriately adjusting for multiple tests (e.g., on multiple outcomes or subgroups). Although simple…

统计方法学 · 统计学 2016-05-25 Joseph J. Lee , Laura Forastiere , Luke Miratrix , Natesh S. Pillai

The ongoing replication crisis in science has increased interest in the methodology of replication studies. We propose a novel Bayesian analysis approach using power priors: The likelihood of the original study's data is raised to the power…

统计方法学 · 统计学 2023-09-28 Samuel Pawel , Frederik Aust , Leonhard Held , Eric-Jan Wagenmakers

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A…

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

The purpose of this article is to infer patient level outcomes from population level randomized control trials (RCTs). In this pursuit, we utilize the recently proposed synthetic nearest neighbors (SNN) estimator. At its core, SNN leverages…

A clustered adaptive intervention (cAI) is a pre-specified sequence of decision rules that guides practitioners on how best - and based on which measures - to tailor cluster-level intervention to improve outcomes at the level of individuals…

统计方法学 · 统计学 2025-05-05 Yao Song , Kelly Speth , Amy Kilbourne , Andrew Quanbeck , Daniel Almirall , Lu Wang

Phase II basket trials are popular tools to evaluate efficacy of a new treatment targeting genetic alteration common to a set of different cancer histologies. Efficient designs are obtained by pooling data from the different arms (e.g.,…

统计方法学 · 统计学 2022-11-01 Massimo Ventrucci , Alessandro Vagheggini

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

统计方法学 · 统计学 2026-05-01 Elizabeth Tipton , Michalis Mamakos

This study investigates treatment effect estimation in the semi-supervised setting, also can be interpreted as prediction-powered inference. In our setting, we can use not only the standard triple of covariates, treatment indicator, and…

机器学习 · 统计学 2026-05-05 Masahiro Kato

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

统计方法学 · 统计学 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

Pre-symptomatic (or Preclinical) Alzheimer's Disease is defined by biomarker evidence of fibrillar amyloid beta pathology in the absence of clinical symptoms. Clinical trials in this early phase of disease are challenging due to the slow…

应用统计 · 统计学 2020-03-10 Dan Li , Samuel Iddi , Paul S. Aisen , Wesley K. Thompson , Michael C. Donohue

Propensity Score Matching (PSM) is an useful method to reduce the impact ofTreatment - Selection Bias in the estimation of causal effects in observational studies. After matching, the PSM significantly reduces the sample under…

统计方法学 · 统计学 2019-02-01 Daniel García Iglesias

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

统计方法学 · 统计学 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

An important goal of precision medicine is to personalize medical treatment by identifying individuals who are most likely to benefit from a specific treatment. The Likely Responder (LR) framework, which identifies a subpopulation where…

统计方法学 · 统计学 2026-03-13 Annan Deng , Carole Siegel , Hyung G. Park

The progression-free survival ratio (PFSr) is a widely used measure in personalized oncology trials. It evaluates the effectiveness of treatment by comparing two consecutive event times - one under standard therapy and one under an…

统计方法学 · 统计学 2025-12-23 Merle Munko , Simon Mack , Marc Ditzhaus , Stefan Fröhling , Dennis Dobler , Dominic Edelmann

We propose a new method to estimate causal effects from nonexperimental data. Each pair of sample units is first associated with a stochastic 'treatment' - differences in factors between units - and an effect - a resultant outcome…

统计方法学 · 统计学 2022-11-08 Andre F. Ribeiro , Frank Neffke , Ricardo Hausmann

Randomized experiments are widely used to estimate the causal effects of a proposed treatment in many areas of science, from medicine and healthcare to the physical and biological sciences, from the social sciences to engineering, to public…

统计方法学 · 统计学 2022-11-30 Christina Lee Yu , Edoardo M Airoldi , Christian Borgs , Jennifer T Chayes