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Exposure mappings are widely used to model potential outcomes in the presence of interference, where each unit's outcome may depend not only on its own treatment, but also on the treatment of other units as well. However, in practice these…

Methodology · Statistics 2018-07-02 David Choi

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

Applications · Statistics 2014-07-22 Xiao Lin , Gabriel Terejanu

Mixed-effect models are flexible tools for researchers in a myriad of fields, but that flexibility comes at the cost of complexity and if users are not careful in how their model is specified, they could be making faulty inferences from…

Methodology · Statistics 2023-08-28 Keith R. Lohse , Allan J. Kozlowski , Michael J. Strube

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

Methodology · Statistics 2016-01-06 Holger Dette , Katrin Kettelhake , Kirsten Schorning , Weng Kee Wong , Frank Bretz

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

The optimization of composition and processing to obtain materials that exhibit desirable characteristics has historically relied on a combination of scientist intuition, trial and error, and luck. We propose a methodology that can…

Machine Learning · Statistics 2017-07-20 Julia Ling , Max Hutchinson , Erin Antono , Sean Paradiso , Bryce Meredig

In the first stage of a two-stage study, the researcher uses a statistical model to impute the unobserved exposures. In the second stage, imputed exposures serve as covariates in epidemiological models. Imputation error in the first stage…

Applications · Statistics 2021-07-19 Ron Sarafian , Itai Kloog , Jonathan D. Rosenblatt

Estimating sample size and statistical power is an essential part of a good study design. This R package allows users to conduct power analysis based on Monte Carlo simulations in settings in which consideration of the correlations between…

Methodology · Statistics 2024-04-16 Phuc H. Nguyen , Stephanie M. Engel , Amy H. Herring

The growing availability of observational databases like electronic health records (EHR) provides unprecedented opportunities for secondary use of such data in biomedical research. However, these data can be error-prone and need to be…

Methodology · Statistics 2024-05-28 Sarah C. Lotspeich , Gustavo G. C. Amorim , Pamela A. Shaw , Ran Tao , Bryan E. Shepherd

How can we find a general way to choose the most suitable samples for training a classifier? Even with very limited prior information? Active learning, which can be regarded as an iterative optimization procedure, plays a key role to…

Machine Learning · Computer Science 2019-04-16 Bo Du , Zengmao Wang , Lefei Zhang , Liangpei Zhang , Wei Liu , Jialie Shen , Dacheng Tao

Familiar statistical tests and estimates are obtained by the direct observation of cases of interest: a clinical trial of a new drug, for instance, will compare the drug's effects on a relevant set of patients and controls. Sometimes,…

Methodology · Statistics 2010-12-09 Bradley Efron

In bioequivalence design, power analyses dictate how much data must be collected to detect the absence of clinically important effects. Power is computed as a tail probability in the sampling distribution of the pertinent test statistics.…

Methodology · Statistics 2025-01-27 Luke Hagar , Nathaniel T. Stevens

Sample size calculation is crucial in biomedical in vivo research investigations mainly for two reasons: to design the most resource-efficient studies and to safeguard ethical issues when alive animals are subjects of testing. In this…

Applications · Statistics 2025-05-27 Hasan Al-Nashash , Jiajin Wei , Ke Yang , Ayman Alzaatreh , Mohsen Adeli , Tiejun Tong , Angelo All

Randomized A/B tests within online learning platforms represent an exciting direction in learning sciences. With minimal assumptions, they allow causal effect estimation without confounding bias and exact statistical inference even in small…

Methodology · Statistics 2023-06-13 Adam C. Sales , Ethan B. Prihar , Johann A. Gagnon-Bartsch , Neil T. Heffernan

In this paper we propose a new template for empirical studies intended to assess causal effects: the outcome-wide longitudinal design. The approach is an extension of what is often done to assess the causal effects of a treatment or…

Methodology · Statistics 2018-10-25 Tyler J. VanderWeele , Maya B. Mathur , Ying Chen

There are a number of measures of direct and indirect effects in the literature. They are suitable in some cases and unsuitable in others. We describe a case where the existing measures are unsuitable and propose new suitable ones. We also…

Methodology · Statistics 2023-10-12 Jose M. Peña

This paper shows how to use a randomized saturation experimental design to identify and estimate causal effects in the presence of spillovers--one person's treatment may affect another's outcome--and one-sided non-compliance--subjects can…

We consider experiments for comparing treatments using units that are ordered linearly over time or space within blocks. In addition to the block effect, we assume that a trend effect influences the response. The latter is modeled as a…

Statistics Theory · Mathematics 2008-12-18 Dibyen Majumdar , John Stufken

Estimating the effect of treatments from natural experiments, where treatments are pre-assigned, is an important and well-studied problem. We introduce a novel natural experiment dataset obtained from an early childhood literacy nonprofit.…

Machine Learning · Statistics 2024-09-10 R. Teal Witter , Christopher Musco

Optimal design for model training is a critical topic in machine learning. Active Learning aims at obtaining improved models by querying samples with maximum uncertainty according to the estimation model for artificially labeling; this has…