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Indirect experiments provide a valuable framework for estimating treatment effects in situations where conducting randomized control trials (RCTs) is impractical or unethical. Unlike RCTs, indirect experiments estimate treatment effects by…

Machine Learning · Computer Science 2023-12-06 Yash Chandak , Shiv Shankar , Vasilis Syrgkanis , Emma Brunskill

Data-adaptive (machine learning-based) effect estimators are increasingly popular to reduce bias in high-dimensional bioinformatic and clinical studies (e.g. real-world data, target trials, -omic discovery). Their relative statistical…

Methodology · Statistics 2022-06-13 Xiang Meng , Jonathan Huang

Simulation studies allow us to explore the properties of statistical methods. They provide a powerful tool with a multiplicity of aims; among others: evaluating and comparing new or existing statistical methods, assessing violations of…

Applications · Statistics 2020-05-05 Alessandro Gasparini , Tim P. Morris , Michael J. Crowther

Research often necessitates of samples, yet obtaining large enough samples is not always possible. When it is, the researcher may use one of two methods for deciding upon the required sample size: rules-of-thumb, quick yet uncertain, and…

Methodology · Statistics 2016-04-08 Jose D. Perezgonzalez

Multi-arm designs provide an effective means of evaluating several treatments within the same clinical trial. Given the large number of treatments now available for testing in many disease areas, it has been argued that their utilisation…

Computation · Statistics 2019-06-24 Michael J Grayling , James MS Wason

How should researchers analyze randomized experiments in which the main outcome is latent and measured in multiple ways but each measure contains some degree of error? We first identify a critical study-specific noncomparability problem in…

Econometrics · Economics 2026-01-13 Jiawei Fu , Donald P. Green

This paper considers the design of observational longitudinal studies with a continuous response and a binary time-invariant exposure, where, typically, the exposure is unbalanced, the mean response in the two groups differs at baseline and…

Methodology · Statistics 2022-09-27 Xavier Basagana , Donna Spiegelman

Measurement error in observational datasets can lead to systematic bias in inferences based on these datasets. As studies based on observational data are increasingly used to inform decisions with real-world impact, it is critical that we…

Machine Learning · Statistics 2019-01-29 Roy Adams , Yuelong Ji , Xiaobin Wang , Suchi Saria

In many contexts it is extremely costly to perform enough high quality experimental measurements to accurately parameterize a predictive quantitative model. However, it is often much easier to carry out large numbers of experiments that…

Data Analysis, Statistics and Probability · Physics 2017-11-22 Alpha A. Lee , Michael P. Brenner , Lucy J. Colwell

When designing experimental studies with human participants, experimenters must decide how many trials each participant will complete, as well as how many participants to test. Most discussion of statistical power (the ability of a study…

Neurons and Cognition · Quantitative Biology 2021-08-30 Daniel H. Baker , Greta Vilidaite , Freya A. Lygo , Anika K. Smith , Tessa R. Flack , Andre D. Gouws , Timothy J. Andrews

In drug discovery, highly automated high-throughput laboratories are used to screen a large number of compounds in search of effective drugs. These experiments are expensive, so one might hope to reduce their cost by only experimenting on a…

Machine Learning · Computer Science 2025-04-15 Ihor Neporozhnii , Julien Roy , Emmanuel Bengio , Jason Hartford

Sequential Multiple-Assignment Randomized Trials (SMARTs) play an increasingly important role in psychological and behavioral health research. This experimental approach enables researchers to answer scientific questions about how to…

Methodology · Statistics 2023-06-21 John J. Dziak , Daniel Almirall , Walter Dempsey , Catherine Stanger , Inbal Nahum-Shani

One way to investigate the precision of estimates likely to result from planned experiments and planned epidemiological studies is to simulate a large number of possible outcomes and analyse the sets of possible results. This appears to be…

Computation · Statistics 2013-06-28 G. K. Robinson , L. M. Ryan

Emotional states, as indicators of affect, are pivotal to overall health, making their accurate prediction before onset crucial. Current studies are primarily centered on immediate short-term affect detection using data from wearable and…

Simulations are valuable tools for empirically evaluating the properties of statistical methods and are primarily employed in methodological research to draw general conclusions about methods. In addition, they can often be useful to…

Other Statistics · Statistics 2025-10-08 Anne-Laure Boulesteix , Patrick Callahan , Luzia Hanssum , Vincent Gaertner , Eva Hoster

Trials enroll a large number of subjects in order to attain power, making them expensive and time-consuming. Sample size calculations are often performed with the assumption of an unadjusted analysis, even if the trial analysis plan…

Methodology · Statistics 2021-07-06 Alejandro Schuler

We introduce a new framework for sample-efficient model evaluation that we call active testing. While approaches like active learning reduce the number of labels needed for model training, existing literature largely ignores the cost of…

Machine Learning · Statistics 2021-06-15 Jannik Kossen , Sebastian Farquhar , Yarin Gal , Tom Rainforth

The question of selecting the "best" amongst different choices is a common problem in statistics. In drug development, our motivating setting, the question becomes, for example: what is the dose that gives me a pre-specified risk of…

Statistics Theory · Mathematics 2018-03-15 Pavel Mozgunov , Thomas Jaki

Confounding and exposure measurement error can introduce bias when drawing inference about the marginal effect of an exposure on an outcome of interest. While there are broad methodologies for addressing each source of bias individually,…

Methodology · Statistics 2025-01-29 Brian D. Richardson , Bryan S. Blette , Peter B. Gilbert , Michael G. Hudgens

Group testing techniques are widely used in resource-constrained settings, such as infectious-disease screening, blood safety, DNA library screening, and industrial inspection, where the efficient use of limited testing resources depends…

Methodology · Statistics 2026-04-29 Chi-Kuang Yeh , Weng Kee Wong , Julie Zhou
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