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We consider planning longitudinal covariate measurements in follow-up studies where covariates are time-varying. We assume that the entire cohort cannot be selected for longitudinal measurements due to financial limitations and study how a…

Methodology · Statistics 2024-07-03 Jaakko Reinikainen , Juha Karvanen

Survey researchers are increasingly turning to multimode data collection to deal with declines in survey response rates and increasing costs. An efficient approach offers the less costly modes (e.g., web) followed with a more expensive mode…

Methodology · Statistics 2023-03-24 J. Michael Brick , Jill M. DeMatteis

We introduce a new approach to variable selection, called Predictive Correlation Screening, for predictor design. Predictive Correlation Screening (PCS) implements false positive control on the selected variables, is well suited to small…

Machine Learning · Statistics 2013-04-11 Hamed Firouzi , Bala Rajaratnam , Alfred Hero

With reference to a stratified case-control procedure based on a binary variable of primary interest, we derive the expression of the distortion induced by the sampling design on the parameters of the logistic model of a secondary variable.…

Methodology · Statistics 2024-01-18 Marco Doretti , Minna Genbäck , Elena Stanghellini

The goal of subsampling is to select an informative subset of all observations, when using the full data for statistical analysis is not viable. We construct locally $ D $-optimal subsampling designs under a Poisson regression model with a…

Statistics Theory · Mathematics 2024-03-28 Torsten Reuter , Rainer Schwabe

Causal inference is crucial for understanding the true impact of interventions, policies, or actions, enabling informed decision-making and providing insights into the underlying mechanisms that shape our world. In this paper, we establish…

Methodology · Statistics 2024-03-26 Jingyue Huang , Changbao Wu , Leilei Zeng

Two-stage randomization is a powerful design for estimating treatment effects in the presence of interference; that is, when one individual's treatment assignment affects another individual's outcomes. Our motivating example is a two-stage…

Applications · Statistics 2017-05-02 Guillaume Basse , Avi Feller

While much of the causal inference literature has focused on addressing internal validity biases, both internal and external validity are necessary for unbiased estimates in a target population of interest. However, few generalizability…

Methodology · Statistics 2023-04-07 Irina Degtiar , Tim Layton , Jacob Wallace , Sherri Rose

Increasingly, medical research is dependent on data collected for non-research purposes, such as electronic health records data (EHR). EHR data and other large databases can be prone to measurement error in key exposures, and unadjusted…

Methodology · Statistics 2020-05-13 Kyunghee Han , Thomas Lumley , Bryan E. Shepherd , Pamela A. Shaw

Adapting the final sample size of a trial to the evidence accruing during the trial is a natural way to address planning uncertainty. Designs with adaptive sample size need to account for their optional stopping to guarantee strict type-I…

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

Computation · Statistics 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

Estimating causal effects under interference is pertinent to many real-world settings. Recent work with low-order potential outcomes models uses a rollout design to obtain unbiased estimators that require no interference network…

Methodology · Statistics 2025-02-12 Mayleen Cortez-Rodriguez , Matthew Eichhorn , Christina Lee Yu

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…

Machine Learning · Statistics 2026-05-05 Masahiro Kato

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

The case-control design is often used to test associations between the case-control status and genetic variants. In addition to this primary phenotype a number of additional traits, known as secondary phenotypes, are routinely recorded and…

2-in-1 design (Chen et al. 2018) is becoming popular in oncology drug development, with the flexibility of using different endpoints at different decision time. Based on the observed interim data, sponsors choose either to seamlessly…

Methodology · Statistics 2024-04-15 Runjia Li , Liwen Wu , Rachael Liu , Jianchang Lin

The proximal causal inference framework enables the identification and estimation of causal effects in the presence of unmeasured confounding by leveraging two disjoint sets of observed strong proxies: negative control treatments and…

Methodology · Statistics 2025-12-16 Antonio Olivas-Martinez , Peter B. Gilbert , Andrea Rotnitzky

In regression models fitted to data from complex survey designs, sampling weights often incorporate non-essential variation, inflating variance estimates. Stabilized weights mitigate this issue by adjusting sampling weights to account for…

Methodology · Statistics 2026-05-18 Tong Chen , Joshua Slone , Gustavo Amorim , Pamela A. Shaw , Bryan E. Shepherd , Thomas Lumley

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp
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