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The age of big data has produced data sets that are computationally expensive to analyze and store. Algorithmic leveraging proposes that we sample observations from the original data set to generate a representative data set and then…

Applications · Statistics 2018-03-13 Katelyn Gao

This paper explores the use of a fuzzy regression discontinuity design where multiple treatments are applied at the threshold. The identification results show that, under the very strong assumption that the change in the probability of…

Econometrics · Economics 2021-04-20 Hector Galindo-Silva , Nibene Habib Some , Guy Tchuente

The average treatment effect (ATE), the mean difference in potential outcomes under treatment and control, is a canonical causal effect. Overlap, which says that all subjects have non-zero probability of either treatment status, is…

Methodology · Statistics 2026-05-14 Herbert P. Susmann , Alec McClean , Iván Díaz

Concerns over reproducibility in science extend to research using existing healthcare data; many observational studies investigating the same topic produce conflicting results, even when using the same data. To address this problem, we…

Applications · Statistics 2018-03-30 Martijn J. Schuemie , Patrick B. Ryan , George Hripcsak , David Madigan , Marc A. Suchard

In order to estimate the proportion of `immune' or `cured' subjects who will never experience failure, a sufficiently long follow-up period is required. Several statistical tests have been proposed in the literature for assessing the…

Methodology · Statistics 2026-01-12 Tsz Pang Yuen , Eni Musta

In a comprehensive cohort study of two competing treatments (say, A and B), clinically eligible individuals are first asked to enroll in a randomized trial and, if they refuse, are then asked to enroll in a parallel observational study in…

Methodology · Statistics 2019-10-09 Yi Lu , Daniel O. Scharfstein , Maria M. Brooks , Kevin Quach , Edward H. Kennedy

In a clinical trial with a survival outcome, an interim analysis is often performed to allow for early stopping for efficacy. If the interim analysis is early in the trial, one might conclude that a new treatment is more effective (compared…

Methodology · Statistics 2023-05-09 Marianne A Jonker , Steven Teerenstra

Generalizing treatment effects from a randomized trial to a target population requires the assumption that potential outcome distributions are invariant across populations after conditioning on observed covariates. This assumption fails…

Methodology · Statistics 2026-04-16 Amir Asiaee , Samhita Pal , Cole Beck , Jared D. Huling

Random effects meta-analysis is a widely applied methodology to synthetize research findings of studies in a specific scientific question. Besides estimating the mean effect, an important aim of the meta-analysis is to summarize the…

Applications · Statistics 2026-01-28 Peter Matrai , Tamas Koi , Zoltan Sipos , Nelli Farkas

In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…

Methodology · Statistics 2025-06-05 Boyu Ren , Federico Ferrari , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

We propose several prediction intervals procedures for the individual treatment effect with either finite-sample or asymptotic coverage guarantee in a non-parametric regression setting, where non-linear regression functions,…

Methodology · Statistics 2020-06-03 Danijel Kivaranovic , Robin Ristl , Martin Posch , Hannes Leeb

It is recommended that measures of between-study effect heterogeneity be reported when conducting individual-participant data meta-analyses (IPD-MA). Methods exist to quantify inconsistency between trials via I^2 (the percentage of…

We consider repeated measurement designs when a residual or carry-over effect may be present in at most one later period. Since assuming an additive model may be unrealistic for some applications and leads to biased estimation of treatment…

Statistics Theory · Mathematics 2014-11-20 R. A. Bailey , P. Druilhet

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

This work proposes a statistical model for crossover trials with multiple skewed responses measured in each period. A 3 $\times$ 3 crossover trial data where different drug doses were administered to subjects with a history of seasonal…

Methodology · Statistics 2026-04-09 Savita Pareek , Kalyan Das , Siuli Mukhopadhyay

Estimating the effect of a change in a particular risk factor and a chronic disease requires information on the risk factor from two time points; the enrolment and the first follow-up. When using observational data to study the effect of…

Progressive censoring scheme has received considerable attention in recent years. In this paper we introduce a new type-II progressive censoring scheme for two samples. It is observed that the proposed censoring scheme is analytically more…

Methodology · Statistics 2016-09-20 Shuvashree Mondal , Debasis Kundu

Uncertainty quantification of causal effects is crucial for safety-critical applications such as personalized medicine. A powerful approach for this is conformal prediction, which has several practical benefits due to model-agnostic…

Machine Learning · Computer Science 2026-02-04 Maresa Schröder , Dennis Frauen , Jonas Schweisthal , Konstantin Heß , Valentyn Melnychuk , Stefan Feuerriegel

Causal effects are commonly defined as comparisons of the potential outcomes under treatment and control, but this definition is threatened by the possibility that the treatment or control condition is not well-defined, existing instead in…

Methodology · Statistics 2019-04-26 Raiden B. Hasegawa , Sameer K. Deshpande , Dylan S. Small , Paul R. Rosenbaum

We construct an asymptotic prediction interval for the population-wise error rate (PWER), which is a multiple type I error criterion for clinical trials with overlapping patient populations. The PWER is the probability that a randomly…

Methodology · Statistics 2026-02-09 Remi Luschei , Werner Brannath