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Adaptive experiment designs can dramatically improve statistical efficiency in randomized trials, but they also complicate statistical inference. For example, it is now well known that the sample mean is biased in adaptive trials.…

Machine Learning · Statistics 2021-02-16 Vitor Hadad , David A. Hirshberg , Ruohan Zhan , Stefan Wager , Susan Athey

The problem of optimal allocation of samples in surveys using a stratified sampling plan was first discussed by Neyman in 1934. Since then, many researchers have studied the problem of the sample allocation in multivariate surveys and…

Discrete Mathematics · Computer Science 2013-09-25 Jose Andre de Moura Brito , Gustavo Silva Semaan , Pedro Luis do Nascimento Silva , Nelson Maculan

Most recent machine learning research focuses on developing new classifiers for the sake of improving classification accuracy. With many well-performing state-of-the-art classifiers available, there is a growing need for understanding…

Machine Learning · Computer Science 2020-09-30 Jaehoon Koo , Diego Klabjan , Jean Utke

We introduce a new multiple type I error criterion for clinical trials with multiple populations. Such trials are of interest in precision medicine where the goal is to develop treatments that are targeted to specific sub-populations…

Methodology · Statistics 2021-02-05 Werner Brannath , Charlie Hillner , Kornelius Rohmeyer

Incorporating external data can improve the efficiency of clinical trials, but distributional mismatches between current and external populations threaten the validity of inference. While numerous dynamic borrowing methods exist, the…

Methodology · Statistics 2026-02-26 Yui Kimura , Shu Tamano

External information borrowing is often considered in order to improve a clinical trial's efficiency. The Bayesian approach borrows such external information by specifying an informative prior distribution. A potential issue with this…

Methodology · Statistics 2025-08-25 Silvia Calderazzo , Manuel Wiesenfarth , Vivienn Weru , Annette Kopp-Schneider

In clinical prediction modeling, model updating refers to the practice of modifying a prediction model before it is used in a new setting. In the context of logistic regression for a binary outcome, one of the simplest updating methods is a…

Applications · Statistics 2022-04-12 Mohsen Sadatsafavi , Hamid Tavakoli , Abdollah Safari

We want to recover the regression function in the single-index model. Using an aggregation algorithm with local polynomial estimators, we answer in particular to the second part of Question~2 from Stone (1982) on the optimal convergence…

Statistics Theory · Mathematics 2007-12-04 Stéphane Gaïffas , Guillaume Lecué

Hybrid controlled trials (HCTs), which augment randomized controlled trials (RCTs) with external controls (ECs), are increasingly receiving attention as a way to address limited power, slow accrual, and ethical concerns in clinical…

Methodology · Statistics 2025-05-02 Jiajun Liu , Ke Zhu , Shu Yang , Xiaofei Wang

We present a new experimental design procedure that divides a set of experimental units into two groups in order to minimize error in estimating an additive treatment effect. One concern is minimizing error at the experimental design stage…

Methodology · Statistics 2021-02-02 Abba M. Krieger , David Azriel , Adam Kapelner

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

Statistics Theory · Mathematics 2011-05-25 Jay Bartroff , Tze Leung Lai

This paper investigates the size performance of Wald tests for CAViaR models (Engle and Manganelli, 2004). We find that the usual estimation strategy on test statistics yields inaccuracies. Indeed, we show that existing density estimation…

Econometrics · Economics 2021-02-03 Alain Hecq , Li Sun

A/B testing is critical for modern technological companies to evaluate the effectiveness of newly developed products against standard baselines. This paper studies optimal designs that aim to maximize the amount of information obtained from…

Methodology · Statistics 2023-11-07 Ting Li , Chengchun Shi , Jianing Wang , Fan Zhou , Hongtu Zhu

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

Methodology · Statistics 2026-03-09 Samuel I. Watson , James Martin

In most clinical trials, patients are randomized with equal probability among treatments to obtain an unbiased estimate of the treatment effect. Response-adaptive randomization (RAR) has been proposed for ethical reasons, where the…

Applications · Statistics 2019-09-16 Thevaa Chandereng , Rick Chappell

Optimal two-treatment, $p$ period crossover designs for binary responses are determined. The optimal designs are obtained by minimizing the variance of the treatment contrast estimator over all possible allocations of $n$ subjects to $2^p$…

Methodology · Statistics 2015-05-12 S. Mukhopadhyay , S. P. Singh , A. Dey

We present a novel stochastic approach to binary optimization for optimal experimental design (OED) for Bayesian inverse problems governed by mathematical models such as partial differential equations. The OED utility function, namely, the…

Optimization and Control · Mathematics 2022-06-28 Ahmed Attia , Sven Leyffer , Todd Munson

Several techniques exist to assess and reduce nonresponse bias, including propensity models, calibration methods, or post-stratification. These approaches can only be applied after the data collection, and assume reliable information…

Methodology · Statistics 2020-05-26 Blanka Szeitl , Tamás Rudas

In this paper, we investigate the impact of high-dimensional Principal Component (PC) adjustments on inferring the effects of variables on outcomes, with a focus on applications in genetic association studies where PC adjustment is commonly…

Statistics Theory · Mathematics 2025-06-30 Sohom Bhattacharya , Rounak Dey , Rajarshi Mukherjee

It has long been noticed that the efficacy observed in small early phase studies is generally better than that observed in later larger studies. Historically, the inflation of the efficacy results from early proof-of-concept studies is…

Methodology · Statistics 2020-06-11 Yongming Qu , Yu Du , Ying Zhang , Lei Shen
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