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Primarily motivated by the drug development process, several publications have now presented methodology for the design of multi-arm multi-stage experiments with normally distributed outcome variables of known variance. Here, we extend…

Methodology · Statistics 2017-12-04 Michael Grayling , James Wason , Adrian Mander

Crossover designs randomly assign each unit to receive a sequence of treatments. By comparing outcomes within the same unit, these designs can effectively eliminate between-unit variation and facilitate the identification of both…

Methodology · Statistics 2026-04-21 Zhichao Jiang , Peng Ding

The quality of assessment determines the quality of learning, and is characterized by validity, reliability and difficulty. Mastery of learning is generally represented by the difficulty levels of assessment items. A very large number of…

Computers and Society · Computer Science 2022-06-10 Shilpi Banerjee , N. J. Rao

Estimation frameworks for statistical inference are preferred to hypothesis testing when quantifying uncertainty and precise estimation are more valuable than binary decisions about statistical significance. Study design for…

Methodology · Statistics 2025-10-29 Luke Hagar , Nathaniel T. Stevens

Estimation problems in the presence of deterministic linear nuisance parameters arise in a variety of fields. To cope with those, three common methods are widely considered: (1) jointly estimating the parameters of interest and the nuisance…

Optimization and Control · Mathematics 2018-07-05 Yongchang Hu , Geert Leus

We revisit the finite-sample behavior of single-variable just-identified instrumental variables (just-ID IV) estimators, arguing that in most microeconometric applications, the usual inference strategies are likely reliable. Three…

Econometrics · Economics 2024-12-31 Joshua Angrist , Michal Kolesár

We introduce and give examples of applications of an optimality criterion which can be used for the design and comparison of multiple beam profile emittance and Twiss parameters measurement sections and which is independent from the…

Accelerator Physics · Physics 2013-05-08 V. Balandin , W. Decking , N. Golubeva

We propose a variance-penalized formulation of Bayesian optimal experimental design for nonlinear models that augments the classical expected utility criterion with a penalty on utility variability, yielding a mean--variance objective that…

Methodology · Statistics 2026-04-07 Wanggang Shen , Xun Huan

N-of-1 experiments, where a unit serves as its own control and treatment in different time windows, have been used in certain medical contexts for decades. However, due to effects that accumulate over long time windows and interventions…

Methodology · Statistics 2025-02-25 Tengyuan Liang , Benjamin Recht

Exploiting the information provided by the molecular noise of a biological process has proven to be valuable in extracting knowledge about the underlying kinetic parameters and sources of variability from single cell measurements. However,…

Quantitative Methods · Quantitative Biology 2013-08-30 Jakob Ruess , Andreas Milias-Argeitis , John Lygeros

Combination of several anti-cancer treatments has typically been presumed to have enhanced drug activity. Motivated by a real clinical trial, this paper considers phase I-II dose finding designs for dual-agent combinations, where one main…

Methodology · Statistics 2023-05-09 José L. Jiménez , Haiyan Zheng

The traditional more-is-better dose selection paradigm, developed based on cytotoxic chemotherapeutics, is often problematic When applied to the development of novel molecularly targeted agents (e.g., kinase inhibitors, monoclonal…

Methodology · Statistics 2022-11-04 Liyun Jiang , Ying Yuan

Inference for high-dimensional logistic regression models using penalized methods has been a challenging research problem. As an illustration, a major difficulty is the significant bias of the Lasso estimator, which limits its direct…

Methodology · Statistics 2024-10-29 Yuming Zhang , Stéphane Guerrier , Runze Li

Within an industrial manufacturing process, tolerancing is a key player. The dimensions uncertainties management starts during the design phase, with an assessment on variability of parts not yet produced. For one assembly step, we can gain…

Applications · Statistics 2019-12-20 Ambre Diet , Nicolas Couellan , Xavier Gendre , Julien Martin

Chronic disease progression models are governed by three main parameters: sensitivity, preclinical intensity, and sojourn time. The estimation of these parameters helps in optimizing screening programs and examine the improvement in…

Applications · Statistics 2020-01-22 Ayman Hijazy , András Zempléni

Using mathematical models to assist in the interpretation of experiments is becoming increasingly important in research across applied mathematics, and in particular in biology and ecology. In this context, accurate parameter estimation is…

Statistics Theory · Mathematics 2025-04-29 Jie Qi , Ruth E. Baker

One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose (MTD), followed by a randomization stage where patients are assigned to two or more doses to…

Methodology · Statistics 2024-11-11 Yixuan Zhao , Rachael Liu , Jianchang Lin , Ying Yuan

We present a simulation-based inference approach for two-stage estimators, focusing on extremum estimators in the second stage. We accommodate a broad range of first-stage estimators, including extremum estimators, high-dimensional…

Econometrics · Economics 2024-11-08 Aristide Houndetoungan , Abdoul Haki Maoude

Calibration of expensive simulation models involves an emulator based on simulation outputs generated across various parameter settings to replace the actual model. Noisy outputs of stochastic simulation models require many simulation…

Methodology · Statistics 2025-05-08 Özge Sürer

An important challenge in statistical analysis concerns the control of the finite sample bias of estimators. For example, the maximum likelihood estimator has a bias that can result in a significant inferential loss. This problem is…

Statistics Theory · Mathematics 2019-11-04 Stéphane Guerrier , Mucyo Karemera , Samuel Orso , Maria-Pia Victoria-Feser