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Existing multi-outcome designs focus almost entirely on evaluating whether all outcomes show evidence of efficacy or whether at least one outcome shows evidence of efficacy. While a small number of authors have provided multi-outcome…

Methodology · Statistics 2020-12-21 Martin Law , Michael J. Grayling , Adrian P. Mander

Two-point time-series data, characterized by baseline and follow-up observations, are frequently encountered in health research. We study a novel two-point time series structure without a control group, which is driven by an observational…

Methodology · Statistics 2023-01-19 Xiaowu Dai , Saad Mouti , Marjorie Lima do Vale , Sumantra Ray , Jeffrey Bohn , Lisa Goldberg

The increasing prevalence of rich sources of data and the availability of electronic medical record databases and electronic registries opens tremendous opportunities for enhancing medical research. For example, controlled trials are…

Methodology · Statistics 2015-09-23 Liwen Ouyang , Daniel W. Apley , Sanjay Mehrotra

Clinical trials often collect data on multiple outcomes, such as overall survival (OS), progression-free survival (PFS), and response to treatment (RT). In most cases, however, study designs only use primary outcome data for interim and…

Applications · Statistics 2026-04-28 Massimiliano Russo , Steffen Ventz , Lorenzo Trippa

Because of the high cost of commercial genotyping chip technologies, many investigations have used a two-stage design for genome-wide association studies, using part of the sample for an initial discovery of ``promising'' SNPs at a less…

Parameter inference in ordinary differential equations is an important problem in many applied sciences and in engineering, especially in a data-scarce setting. In this work, we introduce a novel generative modeling approach based on…

Machine Learning · Computer Science 2019-12-06 Philippe Wenk , Gabriele Abbati , Michael A Osborne , Bernhard Schölkopf , Andreas Krause , Stefan Bauer

Phase 1-2 designs provide a methodological advance over phase 1 designs for dose finding by using both clinical response and toxicity. A phase 1-2 trial still may fail to select a truly optimal dose. because early response is not a perfect…

Applications · Statistics 2024-04-03 Cheng-Han Yang , Peter F. Thall , Ruitao Lin

An informative sampling design leads to unit inclusion probabilities that are correlated with the response variable of interest. However, multistage sampling designs may also induce higher order dependencies, which are typically ignored in…

Methodology · Statistics 2019-01-23 Matthew R. Williams , Terrance D. Savitsky

We propose a two-stage design for a clinical trial with an early stopping rule for safety. We use different criteria to assess early stopping and efficacy. The early stopping rule is based on a criteria that can be determined more quickly…

Methodology · Statistics 2013-07-25 Daniel Zelterman

Deep models are designed to operate on huge volumes of high dimensional data such as images. In order to reduce the volume of data these models must process, we propose a set-based two-stage end-to-end neural subsampling model that is…

Machine Learning · Computer Science 2022-05-31 Bruno Andreis , Seanie Lee , A. Tuan Nguyen , Juho Lee , Eunho Yang , Sung Ju Hwang

Optimizing doses for multiple indications is challenging. The pooled approach of finding a single optimal biological dose (OBD) for all indications ignores that dose-response or dose-toxicity curves may differ between indications, resulting…

Methodology · Statistics 2024-10-07 Shuqi Wang , Peter F. Thall , Kentaro Takeda , Ying Yuan

Background. Designing trials to reduce treatment duration is important in several therapeutic areas, including TB and antibiotics. We recently proposed a new randomised trial design to overcome some of the limitations of standard two-arm…

Accurate estimation of parameters is paramount in developing high-fidelity models for complex dynamical systems. Model-based optimal experiment design (OED) approaches enable systematic design of dynamic experiments to generate input-output…

Systems and Control · Computer Science 2014-11-12 Ali Mesbah , Stefan Streif

In this paper, we address the challenging problem of optimal experimental design (OED) of constrained inverse problems. We consider two OED formulations that allow reducing the experimental costs by minimizing the number of measurements.…

Numerical Analysis · Mathematics 2017-08-17 Lars Ruthotto , Julianne Chung , Matthias Chung

Bipartite experiments arise in various fields, in which the treatments are randomized over one set of units, while the outcomes are measured over another separate set of units. However, existing methods often rely on strong model…

Methodology · Statistics 2025-04-16 Sizhu Lu , Lei Shi , Yue Fang , Wenxin Zhang , Peng Ding

Digital monitoring studies collect real-time high frequency data via mobile sensors in the subjects' natural environment. This data can be used to model the impact of changes in physiology on recurrent event outcomes such as smoking, drug…

Methodology · Statistics 2022-04-15 Walter Dempsey

Questions of `how best to acquire data' are essential to modeling and prediction in the natural and social sciences, engineering applications, and beyond. Optimal experimental design (OED) formalizes these questions and creates…

Methodology · Statistics 2026-05-01 Xun Huan , Jayanth Jagalur , Youssef Marzouk

Gradient-free prompt optimization methods have made significant strides in enhancing the performance of closed-source Large Language Models (LLMs) across a wide range of tasks. However, existing approaches make light of the importance of…

Computation and Language · Computer Science 2024-10-03 Muchen Yang , Moxin Li , Yongle Li , Zijun Chen , Chongming Gao , Junqi Zhang , Yangyang Li , Fuli Feng

We consider a dose-optimization design for first-in-human oncology trial that aims to identify a suitable dose for late-phase drug development. The proposed approach, called the Pharmacometrics-Enabled DOse OPtimization (PEDOOP) design,…

Applications · Statistics 2024-06-19 Shijie Yuan , Zhanbo Huang , Jiaxin Liu , Yuan Ji

Modern data collecting methods and computation tools have made it possible to monitor high-dimensional processes. In this article, Phase II monitoring of high-dimensional processes is investigated when the available number of samples…

Methodology · Statistics 2023-01-24 Mohsen Ebadi , Shojaeddin Chenouri , Stefan H. Steiner