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We consider event-driven clinical trials, where the analysis is performed once a pre-determined number of clinical events has been reached. For example, these events could be progression in oncology or a stroke in cardiovascular trials. At…

统计方法学 · 统计学 2021-08-23 Vladimir Anisimov , Stephen Gormley , Rosalind Baverstock , Cynthia Kineza

Clinical trials in the modern era are characterized by their complexity and high costs and usually involve hundreds/thousands of patients to be recruited across multiple clinical centres in many countries, as typically a rather large sample…

统计方法学 · 统计学 2024-11-27 Volodymyr Anisimov , Lucas Oliver

Forecasting recruitments is a key component of the monitoring phase of multicenter studies. One of the most popular techniques in this field is the Poisson-Gamma recruitment model, a Bayesian technique built on a doubly stochastic Poisson…

统计方法学 · 统计学 2023-01-11 Armando Turchetta , Nicolas Savy , David A. Stephens , Erica E. M. Moodie , Marina B. Klein

Design and forecasting of patient enrollment is among the greatest challenges that the clinical research enterprize faces today, as inefficient enrollment can be a major cause of drug development delays. Therefore, the development of the…

统计方法学 · 统计学 2022-12-27 Vladimir Anisimov , Matthew Austin

Accurate forecast of a clinical trial enrollment timeline at the planning phase is of great importance to both corporate strategic planning and trial operational excellence. While predictions of key milestones such as last subject first…

应用统计 · 统计学 2023-01-05 Sheng Zhong , Yunzhao Xing , Mengjia Yu , Li Wang

Given progressive developments and demands on clinical trials, accurate enrollment timeline forecasting is increasingly crucial for both strategic decision-making and trial execution excellence. Naive approach assumes flat rates on…

应用统计 · 统计学 2023-03-15 Mengjia Yu , Sheng Zhong , Yunzhao Xing , Li Wang

We analyse predictions of future recruitment to a multi-centre clinical trial based on a maximum-likelihood fitting of a commonly used hierarchical Poisson-Gamma model for recruitments at individual centres. We consider the asymptotic…

统计方法学 · 统计学 2021-02-15 Rachael Mountain , Chris Sherlock

We introduce a general framework for monitoring, modelling, and predicting the recruitment to multi-centre clinical trials. The work is motivated by overly optimistic and narrow prediction intervals produced by existing time-homogeneous…

统计方法学 · 统计学 2020-09-03 Szymon Urbas , Chris Sherlock , Paul Metcalfe

Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate…

机器学习 · 计算机科学 2025-11-03 Tien Huu Do , Antoine Masquelier , Nae Eoun Lee , Jonathan Crowther

Significant advancements have been made in recent years to optimize patient recruitment for clinical trials, however, improved methods for patient recruitment prediction are needed to support trial site selection and to estimate appropriate…

机器学习 · 计算机科学 2021-11-16 Jingshu Liu , Patricia J Allen , Luke Benz , Daniel Blickstein , Evon Okidi , Xiao Shi

Estimands using the treatment policy strategy for addressing intercurrent events are common in Phase III clinical trials. One estimation approach for this strategy is retrieved dropout whereby observed data following an intercurrent event…

Epidemic models are used to analyze the progression or outcome of an epidemic under different control policies like vaccinations, quarantines, lockdowns, use of face-masks, pharmaceutical interventions, etc. When these models accurately…

定量方法 · 定量生物学 2022-04-19 Carlos Hernandez-Suarez , Osval Montsinos Lopez , Ramon Solano-Barajas

Predictive multiplicity refers to the phenomenon in which classification tasks may admit multiple competing models that achieve almost-equally-optimal performance, yet generate conflicting outputs for individual samples. This presents…

机器学习 · 计算机科学 2024-02-02 Hsiang Hsu , Guihong Li , Shaohan Hu , Chun-Fu , Chen

Accurate trend forecasting in healthcare time series is essential for planning and resource allocation. This paper proposes a Bayesian framework for predicting oncology demand trends, modeling weekly appointments as a Poisson process with a…

This paper considers the Cram\'er-Lundberg model, with the additional feature that the number of clients can fluctuate over time. Clients arrive according to a Poisson process, where the times they spend in the system form a sequence of…

概率论 · 数学 2023-05-25 Peter Braunsteins , Michel Mandjes

The field of learning analytics needs to adopt a more rigorous approach for predictive model evaluation that matches the complex practice of model-building. In this work, we present a procedure to statistically test hypotheses about model…

应用统计 · 统计学 2018-02-19 Josh Gardner , Christopher Brooks

Clinical prediction models are statistical or machine learning models used to quantify the risk of a certain health outcome using patient data. These can then inform potential interventions on patients, causing an effect called performative…

机器学习 · 计算机科学 2024-06-06 Louis Chislett , Louis JM Aslett , Alisha R Davies , Catalina A Vallejos , James Liley

The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires…

统计方法学 · 统计学 2023-08-28 Suzie Cro , James H Roger , James R Carpenter

Rheumatoid arthritis clinical trials are strategically designed to collect the disease activity score of each patient over multiple clinical visits, meanwhile a patient may drop out before their intended completion due to various reasons.…

应用统计 · 统计学 2018-01-29 Violeta G. Hennessey , Luis G. Leon-Novelo , Juan Li , Li Zhu , Eric Chi , Joseph G. Ibrahim

Estimating the size of an elusive target population is of prominent interest in many areas in the life and social sciences. Our aim is to provide an efficient and workable method to estimate the unknown population size, given the frequency…

应用统计 · 统计学 2011-07-28 Irene Rocchetti , John Bunge , Dankmar Böhning
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