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There is currently a focus on statistical methods which can use historical trial information to help accelerate the discovery, development and delivery of medicine. Bayesian methods can be constructed so that the borrowing is "dynamic" in…

统计方法学 · 统计学 2024-09-13 Darren A. V. Scott , Alex Lewin

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

统计方法学 · 统计学 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

To investigate intervention effects on rare events, meta-analysis techniques are commonly applied in order to assess the accumulated evidence. When it comes to adverse effects in clinical trials, these are often most adequately handled…

统计方法学 · 统计学 2026-04-03 Christian Röver , Qiong Wu , Anja Loos , Tim Friede

The use of historical controls offers a valuable alternative when traditional randomized controlled trials are not feasible. However, such approaches may introduce bias due to temporal changes in patient populations, diagnostic criteria,…

统计方法学 · 统计学 2025-12-24 Marco Ratta , Pavel Mozgunov , Sandrine Boulet , Moreno Ursino

Incorporating historical information into the design and analysis of a new clinical trial has been the subject of much recent discussion. For example, in the context of clinical trials of antibiotics for drug resistant infections, where…

统计方法学 · 统计学 2018-06-08 Isaac Gravestock , Leonhard Held

External information, such as prior information or expert opinions, can play an important role in the design, analysis and interpretation of clinical trials. However, little attention has been devoted thus far to incorporating external…

应用统计 · 统计学 2013-04-24 Minge Xie , Regina Y. Liu , C. V. Damaraju , William H. Olson

In pre- and non-clinical toxicology, the reduction of animal use is highly desireable. Although approaches for possible sample size reduction in the concurrent control group were suggested previously under the virtual control groups…

统计方法学 · 统计学 2026-03-13 Max Menssen , Carsten Kneuer , Gyamfi Akyianu , Christian Röver , Tim Friede , Frank Schaarschmidt

Historical data about disease outcomes can be integrated into the analysis of clinical trials in many ways. We build on existing literature that uses prognostic scores from a predictive model to increase the efficiency of treatment effect…

统计方法学 · 统计学 2020-12-25 David Walsh , Alejandro Schuler , Diana Hall , Jon Walsh , Charles Fisher

Given the cost and duration of phase III and phase IV clinical trials, the development of statistical methods for go/no-go decisions is vital. In this paper, we introduce a Bayesian methodology to compute the probability of success based on…

统计方法学 · 统计学 2020-10-27 Ethan M. Alt , Matthew A. Psioda , Joseph G. Ibrahim

Historical data from previous clinical trials, observational studies and health records may be utilized in analysis of clinical trials data to strengthen inference. Under the Bayesian framework incorporation of information obtained from any…

应用统计 · 统计学 2021-03-23 Shirin Golchi

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

统计方法学 · 统计学 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Meta-analysis is widely used to integrate results from multiple experiments to obtain generalized insights. Since meta-analysis datasets are often heteroscedastic due to varying subgroups and temporal heterogeneity arising from experiments…

统计方法学 · 统计学 2026-01-19 Kohsuke Kubota , Shonosuke Sugasawa , Keiichi Ochiai , Takahiro Hoshino

Time to an event of interest over a lifetime is a central measure of the clinical benefit of an intervention used in a health technology assessment (HTA). Within the same trial, multiple end-points may also be considered. For example,…

应用统计 · 统计学 2026-01-13 Nathan Green , Murat Kurt , Andriy Moshyk , James Larkin , Gianluca Baio

We propose a Bayesian Sequential procedure to test hypotheses concerning the Relative Risk between two specific treatments based on the binary data obtained from the two-arm clinical trial. Our development is based on the optimal sequential…

统计方法学 · 统计学 2025-04-07 Jiayue Wang , Ben Boukai

While randomized controlled trials (RCTs) are the gold standard for estimating treatment effects in medical research, there is increasing use of and interest in using real-world data for drug development. One such use case is the…

统计方法学 · 统计学 2021-10-11 Devin Incerti , Michael T Bretscher , Ray Lin , Chris Harbron

Analysis of lifetime data from epidemiological studies or destructive testing often involves current status censoring, wherein individuals are examined only once and their event status is recorded only at that specific time point. In…

统计方法学 · 统计学 2024-10-15 Pavithra Hariharan , P. G. Sankaran

The Cox model is an indispensable tool for time-to-event analysis, particularly in biomedical research. However, medicine is undergoing a profound transformation, generating data at an unprecedented scale, which opens new frontiers to study…

统计方法学 · 统计学 2023-03-07 Alexander W. Jung , Moritz Gerstung

External control arms can inform early clinical development of experimental drugs and provide efficacy evidence for regulatory approval. However, accessing sufficient real-world or historical clinical trials data is challenging. Indeed,…

Surrogate endpoints play an important role in drug development when they can be used to measure treatment effect early compared to the final clinical outcome and to predict clinical benefit or harm. Such endpoints are assessed for their…

Bayesian statistics plays a pivotal role in advancing medical science by enabling healthcare companies, regulators, and stakeholders to assess the safety and efficacy of new treatments, interventions, and medical procedures. The Bayesian…

应用统计 · 统计学 2023-12-04 Se Yoon Lee
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