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Use of historical control data to augment a small internal control arm in a randomized control trial (RCT) can lead to significant improvement of the efficiency of the trial. It introduces the risk of potential bias, since the historical…

Methodology · Statistics 2022-10-05 Jixian Wang , Hongtao Zhang , Ram Tiwari

Bayesian dynamic borrowing methods incorporate historical control data into current clinical trial analyses while allowing the degree of borrowing to depend on the compatibility between historical and current data. Although many methods…

Methodology · Statistics 2026-05-27 Tomohiro Ohigashi , Wataru Murasaki , Masahiko Gosho

Leveraging preclinical animal data for a phase I first-in-man trial is appealing yet challenging. A prior based on animal data may place large probability mass on values of the dose-toxicity model parameter(s), which appear infeasible in…

Applications · Statistics 2020-04-15 Haiyan Zheng , Lisa V. Hampson

In current clinical trial development, historical information is receiving more attention as it provides utility beyond sample size calculation. Meta-analytic-predictive (MAP) priors and robust MAP priors have been proposed for…

Methodology · Statistics 2022-08-03 Tianyu Zhan , Yiwang Zhou , Ziqian Geng , Yihua Gu , Jian Kang , Li Wang , Xiaohong Huang , Elizabeth H. Slate

When incorporating historical control data into the analysis of current randomized controlled trial data, it is critical to account for differences between the datasets. When the cause of the difference is an unmeasured factor and…

Methodology · Statistics 2025-09-10 Tomohiro Ohigashi , Kazushi Maruo , Takashi Sozu , Masahiko Gosho

Incorporating preclinical animal data, which can be regarded as a special kind of historical data, into phase I clinical trials can improve decision making when very little about human toxicity is known. In this paper, we develop a robust…

Methodology · Statistics 2021-01-29 Haiyan Zheng , Lisa V. Hampson , Thomas Jaki

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

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,…

Methodology · Statistics 2025-12-24 Marco Ratta , Pavel Mozgunov , Sandrine Boulet , Moreno Ursino

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…

Methodology · Statistics 2024-09-13 Darren A. V. Scott , Alex Lewin

In the Bayesian framework power prior distributions are increasingly adopted in clinical trials and similar studies to incorporate external and past information, typically to inform the parameter associated to a treatment effect. Their use…

Methodology · Statistics 2025-08-12 Roberto Macrì Demartino , Leonardo Egidi , Nicola Torelli , Ioannis Ntzoufras

The recent 21st Century Cures Act propagates innovations to accelerate the discovery, development, and delivery of 21st century cures. It includes the broader application of Bayesian statistics and the use of evidence from clinical…

Applications · Statistics 2020-02-10 Satrajit Roychoudhury , Beat Neuenschwander

In pharmaceutical and toxicological research, historical control data are increasingly used to validate concurrent control groups, typically via the construction of historical control limits. While methods have been described for continuous…

Methodology · Statistics 2026-03-24 Sören Budig , Frank Schaarschmidt , Max Menssen

Information borrowing from historical data is gaining attention in clinical trials of rare and pediatric diseases, where statistical power may be insufficient for confirmation of efficacy if the sample size is small. Although Bayesian…

Methodology · Statistics 2023-05-25 Masahiro Kojima

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…

Methodology · Statistics 2020-12-25 David Walsh , Alejandro Schuler , Diana Hall , Jon Walsh , Charles Fisher

We propose a novel Bayesian model selection technique on linear mixed-effects models to compare multiple treatments with a control. A fully Bayesian approach is implemented to estimate the marginal inclusion probabilities that provide a…

Applications · Statistics 2015-09-28 Lei Gong , James M. Flegal , Stephen R. Spindler , Patricia L. Mote

Extrapolating treatment effects from related studies is a promising strategy for designing and analyzing clinical trials in situations where achieving an adequate sample size is challenging. Bayesian methods are well-suited for this…

Methodology · Statistics 2025-11-25 Tristan Fauvel , Julien Tanniou , Pascal Godbillot , Marie Génin , Billy Amzal

Toxicity monitoring is essential in Phase II clinical trials to ensure participant safety. While monitoring rules are well-established for single-arm trials, two-cohort trials present unique challenges because toxicities are expected to be…

Methodology · Statistics 2026-03-31 Yu Wang , Aniko Szabo

To answer the call of introducing more Bayesian techniques to organizational research (e.g., Kruschke, Aguinis, & Joo, 2012; Zyphur & Oswald, 2013), we propose a Bayesian approach for meta-analysis with power prior in this article. The…

Methodology · Statistics 2014-07-30 Zhiyong Zhang , Kaifeng Jiang , Haiyan Liu , In-Sue Oh

Closed-loop performance of sequential decision making algorithms, such as model predictive control, depends strongly on the choice of controller parameters. Bayesian optimization allows learning of parameters from closed-loop experiments,…

Systems and Control · Electrical Eng. & Systems 2025-11-18 Sebastian Hirt , Lukas Theiner , Rolf Findeisen

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…

Methodology · Statistics 2018-06-08 Isaac Gravestock , Leonhard Held
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