Drug Evaluation Committee How to Use Simulation in Bayesian Trial Design
Data Science Subcommittee
October 2025
In 2020, the FDA issued "Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products" to discuss the use of simulation in special clinical trial designs such as adaptive design, external controls, and master protocols. The Bayesian approach is being discussed for use in special clinical trial designs such as adaptive design, external controls, and master protocols. In Japan, the "Basic Guidelines for the Application of Bayesian Approach in Clinical Trials for Rare Diseases" was compiled by the AMED Research Project for Regulatory Harmonization and Evaluation of Pharmaceuticals, and the "Notes on the Use of Master Protocol Studies in Drug Development," issued in June 2024, also refers to Bayesian analysis methods. The concept of the Bayesian analysis method is being developed.
While Bayesian analysis may allow for flexibility in testing through decision-making based on mid-study results and efficient drug development by borrowing information from outside sources, it may also increase the possibility of erroneous conclusions (erroneous conclusions) depending on the prior distribution and decision-making criteria. While borrowing external information may allow for efficient drug development, its performance must be carefully evaluated before the study is conducted, as it may increase the likelihood of erroneous conclusions (erroneous conclusion) depending on the prior distribution and decision criteria. Therefore, simulation plays an important role in evaluating the performance of Bayesian study designs.
This deliverable discusses simulation methods to measure the operating characteristics of Bayesian flow test designs for statistical analysts in clinical trials, with the aim of enabling them to plan appropriate Bayesian flow tests under the required circumstances.
Japan Pharmaceutical Manufacturers Association, Committee on Drug Evaluation
Data Science Subcommittee 2024 Task Force 3-2
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