RWE Collaborative Comment Letter on Use of Bayesian Methodology in Clinical Trials

The Institute appreciates FDA’s timely release of its draft guidance for industry entitled, “Use of Bayesian Methodology in Clinical Trials of Drug and Biological Products.” We particularly value the inclusion of examples of when Bayesian approaches have been used, success criteria and details on reporting. We recommend that FDA include examples of where they envision Bayesian approaches being used in the future. Shortly after the release of FDA’s guidance, the National Medical Products Administration (NMPA), released a guidance document focused on the application of Bayesian external information borrowing methods in clinical trials and the European Medicines Agency published a concept paper on Bayesian methods, noting an anticipated release of a reflection paper on the topic in 2028. However, no other regulators have published dedicated guidance on this topic. Therefore, the present guidance is especially welcome given recent reports on the limited number of submissions to FDA that include Bayesian methods, and given overall limited regulatory agency guidance on ways to best apply Bayesian methods for trials subject to regulatory review. We believe this sets an important direction for sponsors and their collaborators to best and more meaningfully apply Bayesian analyses that may involve real-world data (RWD).