Bayesian Adaptive Clinical Trials for Anti-Infective Therapeutics during Epidemic Outbreaks

Qingyang Xu; Shomesh Chaudhuri; Danying Xiao; Andrew W Lo

Of course, in practice regulators consider many factors beyond p-values in making its decisions. However, that process is opaque even to industry insiders, and the role of patient preferences is unclear. The proposed patient-centered approach provides a systematic, objective, adaptive, and repeatable framework for explicitly incorporating patient preferences and burden-of-disease data in the therapeutic approval process. This framework also fulfills two mandates for the FDA, one from the fifth authorization of the Prescription Drug User Fee Act (PDUFA) for an enhanced quantitative approach to the benefit-risk assessment of new drugs (U.S FDA, 2013), and the other from Section 3002 of the 21 st Century Cures Act of 2016 requiring the FDA to develop guidelines for patientfocused drug development, which includes collecting patient preference and experience data and explicitly incorporating this information in the drug approval process.