Accelerating Regulatory Science Initiatives for the Development of Drugs for Alzheimer’s Disease in Japan

Abstract Purpose The Ministry of Health, Labour and Welfare (MHLW) of Japan launched a regulatory science research project in which the aim is to promote the establishment of guidelines for the development of innovative drugs thorough interactions between academia and Japan’s regulatory agency, the Pharmaceuticals and Medical Devices Agency (PMDA). In this project, a research system with the aim of developing a guideline for the clinical evaluation of drugs intended for the treatment of Alzheimer’s disease (AD) was established. Methods Two research groups were set up: (1) the Biomarker and Clinical Evaluation Group to establish biomarker-based criteria for the clinical evaluation of drugs for AD, and (2) the Modeling and Simulation (M&S) Group to create a disease model of AD using M&S techniques based on data from the Alzheimer’s Disease Neuroimaging Initiative (ADNI). Furthermore, a human resource exchange between the University of Tokyo Hospital and the PMDA is conducted to establish a guideline that is suitable for regulatory use. Findings As an interim report of this project, issues that require consideration for the clinical evaluation and development were summarized, including topics such as the use of biomarkers in the inclusion criteria, the efficacy endpoint, and the clinical data package required for application in Japan. Implications As the result of collaboration between the University of Tokyo Hospital and PMDA, this document is the first to summarize perspectives on the development of drugs for AD in Japan.