Experience at the Pharmaceuticals and Medical Devices Agency, knowing the regulation and being involved in the development of treatments for rare diseases

Abstract

After initial training in neurology at various hospitals, my focus on treating intractable neurological diseases led me to join the Pharmaceuticals and Medical Devices Agency (PMDA). My experience at PMDA was transformative, deepening my understanding of drug development processes and global regulatory standards. This experience was pivotal, particularly in the context of rare and intractable diseases, leading to significant contributions in the field through involvement in the Orphan Drug Working Group and collaboration with international regulatory bodies like FDA and EMA. Returning to academia, I applied my insights to establish a disease registration system crucial for advancing treatments for rare diseases and further broadened my research to include regulatory science, clinical trials, and clinical pharmacology. My career now integrates clinical expertise with regulatory knowledge to enhance therapeutic developments for intractable and rare diseases.