Introduction
PMDA has reduced median new drug review times from over 600 days in 2005 to 333 days in 2023, matching US FDA levels. However, “drug loss” remains a challenge—about 65% of NMEs approved in the US are not approved in Japan, often due to low participation in MRCTs (≈57%) and limited engagement from emerging biopharma (EBPs).
Pediatric and Orphan Drug Support
In July 2024, PMDA established the Consultation Center for Pediatric and Orphan Drug Development (CCPODD), offering MHLW-subsidized, fee-reduced consultations and regulatory guidance to address high unmet needs and streamline approvals in these areas.
“Early Consideration” and Guideline Updates
Since FY2024, PMDA publishes “Early Consideration” papers—timely position statements (JP/EN) similar to EU reflection papers—covering evolving scientific topics such as oncology Phase I trial design, biomarker-based DDI evaluation, and PAH drug development.
Facilitating MRCT Participation
New guidelines clarify the case for a waiver to skip Phase 1 study in Japanese for the start of MRCT, enabling smoother MRCT execution in Japan. For orphan drugs, Japanese data may be waived if robust overseas confirmatory trials exist. PMDA also works with designated sites to cut costs and improve trial efficiency.
RWD/RWE Utilization
PMDA encourages use of RWD/RWE, especially in rare diseases, and has issued guidance on planning, analysis, and interpretation for external control use in regulatory submissions.
Global Outreach
PMDA opened overseas offices in Bangkok (July 2024) and Washington DC (Nov 2024) to promote regulatory harmonization, support MRCTs, and guide EBPs entering Japan.
Conclusion
Through targeted support for orphan/pediatric drugs, flexible MRCT policies, proactive scientific guidance, and global engagement, PMDA aims to mitigate drug lag and make Japan a more integrated part of global drug development pipelines.
Source: Kohno Y, et al. Expediting Drug Development in Japan: A PMDA Perspective. Clin Pharmacol Ther. 2025. https://doi.org/10.1002/cpt.70022