PMDA Symposium 2025: Key Takeaways for Pediatric Drug Development
2025.09.17
Introduction
On August 27, 2025, the PMDA hosted a symposium titled “Toward Enhanced Pediatric Pharmacotherapy with Domestic and Global Stakeholders.” Discussions focused on overcoming drug lag and loss in pediatric medicines and aligning Japan’s system with international practices.
Key Updates in Japan
- Pediatric Drug Development Plan Consultation System (2024–): It allows companies to consult with regulatory authorities about pediatric development plans even during adult development, including linking them with pricing incentives.
- Pediatric & Rare Disease Consultation Center (2024): This center provides services on subsidized consultations, including pediatric plan confirmation, orphan designation, and data package support.
- PMD Act Amendment (May 2025): Applicants for new drug approvals are now obliged to make best efforts to establish pediatric development plans.
- PMDA’s New Approach:
- Acceptance of pediatric extrapolation, modeling & simulation (M&S), and real-world data (RWD).
- Facilitate Japanese participation in global pediatric trials without requiring prior Japanese adult data.
- Cross-functional pediatric expertise within PMDA to strengthen consultation and review.
European Regulatory Perspective (EMA)
- EU Paediatric Regulation (2007–): Over 260 pediatric medicines and 40 new formulations authorized by 2016.
- Incentives: SPC extension (6 months), orphan exclusivity (+2 years), PUMA (8+2 years).
- Expectations for PMDA: Strengthened participation in Pediatric Cluster teleconferences, greater PIP/PSP alignment, enhanced data sharing, and joint training programs to harmonize regulatory approaches globally.
Patient Advocacy Input
- Japanese children with cancer and rare diseases still face delays in access.
- Requests for greater transparency, earlier Japan involvement in global protocols, and formal patient/family engagement in development.
Conclusion for Regulatory Teams
The regulatory landscape in Japan is shifting toward earlier and more structured pediatric planning, with PMDA actively supporting alignment with EU/US practices. For companies:
- Engage early with PMDA consultations to leverage incentives.
- Align pediatric strategies with global PIP/PSP frameworks.
- Prepare to incorporate extrapolation, RWD, and innovative trial designs.
References:
PMDA 小児用医薬品シンポジウム「充実した小児薬物療法を目指し、国内外のStakeholderと共に歩む」の開催について | 独立行政法人 医薬品医療機器総合機構
