– Global Comparison and Future Perspectives in the Post-PRV Era –
With the expiration of the U.S. FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program in 2024, a global debate has emerged about how to sustain innovation in rare pediatric disease treatment. In Volume 1, we explored the U.S. policy shift. In this second part, we turn our attention to Japan—analyzing its systems, comparing them internationally, and offering strategic insights for the future.
🏛 Japan’s Systems and Support Framework
Japan has implemented a rare disease drug designation system since 1993, targeting diseases affecting fewer than 50,000 people. Incentives include tax benefits, grants, and expedited review timelines.
In addition, the PMDA offers a dedicated Pediatric Drug Consultation Desk, and AMED (Japan Agency for Medical Research and Development) funds numerous programs supporting infrastructure and translational research.
🇯🇵🇺🇸 Comparing Japan and the U.S.
- Scale of Incentives: While PRVs in the U.S. could be sold for hundreds of millions of dollars, Japan’s model remains grant- and tax-based, making capital acquisition harder for startups.
- Review Speed: U.S. priority review can shorten approval to six months. In Japan, new modalities often face longer timelines due to stricter evaluation.
- Clinical Trial Infrastructure: Japan struggles with limited patient pools and coordination, while global trials are common in the U.S.
🔬 Positive Momentum in Japan
Despite these challenges, progress is visible. Leading institutions like Kyoto University, Keio University, and the National Center for Child Health and Development are advancing bench-to-bedside research. Biotech firms like JCR Pharma and Healios are also expanding in this space through international collaborations.
🧩 Recommendations for Japan
- Strengthen incentive mechanisms, such as a Japan-style PRV
- Improve global trial infrastructure and streamline regulatory processes
- Enhance public awareness and foster collaboration with patient groups
📝 Conclusion
Developing therapies for pediatric rare diseases is not just about policy—it’s a moral imperative. The end of the U.S. PRV program challenges all nations to rethink how to support next-generation innovation.
For Japan, aligning globally through robust systems, proactive funding, and cross-sector collaboration is key to long-term success in this vital therapeutic domain.
