On January 13, various members of the Japanese Health Ministry met and discussed the need to improve orphan drug designation and approval. Increased funding for orphan drug development was debated. Orphan drug designation (ODD) can be achieved in the West with Phase 1 or Phase 2 data. On the other hand, Japan normally requires Phase 3 data. Other topics reviewed included how to alert foreign drug companies with orphan drugs about Japanese pricing earlier, so there is more certainty in the process. In addition, the need for Japan to accept English-language dossier submittals would help expedite approvals.
Japan is slowly realizing its regulatory and pricing system is limiting access to orphan drugs for rare disease patients. Also, there is a lack of Japanese drug companies developing orphan drugs due to a lack of funding, a lack of drug R&D talent, and Japan’s closed ecosystem.