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March 8, 2024
Approximately 5 minutes
Q&A on Planning Pediatric Drug Development During Adult Drug Development in Japan
1. Publication Details
Updated March 29, 2024 (provisional translation as of May 2024). Issued by the Pharmaceutical Safety Bureau, Pharmaceutical Evaluation Division, Ministry of Health, Labour and Welfare. https://www.pmda.go.jp/files/000268524.pdf
2. Background
This Q&A provides clarification on the Director’s Notification (PSB/PED Notification No. 0112-3, January 12, 2024) regarding the planning of pediatric drug development programs during adult drug development. It addresses inquiries and aims to disseminate information through Prefectural Health Departments. The notification encourages but does not mandate pediatric planning to avoid 'drug loss' in Japan compared to the EU and U.S. https://www.pmda.go.jp/files/000268524.pdf
3. Purpose
To outline procedures, eligibility, and processes for preparing and confirming pediatric drug development plans, ensuring timely development. It covers consultations for confirmation, post-confirmation obligations, and handling of changes or non-trial based plans. https://www.pmda.go.jp/files/000268524.pdf
4. Target Products
Applies to drugs developed for adults where pediatric indications may differ, such as molecular target drugs for cancer expected to respond based on mechanism of action. Includes drugs evaluable with adults (aged 10 or 12 and older) and those requiring separate pediatric dosage forms or indications. https://www.pmda.go.jp/files/000268524.pdf
5. Eligibility Types
Preparation of a pediatric plan is desirable but not mandatory. Eligibility includes drugs with differing adult/pediatric indications, development for recognized age groups (including newborns), stepwise expansion, and plans using clinical trials, real-world data, or modeling & simulation without new trials if appropriate for approval. Also covers pediatric drugs evaluable with adults per the 2020 administrative notice. https://www.pmda.go.jp/files/000268524.pdf
6. Consultation Pathway Before Approval
For pre-approval adult applications, use PMDA’s 'Consultation on Confirmation of the Pediatric Drug Development Program' for plan confirmation. For differing indications, use conventional face-to-face consultations (e.g., post-phase II). Post-approval, include the plan in CTD Module 1.5. For non-trial plans or significant changes, use conventional consultations. https://www.pmda.go.jp/files/000268524.pdf
7. Approval Application and Review Expectations
Plans are confirmed via designated or conventional consultations. For adult approvals, include in CTD Module 1.5 if post-filing. Upon pediatric approval, review reports note confirmation. Significant changes require re-confirmation. Records maintained in PMDA consultation records and review reports. https://www.pmda.go.jp/files/000268524.pdf
8. Procedure After Approval (Post-Approval Obligations)
Proceed without delay based on confirmed plan. Timelines: Without pediatric dosage form, submit protocol/start trial within 2 years; file application within 2 years for non-trial plans. With dosage form, within 3 years. Confirmation date is consultation record-fixing or approval date. Describe in approval documents and review reports. https://www.pmda.go.jp/files/000268524.pdf
9. Practical Considerations
Age groups: Plan for needs, stepwise acceptable. Dosage: Confirm same/adjusted. Non-trial: Valid if using data/simulation. Records: Noted in documents with specific phrasing. Flexibility: No fixed periods; focus on prompt start; re-consult changes. https://www.pmda.go.jp/files/000268524.pdf
10. Effective Date
Based on January 12, 2024 notification; Q&A dated March 29, 2024. https://www.pmda.go.jp/files/000268524.pdf
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