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March 8, 2026
Approximately 5 minutes
Partial Revision of Re-Examination Periods for Orphan Drugs 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/000268675.pdf
2. Background
The notification addresses the partial revision of the handling of re-examination periods for prescription drugs, specifically focusing on orphan drugs. It references prior notifications: “Re-Examination Period of Prescription Drugs” (PSEHB Notification No. 0831-11 dated August 31, 2020) and “Handling of Re-Examination Period” (PSEHB/PED Notification No. 0831-16 dated August 31, 2020). The revision stems from the issue of “Partial Revision of ‘Designation of Orphan Drugs’” (Joint PSB/PED Notification No. 0116-1 and PSB/MDED Notification No. 0116-1 dated January 16, 2024), based on reviews by the “Review Committee on Regulatory Affairs to Strengthen Drug Discovery/Ensure Stable Supply.” The revision to the re-examination period for orphan drugs is detailed in an attached old-and-new comparison table, with underlined parts indicating changes. The revised Notification by Director is attached for reference. https://www.pmda.go.jp/files/000268675.pdf
3. Purpose
The purpose is to revise the handling of re-examination periods for orphan drugs to align with updates in orphan drug designation, ensuring appropriate investigation and re-examination timelines under the revised Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices (Act No. 145 of 1960). https://www.pmda.go.jp/files/000268675.pdf
4. Target Products
The revisions target orphan drugs, defined as those meeting specific criteria such as first approval of the designated indication (limited to clearly different indications if same active ingredients as approved drugs) or addition of clearly different doses (e.g., pediatric doses). https://www.pmda.go.jp/files/000268675.pdf
5. Eligibility Types
Eligibility includes orphan drugs with first approval of designated indication or addition of clearly different doses, such as pediatric doses. Extensions apply for drugs requiring pharmacoepidemiological assessment on efficacy or pediatric dose-finding studies. https://www.pmda.go.jp/files/000268675.pdf
6. Consultation Pathways
Consultations for interim measures or extensions are directed to the Pharmaceutical Evaluation Division. For new drugs approved by March 2021 without pediatric dosage plans submitted by review end, consultation via the Pharmaceuticals and Medical Devices Agency consultation system is recommended, with individual consultations required. Post-marketing scenarios necessitate prompt consideration of clinical studies, with extensions examined based on submitted protocols. https://www.pmda.go.jp/files/000268675.pdf
7. Approval Application and Review Expectations
Re-examination must be conducted within 3 months after the investigation period specified in Article 14-4 (1) of the Act. For orphan drugs, the investigation period is 10 years for first approval of designated indication or addition of clearly different doses. Extensions to 10 years may apply for drugs requiring pharmacoepidemiological assessment on efficacy (e.g., life prolongation, QOL improvement) or pediatric dose-finding studies, based on risk management plans or development plans submitted by approval recipients. https://www.pmda.go.jp/files/000268675.pdf
8. Procedure After Approval (Post-marketing Obligations)
Post-approval, re-examination is required within 3 months post-investigation period. For drugs necessitating extended investigation (e.g., pharmacoepidemiological surveillance or pediatric studies), plans must be submitted without delay, and studies initiated promptly. Non-compliance with pediatric plans may revert to standard periods unless consulted. https://www.pmda.go.jp/files/000268675.pdf
9. Practical Considerations
The revision applies from January 16, 2024. Related parties under prefectural administration should be informed. For drugs approved by August 31, 2020, pre-revision provisions apply if pediatric studies are later deemed necessary. Revisions to prior notifications (e.g., PSEHB/PED Notification No. 0831-16) update references to protocols and divisions. https://www.pmda.go.jp/files/000268675.pdf
10. Effective Date
The revision applies from January 16, 2024. The referenced 2020 notification (PSEHB/PED Notification No. 0831-16) came into effect on September 1, 2020. https://www.pmda.go.jp/files/000268675.pdf
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