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March 10, 2026

Approximately 5 minutes

Q&A on Handling of In Vitro Companion Diagnostics and Corresponding Therapeutic Products in Japan

1. Publication Details

Issued July 1, 2013 as an administrative notice supplementing PFSB/ELD Notification No. 0701-10. https://www.pmda.go.jp/files/000153495.pdf

2. Background

This Q&A compiles clarifications on judgment criteria for classifying in vitro companion diagnostics (CoDx), approval application procedures, and coordination between therapeutic products and diagnostics to ensure safe and effective use in personalized medicine. It accompanies the main notification on approval applications for CoDx and corresponding therapeutic products. https://www.pmda.go.jp/files/000153495.pdf

3. Definition of CoDx

CoDx refers to an in vitro diagnostic agent or medical device essential for using the pertinent therapeutic product (excluding those intended simply for disease diagnosis). Classification is determined case-by-case based on trial notification, approval proposal, and PMDA evaluation of necessity for patient selection or dose adjustment. Diagnostics for routine biochemical, hematological, or infection tests are generally not CoDx unless clinical necessity dictates otherwise. https://www.pmda.go.jp/files/000153495.pdf

4. Target Products and Scope

Applies to therapeutic products requiring patient selection via unapproved CoDx and the corresponding CoDx itself. Covers new approval applications, partial change applications for new indications, and cases where an already-approved diagnostic is newly designated as CoDx. https://www.pmda.go.jp/files/000153495.pdf

5. Handling of Approval Applications

When patient selection requires an unapproved CoDx, approval applications for the CoDx and therapeutic product should be submitted contemporaneously. The CoDx application should be filed within one month after the therapeutic product application at the latest. Delayed submissions for unavoidable reasons are reviewed case-by-case after early consultation with PMDA. For partial changes adding new indications requiring CoDx, the same contemporaneous principle applies. https://www.pmda.go.jp/files/000153495.pdf

6. Handling of Clinical Trial Notifications

In the remarks column of the clinical trial notification, summarize the CoDx development status (e.g., approval status, developer name, cooperation progress). For imported CoDx used in trials, describe name, quantity, intended use, and provider. For CoDx medical devices requiring notification, describe status and summarize the corresponding therapeutic product development. PMDA/MHLW may request additional details. https://www.pmda.go.jp/files/000153495.pdf

7. Review System and Coordination

PMDA coordinates reviews between therapeutic product and CoDx offices. Inquiries on CoDx performance may occur during therapeutic product review and vice versa. Developers are encouraged to share information. For difficult cases, consult PMDA early. https://www.pmda.go.jp/files/000153495.pdf

8. Clinical Significance, Concordance Studies and Package Insert Requirements

Clinical significance is evaluated through therapeutic product trials using the CoDx. Concordance studies are required if the CoDx was not used in confirmatory trials. Package inserts must clearly state CoDx use in “INDICATIONS AND USAGE,” “DOSAGE AND ADMINISTRATION,” or precautions sections. The CoDx insert must name corresponding therapeutic products and restrict use to those purposes. https://www.pmda.go.jp/files/000153495.pdf

9. Practical Considerations and Developer Cooperation

Therapeutic product and CoDx developers must cooperate closely and share development status. Biological samples should be collected and stored with informed consent from early trial stages. Consult PMDA case-by-case on specific designs. The Japanese original prevails in any inconsistency. https://www.pmda.go.jp/files/000153495.pdf

10. Effective Date

The Q&A is effective from July 1, 2013. https://www.pmda.go.jp/files/000153495.pdf

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