Revisions to the requirement of the Japanese clinical study data for biosimilar developments in Japan

ABSTRACT

Background

The ‘Questions and Answers (Q&A)’ document regarding Japanese biosimilar guideline elucidated that Japanese participant enrollment in at least one comparative clinical study was required for the marketing authorization application (MAA) of biosimilars in Japan.

Research design and methods

To discuss the requirement of Japanese clinical study data for biosimilar development, the trend in comparative clinical studies conducted for approved biosimilars of monoclonal antibodies and fusion proteins was analyzed, and the consistency of the results between the overall population and the Japanese population according to the publicly available information was reviewed.

Results

The number of comparative clinical studies enrolling Japanese participants was 25 cases, and the type and percentage were 13 (52%) and 12 (48%) cases of comparative pharmacokinetic study and comparative efficacy study, respectively. In all comparative clinical studies, consistent results between the overall population and the Japanese population were shown.

Conclusions

Our study indicated that Japanese participant enrollment in comparative clinical studies may not always be necessary for biosimilar development when certain conditions are satisfied. This has been described in the revised Q&A document published by the Ministry of Health, Labour and Welfare in January 2024.

KEYWORDS:

• Biosimilar
• Japanese clinical study data
• Monoclonal antibody
• Fusion protein
• Ethnic factor
• PMDA

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Declaration of interest

R Kuribayashi, K Goto, A Hariu and Y Kishioka are employees of the Pharmaceuticals and Medical Devices Agency. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

R Kuribayashi was the main author of this article. All authors contributed to the fact-finding and editing of this article. All authors have read and approved the final manuscript.

Disclaimer

The views expressed in this article are those of the authors and do not necessarily reflect the official views of the Pharmaceuticals and Medical Devices Agency.

Figure 1. Type and percentage of Japanese participants in a comparative clinical study. PK: Pharmacokinetics

Additional information

Funding

No external funding was used for the preparation of this manuscript.