Cell and gene therapy approvals in Japan and the need for international harmonization

The positions of regulatory agencies on new classes of biomedical products such as cell and gene therapies^1,2,3 have a strong influence on product development but have not been clearly defined. We examined all 17 cell and gene therapies approved as of December 2022 in Japan⁴ and compared them to the fates of similar candidate therapies in other countries. This analysis identified notable differences with respect to the use of randomized clinical trials (RCTs), the numbers of participants enrolled, and approaches for evaluating trial results. These differences can be influenced by the use of various accelerated-approval pathways, such as Conditional Early Approval in Japan, the Regenerative Medicine Advance Therapy Designation in the United States, and Priority Medicines in the European Union^5,6. We conclude that the nascent field of cell and gene therapies would benefit from a concerted effort toward international harmonization with respect to clinical study designs and product approval criteria.

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Acknowledgements

We would like to thank Saki Yokoshiki for help in compiling product lists.

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Authors and Affiliations

Hokkaido University Hospital, Clinical Research and Medical Innovation Center, Sapporo, Japan

Teruyo Arato
Hokkaido University Graduate School of Medicine, Department of Regulatory Science, Sapporo, Japan

Teruyo Arato
Teikyo Heisei University, Faculty of Pharmaceutical Sciences, Tokyo, Japan

Kaori Nomura
Fukushima Medical University, Advanced Clinical Research Center, Fukushima, Japan

Kaori Nomura

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Both authors contributed equally to conception and writing of the paper.

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Correspondence to
Teruyo Arato.

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The authors declare no competing interests.

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Nature Biotechnology thanks Megan Munsie and the other, anonymous, reviewer(s) for their contribution to the peer review of this work.

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Arato, T., Nomura, K. Cell and gene therapy approvals in Japan and the need for international harmonization.
Nat Biotechnol (2024). https://doi.org/10.1038/s41587-023-02053-7

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Published: 08 January 2024
DOI: https://doi.org/10.1038/s41587-023-02053-7