The positions of regulatory agencies on new classes of biomedical products such as cell and gene therapies1,2,3 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 Union5,6. 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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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
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