Regulatory approval for autologous human cells and tissue products in the United States, the European Union, and Japan

Kazuo Yano; Natsumi Watanabe; Kenichiro Tsuyuki; Taisuke Ikawa; Hiroshi Kasanuki; Masayuki Yamato

doi:10.1016/j.reth.2014.10.001

Regulatory approval for autologous human cells and tissue products in the United States, the European Union, and Japan

Regen Ther. 2014 Nov 11:1:45-56. doi: 10.1016/j.reth.2014.10.001. eCollection 2015 Jun.

Authors

Kazuo Yano^{1

2}, Natsumi Watanabe^{1

3}, Kenichiro Tsuyuki³, Taisuke Ikawa³, Hiroshi Kasanuki^{2

3}, Masayuki Yamato^{1

3}

Affiliations

¹ Institute of Advanced Biomedical Engineering and Science, Tokyo Women's Medical University, 8-1 Kawada-cho, Shinjuku-ku, Tokyo, 162-866, Japan.
² Research Institute for Science and Engineering, Waseda University, 8-1 Kawada-cho, Shinjuku-ku, Tokyo, 162-866, Japan.
³ Cooperative Major in Advanced Biomedical Sciences, Joint Graduate School of Tokyo Women's Medical University and Waseda University, 8-1 Kawada-cho, Shinjuku-ku, Tokyo, 162-866, Japan.

Abstract

Human cells and tissue products belong to a relatively new class of medical products. Therefore, limited information is available on the classification and premarket evaluation of human cells and tissue products in the United States (US), the European Union (EU), and Japan. In this study, the definition, legislation, and approval system of these products were surveyed. A total of nine autologous human cells and tissue products approved until October 2013 were collected. The definitions of human cells and tissue products were compatible among the US, the EU and Japan. The products were classified as human cells, tissue, and cellular and tissue-based products (HCT/Ps) in the US, advanced therapy medicinal products (ATMPs) in the EU, and cell/tissue-engineered products in Japan. These products were categorized as biologics and medical device in the US and Japan, and drug in the EU. The issuance of new guidance induced regulatory impact for manufacturer, especially in the US. These products are subjected to the accelerated approval of biological product, the humanitarian device exemption approval, the premarket application approval, the biologics license application approval, and new drug application approval with specific targeting of postapproval registry or surveillance. Of nine autologous human cells and tissue products, four products had been evaluated using clinical experiences or open clinical trials with small subjects, although the rests of products had been evaluated using comparative clinical trials with control treatment. Our survey suggests that autologous human cells and tissue products would need postmarket-oriented evaluation rather than premarket-oriented evaluation for doctors and patients.

Keywords: Autologous cell; Chondrocyte; Epithelial cell; Fibroblast; Macrophage; Regenerative medicine; Regulatory science; Tissue engineering.