Report of the International Regulatory Forum on Human Cell Therapy and Gene Therapy Products

被引：7

作者：

Hayakawa, Takao ^{[1
]}

Harris, Ian ^{[2
]}

Joung, Jeewon ^{[3
]}

Kanai, Nobuo ^{[4
]}

Kawamata, Shin ^{[5
]}

Kellathur, Srinivasan ^{[6
]}

Koga, Junichi ^{[7
]}

Lin, Yi-Chu ^{[8
]}

Maruyama, Yoshiaki ^{[9
]}

McBlane, James ^{[10
]}

Nishimura, Takuya ^{[9
]}

Renner, Matthias ^{[11
]}

Ridgway, Anthony ^{[12
]}

Salmikangas, Paula ^{[13
]}

Sakamoto, Norihisa ^{[9
]}

Sato, Daisaku ^{[9
]}

Sato, Yoji ^{[14
]}

Toda, Yuzo ^{[15
]}

Umezawa, Akihiro ^{[16
]}

Werner, Michael ^{[17
]}

Wicks, Stephen ^{[18
]}

机构：

[1] Kindai Univ, Higashiosaka, Osaka, Japan

[2] Janssen Res & Dev, Raritan, NJ USA

[3] Korean Minist Food & Drug Safety, Cheongwon Gun, Chungcheongbuk, South Korea

[4] Tokyo Womens Med Univ, Tokyo, Japan

[5] Inst Biomed Res & Innovat, Ibaraki, Osaka, Japan

[6] Hlth Sci Author, Singapore, Singapore

[7] Japan Pharmaceut Mfg Assoc, Tokyo, Japan

[8] Taiwanese Food & Drug Adm, Taipei, Taiwan

[9] Pharmaceut & Med Devices Agcy, Tokyo, Japan

[10] Med & Healthcare Prod Regulatory Agcy, London, England

[11] Paul Ehrlich Inst, Langen, Germany

[12] Hlth Canada, Ottawa, ON, Canada

[13] Finnish Med Agcy, Helsinki, Finland

[14] Natl Inst Hlth Sci, Tokyo, Japan

[15] Forum Innovat Regenerat Med, Tokyo, Japan

[16] Natl Inst Child Hlth & Dev, Tokyo, Japan

[17] Alliance Regenerat Med, Washington, DC USA

[18] European Directorate Qual Med & HealthCare, Strasbourg, France

来源：

BIOLOGICALS | 2016年 / 44卷 / 05期

关键词：

Cell therapy; Gene therapy; Transplantation; Regulation; PHARMACEUTICALS; QUALITY; SAFETY;

D O I：

10.1016/j.biologicals.2016.06.002

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

The development of human cell therapy and gene therapy products has progressed internationally. Efforts have been made to address regulatory challenges in the evaluation of quality, efficacy, and safety of the products. In this forum, updates on the specific challenges in quality, efficacy, and safety of products in the view of international development were shared through the exchange of information and opinions among experts from regulatory authorities, academic institutions, and industry practitioners. Sessions identified specific/critical points to consider for the evaluation of human cell therapy and gene therapy products that are different from conventional biological products; common approaches and practices among regulatory regions were also shared. Certain elements of current international guidelines might not be appropriate to be applied to these products. Further, international discussion on the concept of potency and in vivo tumorigenicity studies, among others, is needed. This forum concluded that the continued collective actions are expected to promote international convergence of regulatory approaches of the products. The Pharmaceuticals and Medical Devices Agency and Japanese Society for Regenerative Medicine jointly convened the forum with support from the National Institutes of Biomedical Innovation, Health and Nutrition. Participants at the forum include 300 experts in and outside of Japan.

引用

页码：467 / 479

页数：13

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