title: Report of the International Regulatory Forum on Human Cell Therapy and Gene Therapy Products.
abstract: 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.
authors.affiliation: Kindai University, Japan.
authors.forename: Takao
authors.initials: T
authors.lastname: Hayakawa
authors.affiliation: Janssen Research & Development, USA.
authors.forename: Ian
authors.initials: I
authors.lastname: Harris
authors.affiliation: Korean Ministry of Food and Drug Safety, South Korea.
authors.forename: Jeewon
authors.initials: J
authors.lastname: Joung
authors.affiliation: Tokyo Women's Medical University, Japan.
authors.forename: Nobuo
authors.initials: N
authors.lastname: Kanai
authors.affiliation: Institute of Biomedical Research and Innovation, Japan.
authors.forename: Shin
authors.initials: S
authors.lastname: Kawamata
authors.affiliation: Health Science Authority, Singapore.
authors.forename: Srinivasan
authors.initials: S
authors.lastname: Kellathur
authors.affiliation: Japan Pharmaceutical Manufacturers Association, Japan.
authors.forename: Junichi
authors.initials: J
authors.lastname: Koga
authors.affiliation: Taiwanese Food and Drug Administration, Taiwan.
authors.forename: Yi-Chu
authors.initials: YC
authors.lastname: Lin
authors.affiliation: Pharmaceuticals and Medical Devices Agency, Japan.
authors.forename: Yoshiaki
authors.initials: Y
authors.lastname: Maruyama
authors.affiliation: Medicines and Healthcare Products Regulatory Agency, UK.
authors.forename: James
authors.initials: J
authors.lastname: McBlane
authors.affiliation: Pharmaceuticals and Medical Devices Agency, Japan.
authors.forename: Takuya
authors.initials: T
authors.lastname: Nishimura
authors.affiliation: Paul Ehrlich Institute, Germany.
authors.forename: Matthias
authors.initials: M
authors.lastname: Renner
authors.affiliation: Health Canada, Canada.
authors.forename: Anthony
authors.initials: A
authors.lastname: Ridgway
authors.affiliation: Finnish Medicines Agency, Finland.
authors.forename: Paula
authors.initials: P
authors.lastname: Salmikangas
authors.affiliation: Pharmaceuticals and Medical Devices Agency, Japan.
authors.forename: Norihisa
authors.initials: N
authors.lastname: Sakamoto
authors.affiliation: Pharmaceuticals and Medical Devices Agency, Japan. Electronic address: sato-daisaku@mhlw.go.jp.
authors.forename: Daisaku
authors.initials: D
authors.lastname: Sato
authors.affiliation: National Institute of Health Sciences, Japan.
authors.forename: Yoji
authors.initials: Y
authors.lastname: Sato
authors.affiliation: Forum for Innovative Regenerative Medicine, Japan.
authors.forename: Yuzo
authors.initials: Y
authors.lastname: Toda
authors.affiliation: National Institute for Child Health and Development, Japan.
authors.forename: Akihiro
authors.initials: A
authors.lastname: Umezawa
authors.affiliation: Alliance for Regenerative Medicine, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Werner
authors.affiliation: European Directorate for the Quality of Medicines and HealthCare, France.
authors.forename: Stephen
authors.initials: S
authors.lastname: Wicks
mesh_terms: D064987:Cell- and Tissue-Based Therapy / Q000295:instrumentation / Q000379:methods*; D003226:Congresses as Topic; D015316:Genetic Therapy / Q000295:instrumentation / Q000379:methods*; D006801:Humans
keywords: Cell therapy; Gene therapy; Regulation; Transplantation