Azuma Kentaro, Yamanaka Shinya
Center for iPS Cell Research and Application, Kyoto University, Kyoto 606-8507, Japan.
Gladstone Institute of Cardiovascular Disease, San Francisco, California 94158, USA.
Regen Ther. 2016 Mar 1;4:36-47. doi: 10.1016/j.reth.2016.01.009. eCollection 2016 Jun.
In Japan, a research center network consisting of Kyoto University to provide clinical-grade induced Pluripotent Stem Cells (iPSC) and several major research centers to develop iPSC-based regenerative therapies was formed for the clinical application of iPSCs. This network is under the supervision of a newly formed funding agency, the Japan Agency for Medical Research and Development. In parallel, regulatory authorities of Japan, including the Ministry of Health, Labour and Welfare, and Pharmaceuticals and Medical Devices Agency, are trying to accelerate the development process of regenerative medicine products (RMPs) by several initiatives: 1) introduction of a conditional and time-limited approval scheme only applicable to RMPs under the revised Pharmaceuticals and Medical Devices Act, 2) expansion of a consultation program at the early stage of development, 3) establishment of guidelines to support efficient development and review and 4) enhancement of post-market safety measures such as introduction of patient registries and setting user requirements with cooperation from relevant academic societies and experts. Ultimately, the establishment of a global network among iPSC banks that derives clinical-grade iPSCs from human leukocyte antigens homozygous donors has been proposed. In order to share clinical-grade iPSCs globally and to facilitate global development of iPSC-based RMPs, it will be necessary to promote regulatory harmonization and to establish common standards related to iPSCs and differentiated cells based on scientific evidence.
在日本，为了诱导多能干细胞（iPSC）的临床应用，一个由京都大学提供临床级iPSC以及几个主要研究中心开展基于iPSC的再生疗法研发的研究中心网络已经形成。该网络受新成立的资助机构——日本医疗研究与开发机构的监管。与此同时，日本的监管当局，包括厚生劳动省和药品与医疗器械局，正通过多项举措努力加速再生医疗产品（RMP）的开发进程：1）在修订后的《药品与医疗器械法》中引入仅适用于RMP的有条件和限时批准制度；2）在开发早期扩大咨询项目；3）制定支持高效开发和审查的指南；4）加强上市后安全措施，如引入患者登记制度并与相关学术团体和专家合作设定用户要求。最终，有人提议在从人类白细胞抗原纯合供体获取临床级iPSC的iPSC库之间建立全球网络。为了在全球范围内共享临床级iPSC并促进基于iPSC的RMP的全球开发，有必要推动监管协调，并基于科学证据建立与iPSC和分化细胞相关的共同标准。
