Recent policies that support clinical application of induced pluripotent stem cell-based regenerative therapies

Autor: Shinya Yamanaka, Kentaro Azuma
Jazyk: angličtina
Rok vydání: 2016
Předmět:
0301 basic medicine
Engineering
ESC
embryonic stem cell
RMP
regenerative medicine product
AMED
Japan Agency for Medical Research and Development
J-MACS
Japanese Registry for Mechanically Assisted Circulatory Support
Review Article
R&D
research and development
MEXT
Ministry of Education
Culture
Sports
Science and Technology
Regenerative medicine
GMP
good manufacturing practice
Japan
Agency (sociology)
GCTP
Good Gene
Cell
Cellular and Tissue-based Products Manufacturing Practice
Haplobank
Induced pluripotent stem cell
Riken CDB
Riken Center for Developmental Biology
RM Act
the Act on the Safety of Regenerative Medicine
Medical research
PMD Act
Pharmaceuticals and Medical Devices Act
CiRA
Center for iPS Cell Research and Application
3. Good health
iPS cells
Policy
Risk analysis (engineering)
MHLW
Ministry of Health
Labour and Welfare
NEDO
New Energy and Industrial Technology Development Organization
Research center
Regulation
iPSC
induced pluripotent stem cell
IRB
Institutional Review Board
PAL
Pharmaceutical Affairs Law
INTERMACS
Interagency Registry for Mechanically Assisted Circulatory Support
GAiT
Global Alliance for iPS Cell Therapies
Biomedical Engineering
Harmonization
WHO
World Health Organization
Biomaterials
JST
Japan Science and Technology Agency
03 medical and health sciences
NIBIO
National Institute of Biomedical Innovation
IBRI
Institution of Biomedical Research and Innovation
Global network
FY
fiscal year
Operations management
IND
Investigational New Drug
ICH
The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use
NIHS
National Institute of Health Science
Funding Agency
HLA
human leukocyte antigen
business.industry
PMDA
Pharmaceuticals and Medical Devices Agency
FDA
Food and Drug Administration
BLA
Biological License Approval
030104 developmental biology
CFR
Code of Federal Regulations
METI
Ministry of Economy
Trade and Industry
U.S.
United States
LVAD
left ventricular assist device
PIC/S
The Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme
business
DMF
Drug Master File
Developmental Biology
Zdroj: Regenerative Therapy
ISSN: 2352-3204
Popis: 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.
Highlights • Overview of collaborative research center network program related to iPSC-based regenerative therapies in Japan. • Recent initiatives that promote R&D and review of regenerative medicine products by regulatory authorities of Japan. • Challenges related to global cooperation among iPSC banks overseas under current regulations.
Databáze: OpenAIRE
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