Update on Regulatory Landscape of Regenerative …Pharmaceuticals and Medical Devices Agency 1...
Transcript of Update on Regulatory Landscape of Regenerative …Pharmaceuticals and Medical Devices Agency 1...
1Pharmaceuticals and Medical Devices Agency
Update on Regulatory Landscape of
Regenerative Medicine in Japan
DISCLAIMER : The contents of this presentation represent the view of this
presenter only, and do not represent the views and/or policies of the PMDA
4th India - Japan Medical Products Regulation Symposium 2019
5 February 2020
Yoshiaki Maruyama, Ph.D.Review Director,
Office of Cellular and Tissue-based Products
PMDA, Japan
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Regenerative Medicine
All medical technologies using
processed cells which safety and
efficacy have not yet been
established
The Act on Pharmaceuticals and
Medical Devices (PMD Act)
Production and marketing of
regenerative and cellular
therapeutic products by firms
The Act on the Safety of
Regenerative Medicine
Medical Care or
Academic Research Purpose
Commercial Product
Marketing Authorization Purpose
Enacted on
25 November 2014
Two Acts Regulating Regenerative Medical Technology & Product
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Technology excluded by Cabinet OrderYes
Out of the scope of application of the Act
YesNo
Cells to which gene was introducedYes
No
Class I
Class I
YesStem cells are used
No
Allogeneic cellsYes
No
Class I
No
Xenogeneic cellsYes
Class I
No
Purpose is reconstruction, repair or formation
of human body structure of function
No
Yes
Homologous use Class IINo
Yes
Human embryotic stem cells, iPS cells, cells similar to iPS cells
Class III
Class IICell cultureYes
No
Homologous use Class IINo
YesClass III
Class IICell cultureYes
No
Homologous use Class IINo
Yes
Class III
Risk Classification Regenerative Medical Technology
Note:In vivo gene therapy is currently out of scope
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MHLW
Health Science CouncilHospitals / Clinics
submission evaluation
MHLW
submission
opinion
Provision (Within
90 days)
Change order (Within 90 days)
Certified special committee for regenerative medicine
Plan
High Risk (class I)
Middle Risk (class II)
Low Risk (class III)
Hospitals / Clinics
Plan
evaluation
submissionProvision
Hospitals / Clinics
Plan
submission
submission evaluation
submission
MHLW
Provision
Certified special committee for regenerative medicine
Certified committee for regenerative medicine
Special committee = 57
Committee = 96
Plans (3,785)
Clinical
research
Medical
care
0 19
443 70
3,342 64
(As of 30 November 2019)
Rules for Hospitals and Clinics
Safety Act
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Medical institutionCorporate factory. etc
Collection
Transplant
Processing, storage
Outside hospital Within hospital
Processing, storage
Notified = 2,711 sites Licensed (Local)
= 66 sites
Accreditation (Overseas) = 6 site (Korea) = 1sites (China) = 2 sites (Taiwan)
Cell Processing Facility
(As of 30 November 2019)
Safety Act
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Organizations and Authorities in Japan
Health Policy Bureau
Pharmaceutical Safety and Environmental Health Bureau
R&D Division
Site Inspections
Product Review
Approval Decision, License Execution
Office of Safety II
HospitalsCPFsManufacturing
SitesMAHs
Certified committees
MHLW
PMDA
Office of Cellular and Tissue based Products
Office of Manufacturing Quality and Compliance
Safety Act
Medical Device Evaluation Division
PMD Act4th India - Japan Medical Products Regulation Symposium 2019
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Approved Plans (iPS)
2019-
Under The Act on the Safety of
Regenerative Medicine
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Feb/2019
Safety Act
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Aug/2019Mar/2019 Safety Act
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Jan/2020Safety Act
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Regenerative Medicine
All medical technologies using
processed cells which safety and
efficacy have not yet been
established
The Act on Pharmaceuticals and
Medical Devices (PMD Act)
Production and marketing of
regenerative and cellular
therapeutic products by firms
The Act on the Safety of
Regenerative Medicine
Medical Care or
Academic Research Purpose
Commercial Product
Marketing Authorization Purpose
Enacted on
25 November 2014
Two Acts Regulating Regenerative Medical Technology & Product
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Consultation and Review Pathway
Regulatory Science Strategy
Consultations
After completion of
exploratory study meeting
Consultations on quality assurance
Etc.
Quality
Non-clinical
Clinical trial
Development
Consultation
Application for
Marketing
Authorization
Review of Clinical Trial Protocol
(30 days-IND review)
* To prevent the occurrence or spread of
hazard to the public.
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Face-to-face consultations
on R&D strategyFY2014 FY2015 FY2016 FY2017 FY2018 Total
Regenerative medical products#1 2 11 14 13 5 45
Quality and safety of regenerative
medical products#2 18 [44] 29 [55] 26 [64] 29 [71] 25 [54] 158 [340]
Total 46 66 78 84 59 385
Number of INDs#4 FY2014 FY2015 FY2016 FY2017 FY2018 Total
First Notification 3 [1] 10 [2] 16 [7] 13 [8] 18 [8] 60 [26]
Others 1 [1] 3 [2] 5 [0] 14 [10] 17 [3] 40 [16]
Face-to-face consultations for INDs#3 6 18 28 38 47 137
PMD Act
#1: This consultation category was introduced on November 25, 2014.#2: Some consultations were divided into multiple sessions over several days, to confirm the quality and safety of regenerative medical products before the submission of clinical trial notifications. The figures in brackets indicate the total number of sessions.#3: For prior assessment consultations for regenerative medical products, the number of consultation categories was summed. (Set categories: quality/safety/efficacy, exploratory trial, confirmatory trial)#4: Timing: The first notifications; 31 days before, Others; 2 weeks before. The figures in brackets indicate the Investigator -initiated Clinical Trials.
Number of Consultations and INDs
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Today Approved Regenerative Medical Products
Maruyama et al., Cell & Gene Therapy Insights 2019; 5(6), 561–568
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https://www.novartis.co.jp/news/media-releases/prkk20180423-1
Press release in Japanese)
Mar/2019
Indication:
ALL
DLBCL
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Desirable development model of regenerative medical products for conditional and time-limited approval
Maruyama et al., Cell & Gene Therapy Insights 2019; 5(6), 561–568
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https://www.anges.co.jp/en/pdf.php?pdf=100990.pdf
https://www.anges.co.jp/en/project/proj_develop.html
Indication:
Ulcer healing for CLI patient
Conditional &
Time-Limited
Approval
Mar/2019
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Assigned SAKIGAKE
Designation4th round, announced by 8th April 2019
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Lead the world in the practical application of innovative medical products
Accelerate R&D through supporting each stage
The Strategy of SAKIGAKE covers from basic research to clinical
research/trials, approval reviews, safety measures, insurance coverage,
improvement of infrastructure and the environment for corporate activities,
and global expansion.
Strengthen the structure of PMDA
(consultation, review, safety measures in terms of quality and quantity)
Promotion of Regulatory Science
(Developing guidelines/assessment for the state-of-the-art technology)
Strategy of SAKIGAKE
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4th Round
2019
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4th Round
2019
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