PMDA consultation



Ministry of Health, Labor and Welfare (MHLW)

“Authorizer” a/o “Organizer”

  • Marketing Authorization
  • Safety Measures for emergent and significant case
  • Examination and Authorization
    • Orphan Drug Designation
    • Type 1 Use of Living Modified Organisms (LOMs) #1

(#1 Cartagena Act: Act on the Conservation and Sustainable Use of Biological Diversity through Regulations on the Use of LMOs
Type 1 use: Use without preventing the dispersal of LMO into the air, water or soiloutside facilities.)

Gene and Cell Therapy

Pharmaceuticals and Medical Devices Agency (PMDA)

“Reviewer” a/o “Guide”

  • Scientific Review
  • GxP (GCTP/GCP/GLP/GMP etc.) inspection
  • Consultation on
    • Research and Development Strategy
    • Quality#2 and Non-Clinical Safety
    • Clinical Trial Protocol
    • Application and Data Package

(#2 Standard for Biological Ingredients: Real Japanese standard, Major Gap compared with US(FDA) or EU(EMA))

PMDA Consultation

Basic Track of PMDA Consultation


Act on the Safety of Regenerative Medicine (ASRM)

[Enacted in November 2014]
To establish steps for the practice and ensure the safe and ethical administration of regenerative medical technologies including Gene therapy

<Key Points>

  • Processed cellular products should be produced under the guidance of a medical institution, for the purposes of “clinical research” or “medical treatment at one’s own expense”, and meant for the treatment of a specific patient.
  • Foreign CPC need to receive an inspection by PMDA to get a license.

Pharmaceuticals and Medical Devices Act (PMD Act)

[Revised in November 2014]
To provide a route to market and establish regulations for regenerative medical products that is more in sync with the current industry/patient needs.

<Key Points>

Conditional Time-limited Approval

A system of approval put in place for those regenerative medical products that have all of the following conditions, and allow for the sale of products for up to 7 years:

  • No any major safety concerns
  • Probably effective
  • Not uniform in nature

Conditional and Time-limited Authorization of Regenerative Medical Products

Regulatory Science Strategic / Comprehensive Consultation

Assistances/Supports for Preparatory Meeting, “Zizen-Mendan”

  • Product reviewing
  • Guidelines and consulting items identification
  • Briefing documents preparation
  • Meeting arrangement

Assistances/Supports for Face-to-Face Advice Meeting, “Taimen-Jyogen”

  • Briefing books preparation
  • [Quality]Preliminary compliance survey for the “Japanese” Standards for Biological Ingredients
  • [Quality]Eligibility evaluation for products supecification, quality control, in-process control and impurities management
  • [Safety]Sufficency analysis of non-clinical safety studies
  • [Safety]Assessing the nessecity of Type 1 or 2 Use Application under the CARTAGENA-ACT
  • [Clinical]Drawing the Diagram of Overall Development Roadmap
  • [Clinical]Detailed Description of Clinical Trial Protocol
  • Meeting arrangement
  • Archaive of PMDA’s official comments

Process of Regulatory Science Strategic / Comprehensive Consultation

Process of Pharmaceutical Affairs Consultation on R&D Strategy