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Report of the International Regulatory Forum of Human Cell Therapy and Gene Therapy Products

Pharmaceuticals and Medical Devices Agency (PMDA) and The Japanese Society for Regenerative Medicine (JSRM) jointly convened International Regulatory Forum of Human Cell Therapy and Gene Therapy Products on 16 March 2016 with support from National Institutes of Biomedical Innovation, Health and Nutrition (NIBIOHN), with regulatory trends in cell therapy and gene therapy products as the theme. The invitees included U.S.FDA and EMA and other regulatory authorities for cell therapy and gene therapy products in Europe and Asia and leading researches from in and outside of Japan.
This forum has been concluded successfully as follows:
Date: March 16, 2016
Venue: Osaka International Convention Center (Osaka City, Osaka Prefecture)
Dr. Tatsuya Kondo, Chief Executive of PMDA,
Mr. Kazuhiko Mori, Councillor for Pharmaceutical Safety, Minister's Secretariat, Ministry of Health, Labour and Welfare
Dr. Daisaku Sato, Director, Office of Cellular and Tissue-based Products, PMDA
Dr. Kohji Nishida, The Japanese Society for Regenerative Medicine (JSRM) (Professor, Osaka University)
Dr. Suminori Kono (Executive Director, NIBIOHN)
Dr. Takao Hayakawa (Professor, Kindai University)
Dr. Yoji Sato (Head of Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan)
Dr. Akihiro Umezawa (Director, Department of Reproductive Biology and Pathology, National Research Institute for Child Health and Development)
Dr. Shin Kawamata (Director, R&D Center for Cell Therapy, Foundation for Biomedical Research and Innovation)
Mr. Yuzo Toda (FIRM Chairperson)
from Japan, and 300 people from regulatory agencies, academic institutions and the industry in and outside of Japan participated in the forum.
group photo
 Chief Executive

The key attendees from outside of Japan included:
Dr. Ian Harris (Janssen Research & Development, USA)
Dr. Jeewon Joung (MFDS, Korea)
Dr. Srinivasan Kellathur (HSA, Singapore)
Dr. Yi-Chu Lin (TFDA, Taiwan)
Dr. James McBlane (MHRA, UK)
Dr. Steven S. Oh (FDA, USA)
Dr. Matthias Renner (PEI, Germany)
Dr. Anthony Ridgway (Health Canada, Canada)
Dr. Paula Salmikangas (CAT/EMA, EU)
Mr. Michael Werner (ARM, USA) 
Dr. Stephen Wicks (EDQM, EU)

Forum Overview

Nowadays, development of human cell therapy and gene therapy products has been progressed internationally, and efforts have been made to address regulatory challenges in the evaluation of quality, efficacy and safety of the products in clinical trials and reviews. In this forum, updates on the specific challenges in quality, efficacy and safety of regenerative medical products in the views of development in interenational scale were shared through the exchange of information and opinions among experts from regulatory authorities, academia and industry from in and outside of Japan.

All the presentations and discussions were conducted in English. The recognition of the current challenges which are in common across all cell therapy products in the quality, pre-clinical and clinical evaluation as well as necessary points to consider from scientific perspective was shared through active discussions among the participants from in and outside of Japan.

Each regulatory authority in both advanced countiries and Asian region has been implementing category specific regulations for cell therapy and gene therapy products. And non conventional approaches for regulatory requirements based on characteristics of the product were shared, i.e. flexible and risk based approaches in reference to ICH Q5A, Q5D, Q5E, Q6B, and S6 Guidelines.

In particular, a common recognition was reached on scientific approaches required for cell therapy and gene therapy products to address issues including the principle of potency as a quality attribute, practical problems in the impurity evaluation, the need and the limit of pre-clinical evaluation, pre-clinical tumorigenicity testing methods, and clinical study designs.

On the other hand, even the field of cell therapy and gene therapy has been progressed in terms of technology, it is still in the process of advancing in terms of regulatory science. It was confirmed that regulatory convergence rather than guideline harmonization should be promoted due to variation of those products, and efforts for scientific alignment among international regulatory authorities should be continued. In addition, the proposal of ‘minimum consensus package’ in reference to the Guideline for the Quality, Safety Assurance of Cellular and Tissue-based Products issued in Japan, was introduced as common technical requirements to be applicable internationally in the future.

This forum is expected to offer the opportunity to trigger the international dissemination of regulatory science for regenerative medical products and a promotion of international convergence in the evaluation of quality, efficacy and safety of the products through the activities of IPRF, APEC, WHO and so on.

The outcome of the forum will be published in an international journal to present the current scientific principles on the cell therapy regulation.


Presentation Slides

The program is here.

Session 1  Keynote Lecture

FDA’s perspective on cellular and gene therapy regulation

Steven S. Oh (Chief, Cell Therapy Branch, OCTGT, CBER, FDA, USA) (teleconference)

European perspective on ATMPs

Paula Salmikangas (Chair, CAT/EMA, EU)

Session 2  Safety Evaluation Approaches for hCTPs in Reference to ICH S6 and S7 Guidelines

2.1 Process related impurity safety evaluation

Takuya Nishimura (PMDA, Japan)

2.2 Pre-clinical safety evaluation

James McBlane (MHRA, UK)

2.3 Tumorigenicity

Shin Kawamata (Institute of Biomedical Research and Innovation (IBRI), Japan)

2.4 Pre-clinical proof of concept and other pre-clinical issues

Ian Harris (Janssen Research & Development, USA)

Session 3  Quality Eapproaches for hCTPs in Reference to ICH Q5 and Q6 Guidelines

3.1 Potency

Anthony Ridgway (Health Canada, Canada)

3.2 Raw materials and microbiological control

Stephen Wicks (EDQM, EU)

3.3 Specification and process validation

Yoshiaki Maruyama (PMDA, Japan)

3.4 Vector characterization of genetically-modified cell therapies

Matthias Renner (PEI, Germany)

Session 4  Efficacy Evaluation Approaches for hCTPs

4.1 Study design

Nobuo Kanai (Tokyo Women’s Medical Univ. Japan)

4.2 Clinical issues

Graziella Pellegrini (Università degli Studi di Modena e Reggio Emilia, Italy) (Cancelled)

4.3 Clinical and regulatory issues in the US

Michael Werner (ARM, USA)

Session 5  International Development and Regulatory Issues

5.1 Endeavour to minimum consensus package

Takao Hayakawa (Kindai Univ., Japan)

5.2 Regulatory Panel

Regulatory update on cell and gene therapy products in Korea

Jeewon Joung (MFDS, Korea)

Cell- and tissue-based therapeutic product regulations in Singapore and regulatory convergence

Srinivasan Kellathur (HSA, Singapore)

Regulation of human cell therapy and gene therapy products in Taiwan

Yi-Chu Lin (TFDA, Taiwan)

Regulatory trends in regenerative medicine in Japan

Daisaku Sato (PMDA, Japan)

Session 6  Summary

Overall Summary IRF2016

Daisaku Sato (PMDA, Japan)

Meeting Report

To be announced.

>Click here for the outline of the forum

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