Recently, as the number of approved Regenerative Medical Products (RMPs) in Japan has been rising, companies are expected to face a number of challenges specific to the Cell Therapy and/or Gene Therapy Products. Meanwhile, the collaborations between industry, government, and academia to resolve these challenges are drawing the spotlights, such as the framework to consider the laws and regulations related to the RMPs among regulatory authorities and industries. In this session, current regulatory considerations on the unique/specific challenges of RMPs through the product life cycle from development to post-marketing stages, such as regulatory affair, change control, manufacturing, technical transfer, quality control, Japanese Standards for Biological Ingredients, raw material procurements, or logistics, will be discussed, with overviews of case studies and lessons learned from actual developments. As a part of initiatives to resolve the unique/specific challenges of RMPs through the product life cycle, the developing/draft guidance for the preparation of quality section of the application file for RMPs will be shared by PMDA to exchange views on that. Furthermore, the current Japanese and European operations of the Law/Act concerning Cartagena Protocol on Biosafety for the development of gene therapy products will be shared, and the differences in the regulatory consideration, such as scope, criteria, and approaches of review process through the development of gene therapy products, will be discussed.
Key Questions: • What are the challenges in the development of cell therapy products or gene therapy products unique to Japan? • What are the differences in the operation of Cartagena Act between Japan and EU to develop gene therapy products? Are there any desired improvement or streamline efforts in the operation of Cartagena Act for the clinical development of the gene therapy products in Japan? • What are the desirable suggestions regarding the developing/draft guidance for the preparation of quality section of the application file for RMPs, from Industry perspective? Or, what are the unique/specific challenges in RMPs to prepare the quality part of application dossiers? • Are there any desirable improvement/updating of regulation to encourage the development of RMPs in Japan? • Are there any differences and/or challenges in review/evaluation process concerning the cell/gene therapy products compared to those of Drugs? • Compared GMP compliance inspection for Drugs, are there any differences and/or challenges in GCTP compliance inspection concerning cell/gene therapy products? • At present, there is no region nor country which has signed mutual recognition agreements with Japan concerning GCTP inspections and batch certification of RMPs. What kinds of challenges would be expected to achieve the MRAs for cell/gene therapy products? Are there any expectation/request from Industry concerning the MRAs for the cell/gene therapy products? • Compared to Drugs, the amount of analytical sampling could significantly impact on the yield, due to its smaller lot size. At present, the importer countries are required to conduct the batch release testing of the imported RMPs in some countries including Japan, and the impact of the amount of analytical sampling on the yield could be substantial. Regarding the imported cell/gene therapy products, are there any differences in the regulatory requirement concerning batch release testing in the importer countries or the status of waiving re-testing upon importation based on product type or country/territories? Are there any challenges or desirable streamlining from perspectives of regulator, industry, or patients? Are there any expectations to mitigate the impact of batch release testing or re-testing in the importer countries from regulatory or industry? • Compared to the PQS of Drugs, are there any differences or challenges in the quality system for cell/gene therapy products? • Compared to Drugs, are there any specific challenges concerning supply chain including a case for storage/transportation at ultra-low temperature condition, and a case for a product requiring rapid delivery?
Session Speakers: 14:05 - 14:30 Global Regulatory Considerations of Allogeneic Cell Therapies Yoko Momonoi, Takeda Pharmaceutical Company Limited, United States
14:30 - 14:55 Points to Consider and Challenges in CMC for Regenerative Medical Products Mitsuo Kitada and Naoyuki Hanada, Novartis Pharma K.K., Japan
14:55 - 15:20 Composition of the Application Material on Quality for the Marketing Authorization Application of Regenerative Medical Products Atsushi Nishikawa, PMDA-Pharmaceuticals and Medical Devices Agency, Japan
15:35 - 16:00 Current Status of GMO Regulation in Japan Seiichi Kanzaki, PMDA-Pharmaceuticals and Medical Devices Agency, Japan
16:00 - 16:25 Differences in Regulations on Gene Modified Organisms Between the Europe, Japan, and the United States Gentaro Tajima, Pfizer R&D Japan G.K., Japan