New Regulatory Framework in Japan

Regulatory perspectives and considerations for cell & gene therapies

Japan has been at the forefront in the innovation and development cell and gene therapies. To accelerate the development, innovation and commercialization of regenerative medicine products, Japan has championed a radical approach by passing new legislations known as the act on the safety of regenerative medicine”(ASRM) and the pharmaceuticals, medical devices and other therapeutic products act (PMDA), which came into effect in November 2014. The PMDA and ASRM identify the need for collaboration between the industry and medical institutions and the need for guidelines securing the safety of new stem cell therapies respectively. The new PMDA includes drugs, medical devices as well as regenerative medical products defined as ‘processed live human/animal cells’ that are intended to be used for (1) the reconstruction, repair, or formation of structures or functions of the human body or (2) the treatment or prevention of human diseases, and gene therapy.

The ASRM regulates medical professionals’ practices and clinical studies related to regenerative medicine and charts the legal regulations for research and daily practice of cell therapy without approved regenerative medical products. The ASRM specifies manufacturing and quality requirements for cell and gene therapies, also called as good, gene, cellular and tissue-based product manufacturing practice (GCTP) to ensure safety of the manufacturing facilities and equipment, as well as the aspects of quality control. The PMDA enlists a new pathway to get conditional and time-limited approval for “regenerative medical products,” accounting for the high degree of variability in cell and therapies under the classification.

The new legislative framework in Japan requires compliance of cell processing facilities in Japan to receive a license from the Ministry of Health Labor and Welfare (MHLW) to operate and the facilities may be audited by PMDA. Cell processing facilities outside Japan require accreditation from MHLW in order to supply processed cells for regenerative medicine to medical intuitions in Japan and all licensed facilities are mandated to produce annual reports to the MHLW regarding the number of cells processed, specific claims on the processed cells and any incidences of diseases. With the new law, it is now possible for medical institutes to outsource cell/tissue processing operations to an external provider, which was previously not allowed.

Conditional, time-limited marketing authorization of the product is possible upon successful demonstration of the safety and evidence predicting likely efficacy. However, a conditional and time-limited approval must be followed by a confirmatory clinical study and follow-up patient safety measures (with respect to restrictions on clinical institutions using the treatment), enabling a further approval process within a maximum period of 7 years. This is an advantage as the product can be made available to patients even with a limited demonstration on safety and efficacy. This strategy is novel to Japan and can be particularly attractive for foreign companies wanting to enter Japanese market via a strategic or financial partnership with a Japanese company. Foreign companies interested in launching cell and gene therapeutics in Japan need to file for a clinical trial with the MHLW. The decision if the trial qualifies for a conditional approval rather than the next phase of a clinical trial lies at the discretion of PMDA on a case-by–case basis. Consultations with the PMDA are required before the initiation of clinical trial under the PMDA provide a clear picture regarding the regulatory clearances required when submitting a clinical trial plan. In effect, the new regulations in Japan encourage international collaboration and are a step towards attracting investment and accelerating approvals and commercialization of cell and gene therapeutics.


The above post is an excerpt from our recent whitepaper “Regulatory Perspectives and Considerations for Cell & Gene Therapies“. This paper summarizes the fundamental considerations associated with the development and commercialization of cell and gene therapeutics and evaluate the regulatory landscape, with a special focus on the new legislative framework in Japan and the regulation of advanced therapy medicinal products in Europe. Access the complete paper here. 

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