Stem Cells Live: Japan’s New Legislation – will companies benefit from it?

Dr Hasakazu Hirata Tissue-Based Products for the Pharmaceutical and Medical Device Agency PMDA WSCRM Terrapinn

“It is not a phase I clinical trial”, said Dr Hasakazu Hirata, during a keynote interview with Prof Chris Mason. Dr Hirata is Review Director at the Office of Cellular and Tissue-Based Products for the Pharmaceutical and Medical Device Agency (PMDA), the FDA and EMA equivalent in Japan.  He talked about the new legislation being promoted by the Japanese government.

Starting from 1 November 2014, companies will be allowed to apply for conditional marketing approval in Japan. The key requirement, for companies from all around the world willing to benefit from this accelerated path to market, is to provide data from a clinical study predicting likely efficacy and confirm short-time safety.  He also appreciates the difficulty companies have in the early stage of development to evaluate which data will be necessary to prove efficacy as this will largely depend on the type of therapy.

“Clinical data from a small Japanese population will also be necessary”, he added. Early engagement with PMDA early in the development of the product is then highly recommended.

Will all these products get reimbursement in Japan? It is hard to tell. The Japanese government is currently working on a set of rules which will require minimum criteria.

This 7 year period (10 for orphan drug designation) of conditional approval will allow the company to get more clinical data necessary for final approval in Japan and could also be used in the EU, US, Canada.

Mesoblast limited has already made an agreement with JCR Pharmaceuticals Co. Ltd to manufacture, develop and market Mesoblasts for the treatment of steroid-refractory GvHD in children and adults following bone marrow transplantation.

Athersys is also looking at Japan to bring their proprietary cell product, Multistem, to market quicker. If successful, the company could revolutionize the current treatment for stoke, which is considered one of the biggest challenge in the Japanese healthcare system.

Hasakazu is confident that this new legislation will be beneficial to Japanese population as it will enable access to new and better medicines.

Fabio D’Agostino for Total BioPharma at the World Stem Cell and Regenerative Medicine Congress, London.

Fabio is a student of the Biopharmaceutical Bioprocessing Technology Centre based in Newcastle University’s School of Chemical Engineeringand Advanced Materials.


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