Dr Hirata of the Pharmaceutical and Medicinal Device Authority (PMDA) in Japan detailed how legislative changes in Japan due for enactment by the end of November will be pivotal in throwing open the doors for cell and tissue based therapy manufacturing in or with Japanese manufacturing companies.
Novel therapeutics ready to conduct efficacy (phase 1/2) studies will be welcomed to apply for conditional market licensing in Japan from November 2014. Companies successful in achieving this status will be likely to be eligible for reimbursement from the Japanese government although assurances have yet to be provided.
This legislation goes a long way to de-risking investment into novel therapeutics by raising margins and reducing the cost of clinical trials if – companies are successful in attaining reimbursement at the stage of efficacy studies. With a 7 year cap on the conditional marketing licence RM companies will have time to complete efficacy/ phase 1/2 studies. Data for these studies must include Japanese patients but not exclusively as long as the product quality profile can be demonstrated to be stable between sites around the world.
The criteria for successful efficacy studies remains flexible enough to accommodate the complexities of proving novel therapeutics but how necessary will stricter guidelines be for predicting a successful application and investment?
Vishal Sharma for Total BioPharma at the World Stem Cells and Regenerative Medicine Congress.