FIRM looks to fix blips in Japan’s fast-track regenerative medicine system
Regenerative medicine (RM) has been a big focus for the Japanese government. The country introduced the revised pharmaceutical affairs law and new regenerative medicines legislation in November 2014, meaning such products could be reviewed and approved in just two years, if deemed to be effective.
But because the field is so new, trials are more complex, making it difficult to progress the RM industry. And, following significant government investment, industry advocacy group the Forum for Innovative Regenerative Medicine (FIRM) is looking to step in to help tackle the on-going problems.
Given many novel cell therapies are autologous, statistically measuring the efficacy of product is challenging, as it is highly dependent on the individual. Therefore recruitment for these trials is difficult.
Speaking at the Life Sciences Baltics conference 2016 in Vilnius, Lithuania, Fujifilm Corporation’s Takuya Yokokawa who head up a steering committee at FIRM said the group along with Japan’s Pharmaceutical and Medical Devices Agency (PMDA) has “to think outside the box” to deal with the issues and high costs of running RM therapy and control studies.
The idea is to improve industry communication; to help those running trials combat the difficulty of outsourcing for resources and talent, as well as help to define liability categories within RM.
FIRM has already drawn on over 190 companies and institutes across different industries in a global-collaboration project so far, and in the last year also forged a significant partnership with America’s Alliance for Regenerative Medicine (ARM).
And on the manufacturing side, Yokokawa said FIRM is specifically looking to help develop new frameworks able to cope with GMP and QMS (to enable CGTP), to move towards standardisation.
For example, through better guidelines for automated cell culture equipment and cell transportation between sites.
‘Early approval’ system
FIRM was involved in creating the conditional ‘early approval system’ for RM therapies in Japan, aimed at speeding up the arrival of cell and gene therapies to the patient.
The new framework means RM products can enter smaller Phase I/II clinical trials whilst at the same time getting conditional marketing and sales approval of the therapy for a period of up to seven years. During this time additional efficacy data is still collected – essentially bypassing Phase III for approval.
However, two companies - Terumo and JCR Pharmaceuticals - have already started the ball rolling, with their products having obtained conditional early marketing approval in September and November 2015, respectively.
JCR Pharmaceuticals and Mesoblast’s allogenic stem cell product, TemCell, was upgraded to full marketing approval following significant results from a smaller trial.