Because of the significant costs associated with stem cell production scale-up, one of the challenges of clinical development is the potentially crippling effect of late-stage failure and the inability to leverage some of that financial risk by accessing patient populations before the end of Phase 3 studies. There is some hope on the horizon, however, according to Gregory Bonfiglio, founder and managing partner of Proteus Regenerative Medicine, who spoke about recent changes in Japan’s regulations regarding regenerative medicine.
Under the new law, announced late last year by the Pharmaceuticals & Medical Devices Agency, if a cell therapy product can show safety and some degree of efficacy in early-phase trials, it can be given a conditional approval, which will allow the product to be brought to market and the company to generate revenues. These revenues can then be used to fund later stage trials that will (hopefully) support final approval and market authorization. A graphic explanation of the new paradigm is available here.
According to Darin Weber, executive vice president of global regulatory affairs at Mesoblast, the new legislation isn’t just about Japan but rather highlights a general convergence in global attitudes about the importance of cell therapies in addressing a growing list of unmet medical needs. At the same time, he doesn’t see the floodgates opening any time soon, but rather expects regulatory bodies to continue to be conservative in their approach to which new therapeutics will get through.