Daisaku Sato

Councilor for Pharmaceutical Affairs, Ministry of Health, Labor and Welfare

Sato began the exchange with an overview of the review-and-approval process for regenerative medical products. He showed in a slide how the 2014 revision of Japan’s Act on Pharmaceuticals and Medical Devices had occasioned a surge in the number of regenerative medicine product candidates in clinical trials. That revision had become necessary because the traditional approval process was unsuited to regenerative medicine and, as a result, required an inordinately long time for products in that sector.

The revision of the Act on Pharmaceuticals and Medical Devices (formally the Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices) included the introduction of a fast-track authorization process. Under the revised act, the Ministry of Health, Labor, and Welfare can authorize an applicant to manufacture and market a regenerative medicine product once clinical trials have verified its safety. That authorization dispenses with the former requirement for large-scale, follow-up clinical trials to double-check candidates’ efficacy and safety. It is for a fixed term and is subject to expert opinion from the Pharmaceutical Affairs and Food Sanitation Council.

Japan’s fast-tracking for regenerative medicine products has also included shortening the review that follows the completion of Phase III clinical trials and precedes the start of marketing. The target review time was 12 months before the revision of the Act on Pharmaceuticals and Medical Devices. It is now nine months for “orphan” products, which are for treating serious but rare health problems, and six months for pioneering products.

A total of 19 regenerative medical products have won approval to market in Japan — 17 since the revision of the Act on Pharmaceuticals and Medical Devices. Cell therapy products account for 16 of the 19, and gene therapy products for three. Sato acknowledged that Japan’s fast-tracking for cell and gene therapies has received criticism for subjecting patients to treatments of dubious clinical value. He argued, however, that the value of making potentially valuable treatments available sooner rather than later justified the initiative. Fostering Japan’s domestic capacity for developing regenerative medical products was part of the motivation for deregulating the approval process, and Sato noted that products of domestic origin accounted for 11 of the 19 approvals in that sector.

Hideyuki Okano

President, Japanese Society for Regenerative Medicine
Director, Keio University Regenerative Medicine Research Center
Chief Scientific Officer, K Pharma

Okano seconded Sato in regard to the value of Japan’s fast-tracking regenerative medical products. In addition to heading the Japanese Society for Regenerative Medicine, Okano is the director of the Keio University Regenerative Medicine Research Center and chief scientific officer at the Keio-based biotech venture K Pharma. He cited, meanwhile, three challenges that demand attention in pursuing further advances in regenerative medicine:

1. Fundamental progress in stem cell research and genomics
2. The application of genomic engineering technologies to tissue engineering
3. Research on fundamental technologies, such as mass cultivation techniques and cell separation equipment

“We need to do more,” emphasized Okano, “than just write academic papers about the products of our research. We also need to do more than achieve first-in-human clinical studies with a few subjects. We need to create mass production systems that allow for deploying our advances in hundreds of thousands or even millions of patients.”

Okano offered regenerative therapy as an example of a promising field for cultivating. Regenerative therapy is treatment for revitalizing cells, tissue, and organs that have become dysfunctional. It promises to address medical problems that are untreatable with conventional medicines. Okano cited the need for developing new vector systems for confirming the efficacy and safety of genetic technologies and then deploying those technologies in designer cells.