Announcement of the Final Report Compiled by the Study Group on Commercialization and Industrialization of Regenerative Medicine

It is highly expected around the world for regenerative medicine to pave the way to fundamental treatment of diseases considered to be incurable by existing methods, such as operations and medications. In the future, it will also be able to contribute to suppressing the cost of social security by curing chronic diseases or diseases accompanying aging.

Nevertheless, Japan's efforts being made toward commercialization of the technology are slow, even though research activities in the field are at the world's top level, and it is a concern that this slowness is widening the gap between Japan and other countries such as Western societies.

To overcome such concern, it has become an urgent necessity to organize safety and other standards considering characteristics of regenerative medicine and to establish systematic frameworks to efficiently process cells or control cell quality, as well as to organize business environments in which a business model is able to be established.

1. In regenerative medicine conducted by doctors and medical organizations under the Medical Practitioners Act and the Medical Care Act, such doctors and medical organizations have to carry out all of the operations from extracting cells and tissues to processing, examining, and transplanting them, which results in ineffective and expensive treatments. For improving such a situation, it is necessary to organize a system under which such doctors and organizations are able to commission cell and tissue processing operations required for regenerative medicine to external businesses (cell-processing organizations). At the same time, establishment of security measures, such as authorization of such cell-processing organizations by a third party, is also required to secure adequacy of the organizations.
2. Although regenerative medicine products are to be sold after being approved under the Pharmaceutical Affairs Act, it would not be appropriate to apply the existing examination and approval procedures for medicines without changing the procedures to such regenerative medicine products, considering the non-uniformity of raw materials mainly due to using patients' own cells, and other elements. To tackle such a challenge, it is required to introduce an early approval system on the presumption that such products' security is acquired and effectiveness is estimated, as well as to organize security and other standards considering the characteristics of the regenerative medicine.

Future market expectation of regenerative medicine (approximate figures)

Cost related to manufacturing and processing in regenerative medicine and effectiveness of cost reduction

1) Approval of manufacturing and selling regenerative medicine products

Some approaches will have a great effect on cost reduction, such as the shortening of clinical trial periods, establishment of an early approval system, reduction of burdens in the procedures of approval of partial changes, as well as flexible change and streamlining of manufacturing processes, and reduction of burdens imposed in the survey on commercialized products.

• For the cost of manufacturing regenerative medicine products, nearly 20 % of the total cost may be reduced.
  (For 500 cases examined per year, 3.6 million yen per product may be reduced to 2.9 million yen.)
  (For 1,000 cases examined per year, 2.3 million yen per product may be reduced to 1.8 million yen.)
• For the cost related to clinical trial, nearly 60 % of the total cost may be reduced.
  (1.23 billion yen for seven years may be reduced to 538 million yen for two years.)

2) Cell-processing organizations for regenerative medicine

If businesses are stably operated according to the increase in the number of commissioned cell-processing by utilizing automatic culturing devices and other methods, the possibility to provide cell products at more reasonable prices will increase.

• For 500 cases examined per year, cell-processing may cost 1.5 million to 2 million yen per product.

Expansion of industries related regenerative medicine

It is necessary to establish reasonable standards for quality and safety exclusively for peripheral devices for regenerative medicine that are used for clinical uses.

To achieve this goal, it is required to define requirements for such peripheral devices by streamlining industry standards by the Forum for Innovative Regenerative Medicine (FIRM). At the same time, standardization of such industry standards will be implemented, such as JIS/ISO, if necessary.

The following challenges are to be overcome. METI will continue to encourage related ministries and agencies to organize desirable systems and also to promote enterprises to enter the regenerative medicine field.

• Promoting understanding of characteristics of treatment by regenerative medicine
• Developing standards and security measures required for cell-processing organizations
• Rationalizing and increasing transparency of examination procedures for regenerative medicine
• Developing technology for commercializing regenerative medicine
• Encouraging efforts of FIRM as an economic organization to expand the market
• Studying risks and cost burdens that are suitable for the characteristics of regenerative medicine

• The first meeting (July 13, 2012): Confirming of the current situation surrounding regenerative medicine and extracting problems thereof
• The second meeting (September 21, 2012): Streamlining of subjects concerning systems related to regenerative medicine
• The third meeting (November 5, 2012): Compiling of the interim report
• The fourth meeting (February 22, 2013): Compiling of the final report

February 22, 2013

Bio-Industry Division, Manufacturing Industries Bureau