LONDON – An expedited approval system for regenerative medicines coming into effect in Japan in November, will provide a possible route for cell therapy companies from around the world to get conditional approval and reimbursement on the basis of phase I/II data confirming safety and indicating a product is likely to be effective.

The conditional approvals will run for seven years, during which time postmarket monitoring must be carried out. In parallel, companies will be expected to conduct phase IIb/III trials and to submit a dossier for full approval.

The UK Bioindustry Association (BIA) has been exploring ways in which British companies could take advantage of the new law to get early revenues in Japan, while continuing formal trials to support full marketing approvals in both Europe and Japan.

Chris Mason, director of the Regenerative Medicines Bioprocessing Unit at University College London and chair of the BIA's Regenerative Medicines Committee, has visited Japan with some BIA member companies to talk to regulators there, and has come up with a proposed scheme for using the best of the new Japanese system, and existing UK and European regulations, to optimize and speed up development of cell therapies.

"There are good synergies and a willingness to partner," Mason said. On the back of the trip to Japan, two UK companies are "progressing dialogues" to use the new legislation, he told BioWorld Today on the sidelines of the World Stem Cells and Regenerative Medicine Congress 2014 in London last week.

The creation of the expedited approvals route was inspired by the award in 2012 of the Nobel prize to Shinya Yamanaka for his discovery of induced pluripotent stem cells. This provided the momentum for Japan's Regenerative Medicines Promotion law, which it is hoped will enable the country to capitalize on Yamanaka's breakthrough.

The Nobel was awarded jointly with British scientist John Gurdon, who laid the foundation stone of stem cell therapy back in 1962, in his discovery that the specialization of cells is reversible.

"Japan has seized a moment of national pride," said Steve Bates, CEO of the BIA. "Winning the Nobel prize changed the game in Japan, and should in the UK, too," he told BioWorld Today.

Under Mason's proposed scheme, preclinical studies would be carried out in the UK, but with agreement from the Japanese regulator, the Ministry of Health, Labor and Welfare, that adequate data would be generated. Similarly, phase I/II studies would be carried out in the UK. Given positive results, companies could then apply for conditional approval in Japan.

Getting reimbursement would ensure funding for phase IIb/III studies and provide validation for investors and potential licensees.

Bates noted that in addition, the registry of patients treated during the seven-year conditional approval would generate important information for health technology assessment bodies. The information in the Japanese registries could also be used in support of the application to carry out a phase IIb/III trial in the UK, with the resulting data supporting full marketing applications in both the UK and Japan.

Mason said this UK/Japan development strategy would likewise work for Japanese companies, noting the conditional approval system will make it difficult to carry out the double-blind, controlled trials required to get full approval.

"Are patients [in Japan] really going to want to enter a clinical trial where they may receive placebo, if the product already has conditional approval?" he said.

Putting this into practice will require high level cooperation between the UK and Japanese regulators, but Bates said he believes there is the political will and support for the scheme.

There remain some question marks, most notably confirmation that the data supporting conditional approval need not be generated in Japan, and that UK-generated data would be accepted by Japanese regulators. In addition, discussions are ongoing about reimbursement under the expedited approvals system.

Mason has been sharing his thoughts on how cell therapy companies might best benefit from Japan's new Regenerative Medicine Regulation widely, in order to promote a dialogue. "Companies in the U.S. are now thinking Japan is the second market; putting Europe on hold," he said. Meanwhile, Japan would become the number two market, rather than the U.S., for UK regenerative medicine companies.