Gene therapy is permitted and approval can be fast-tracked through an accelerated system that is unique to Japan. After short, small clinical trials for safety and effectiveness, regenerative medicines (therapies that repair tissues and organs, including gene therapy) can be approved on a conditional basis that allows for the marketing, sale, and use of the treatment. Additional data collection is required for seven years, after which researchers can apply for final marketing approval.
Some scientists have voiced concerns that the approval system risks marketing unsafe or ineffective drugs and that it is unclear how evidence of safety and effectiveness will be collected after treatments are on the market.
Gene and stem cell therapies are regulated by two agencies. The Ministry of Health, Labor and Welfare (MHLW) is responsible for implementing safety standards for drugs. The Pharmaceuticals and Medical Devices Agency (PMDA) is an independent agency that works with MHLW to review drug applications and monitor post-market safety. PMDA functions under the Pharmaceutical and Medical Devices Act, adopted in 2014. The Act introduces conditional approval of treatments that have gone through minimal clinical testing, which allows for the immediate marketing and use of the treatment. Companies must continue to collect data on the treatment’s effectiveness for seven years in order for the treatment to receive final marketing approval.
- Stem cell treatment for paralysis: Sapporo Medical University received accelerated approval for the sale of a stem cell treatment for spinal cord injuries, called Stemirac, the first approval worldwide for a therapy for these types of injuries. Some scientists voiced concerns that there is insufficient evidence that the therapy works and is safe.
- Stem cell treatment for lack of blood to limbs: Sapporo Medical University used blood-forming stem cells to treat critical limb ischaemia, a condition in which a limb does not receive enough blood due to a blockage in an artery.
- Cornea repair: Osaka University used reprogrammed stem cells to repair the cornea to treat a patient’s vision loss.
- Human organs in animals: University of Tokyo received approval to grow human organs in rats and mice. The research is in early stages, but the goal is to develop human pancreases that can be transplanted into humans.
2017: Conditional Prompt Marketing Approval Designation, which allows developers to gain conditional marketing approval with minimal clinical trial requirements if enough patients with a rare disorder cannot be recruited, is introduced.
2015: The Guidelines of Clinical Research Regarding Gene Therapy, which regulates clinical research on gene therapy, passes.
2014: Japanese government adopts accelerated approval system for regenerative medicines, including gene therapy and stem cell treatments. The Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act (PMD Act) introduces conditional approval, which requires only minimal safety and efficacy data. The Act on the Safety of Regenerative Medicine accelerates the clinical application and commercialization of regenerative medicine.
2013: Regenerative Medicine Promotion Act passes in response to the discovery of induced pluripotent stem (iPS) cells (adult stem cells that can develop into any type of cell) by Shinya Yamanaka in 2006.
2006: Guideline for Human Stem Cell Clinical Research is published.