On the 20th Anniversary of Promotion for the Development of Orphan Drugs and Orphan Medical Devices in Japan : Results of the Last 10 Years and Future Challenges

Abstract

The Japanese Orphan Drug Development Support Program was established in 1993. During the 20 years after its establishment, the Ministry of Health, Labour and Welfare (MHLW) has designated 300 medicines and 23 medical devices as orphan. Of these, 192 medicines and 13 medical devices have been approved. Over the last 10 years from April 2003 to March 2013, 97 medicines were approved including 27 medicines for rare diseases, 17 anticancer therapeutic medicines, 13 medicines for HIV/AIDS and 10 medicines to meet multiple medical needs. The appreciable outcomes of these medicines are as follows. Tocilizumab (genetic recombination) is a humanized anti-human interleukin-6 receptor monoclonal antibody of the IGG1 subclass. It was discovered by Osaka University and Chugai Pharmaceutical Co., Ltd., and was approved for improvement of various symptoms associated with Castleman’s disease in 2005. Pirfenidone was approved for idiopathic pulmonary fibrosis in 2008. Mogamulizumab (genetic recombination), a humanized anti-CCR4 monoclonal antibody, was approved for relapsed or refractory CCR4-positive adult T-cell leukemia/lymphoma in 2012. The above three medicines first commenced in Japan. Pegaptanib sodium was approved for subfoveal choroidal neovascularization secondary to age-related muscular degeneration in 2008. Four alum-adjuvanted influenza (H5 N1) prepandemic vaccines were approved for avian influenza pandemic preparedness in 2007, 2010 and 2013. Regarding medical devices, nine medical devices were approved. For example, an implantable ventricular assist device was approved in 2010. It is used to improve the blood circulation until cardiac transplantation is performed in patients who have severe heart failure for which cardiac transplantation is indicated. Since rare diseases are a global issue, working with international partners and sharing information on orphan drugs have been recognized as issues. This includes organizations such as the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA)． The network involving patients and members of the rare diseases community has been improved. For example, the Cancer Philosophy Clinic was set up as a non-profit organization in 2011. Recently, 31 sites have been opened to care for terminally ill cancer patients. Finally, future proposals for the next decade are also outlined.