Rare diseases and orphan drugs in Japan: developing multiple strategies of regulation and research

Abstract

As far back as 1972, measures to combat rare diseases have been part of the Japanese national health system. Thanks to extensive support from the government, measures to combat rare diseases in Japan have made considerable progress over the past 40 years, including specific orphan drug legislation enacted in 1993 to encourage the development of and research on orphan drugs, pricing and reimbursement systems to facilitate access to orphan drugs, specific research programs to promote research on and development of orphan drugs, and a government-supported information centre to promote the understanding of rare diseases. Multiple strategies for regulation of orphan drugs and research on rare diseases have been adopted in Japan. Moreover, a new project to establish a national rare diseases database to collect a wide range of information on patients with rare diseases was launched in 2013 with government support. And in order to ensure quality data, designated doctors specialised in rare diseases will be responsible for directly sending data to the national rare disease database starting in 2015. Quality national data will lead to new opportunities to research and treat rare diseases and also encourage the discovery of orphan drugs in the near future.

Keywords::

• database
• drug discovery
• health system
• orphan drug legislation