Measures to combat rare diseases and promote orphan drug development in Japan: government-funded special biomedical research programs to enhance basic and applied research

Introduction: As a measure to combat rare diseases and promote the development of orphan drugs, special research programs to strengthen basic and applied research on rare diseases have been well implemented in some countries, including Japan. Areas covered: This review reflects the current status of systems to research rare diseases in Japan. In particular, it focuses on the government-funded special biomedical research programs to strengthen basic and applied research, the 'Program to Research Treatments for Specified Diseases' and 'Centers to Conduct Early/Exploratory Clinical Trials in Specific Research Areas'. Expert opinion: Government-funded special biomedical research programs to enhance basic and applied research have been emphasized and are expected to play an even greater role in combating rare diseases in Japan in the future.