Orphan Drugs in Japan

In order to promote the development of orphan drugs, the Pharmaceutical Affairs Law and the Law Concerning the Drug Fund for ADR Relief and R&D Promotion were partially amended in April 1993 and a new system was established on October 1, 1993. In the new system, drug companies which wish to develop drugs for the treatment of rare and severe diseases are encouraged to apply to the Ministry of Health and Welfare (MHW) for orphan drug status. Once a drug is designated an orphan drug by MHW, the following preferential measures are given: 1. grants for research and development (R&D) expenses, 2. tax reduction on R&D costs, 3. guidance and advice on the development of the drug, 4. acceleration of new drug application (NDA) review, and 5. extension of the term for reexamination to 10 years.

The Organization for Drug ADR Relief, R&D Promotion and Product Review (Hereinafter referred to as “drug organization”) works closely with MHW and carries out some of these measures. MHW has designated 78 drugs orphan drugs since 1993. The drug organization made grants totaling 400 million Japanese yen in fiscal 1994.