Intractable Rare Dis Res. 2012;1(2):95-97. (DOI: 10.5582/irdr.2012.v1.2.95)

New opportunity for orphan drug development in Japan: Early exploratory clinical trial bases promote drug translation from basic studies to clinical application.

Song PP, Gao JJ, Kokudo N, Tang W


In Japan, although orphan drug legislation has been established in 1993 to encourage drug research and development (R&D) for intractable and rare diseases, nearly half of the orphan drugs in the Japanese market originated from the European Union (EU) or the United States of America (USA). Availability of orphan drugs for intractable and rare diseases is compounded by the "drug lag" phenomenon, which is mainly caused by the imperfect clinical trial environment in Japan. In recent years, the Japanese government paid great attention to development of innovative drugs and medical devices which originated from Japan. With financial support and institutional guarantees from government, the project of "Early Exploratory Clinical Trial Bases for Specific Research Areas" was launched in 2011 and 5 institutions were selected as the national early exploratory clinical trial bases for specific research areas including cancer, cerebral and cardiovascular diseases, neuropsychiatric disorders, and immunological intractable diseases. The early exploratory clinical trial bases offer a new opportunity for drug development for immunological and neuropsychiatric intractable diseases, thereby promoting orphan drug translation from basic studies to clinical use.

KEYWORDS: Intractable and rare diseases, orphan drugs, clinical trial, autoimmune disease, neuropsychiatric disorders

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