Intractable & Rare Diseases Research
Online ISSN : 2186-361X
Print ISSN : 2186-3644
ISSN-L : 2186-3644
New opportunity for orphan drug development in Japan: Early exploratory clinical trial bases promote drug translation from basic studies to clinical application
Peipei SongJianjun GaoNorihiro KokudoWei Tang
Author information

2012 Volume 1 Issue 2 Pages 95-97


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.

Information related to the author
© 2012 International Research and Cooperation Association for Bio & Socio-Sciences Advancement
Previous article