Development of Orphan Drugs in Japan: Effects of a Support System for Development of Orphan Drugs in Japan *

In order to promote development of orphan drugs, the Japanese Ministry of Health and Welfare introduced support measures for drug development in 1985 and 1993. To see if these support measures were really effective for drug development, we compared the number of orphan drugs and review periods required for their approval in three different periods. The number of approved drugs in a year of Term 3 (after 1993) was approximately 3.3 times that of Term 1 (before 1985). The median value of the review period required for approval of drugs in Term 3 was 15.5 months, shorter than that in Term 1 by 10.5 months. It was clearly shown that the number of drugs developed was increased and the review period was significantly reduced because of the support measures. Now it is possible for patients suffering from orphan diseases to get access to new and more effective drugs sooner.