Abstract
In 1985, the MHW made an announcement by Division Director's Notice to simplify the data necessary for the application for approval of an orphan drug. In 1993, the Pharmaceutical Affairs Law was amended and the Orphan Drug Development Support Program was introduced. In this study, we compared the situation of orphan drug development among stage I (1980-1985), stage II (1985-1993) and stage III (1993-1998).
Thirty-four orphan drugs were approved during stage III (7.6 drugs/year) showing a 3.2- and 1.9-fold increase from stage I (2.4 drugs/year) and stage II (3.9 drugs/year), respectively. Orphan drugs are granted fast-track approval status. The median of the evaluation period (from application submission to approval) of approved drugs in stage III was 16 months and faster than the previous stage.
The Orphan Drug Development Support Program has encouraged orphan drug development. Orphan sponsors recognize that among several incentives under the support program's fast-track approval system, and consultation with and advice from the Drug Organization are most effective.