A Comparative Study on the Orphan Drug Policies

Abstract

This study investigated orphan drug policies of Korea, US, EU, and Japan and compared them. We focused on orphan drug designation, R&D support program, marketing authorization, and market exclusivity. The US, EU, and Japanese governments have provided various incentives for orphan drug development including research grants, tax credits for development costs, protocol assistances, and market exclusivity. Those countries designate products as orphan drugs eligible for the supporting programs during the R&D phase. In Korea, the primary purpose of orphan drug policy has been accelerating the provision of orphan drugs most of which are imported drugs developed in other countries. Therefore, designation of orphan drug has been only possible at the marketing approval phase and there have been few R&D support programs. In addition, the regulatory authority has partly skipped evaluation of efficacy and safety. These days, however, there has been much progress in new drug research capacity and increasing number of ongoing projects for orphan drug development in Korea. Considering these changes, orphan drug policy of Korea needs to shift to accelerating drug development and ensuring safety and efficacy through the regulatory process.