Comparative Study of Orphan Drug Designation and Incentive Policies in the United States, Japan, and Korea

The Korean orphan drug industry, constrained by low market viability and high developmental barriers, tends to rely considerably on imported drugs. The purpose of this study is to draw meaningful implications through a comparative analysis of the rare diseases, designation regulations and incentive systems for orphan drugs in the United States (U.S.), Japan, and Korea. Regulatory frameworks, incentive structures, and recent legal amendments were analyzed using data from each country’s regulatory agencies. Globally, over 10,000 rare diseases are estimated, with the number rising as science advances. Japan designates ‘Nanbyo’, and Korea designates ‘nationally managed rare diseases’, both offering government support for patients’ medical costs. By classifying and comparing the orphan drug designation regulations of U.S., Japan, and Korea according to patient population criterion, marketability, disease severity, and clinical superiority requirements, the greatest difference was found in marketability. All three countries provide incentives such as market exclusivity, fast-track approval systems, drug pricing and reimbursement, and financial support to encourage orphan drug development, though the specific measures vary by nation. In Korea, stronger institutional support and broader financial incentives are required to further advance orphan drug development.