Matched analysis on orphan drug designations and approvals: cross regional analysis in the United States, the European Union, and Japan

• Analysis on the ‘entire’ orphan drug designations in the US, EU and Japan.
• Analysis on the ‘cross-regionally common’ designations among the US, EU and Japan.
• Regional differences in designation lag, drug type, applicant type and regulations.
• Continued increase in total annual designations, and similar trend in new modalities.
• Orphan drug designation still bears a crucial role in drug development.

Orphan drugs have become a key area of focus in drug development for resolving unmet medical needs. The Orphan Drug Act in the USA and similar legislation in Japan, the European Union (EU), and several other countries has been enacted since 1983. This study provides a quantitative review of all orphan drug designations and approvals since the implementation of orphan drug legislation in key three regions. This study also identified and reviewed ‘commonly designated’ drugs across regions. Out of approximately 5000 designations, approximately 800 designations were common among the USA, EU, and/or Japan. Regional similarities, differences, and trends were identified. It is important to understand these aspects and the crucial role of orphan drug designation in global drug development.