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

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.

[1]  N. Dahiya,et al.  The Evolving Drug Development Landscape: From Blockbusters to Niche Busters in the Orphan Drug Space , 2014, Drug development research.

[2]  Cheng Zhu,et al.  Drug repositioning for orphan diseases , 2011, Briefings Bioinform..

[3]  D. Swinney,et al.  The discovery of medicines for rare diseases. , 2014, Future medicinal chemistry.

[4]  M. Kinch,et al.  An analysis of FDA-approved drugs for oncology. , 2014, Drug discovery today.

[5]  Jyoti Tiwari.  Navigating through orphan medicinal product regulations in EU and US--similarities and differences. , 2015, Regulatory toxicology and pharmacology : RTP.

[6]  Jianjun Gao,et al.  Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives. , 2012, Intractable & rare diseases research.

[7]  J. Krischer,et al.  Research into rare diseases of childhood. , 2014, JAMA.

[8]  Martin Možina,et al.  European regulation on orphan medicinal products: 10 years of experience and future perspectives. , 2011, Nature reviews. Drug discovery.

[9]  Pedro Franco,et al.  Orphan drugs: the regulatory environment. , 2013, Drug discovery today.

[10]  F. Augustovski,et al.  The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America , 2015, F1000Research.

[11]  New Financial and Research Models for Pediatric Orphan Drug Development: Focus on the NCATS TRND Program , 2014, Pharmaceutical Medicine.

[12]  Timothy R. Coté,et al.  Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years , 2010, Nature Reviews Drug Discovery.

[13]  H. Leufkens,et al.  Characteristics of orphan drug applications that fail to achieve marketing approval in the USA. , 2011, Drug discovery today.

[14]  M. Kinch,et al.  An analysis of FDA-approved drugs for metabolic diseases. , 2015, Drug discovery today.