Abstract
The 1983 US Orphan Drug Act has stimulated the development of new therapies for rare diseases. To provide the first comprehensive overview of orphan-designated products and their indications, this article quantitatively analyses the characteristics and distribution of orphan designations and approvals by the US Food and Drug Administration from 1983 to August 2008. Of the 1,892 orphan-designated products, 326 received marketing approval, representing 247 different drugs and more than 200 different diseases. About half of the approvals had occurred by 4 years after designation was granted. The most common patient population size for orphan designations and approvals was fewer than 10,000 patients, and cancer was the most common disease area. The implications of such findings for future development and marketing of therapies for rare diseases are discussed.
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References
Orphan Drug Act, H.R. 5238, Public Law No. 97–414, 97th Congress (1983).
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Acknowledgements
We thank G. Gupta for statistical consultation, J. Fritsch and H. Startzman for providing useful background on orphan designations at the FDA, K. Robertson for provision of FDA data abstracts and A. Kelkar for the data and analysis on NMEs.
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At the time the work on this project was performed, all authors were FDA employees or fellows. Subsequently, M. Miles Braun was employed by MedImmune, LLC and Sanofi Pasteur.
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Braun, M., Farag-El-Massah, S., Xu, K. et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov 9, 519–522 (2010). https://doi.org/10.1038/nrd3160
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DOI: https://doi.org/10.1038/nrd3160
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