The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers - 15/08/19
Abstract |
The Orphan Drug Act of 1983 (ODA) put in place a set of financial and marketing incentives to stimulate the development of drugs to treat rare diseases, and since its passage, more than 600 orphan drug and biologic products have been brought to market in the United States. Rapid growth in orphan drug approvals in conjunction with high orphan drug prices have triggered concern that drug makers are exploiting certain aspects of the ODA for financial gain and that some pharmaceutical drugs are receiving orphan status where it is not warranted. The landscape of approved therapies for rare skin diseases has not been well described. In this article, we provide a descriptive analysis of the United States Food and Drug Administration-approved orphan drugs for the treatment of rare dermatologic conditions and skin-related cancers since the enactment of the ODA. We discuss policy issues that emerge from the analysis and suggest areas for future research. Next, we elucidate ODA loopholes using dermatologic drugs as examples and propose potential reforms. Finally, we consider future directions for orphan drug development in the field of dermatology.
Le texte complet de cet article est disponible en PDF.Key words : drug approvals, Food and Drug Administration, orphan drugs, Orphan Drug Act, rare skin diseases, pharmaceutical drugs
Abbreviations used : FDA, HS, ODA, US
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Funding sources: This work was supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (K24-AR-069760 to Dr Asgari). |
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Conflicts of interest: Dr Asgari receives grant funding to her institution from Pfizer. Drs Karas, Lu, and Agrawal have no conflicts of interest to disclose. |
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Reprints not available from the authors. |
Vol 81 - N° 3
P. 867-877 - septembre 2019 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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