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J Am Acad Dermatol. 2019 May 16. pii: S0190-9622(19)30792-3. doi: 10.1016/j.jaad.2019.05.025. [Epub ahead of print]

The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.

Author information

1
Department of Population Medicine, Harvard Medical School and the Harvard Pilgrim Health Care Institute, Boston, Massachusetts. Electronic address: laura.o.karas@gmail.com.
2
Department of Population Medicine, Harvard Medical School and the Harvard Pilgrim Health Care Institute, Boston, Massachusetts.
3
Divisions of Genetics & Genomics and Newborn Medicine, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School Boston, Massachusetts.
4
Department of Population Medicine, Harvard Medical School and the Harvard Pilgrim Health Care Institute, Boston, Massachusetts; Department of Dermatology, Massachusetts General Hospital, Boston, Massachusetts.

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.

KEYWORDS:

Food and Drug Administration; Orphan Drug Act; drug approvals; orphan drugs; pharmaceutical drugs; rare skin diseases

PMID:
31103566
DOI:
10.1016/j.jaad.2019.05.025

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