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The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?

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  • Wellman-Labadie, Olivier
  • Zhou, Youwen

Abstract

Objectives This study investigates issues associated with the United States Orphan Drug Act.Methods A comprehensive orphan drug database was compiled from FDA data and corporate annual reports of major pharmaceutical companies. Analysis allowed the generation of a descriptive orphan drug portrait as well as documentation of orphan drugs along their lifecycle.Results Currently, 2002 products have obtained orphan drug designation with 352 drugs obtaining FDA approval. Approximately 33% of orphan drugs are oncology products. On average, products obtain 1.7 orphan designations with approximately 70% obtaining a single designation. At least 9% of orphan drugs have reached blockbuster status with two-thirds having two or more designations. An additional 25 orphan drugs had sales exceeding US$ 100 million in 2008 alone. Since 1983, at least 14 previously discontinued products have been recycled as orphan drugs.Conclusions The United States Orphan Drug Act has created issues which, in some cases, have led to commercial and ethical abuses. Orphan Drug Act reform is necessary but current incentives, including 7 year market exclusivity, should be maintained in order to favour patients as well as economic prosperity. Suggested reforms include price regulation, subsidy paybacks for profitable drugs and the establishment of an International Orphan Drug Office.

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  • Wellman-Labadie, Olivier & Zhou, Youwen, 2010. "The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?," Health Policy, Elsevier, vol. 95(2-3), pages 216-228, May.
    • Handle: RePEc:eee:hepoli:v:95:y:2010:i:2-3:p:216-228
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    References listed on IDEAS

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    1. Rosenberg-Yunger, Zahava R.S. & Daar, Abdallah S. & Singer, Peter A. & Martin, Douglas K., 2008. "Healthcare sustainability and the challenges of innovation to biopharmaceuticals in Canada," Health Policy, Elsevier, vol. 87(3), pages 359-368, September.
    2. Yin, Wesley, 2008. "Market incentives and pharmaceutical innovation," Journal of Health Economics, Elsevier, vol. 27(4), pages 1060-1077, July.
    3. Joseph Golec & John Vernon, 2009. "Financial risk of the Biotech Industry versus the Pharmaceutical Industry," Applied Health Economics and Health Policy, Springer, vol. 7(3), pages 155-165, September.
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    1. Dyfrig A Hughes & Jannine Poletti-Hughes, 2016. "Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study," PLOS ONE, Public Library of Science, vol. 11(10), pages 1-12, October.
    2. Marialuisa Saviano & Sergio Barile & Francesco Caputo & Mattia Lettieri & Stefania Zanda, 2019. "From Rare to Neglected Diseases: A Sustainable and Inclusive Healthcare Perspective for Reframing the Orphan Drugs Issue," Sustainability, MDPI, vol. 11(5), pages 1-21, March.
    3. Aaron S Kesselheim & Jessica A Myers & Daniel H Solomon & Wolfgang C Winkelmayer & Raisa Levin & Jerry Avorn, 2012. "The Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugs," PLOS ONE, Public Library of Science, vol. 7(2), pages 1-7, February.
    4. Petra Maresova & Blanka Klimova & Kamil Kuca, 2016. "Financial and legislative aspects of drug development of orphan diseases on the European market -- a systematic review," Applied Economics, Taylor & Francis Journals, vol. 48(27), pages 2562-2570, June.
    5. Todd Gammie & Christine Y Lu & Zaheer Ud-Din Babar, 2015. "Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries," PLOS ONE, Public Library of Science, vol. 10(10), pages 1-24, October.
    6. Salas-Vega, Sebastian & Shearer, Emily & Mossialos, Elias, 2020. "Relationship between costs and clinical benefits of new cancer medicines in Australia, France, the UK, and the US," Social Science & Medicine, Elsevier, vol. 258(C).
    7. Picavet, Eline & Cassiman, David & Simoens, Steven, 2012. "Evaluating and improving orphan drug regulations in Europe: A Delphi policy study," Health Policy, Elsevier, vol. 108(1), pages 1-9.

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