Abstract
Changes in regulatory policy and legislative incentives to promote the development of drugs for rare diseases — orphan drugs — have led to increases in the number of orphan drug designations, but the rate of such products reaching the market remains frustratingly flat. This article highlights areas in which novel approaches could facilitate regulatory approval and access to treatments for rare diseases.
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References
Braun, M. M. et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nature Rev. Drug Discov. 9, 519–522 (2010).
The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nature Rev. Drug Discov. 10, 341–349 (2011).
Acknowledgements
The author would like to acknowledge the GlaxoSmithKline Rare Diseases team who assisted with the preparation of the manuscript and provided helpful comments. Medical writing support was provided by S. Turrell and R. Sherman of Fishawack Communications.
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Marc Dunoyer is an employee of GlaxoSmithKline.
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Dunoyer, M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov 10, 475–476 (2011). https://doi.org/10.1038/nrd3493
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DOI: https://doi.org/10.1038/nrd3493
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