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Accelerating orphan drug development

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Abstract

Interest in developing drugs for rare diseases has increased substantially in recent years. This article from the US Food and Drug Administration highlights the role of regulators in catalysing further progress in this field.

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References

  1. 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).

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  8. Institute of Medicine of the National Academies. Rare Diseases and Orphan Products: Accelerating Research and Development (eds Field, M. J. and Boat, T. F.) (The National Academies Press, Washington, DC, 2010).

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Authors and Affiliations

  1. Timothy R. Coté, M.D., M.P.H., and Kui Xu, M.D., Ph.D., are at the Office of the Commissioner, Office of Orphan Products Development, Silver Spring, Maryland 20993, USA.,

    Timothy R. Coté & Kui Xu

  2. Anne R. Pariser, M.D., is at the Center for Drug Evaluation and Research, Office of New Drugs, US Food and Drug Administration, Silver Spring, Maryland 20993, USA.,

    Anne R. Pariser

Authors
  1. Timothy R. Coté
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  2. Kui Xu
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  3. Anne R. Pariser
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Corresponding author

Correspondence to Timothy R. Coté.

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The authors declare no competing financial interests.

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Coté, T., Xu, K. & Pariser, A. Accelerating orphan drug development. Nat Rev Drug Discov 9, 901–902 (2010). https://doi.org/10.1038/nrd3340

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