Regulatory policy for the development of targeted therapies for low-frequency molecular subtypes of disease

Nature Reviews Drug Discovery volume 17, pages79–80(2018)Cite this article

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Numerous challenges arise when developing targeted therapies for diseases comprising low-frequency molecular subtypes. In this article, we describe a pragmatic, science-based regulatory policy for the development and approval of targeted therapies in such cases.

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Affiliations

  1. Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, 20993, Maryland, USA

    Robert N. Schuck, Michael Pacanowski & Issam Zineh

  2. Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, 20993, Maryland, USA

    Janet Woodcock

Authors
  1. Robert N. Schuck
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  2. Michael Pacanowski
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  3. Janet Woodcock
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  4. Issam Zineh
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Corresponding author

Correspondence to Issam Zineh.

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The authors declare no competing financial interests.

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US Food and Drug Administration. Paving the way for personalized medicine

US Food and Drug Administration. Guidances (Drugs)

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Schuck, R., Pacanowski, M., Woodcock, J. et al. Regulatory policy for the development of targeted therapies for low-frequency molecular subtypes of disease. Nat Rev Drug Discov 17, 79–80 (2018). https://doi.org/10.1038/nrd.2017.231

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