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

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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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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