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

Nature Reviews Drug Discovery volume 17, pages 7980 (2018) | Download Citation

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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References

  1. 1.

    , & Next-generation medicines: past regulatory experience and considerations for the future. Clin. Pharmacol. Ther. 95, 247–249 (2014).

  2. 2.

    , & Patient-centric trials for therapeutic development in precision oncology. Nature 526, 361–370 (2015).

  3. 3.

    et al. The FDA's experience with ivacaftor in cystic fibrosis: establishing efficacy using in vitro data in lieu of a clinical trial. Ann. Am. Thorac. Soc. (2017).

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Author information

Affiliations

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

    • Robert N. Schuck
    • , Michael Pacanowski
    •  & Issam Zineh
  2. Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993, USA.

    • Janet Woodcock

Authors

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Competing interests

The authors declare no competing financial interests.

Corresponding author

Correspondence to Issam Zineh.

About this article

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DOI

https://doi.org/10.1038/nrd.2017.231

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