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
Pacanowski, M. A., Leptak, C. & Zineh, I. Next-generation medicines: past regulatory experience and considerations for the future. Clin. Pharmacol. Ther. 95, 247–249 (2014).
Biankin, A. V., Piantadosi, S. & Hollingsworth, S. J. Patient-centric trials for therapeutic development in precision oncology. Nature 526, 361–370 (2015).
Durmowicz, A. G. 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. https://dx.doi.org/10.1513/AnnalsATS.201708-668PS (2017).
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US Food and Drug Administration. Paving the way for personalized medicine
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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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DOI: https://doi.org/10.1038/nrd.2017.231