Approvals in 2018: a histology-agnostic new molecular entity, novel end points and real-time review

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In 2018, the FDA approved 19 new drug and biologic applications, 38 supplemental drug and biologic applications and 4 biosimilar applications in oncology. These advances in anticancer therapy included a landmark approval of the first histology-agnostic, biomarker-defined new molecular entity and approvals based on real-time data review and novel end points, such as minimal residual disease rate and metastasis-free survival.

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Fig. 1: FDA haematology–oncology (cancer) drug approvals in 2018.


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    Drilon, A. et al. Efficacy of larotrectinib in TRK fusion-positive cancers in adults and children. N. Engl. J. Med. 378, 731–739 (2018).

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    US Food and Drug Administration. Expansion cohorts: use in first-in-human clinical trials to expedite development of oncology drugs and biologics. FDA (2018).

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    Beaver, J. A., Kluetz, P. F. & Pazdur, R. Metastasis-free survival — a new end point in prostate cancer trials. N. Engl. J. Med. 378, 2458–2460 (2018).

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    US Food & Drug Administration. Nonmetastatic, castration-resistant prostate cancer: considerations for metastasis-free survival endpoint in clinical trials. FDA (2018).

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    US Food & Drug Administration. Clinical trial endpoints for the approval of cancer drugs and biologics. FDA (2018).

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    US Food & Drug Administration. Hematologic malignancies: regulatory considerations for use of minimal residual disease in development of drug and biological products for treatment. FDA (2018).

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    US Food & Drug Administration. FDA alerts health care professionals and oncology clinical investigators about an efficacy issue identified in clinical trials for some patients taking Keytruda (pembrolizumab) or Tecentriq (atezolizumab) as monotherapy to treat urothelial cancer with low expression of PD-L1. FDA (2018).

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    Gormley, N. J. & Pazdur, R. Immunotherapy combinations in multiple myeloma — known unknowns. N. Engl. J. Med. 379, 1791–1795 (2018).

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    Blumenthal, G. M. et al. Real-time oncology review and the assessment aid: increasing review efficiency through standardization and earlier data access. Friends of Cancer Research (2018).

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The authors thank K. Goldberg for her assistance in editing this manuscript.

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Correspondence to Gideon M. Blumenthal.

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The authors declare no competing interests.

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Blumenthal, G.M., Pazdur, R. Approvals in 2018: a histology-agnostic new molecular entity, novel end points and real-time review. Nat Rev Clin Oncol 16, 139–141 (2019) doi:10.1038/s41571-019-0170-z

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