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

Article metrics

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.

Access options

Rent or Buy article

Get time limited or full article access on ReadCube.

from$8.99

All prices are NET prices.

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

References

  1. 1.

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

  2. 2.

    US Food and Drug Administration. Expansion cohorts: use in first-in-human clinical trials to expedite development of oncology drugs and biologics. FDA https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM616325.pdf (2018).

  3. 3.

    US Food and Drug Administration. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics. FDA https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM621817.pdf (2018).

  4. 4.

    Blumenthal, G. M. & Pazdur, R. Approvals in 2017: gene therapies and site-agnostic indications. Nat. Rev. Clin. Oncol. 15, 127–128 (2018).

  5. 5.

    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).

  6. 6.

    US Food & Drug Administration. Nonmetastatic, castration-resistant prostate cancer: considerations for metastasis-free survival endpoint in clinical trials. FDA https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM625703.pdf (2018).

  7. 7.

    US Food & Drug Administration. Clinical trial endpoints for the approval of cancer drugs and biologics. FDA https://www.fda.gov/downloads/drugsGuidanceComplianceRegulatoyInformation/Guidance/UCM071590.pdf (2018).

  8. 8.

    US Food & Drug Administration. Hematologic malignancies: regulatory considerations for use of minimal residual disease in development of drug and biological products for treatment. FDA https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM623333.pdf (2018).

  9. 9.

    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 https://www.fda.gov/Drugs/DrugSafety/ucm608075.htm (2018).

  10. 10.

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

  11. 11.

    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 https://www.focr.org/sites/default/files/ROTR%20White%20Paper%201.pdf (2018).

  12. 12.

    Sutter, S. Project renewal: US FDA’s plan to update generic cancer drug labels depends on NDA holders. Pink Sheet Pharma intelligence https://pink.pharmaintelligence.informa.com/PS124262/Project-Renewal-US-FDAs-Plan-To-Update-Generic-Cancer-Drug-Labels-Depends-On-NDA-Holders (2018).

  13. 13.

    Usdin, S. FDA to facilitate access to unapproved drugs. BioCentury https://www.biocentury.com/biocentury/regulation/2018-12-14/how-fda-plans-help-patients-get-expanded-access-unapproved-drugs (2018).

Download references

Acknowledgements

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

Author information

Correspondence to Gideon M. Blumenthal.

Ethics declarations

Competing interests

The authors declare no competing interests.

Supplementary information

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

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

Download citation

Further reading