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An appraisal of FDA approvals for adult solid tumours in 2017–2021: has the eagle landed?

Abstract

In 2016, the then US President Barack Obama announced the Cancer Moonshot with a view to making 10 years’ worth of progress in cancer prevention, diagnosis and treatment in only 5 years. This Perspective evaluates the FDA approvals of therapeutic agents for use in solid tumour oncology for the period 2017–2021 against the aspirations of the Cancer Moonshot. In the past 5 years, the FDA issued an unprecedented 161 new approvals of therapeutic agents for various indications in adult patients with solid tumours. However, less than a third (27%) of the newly approved medicines are supported by unequivocal evidence of an overall survival benefit; most are supported by positive signals from surrogate end points. Herein, the European Society for Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 was used to evaluate the clinical value of the therapies granted FDA approval during the period 2017–2021. The results of this appraisal indicate a low level of clinical benefit for a substantial proportion (~20%) of the new indications, with most (~44%) providing intermediate benefit. The data suggest that, beyond increases in the sheer quantity of approvals, considerable improvement in the quality of the approved treatments is required to more confidently ensure that the clinical benefits are real and substantial enough to clearly justify the risks to patients.

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Fig. 1: Overview of FDA approvals granted in 2017–2021 of therapies for use in adults with solid tumours.
Fig. 2: Trends in the FDA-approval pathways and disease settings of treatments indicated for use in adults with solid tumours based on approvals granted during the period 2017–2021.
Fig. 3: Immuno-oncology agents approved by the FDA in 2017–2021 for the treatment of solid tumours in adults.
Fig. 4: ESMO-MCBS v1.1 grade distribution of immuno-oncology agents approved by the FDA in 2017–2021 for the treatment of solid tumours in adults.
Fig. 5: Trends in the primary end points supporting FDA approvals granted during the period 2017–2021 of therapeutic agents for use in adults with solid tumours.
Fig. 6: Temporal trends in the ESMO-MCBS v1.1 grade distribution of FDA approvals granted during the period 2017–2021 of agents for the treatment of advanced-stage solid tumours in adults.

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Acknowledgements

I thank the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) administrative coordinators N. Latino and M. Galotti for general support and for drafting the figures, and B. Gyawali, E. de Vries, H. Dressler, H. Goldwasser and A. Gabizon for providing feedback on early drafts of this manuscript. I am also grateful for general support from the Shaare Zedek Medical Center, ESMO, and my tolerant and supportive family. I am solely responsible for any errors in the information presented herein.

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Correspondence to Nathan I. Cherny.

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N.I.C. is Chair of ESMO-MCBS working group. The author declares no financial competing interests.

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Nature Reviews Clinical Oncology thanks C. Booth, C. A. Uyl-de Groot and the other, anonymous, reviewer(s) for their contribution to the peer review of this work.

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Cherny, N.I. An appraisal of FDA approvals for adult solid tumours in 2017–2021: has the eagle landed?. Nat Rev Clin Oncol 19, 486–492 (2022). https://doi.org/10.1038/s41571-022-00636-y

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