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Exploring a new pathway for biomarker-based approval of immunotherapies

Nature Reviews Clinical Oncology (2023)Cite this article

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The revolution of immunotherapies in oncology has not been matched by the development of companion diagnostic biomarkers able to identify the ideal target populations. Aside from the established pathways for regulatory approval relying on predefined biomarkers, a novel approach based on post hoc biomarker analysis could potentially be instrumental in enhancing the cost-effectiveness of cancer immunotherapy.

The development of immunotherapies and molecularly targeted therapies has revolutionized oncology. Between 2017 and 2021, the FDA issued 161 approvals of therapeutic agents for new indications in adults with solid tumours (74% regular and 36% accelerated)1. During this period, ~85% of small-molecule therapies and ~30% of immunotherapies were approved for the treatment of specific molecular cancer subtypes identified using companion diagnostic biomarkers1. This huge step forward in precision oncology is partially attributable to the success of the FDA accelerated approval programme, which has provided patients with early access to promising treatments2. Nonetheless, some issues need to be considered to further realize the promise of precision oncology, particularly with the expansion of immunotherapy-based strategies.

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Fig. 1: Current pathways for FDA approval of companion diagnostic biomarkers for cancer therapies.

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Acknowledgements

The work of J.M.L. is supported by grants from the US NIH (RO1DK56621 and RO1DK128289), Cancer Research UK, Fondazione AIRC and Fundación Científica de la Asociación Española Contra el Cáncer (HUNTER, ref. C9380/A26813), the Samuel Waxman Cancer Research Foundation, Generalitat de Catalunya (AGAUR, SGR-1358), and the Spanish National Health Institute (MICINN, PID2019-105378RB-I00).

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Authors and Affiliations

  1. Mount Sinai Liver Cancer Program, Division of Liver Diseases, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA

    Josep M. Llovet

  2. Translational Research in Hepatic Oncology, Liver Unit, Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Hospital Clinic, University of Barcelona, Barcelona, Spain

    Josep M. Llovet

  3. Institució Catalana de Recerca i Estudis Avançats (ICREA), Barcelona, Spain

    Josep M. Llovet

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Correspondence to Josep M. Llovet.

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Competing interests

J.M.L. has received research support from Bayer HealthCare Pharmaceuticals, Bristol Myers Squibb, Eisai and Ipsen, and consulting fees from AstraZeneca, Bayer HealthCare Pharmaceuticals, Bluejay Therapeutics, Boston Scientific, Bristol Myers Squibb, Captor Therapeutics, Eisai, Eli Lilly, Exelixis, Genentech, Glycotest, Ipsen, Merck, Mina Alpha, Omega Therapeutics and Roche.

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Llovet, J.M. Exploring a new pathway for biomarker-based approval of immunotherapies. Nat Rev Clin Oncol (2023). https://doi.org/10.1038/s41571-023-00731-8

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  • DOI: https://doi.org/10.1038/s41571-023-00731-8

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