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Accelerated approvals hit the target in precision oncology

Nature Medicine volume 28, pages 1976–1979 (2022)Cite this article

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The US Food and Drug Administration (FDA)’s accelerated approval pathway is most frequently used for oncology indications, with roughly one-quarter of these approvals for precision medicines that target solid tumors1,2. This pathway expedites access to drugs that have an effect on clinical endpoints measured earlier than survival, and relies on post-approval studies to verify clinical benefit. Initially developed in 1992 to address the HIV and AIDS crisis, the accelerated approval pathway was first applied to oncology in the era of cytotoxic chemotherapy. Since then, the emergence of precision medicines has transformed the field of oncology.

Precision oncology aims to match the right drugs or treatments to the right patients, based on a genetic or molecular understanding of their disease. This targeted approach is unique in its exacting and durable effect on tumor size, and has enabled overall response rates to frequently serve as an early clinical endpoint to support accelerated approval. The opportunity to leverage accelerated approval has provided patients with cancer with early and broad access to potentially life-saving precision therapies. The application of this program in precision oncology has unique challenges. Rare patient populations and high response rates in this setting frequently limit the ability to perform randomized trials and compare overall survival, considered to be the gold standard endpoint in oncology clinical trials. A review of three decades of accelerated approvals in precision oncology for solid tumors suggests that this program has been highly successful.

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Acknowledgements

V.S. is an Andrew Sabin Family Foundation Fellow at The University of Texas MD Anderson Cancer Center, acknowledges support of The Jacquelyn A. Brady Fund, is supported by a US National Institutes of Health (NIH) grant (no. R01CA242845 and R01CA273168). MD Anderson Cancer Center Department of Investigational Cancer Therapeutics is supported by the Cancer Prevention and Research Institute of Texas (no. RP1100584), the Sheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer Therapy (no. 1U01 CA180964), a NCATS (Center for Clinical and Translational Sciences) grant (no. UL1 TR000371), and the MD Anderson Cancer Center support grant (no. P30 CA016672). The content is solely the responsibility of the authors and does not necessarily represent the official views of the US NIH or FDA.

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Authors and Affiliations

  1. The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Vivek Subbiah

  2. Harvard Medical School, Massachusetts General Hospital, Boston, MA, USA

    Lori J. Wirth

  3. Medical College of Wisconsin Cancer Center and Genome Science and Precision Medicine Center, Milwaukee, WI, USA

    Razelle Kurzrock

  4. Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA

    Richard Pazdur, Julia A. Beaver & Harpreet Singh

  5. Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Harpreet Singh & Gautam U. Mehta

Authors
  1. Vivek Subbiah
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  2. Lori J. Wirth
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  3. Razelle Kurzrock
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  4. Richard Pazdur
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  5. Julia A. Beaver
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  7. Gautam U. Mehta
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Correspondence to Vivek Subbiah or Gautam U. Mehta.

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Competing interests

V.S. reports research funding or grant support for clinical trials from AbbVie, Agensys, Inc., Alfasigma, Altum, Amgen, Bayer, BERG Health, Blueprint Medicines Corporation, Boston Biomedical, Inc., Boston Pharmaceuticals, Celgene Corporation, D3 Bio, Inc., Dragonfly Therapeutics, Inc., Exelixis, Fujifilm, GlaxoSmithKline, Idera Pharmaceuticals, Inc., Incyte Corporation, Inhibrx, Loxo Oncology/ Eli Lilly, MedImmune, MultiVir, Inc., NanoCarrier, Co., National Comprehensive Cancer Network, NCI-CTEP, Novartis, PharmaMar, Pfizer, Relay Therapeutics, Roche/Genentech, Takeda, Turning Point Therapeutics, UT MD Anderson Cancer Center and Vegenics Pty Ltd.; travel support from ASCO, ESMO, Helsinn Healthcare, Incyte Corporation, Novartis and PharmaMar; consultancy or advisory board participation for Helsinn Healthcare, Incyte Corporation, Loxo Oncology/Eli Lilly, MedImmune, Novartis, QED Therapeutics, Relay Therapeutics, Daiichi-Sankyo, and R-Pharm US; and other relationships with Medscape. L.W. receives consulting income from Bayer, Coherus, Curie Therapeutics, Eisai, Exelixis, Lilly and Novartis and has received fees for sitting on a Data Monitoring Committee for PDS Biotechnology. R.K. has received research funding from Biological Dynamics, Boehringer Ingelheim, Debiopharm, Foundation Medicine, Genentech, Grifols, Guardant, Incyte, Konica Minolta, Medimmune, Merck Serono, Omniseq, Pfizer, Sequenom, Takeda and TopAlliance; as well as consultant fees, speaker fees or is an advisory board member for Actuate Therapeutics, AstraZeneca, Bicara Therapeutics, Biological Dynamics, Daiichi Sankyo, Inc., EISAI, EOM Pharmaceuticals, Iylon, Merck, NeoGenomics, Neomed, Pfizer, Prosperdtx, Roche, TD2/Volastra, Turning Point Therapeutics and X-Biotech; has an equity interest in CureMatch Inc., CureMetrix,and IDbyDNA; serves on the Board of CureMatch and CureMetrix, and is a co-founder of CureMatch. All other co-authors have no other conflicts.

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Subbiah, V., Wirth, L.J., Kurzrock, R. et al. Accelerated approvals hit the target in precision oncology. Nat Med 28, 1976–1979 (2022). https://doi.org/10.1038/s41591-022-01984-z

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