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Trends in the approval of cancer therapies by the FDA in the twenty-first century

Nature Reviews Drug Discovery volume 22, pages 625–640 (2023)Cite this article

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Abstract

The cancer treatment landscape has changed dramatically since the turn of the century, resulting in substantial improvements in outcomes for patients. This Review summarizes trends in the approval of oncology therapeutic products by the United States Food and Drug Administration (FDA) from January 2000 to October 2022, based on a categorization of these products by their mechanism of action and primary target. Notably, the rate of oncology indication approvals has increased in this time, driven by approvals for targeted therapies, as has the rate of introduction of new therapeutic approaches. Kinase inhibitors are the dominant product class by number of approved products and indications, yet immune checkpoint inhibitors have the second most approvals despite not entering the market until 2011. Other trends include a slight increase in the share of approvals for biomarker-defined populations and the emergence of tumour-site-agnostic approvals. Finally, we consider the implications of the trends for the future of oncology therapeutic product development, including the impact of novel therapeutic approaches and technologies.

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Fig. 1: Overview of oncology therapeutic products approved by the FDA since 2000 by mechanism of action.
Fig. 2: Trends in oncology therapeutic indication approvals since 2000.
Fig. 3: Trends in approvals across product classes.
Fig. 4: Trends in approvals for biomarker-defined populations.
Fig. 5: Trends for single-agent and combination approvals.
Fig. 6: Trends in regulatory pathways for oncology approvals.

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Acknowledgements

This work was funded in part by appointments to the Research Participation Program at the Office of Oncologic Diseases, Center for Drug Evaluation and Research at the FDA administered by the Oak Ridge Institute for Science and Education through an interagency agreement between the U.S. Department of Energy and the FDA. The authors thank S. Balasubramaniam and G. Kim for helpful discussions during early stages of study design.

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  1. Office of Oncologic Diseases, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA

    Emma C. Scott, Andrea C. Baines, Yutao Gong, Rodney Moore Jr, Gulsum E. Pamuk, Haleh Saber, Ashim Subedee, Matthew D. Thompson, Wenming Xiao & Julia A. Beaver

  2. National Cancer Institute, Rockville, MD, USA

    Ashim Subedee

  3. Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, MD, USA

    Richard Pazdur, Julie Schneider & Julia A. Beaver

  4. Office of Biotechnology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA

    V. Ashutosh Rao

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Contributions

E.C.S., A.C.B., Y.G., R.M., G.E.P., H.S., A.S., M.D.T., W.X. and J.A.B. researched data for the article. E.C.S., R.P., V.A.R., J.S. and J.A.B. contributed substantially to discussion of content. E.C.S. and J.A.B. contributed to writing the manuscript. All authors except Y.G. and A.S. reviewed and edited the manuscript.

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Correspondence to Emma C. Scott.

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Competing interests

Y.G., A.S. and J.A.B. completed work on this publication while employees at the FDA. At the time of publishing, Y.G. is an employee and shareholder at BeiGene, A.S. is an employee at the U.S. Department of Health and Human Services, and J.A.B. is an employee and shareholder at Treeline Biosciences. The other authors declare no competing interests.

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Nature Reviews Drug Discovery thanks Melissa Junttila, Joachim Rudolph and Doriano Fabbro for their contribution to the peer review of this work.

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Glossary

Cytotoxic drugs

Small-molecule drugs with the primary mode of action of inducing cellular toxicity, generally by interacting with DNA or components of the cell cycle. They affect rapidly dividing cells and are usually genotoxic.

Indication

The approved indication for a given product. This includes the use (for example, for treatment) and disease or condition for which the product is approved, as well as additional information, when applicable, such as use in conjunction with a primary mode of therapy (for example, in combination with another product(s)), the indicated population (for example, by age or biomarkers), and use in specific situations (for example, for use in previously treated patients).

Targeted biologics

Biological products, including monoclonal antibodies, other antibody constructs and conjugates, cellular therapies, enzymes, fusion proteins and viral therapies. They typically recognize specific peptide sequences in proteins present on the surface of cancer cells and have high target specificity.

Targeted drugs

Drugs that inhibit or interfere with defined molecular targets in cancer cells (such as kinases, receptors and other molecules) that are involved in intercellular or intracellular signalling pathways. They are primarily small-molecule drugs, but also include short peptides and radioactive agents without an antibody moiety.

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Scott, E.C., Baines, A.C., Gong, Y. et al. Trends in the approval of cancer therapies by the FDA in the twenty-first century. Nat Rev Drug Discov 22, 625–640 (2023). https://doi.org/10.1038/s41573-023-00723-4

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