Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Mar 1;5(3):e222265.
doi: 10.1001/jamanetworkopen.2022.2265.

Cancer Drug Approvals That Displaced Existing Standard-of-Care Therapies, 2016-2021

David J Benjamin  1 , Alexander Xu  2 , Mark P Lythgoe  3 , Vinay Prasad  4
Affiliations

Cancer Drug Approvals That Displaced Existing Standard-of-Care Therapies, 2016-2021

David J Benjamin et al. JAMA Netw Open. .

Abstract

Importance: Although several cancer drugs receive US Food and Drug Administration (FDA) approval each month, it is unclear how many of these cancer drugs transform the treatment landscape significantly by tumor group. Specifically, it remains unclear how many of these newly approved cancer drugs displace the existing standard-of-care therapies for their indication vs being added to existing therapies.

Objective: To examine how many cancer drugs displace the standard-of-care therapies vs being added to existing therapy or filling breaks in systemic treatments in the metastatic setting, adjuvant setting, or maintenance setting.

Design, setting, and participants: Retrospective cross-sectional study using landmark trials leading to FDA approval of cancer drugs between May 1, 2016, and May 31, 2021. The study evaluated all FDA approvals for cancer drugs between May 1, 2016, and May 31, 2021, using the FDA Oncology (Cancer)/Hematologic Malignancies Approval Notifications website. All clinical trials leading to FDA approval of cancer drugs during this period were examined.

Main outcomes and measures: A drug was determined to have displaced the prior standard-of-care therapy by evaluating the comparator arm (or lack thereof) in the clinical trial leading to the drug's approval and also by reviewing National Comprehensive Cancer Network Guidelines. Cancer drug approvals were categorized as first-line displacing if a drug was approved for use in the first-line setting and displaced the prior standard-of-care drug for an indication, first-line drug alternatives/new if a drug was approved for use in the first-line setting but did not displace the standard of care at the time of approval or was a new drug that was first of its class for an approved indication, add on if a drug was approved in combination with a previously approved therapy for a disease or if a drug was approved for use in the adjuvant or maintenance settings, and later line if a drug was approved for use in the second-, third-, or later-line settings.

Results: Between May 1, 2016, and May 31, 2021, there were 207 FDA cancer drug approvals in oncology and malignant hematology. Of these 207 approvals, 28 drugs (14%) were first-line displacing therapies. A total of 32 drugs (15%) were first-line drug alternatives/new drugs. A total of 61 drugs (29%) were add-on therapies. Finally, 86 drugs (42%) were approved as later-line therapies.

Conclusions and relevance: In this study, most cancer drug approvals between 2016 and 2021 were in the later-line settings as opposed to displacing the current standard-of-care therapy for the approved indication. These later-line drugs may benefit patients with few alternatives but add to the cost of care because competition in the drug markets is a key factor in leading to lower drug prices.

PubMed Disclaimer

Conflict of interest statement

Conflict of Interest Disclosures: Dr Lythgoe reported personal fees from Clovis Oncology outside the submitted work. Dr Prasad reported grants from Arnold Ventures during the conduct of the study and outside the submitted work, and personal fees from Johns Hopkins Press, Medscape, MedPage, UnitedHealthcare, Evicore, New Century, and Patreon outside the submitted work. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Percentage of US Food and Drug Administration (FDA) Approvals Between May 1, 2016, and May 31, 2021, by Setting of Therapy
Figure 2.
Figure 2.. Number of Oncologic Approvals Between May 1, 2016, and May 31, 2021, by Tumor Organ System
See this image and copyright information in PMC

References

    1. Landau D. Why advances in immunotherapy mean the golden age of oncology. Oncol Times. 2019;41(13):24, 32. doi: 10.1097/01.COT.0000574936.15457.93 - DOI
    1. Smith CEP, Prasad V. Assessment of new molecular entities approved for cancer treatment in 2020. JAMA Netw Open. 2021;4(5):e2112558. doi: 10.1001/jamanetworkopen.2021.12558 - DOI - PMC - PubMed
    1. Davis JR, Benjamin DJ, Jonas BA. New and emerging therapies for acute myeloid leukaemia. J Investig Med. 2018;66(8):1088-1095. doi: 10.1136/jim-2018-000807 - DOI - PMC - PubMed
    1. Suzman DL, Agrawal S, Ning YM, et al. FDA approval summary: atezolizumab or pembrolizumab for the treatment of patients with advanced urothelial carcinoma ineligible for cisplatin-containing chemotherapy. Oncologist. 2019;24(4):563-569. doi: 10.1634/theoncologist.2018-0084 - DOI - PMC - PubMed
    1. Greig SL. Osimertinib: first global approval. Drugs. 2016;76(2):263-273. doi: 10.1007/s40265-015-0533-4 - DOI - PubMed

Publication types