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Do cancer biomarkers make targeted therapies cost-effective? A systematic review in metastatic colorectal cancer


  • Mikyung Kelly Seo
  • John Cairns


Background: Recent advances in targeted therapies have raised expectations that the clinical application of biomarkers would improve patient’s health outcomes and potentially save costs. However, the cost-effectiveness of biomarkers remains unclear irrespective of the cost-effectiveness of corresponding therapies. It is thus important to determine whether biomarkers for targeted therapies provide good value for money. This study systematically reviews economic evaluations of biomarkers for targeted therapies in metastatic colorectal cancer (mCRC) and assesses the cost-effectiveness of predictive biomarkers in mCRC. Methods: A literature search was performed using Medline, Embase, EconLit, NHSEED. Papers published from 2000 until June 2018 were searched. All economic evaluations assessing biomarker-guided therapies with companion diagnostics in mCRC were searched. To make studies more comparable, cost-effectiveness results were synthesized as per biomarker tests and corresponding therapies. Methodological quality was assessed using the Quality of Health Economic Studies (QHES) instrument. Results: Forty-six studies were included in this review. Of these, 17 studies evaluated the intrinsic value of cancer biomarkers, whereas the remaining studies focused on assessing the cost-effectiveness of corresponding drugs. Most studies indicated favourable cost-effectiveness of biomarkers for targeted therapies in mCRC. Some studies reported that biomarkers were cost-effective, while their corresponding therapies were not cost-effective. A considerable number of economic evaluations were conducted in pre-defined genetic populations and thus, often failed to fully capture the biomarker’s clinical and economic values. The average QHES score was 73.6. Conclusion: Cancer biomarkers for targeted therapies in mCRC were mostly found to be cost-effective; otherwise, they at least improved the cost-effectiveness of targeted therapies by saving some costs. However, this did not necessarily make their corresponding therapies cost-effective. While companion biomarkers reduced therapy costs, the savings were not sufficient to make the corresponding agents cost-effective. Evaluation of biomarkers was often restricted to the cost of tests and did not consider their clinical values or biomarker prevalence.

Suggested Citation

  • Mikyung Kelly Seo & John Cairns, 2018. "Do cancer biomarkers make targeted therapies cost-effective? A systematic review in metastatic colorectal cancer," PLOS ONE, Public Library of Science, vol. 13(9), pages 1-23, September.
  • Handle: RePEc:plo:pone00:0204496
    DOI: 10.1371/journal.pone.0204496

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    References listed on IDEAS

    1. Don Husereau & Michael Drummond & Stavros Petrou & Chris Carswell & David Moher & Dan Greenberg & Federico Augustovski & Andrew Briggs & Josephine Mauskopf & Elizabeth Loder, 2013. "Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement," PharmacoEconomics, Springer, vol. 31(5), pages 361-367, May.
    2. Drummond, Michael F. & Sculpher, Mark J. & Claxton, Karl & Stoddart, Greg L. & Torrance, George W., 2015. "Methods for the Economic Evaluation of Health Care Programmes," OUP Catalogue, Oxford University Press, edition 4, number 9780199665884, Decembrie.
    3. Martin Frank & Thomas Mittendorf, 2013. "Influence of Pharmacogenomic Profiling Prior to Pharmaceutical Treatment in Metastatic Colorectal Cancer on Cost Effectiveness," PharmacoEconomics, Springer, vol. 31(3), pages 215-228, March.
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    Cited by:

    1. Ana Beatriz D′Avó Luís & Mikyung Kelly Seo, 2021. "Has the development of cancer biomarkers to guide treatment improved health outcomes?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 22(5), pages 789-810, July.
    2. Mikyung Kelly Seo & Mark Strong, 2021. "A Practical Guide to Modeling and Conducting a Cost-Effectiveness Analysis of Companion Biomarker Tests for Targeted Therapies Using R: Tutorial Paper," PharmacoEconomics, Springer, vol. 39(12), pages 1373-1381, December.

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