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

Date Published: September 26, 2018

Publisher: Public Library of Science

Author(s): Mikyung Kelly Seo, John Cairns, Aamir Ahmad.


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.

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.

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.

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.

Partial Text

Colorectal cancer (CRC) is the third most common cancer and the third leading cause of cancer deaths worldwide [1]. In Europe, it is the most common cause of cancer death after lung cancer. In 2012, 241,600 men and 205,200 women were diagnosed with CRC [2], and 113,200 men and 101,500 women died from CRC [2]. In the USA, 136,830 cases newly diagnosed with CRC and 50,310 deaths with CRC were projected in 2014 [3].

The quality of the included studies was assessed by the Quality of Health Economic Studies (QHES) instrument. The QHES scale consists of 16 weighted questions, with a range of scores from 0 (worst quality) to 100 (best quality). The QHES tool was used by two independent assessors to rate the quality of the studies. QHES score per study is provided in S6 Table. Economic evaluations reported in full articles were scored using the QHES instrument (n = 30) and studies reported only in abstract (n = 16) were excluded from quality assessment due to their limited information.

Altogether, 46 papers were included in this systematic review. We identified three systematic reviews previously conducted for targeted therapies in mCRC [75–77], although they are different from ours in terms of the interventions focused. We focused on predictive biomarkers (or, companion biomarkers) and thus, targeted therapies with no licensed companion diagnostics were not included.




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