Standard Dose of Regorafenib Not Cost-Effective in CRC

A dose escalation strategy for regorafenib could improve the cost-effectiveness of the medication in metastatic colorectal cancer (CRC).

Regorafenib is not as cost-effective as other third-line treatments for metastatic colorectal cancer (mCRC) when using the standard dose, according to a review published in Cancer Management and Research.1 Using a dose-escalation strategy could help improve the cost-effectiveness of the treatment.

CRC led to approximately 51,869 deaths in the US in 2020,2 making it a prominent cause of death related to cancer in the country. mCRC occurs in approximately 25% of cases at first diagnosis and at least 50% of patients will eventually develop mCRC. The FDA approved regorafenib to be used as a third-line therapy for mCRC. This review aimed to evaluate how cost-effective the treatment was compared with other third-line treatments after the reduction in price.1

Stacked coins | Image credit: sitthiphong -

Stacked coins | Image credit: sitthiphong -

PubMed, Medline, Scopus, Embase, Cochrane Library, and 9 other databases were used to find eligible studies for the review. Articles were used if they were published in English between January 1, 2012, and October 31, 2023. Studies were included if they included a cost-effectiveness analysis, included patients diagnosed with mCRC who were using regorafenib, and used incremental cost-effectiveness ratios (ICERs) to compare treatments. Studies that were not published in English, did not include an economic evaluation, and did not focus on a specific drug were excluded from the study. Data extracted from the studies included the research type, country, study design, study outcomes, and publication date.

There were 13 studies included in the review, of which 4 studies were conducted in the US, 3 were from China, 2 were from Japan, and the remaining studies were conducted in Italy, England and Wales, Greece, and the Czech Republic. Cost-utility analysis (CUA) was used in 11 of the studies. Life-years (LYs) and quality-adjusted life-years (QALYs) and ICER were used to evaluate the effectiveness of the medications. Randomized controlled trials, meta-analyses, and clinical trials were used to collect the clinical data.

Cost-effectiveness of regorafenib was compared with other methods of care. Minimal incremental benefit at a high incremental cost per QALY was found in regorafenib when evaluating from the US payer perspective. There was a 50% chance of cost-effectiveness for regorafenib at a willingness-to-pay (WTP) threshold of $900,000 per QALY. When compared with best supportive care, regorafenib was not cost-effective at a WTP threshold of $27,576. Regorafenib was found to be a cost-effective therapeutic option for patients with mCRC in the Czech Republic at a WTP threshold of €47,000 per QALY.

When compared with trifluridine/tipiracil (TAS-102), regorafenib was found to be less clinically and cost-effective in 5 studies that compared the standard dose of regorafenib vs TAS-102. An optimal dosing strategy for regorafenib was able to improve its relative cost-effectiveness. TAS-102 combined with bevacizumab was less cost-effective than regorafenib on dose optimization.

Regorafenib was also less cost-effective than fruquintinib, which was associated with an increase of 0.05 QALYs and had a cost savings of $11,454. Serplulimab was also more cost-effective compared with regorafenib in patients who were previously treated for mCRC.

There were some limitations to this study. Studies published in other languages were not included in this review. Medical services, costs of drugs, and WTP were different across the different countries. Publication bias could have an effect on the results of the studies.

Although regorafenib was more cost-effective than best supportive care, it was less cost-effective than other third-line treatments for mCRC. Dose optimization could make regorafenib more cost-effective in the future.


  1. Zhong J, Liu Y, Fu Q, Huang D, Gong W, Zou J. Cost-effectiveness analysis of regorafenib versus other third-line treatments for metastatic colorectal cancer. Cancer Manag Res. 2024;16:593-602. doi:10.2147/CMAR.S464831
  2. Colorectal cancer stat bite. CDC. Updated June 23, 2023. Accessed June 10, 2024.
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