Common Second-Line Treatment for Liver Cancer Deemed Not Cost-Effective

A conventional second-line treatment for hepatocellular carcinoma offers only a marginal increase in quality-adjusted life-years (QALYs) at a significantly high cost, according to an analysis published in Cancer (published online June 29, 2017; doi:10.1002/cncr.30863).


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The multikinase inhibitor regorafenib has shown effectiveness in prolonging survival by 2.8 months as a second-line therapy in patients with hepatocellular carcinoma who progress after first-line sorafenib. However, a cost analysis has yet to be undertaken involving regorafenib in this setting.

Neehar D Parikh, MD, MS, department of internal medicine, University of Michigan, and colleagues conducted a study to assess the cost-effectiveness of second-line regorafenib in progressed hepatocellular carcinoma. Researchers created a simulation model—using data from previously published clinical trials and literature—of patients with unresectable hepatocellular carcinoma and Child-Pugh A cirrhosis who received treatment with regorafenib versus best supportive care. QALYs and the incremental cost-effectiveness ratio (ICER) were calculated. Researchers also determined the regorafenib cost threshold at which cost-effectiveness would be achieved.

Results of the analysis found regorafenib to not be a cost-effective second-line agent in most scenarios for the treatment of hepatocellular carcinoma. The multikinase inhibitor provided an increase of only 0.18 QALYs at a cost of $47,112. The ICER for regorafenib, when compared with best supportive care, was $224,362. Sensitivity analyses did not yield any instances in which regorafenib was cost-effective. In the cost threshold analysis, regorafenib would need to be priced less than $68 per pill to be considered cost-effective at an ICER of $100,000.

After demonstrating only slight increases in QALYs at a significantly high cost, researchers concluded that regorafenib is not a cost-effective second-ling agent in the treatment of progressed hepatocellular carcinoma. The only scenarios in which the value of regorafenib could be increased, they wrote, would be if it was sold at a lower price or by improving the selection of patients who can achieve maximal survival benefit.—Zachary Bessette