Study: Second-Line Therapy for HCC Not Cost-Effective


The multikinase inhibitor regorafenib is not a cost-effective second-line therapy for the treatment of hepatocellular carcinoma (HCC), according to results of a study.

Previous trials have demonstrated that regorafenib is clinically effective as a second-line therapy in patients with HCC who experienced disease progression while on sorafenib, prolonging survival by 2.8 months in these patients. However, less is currently known about the cost-effectiveness of the therapy.

Neehar D. Parikh, MD, MS, University of Michigan, and colleagues constructed a Markov simulation model of patients with unresectable HCC and Child-Pugh A cirrhosis who were treated with regorafenib vs best supportive care. Researchers reviewed published clinical trial data and literature to determine model inputs for regorafenib effectiveness and adverse event rates.

Quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER) of regorafenib therapy were calculated. One-way sensitivity analyses were performed via Monte-Carlo simulation parameters. The regorafenib cost threshold for cost effectiveness was calculated.

The study was published in Cancer (October 2017;123[19]:3725-3731).

Results of the study showed that regorafenib therapy was not cost-effective as a second-line therapy. According to study findings, regorafenib therapy was associated with an increase of 0.18 QALYs at a cost of $47,112. The ICER for regorafenib therapy was found to be $224,362, compared with best supportive care.


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Ultimately, according to 1-way sensitivity analysis, there were no scenarios in which regorafenib therapy was cost-effective. Cost threshold analysis indicated that, in order to be cost effective at an ICER of $100,000, regorafenib therapy would have to be priced at $67 or less per pill.

“Regorafenib is not cost effective as a second-line agent in the treatment of HCC, with a marginal increase in QALYs at a high cost,” the researchers wrote. “Lowering the cost of regorafenib or improving the selection of patients who can achieve maximal survival benefit would improve its value as a second-line treatment option for patients with HCC.”—Christina Vogt