Hepatocellular carcinoma (HCC) is the most common form of liver cancer and the fourth leading cause of cancer-related death globally. There are unmet needs for effective systematic therapy. The findings of the RESORCE trial highlighted the improvement in overall survival with regorafenib in advanced HCC patients progressing on sorafenib treatment. This study aimed to assess the cost-effectiveness of regorafenib compared with best supportive care (BSC) for advanced HCC from the Canadian healthcare system perspective.

We developed a Markov model based on four health states: live with adverse events, live without adverse events, progression, and dead. Health outcomes were measured using life-years (LYs), and quality-adjusted life-years (QALYs), and costs were presented in Canadian dollars (CAD). Clinical inputs were derived from the RESORCE trial. A 1.5 percent discount rate was applied to costs and outcomes. One-way and probabilistic sensitivity analyses were performed to assess the uncertainty in findings due to variability in parameters. TreeAge Pro software was used for model implementation.

The use of regorafenib results in a gain of 0.38 LYs and 0.25 QALYs as compared to BSC with a high incremental cost of CAD26,954 (USD22,313). The ICER for regorafenib compared with BSC was CAD105,850/QALY (USD87,624/QALY) in the base-case analysis. Further, probabilistic sensitivity analyses revealed regorafenib not to be cost-effective at a willingness-to-pay threshold of CAD50,000/QALY.

Regorafenib was not found to be cost-effective in the treatment of advanced HCC because of the lower health benefits and higher incremental costs. Lowering the official price of regorafenib or use for only selected patients who can achieve maximum benefits would enhance its cost-effectiveness and treatment preference value.