OBJECTIVES: There are about 466,100 new cases diagnosed and 422,100 patients died from liver cancer in China annually. Hepatocellular carcinoma (HCC) accounts for 85%~90% of liver cancer. Regorafenib is a potent oral multi-kinase inhibitor approved in 2017 in China as a second-line therapy for patients with HCC. While the clinical efficacy of regorafenib for HCC has been well documented, the economic value of adopting this new therapy is still unclear in China. This study aimed to examine the cost-effectiveness of regorafenib plus best supportive care (BSC) compared to placebo plus BSC in Chinese setting.

METHODS: A previously published Markov model with a lifetime horizon was developed to evaluate the cost-effectiveness of regorafenib from a third-party payer perspective. The primary model outputs were total costs, quality-adjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER). Only direct medical costs were considered. Clinical efficacy data were obtained from the pivotal clinical trial (RESORCE). One-way sensitivity and probability sensitivity analyses were conducted to examine the robustness of the base-case findings.

RESULTS: In the base-case analysis, the lifetime total costs were higher for regorafenib plus BSC at ¥200,593 compared with ¥113,358 for BSC. The QALYs gained were 1.30 and 0.79 for patients treated with regorafenib plus BSC and BSC only, respectively. The ICER of regorafenib plus BSC vs BSC was ¥172,670/QALY, which is under the threshold of 3 times per capita GDP of China in 2017 (¥178,980). One-way sensitivity analysis showed that ICER was sensitive to the increased overall survival in regorafenib-treated patients. Findings from the probabilistic sensitivity analysis indicated that the likelihood of regorafenib plus BSC being cost-effective was 53%.

CONCLUSIONS: Compared with BSC, HCC treatment with regorafenib is more expensive but also with greater QALYs gains. On balance, regorafenib is an attractive cost-effective option for the 2^nd line treatment of HCC in China.