OBJECTIVES : Sorafenib is currently the only targeted therapy which showed significant survival benefit over placebo for first-line treatment of uHCC in China. This study aimed to examine the cost-effectiveness of sorafenib compared to best support care (BSC) from a third-party payer perspective in China.

METHODS : A Markov model was developed by deriving transition probabilities for sorafenib group from ORIENTAL trial as scenario 1 and GIDEON trial as scenario 2. Health effect was expressed as QALYs gained. Resource utilization included only direct medical costs. The inpatient and outpatient medical costs are collected through a combination of hospital databases and clinical expert interviews. Costs and effects were evaluated over a patient’s lifetime and discounted at 5% per year. One-way and probabilistic sensitivity analyses (PSA) were conducted to examine the robustness of the findings.

RESULTS : In scenario 1, QALYs gained were increased for sorafenib compared to BSC (0.50 QALYs vs 0.38 QALYs, respectively). From the third-party payer perspective (on average 70% medical expenses reimbursed by local social medical insurance program), lifetime total costs were $US 8,736 for sorafenib and $US 3,834 for BSC. The incremental cost-effectiveness ratio (ICER) was $US 43,612 /QALY gained. In scenario 2, the lifetime costs and health outcome for sorafenib were estimated to be $US 12,266 and 0.95 QALYs. By keeping BSC the same as in scenario 1, the ICER decreased to $US 14,891 /QALY. PSA results showed that the probability of being cost-effective for sorafenib was 32% in scenario 1 and 100% in scenario 2, if the willingness-to-pay for a QALY was $US 25,145 (three times of GDP per capita in China in 2016 ).

CONCLUSIONS : The cost-effectiveness of sorafenib in comparison with BSC for treating uHCC differs significantly between the two scenarios. More data from the real world is needed in the future to reduce the uncertainty of the results.