Economic evaluation of sorafenib in unresectable hepatocellular carcinoma

Background and Aim: A double-blind, randomized phase III trial of sorafenib in advanced hepatocellular carcinoma demonstrated that sorafenib significantly prolonged overall survival compared to placebo (median overall survival = 10.7 months vs 7.9 months, P < 0.001). Sorafenib is the first and only systemic agent demonstrating survival benefit in these patients. The aim of this study was to assess the cost-effectiveness of sorafenib versus best supportive care in the treatment of advanced hepatocellular carcinoma in the USA. Methods: A Markov model was developed following time-to-progression and survival using phase III trial data. Health effects are expressed as life-years gained. Resource utilization included drugs, physician visits, laboratory tests, scans, and hospitalizations. Unit costs, expressed in 2007 $US, came from diagnosis-related groupings, fee schedules, and the Red Book. Costs and effects were evaluated over a patient's lifetime and discounted at 3%. Results: Results are presented as incremental cost/life-year gained. Deterministic and probabilistic sensitivity analyses were conducted. Life-years gained were increased for sorafenib compared to best supportive care (mean +/- standard deviation: 1.58 +/- 0.17 vs 1.05 +/- 0.10 life-years gained/sorafenib patient and best supportive care, respectively). Lifetime total costs were $US40 639 +/- $US3052 for sorafenib and $US7 804 +/- $US1349 for best supportive care. The incremental cost-effectiveness ratio was $US62 473/life-year gained. Conclusions: The economic evaluation indicates that sorafenib is cost-effective compared to best supportive care, with a cost-effectiveness ratio within the established threshold that US society is willing to pay (i.e. $US50 000-$US100 000) and significantly lower than alternative thresholds suggested in recent years ($US183 000-$US264 000/life-year gained, or $US300 000/quality-adjusted life-year) in oncology.