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.