Economic evaluation of sorafenib in unresectable hepatocellular carcinoma

J Gastroenterol Hepatol. 2010 Nov;25(11):1739-46. doi: 10.1111/j.1440-1746.2010.06404.x.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / economics*
  • Antineoplastic Agents / therapeutic use*
  • Benzenesulfonates / economics*
  • Benzenesulfonates / therapeutic use*
  • Carcinoma, Hepatocellular / drug therapy*
  • Carcinoma, Hepatocellular / economics*
  • Carcinoma, Hepatocellular / mortality
  • Cost-Benefit Analysis
  • Humans
  • Liver Neoplasms / drug therapy*
  • Liver Neoplasms / economics*
  • Liver Neoplasms / mortality
  • Markov Chains
  • Models, Economic
  • Niacinamide / analogs & derivatives
  • Phenylurea Compounds
  • Pyridines / economics*
  • Pyridines / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Sorafenib
  • Survival Analysis


  • Antineoplastic Agents
  • Benzenesulfonates
  • Phenylurea Compounds
  • Pyridines
  • Niacinamide
  • Sorafenib