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Regorafenib treatment for patients with hepatocellular carcinoma who progressed on sorafenib—A cost-effectiveness analysis

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  • Amir Shlomai
  • Moshe Leshno
  • Daniel A Goldstein

Abstract

Background and aims: Hepatocellular carcinoma (HCC) is one of the leading causes of cancer related deaths. Patients with advanced HCC are treated with sorafenib. A recent randomized controlled trial demonstrated a survival benefit for regorafenib treatment in patients with advanced HCC who had progressed on sorafenib. We aimed to evaluate the cost-effectiveness of this approach. Methods: To evaluate the cost effectiveness of regorafenib, we used a Markov model that incorporates health outcomes, measured by life-years and quality-adjusted life-years (QALYs). Drug costs were based on 2017 discounted prices. Model robustness was validated by probabilistic sensitivity analyses using Monte Carlo simulations. Results: The use of regorafenib results in a gain of 19.76 weeks of life (0.38 Life Years) as compared to placebo. When adjusted for quality of life, using regorafenib produced a gain of 0.25 quality adjusted life years (QALYs). The incremental cost-effectiveness ratio for regorafenib compared with best supportive care was between $201,797 and $268,506 per QALY. Conclusion: The modest incremental benefit at a relatively high incremental cost of regorafenib treatment suggests that it is not cost-effective at commonly accepted willingness to pay thresholds.

Suggested Citation

  • Amir Shlomai & Moshe Leshno & Daniel A Goldstein, 2018. "Regorafenib treatment for patients with hepatocellular carcinoma who progressed on sorafenib—A cost-effectiveness analysis," PLOS ONE, Public Library of Science, vol. 13(11), pages 1-11, November.
    • Handle: RePEc:plo:pone00:0207132
    DOI: 10.1371/journal.pone.0207132
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