Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis

Background and aims: The multi-kinase inhibitor sorafenib is a first-line drug for patients with advanced hepatocellular carcinoma (HCC). Treatment options for patients whose disease has progressed on sorafenib are limited. In a recent randomized controlled trial (CELESTIAL trial), patients with adv...

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Main Authors: Amir Shlomai, Moshe Leshno, Daniel A. Goldstein
Format: Article
Language:English
Published: SAGE Publishing 2019-09-01
Series:Therapeutic Advances in Gastroenterology
Online Access:https://doi.org/10.1177/1756284819878304
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author Amir Shlomai
Moshe Leshno
Daniel A. Goldstein
author_facet Amir Shlomai
Moshe Leshno
Daniel A. Goldstein
author_sort Amir Shlomai
collection DOAJ
description Background and aims: The multi-kinase inhibitor sorafenib is a first-line drug for patients with advanced hepatocellular carcinoma (HCC). Treatment options for patients whose disease has progressed on sorafenib are limited. In a recent randomized controlled trial (CELESTIAL trial), patients with advanced HCC who had failed prior systemic therapy had moderate progression-free survival and overall survival advantages when treated with the multi-kinase inhibitor cabozantinib. However, since this treatment is costly and is accompanied by significant adverse events in a large proportion of patients, its cost-effectiveness in these patients should be determined. Methods: We developed a Markov model incorporating health outcomes, measured by life-years and quality-adjusted life-years (QALYs) to evaluate the cost-effectiveness of cabozantinib compared with placebo in patients who have failed prior systemic therapy. Results: Treatment with cabozantinib results in a mean gain of 11.6 weeks of life (0.22 life-years) as compared with placebo. When quality of life was incorporated, treatment with cabozantinib produced a gain of 0.16 QALYs. The total mean incremental cost of cabozantinib was US$76,406 per patient. The incremental cost-effectiveness ratio for cabozantinib compared with best supportive care was US$469,374/QALY using the recommended dose of 60 mg cabozantinib daily. Conclusion: Our results suggest that the use of cabozantinib in patients with advanced HCC who have progressed on prior treatment, results in a modest incremental benefit with high incremental costs, suggesting that it is not cost-effective at conventional willingness to pay thresholds.
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spelling doaj.art-3375dcf1ff334825a476f2ca161af60b2022-12-21T23:52:31ZengSAGE PublishingTherapeutic Advances in Gastroenterology1756-28482019-09-011210.1177/1756284819878304Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysisAmir ShlomaiMoshe LeshnoDaniel A. GoldsteinBackground and aims: The multi-kinase inhibitor sorafenib is a first-line drug for patients with advanced hepatocellular carcinoma (HCC). Treatment options for patients whose disease has progressed on sorafenib are limited. In a recent randomized controlled trial (CELESTIAL trial), patients with advanced HCC who had failed prior systemic therapy had moderate progression-free survival and overall survival advantages when treated with the multi-kinase inhibitor cabozantinib. However, since this treatment is costly and is accompanied by significant adverse events in a large proportion of patients, its cost-effectiveness in these patients should be determined. Methods: We developed a Markov model incorporating health outcomes, measured by life-years and quality-adjusted life-years (QALYs) to evaluate the cost-effectiveness of cabozantinib compared with placebo in patients who have failed prior systemic therapy. Results: Treatment with cabozantinib results in a mean gain of 11.6 weeks of life (0.22 life-years) as compared with placebo. When quality of life was incorporated, treatment with cabozantinib produced a gain of 0.16 QALYs. The total mean incremental cost of cabozantinib was US$76,406 per patient. The incremental cost-effectiveness ratio for cabozantinib compared with best supportive care was US$469,374/QALY using the recommended dose of 60 mg cabozantinib daily. Conclusion: Our results suggest that the use of cabozantinib in patients with advanced HCC who have progressed on prior treatment, results in a modest incremental benefit with high incremental costs, suggesting that it is not cost-effective at conventional willingness to pay thresholds.https://doi.org/10.1177/1756284819878304
spellingShingle Amir Shlomai
Moshe Leshno
Daniel A. Goldstein
Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
Therapeutic Advances in Gastroenterology
title Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
title_full Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
title_fullStr Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
title_full_unstemmed Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
title_short Cabozantinib for patients with advanced hepatocellular carcinoma: a cost-effectiveness analysis
title_sort cabozantinib for patients with advanced hepatocellular carcinoma a cost effectiveness analysis
url https://doi.org/10.1177/1756284819878304
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