Cost-Effectiveness of Cabozantinib in the Second-Line Treatment of Advanced Hepatocellular Carcinoma

Authors:
Enrique Soto-Perez-de-Celis Department of Geriatrics, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico;

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Pedro N. Aguiar Jr Centro de Estudos e Pesquisa de Hematologia e Oncologia, Faculdade de Medicina do ABC, Santo André, Brazil;

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Mónica L. Cordón Department of Hemato-Oncology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico; and

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Yanin Chavarri-Guerra Department of Hemato-Oncology, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico; and

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Gilberto de Lima Lopes Jr Sylvester Comprehensive Cancer Center, University of Miami, Miami, Florida.

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Background: Treatment options are limited for patients with advanced hepatocellular carcinoma (HCC) that progresses after treatment with sorafenib. Cabozantinib, an oral small molecule inhibitor of multiple tyrosine kinase receptors, recently showed improved overall survival (OS) compared with placebo in sorafenib-pretreated patients with advanced HCC in the CELESTIAL trial. This study assessed the cost-effectiveness of cabozantinib for second-line treatment of patients with advanced HCC from a US healthcare system perspective. Patients and Methods: Cost and utility data were extracted from the CELESTIAL trial and used to determine the cost-effectiveness of cabozantinib compared with placebo plus best supportive care. The main outcome of this study was the incremental cost-effectiveness ratio (ICER), expressed as cost per quality-adjusted life year (QALY) gained by using cabozantinib compared with placebo plus best supportive care in sorafenib-pretreated HCC. Results: In the base-case analysis using data from the CELESTIAL trial, the incremental QALY and ICER were 0.067 and $1,040,675 for cabozantinib compared with placebo and best supportive care. OS reported in the CELESTIAL trial (hazard ratio, 0.76; 95% CI, 0.63–0.92) had the strongest association with the ICER. In one-way sensitivity analyses, there were no scenarios in which cabozantinib was cost-effective. In a cost-threshold analysis, cabozantinib would have to be priced at least $50 per pill to be cost-effective considering a willingness to pay of $100,000 per QALY. Although the CELESTIAL trial demonstrated that cabozantinib improves OS compared with placebo in patients with HCC that progresses after treatment with sorafenib, our analysis shows that cabozantinib is not a cost-effective therapy in this scenario. Conclusions: At current costs, cabozantinib is not cost-effective for second-line therapy of HCC in the United States.

Submitted August 29, 2018; accepted for publication January 16, 2019.

Author contributions: Study concept: Soto-Perez-de-Celis, Aguiar, Cordón, Lopes. Data curation: Soto-Perez-de-Celis, Aguiar, Cordón. Formal analysis: Aguiar. Investigation: All authors. Methodology: Soto-Perez-de-Celis, Aguiar. Manuscript preparation: All authors. Critical revision: All authors.

Disclosures: The authors have not received any financial consideration from any person or organization to support the preparation, analysis, results, or discussion of this article.

Correspondence: Gilberto de Lima Lopes Jr, MD, MBA, Sylvester Comprehensive Cancer Center, University of Miami, 1120 NW 14th Street, Suite 650J, Miami, FL 33135. Email: glopes@med.miami.edu
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