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Cost-Effectiveness of Cabozantinib in the Second-Line Treatment of Advanced Hepatocellular Carcinoma
Enrique Soto-Perez-de-Celis1, Pedro N Aguiar2, Mónica L Cordón3
1Department of Geriatrics, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
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.
Insights
Cabozantinib offers improved overall survival for advanced hepatocellular carcinoma (HCC) patients post-sorafenib treatment. However, this study found cabozantinib not to be a cost-effective second-line therapy in the US at current pricing.
Area of Science:
- Oncology
- Health Economics
- Pharmacoeconomics
Background:
- Limited treatment options exist for advanced hepatocellular carcinoma (HCC) progressing after sorafenib.
- Cabozantinib demonstrated improved overall survival (OS) in the CELESTIAL trial for such patients.
- This study evaluates the cost-effectiveness of cabozantinib as a second-line HCC treatment from a US healthcare perspective.
Purpose of the Study:
- To assess the cost-effectiveness of cabozantinib versus placebo plus best supportive care for second-line treatment of advanced HCC.
- To determine the incremental cost-effectiveness ratio (ICER) for cabozantinib in this patient population.
Main Methods:
- Cost and utility data were extracted from the CELESTIAL trial.
- The incremental cost-effectiveness ratio (ICER) was calculated as cost per quality-adjusted life year (QALY) gained.
- Sensitivity and cost-threshold analyses were performed.
Main Results:
- The base-case analysis yielded an ICER of $1,040,675 per QALY gained for cabozantinib.
- Overall survival (OS) from the CELESTIAL trial was the most influential factor on the ICER.
- No scenarios in sensitivity analyses demonstrated cabozantinib to be cost-effective; a price reduction to $50/pill was estimated as necessary.
Conclusions:
- Cabozantinib is not a cost-effective second-line therapy for advanced HCC in the United States at its current cost.
- Despite improving OS, the high cost of cabozantinib limits its value in this treatment setting.
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