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

Abstract

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.

Targeted therapy for hepatocellular carcinoma

“…Consequently, several studies have addressed the cost-effectiveness of cabozantinib using the cost and utility data extracted from the CELESTIAL trial. The conclusion from these studies is consistent and confirms that at its current cost point, the gain of quality-adjusted life-years for cabozantinib (QALYs, 0.067–0.16) and the incremental cost-effectiveness ratio (ICER, $156 437–$1,040,675) mean that it is not a cost-effective treatment option for patients with sorafenib-refractory HCC, 112 – 114 compared with regorafenib (QALY, 0.18–0.25 and ICER, $201,797–$224,362). 115 , 116 …”

Real-Life Clinical Data of Cabozantinib for Unresectable Hepatocellular Carcinoma

“…Indeed, the optimal management of these patients should rely on predictive rather than prognostic information. In this way, there would be the possibility to identify patients for whom the treatment is futile on 1 hand (also reducing the problem of cost-effectiveness of cabozantinib) [22, 23] and patients with the potential of becoming long survivors on the other hand. Unfortunately, such biomarkers have to be identified yet in the case of TKIs for HCC and in particular of cabozantinib.…”

Targeting hepatocyte growth factor/c‐mesenchymal–epithelial transition factor axis in hepatocellular carcinoma: Rationale and therapeutic strategies

“…[48][49][50][51] However, according to several retrospective cost-effectiveness analyses extracted from the CELESTIAL trial, the cabozantinib treatment yield high incremental costs and financially inviable for patients in the United States, United Kingdom, and China. [52][53][54] The findings remind an important element, which is to weigh a trade-off between costs and clinical benefits, and the willingness of conventional cost-effectiveness thresholds while developing a new treatment.…”