Cost‐effectiveness analysis of cabozantinib as second‐line therapy in advanced hepatocellular carcinoma

Abstract

Background: In the CELESTIAL trial for patients with advanced hepatocellular carcinoma (HCC), cabozantinib showed improved survival compared with placebo but comes at a price. We aimed to investigate the cost-effectiveness of cabozantinib for sorafenib-resistant HCC from the payer's perspective of the USA, UK and China. Methods:We developed Markov models to simulate the patients pre-treated with first-line sorafenib following the CELESTIAL trial. Quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) were calculated for the treatment with cabozantinib or best supportive care. The list price for drugs was acquired from the Red Book, the British National Formulary, West China hospital and reported literature. Adverse events, utilities weights, and transition likelihood between states were sourced from the published randomized phase III trial. A willing-to-pay threshold was set $150 000/QALY in the USA, $70 671/QALY (£50 000/QALY) in the UK and $26 481/QALY (3x GDP per capita) in China. Deterministic and probabilistic sensitivity analyses were developed to test the models' uncertainty. Results:In the base case, treatment with cabozantinib increased effectiveness by 0.13 QALYs, resulting in an ICER vs best supportive care of $833 497/QALY in the USA, $304 177/QALY in the UK and $156 437/QALY in China. The models were most sensitive to assumptions about transitions to progression with both cabozantinib and best supportive care, the utility associated with being progression free. These results were robust across a range of scenarios and sensitivity analyses, including deterministic and probabilistic analyses.Conclusions: Cabozantinib at its current cost would not be a cost-effective treatment option for patients with sorafenib-resistant HCC from the payer's perspective in the USA, UK or China. Substantial discounts are necessary to meet conventional costeffectiveness thresholds. K E Y W O R D S cabozantinib, cost-effective, hepatocellular carcinoma, incremental cost-effectiveness ratio, Markov model, second-line therapy S U PP O RTI N G I N FO R M ATI O N Additional supporting information may be found online in the Supporting Information section at the end of the article. How to cite this article: Liao W, Huang J, Hutton D, et al. Cost-effectiveness analysis of cabozantinib as second-line therapy in advanced hepatocellular carcinoma. Liver Int.

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 sorafenibrefractory HCC, [112][113][114] compared with regorafenib (QALY, 0.18-0.25 and ICER, $201,797-$224,362). 115,116 Ramucirumab Ramucirumab is a fully human recombinant IgG1 monoclonal antibody targeting the VEGF2 receptor.…”

Cost-Effectiveness of Tucatinib in Human Epidermal Growth Factor Receptor 2–Positive Metastatic Breast Cancer From the US and Chinese Perspectives

“…Diaby et al ( 54 ) reported a cost of $158,961.4 per QALY for patients treated with second-line for MBC. The use of innovative drugs that have been proven to be effective in clinical trials may lead to a substantial increase in medical expenditure, while the abandonment of these drugs means rejection of possible beneficial treatment ( 29 ). Therefore, cost-effectiveness analysis from different perspectives has become an important part of a broader discussion in how we allocate resources to treat cancer.…”

Optimal sequence of systemic therapy after sorafenib failure in patients with hepatocellular carcinoma

“…One study reported that cabozantinib would not be cost-effective as the second-line therapy in advanced HCC. 13 In the near future, the systemic treatment paradigm will be changed as lenvatinib becomes increasingly used as a first-line therapy, and the combination of atezolizumab with bevacizumab has shown promising results as the first-line treatment in a recent phase 3 trial. With rearrangement of first-line systemic therapies in practice, the need to select the optimal second-line treatment will again be raised.…”

“…When choosing the type of systemic therapy, it is important to consider the cost-effectiveness. One study reported that cabozantinib would not be cost-effective as the second-line therapy in advanced HCC [ 13 ]. In the near future, the systemic treatment paradigm will be changed as lenvatinib becomes increasingly used as a first-line therapy, and the combination of atezolizumab with bevacizumab has shown promising results as the first-line treatment in a recent phase 3 trial.…”