OBJECTIVES: To compare the cost-effectiveness in Scotland of cabozantinib with axitinib and everolimus in adult patients with aRCC following prior vascular endothelial growth factor receptors (VEGFR) targeted therapy.

METHODS: An economic model was developed to assess the cost-effectiveness using a lifetime time horizon, which equated to 30 years. Efficacy (time to discontinuation, progression free survival (PFS), overall survival) data came from the phase III trial METEOR (NCT01865747) comparing cabozantinib to everolimus. Equal efficacy for axitinib and everolimus was assumed due to the absence of head-to-head studies and differences in the design of pivotal trials, which precluded an indirect comparison. Tolerability (grade 3 and 4 adverse events) data came from two phase III trials: METEOR and AXIS (NCT00678392). Treatment duration for cabozantinib and everolimus was modelled through estimating time to treatment discontinuation (TTD), while treatment duration for axitinib was assumed to reflect PFS. For all efficacy endpoints, parametric curves were independently fitted to Kaplan-Meier curves of the three treatments to estimate outcomes during and beyond the trial period. Utilities were taken from METEOR. Costs data were specific to Scotland and list prices from the British National Formulary were used for drugs for this analysis.

RESULTS: In the base case, treatment with cabozantinib was estimated to cost an average of 86,378 GBP per patient while providing them with 2.80 life-years (LY) and 2.21 quality-adjusted life-years (QALY). It resulted in an incremental cost effectiveness ratio (ICER) of 93,221 GBP/LY and 115,473 GBP/QALY compared to everolimus, and an ICER of 78,716 GBP/LY and 97,224 GBP/QALY compared to axitinib.

CONCLUSIONS: Treatment with cabozantinib was more costly but also more effective in terms of LYs and QALYs gained than treatment with everolimus or axitinib. These conclusions held true across a range of scenarios and sensitivity analyses, including one-way and probabilistic analyses