OBJECTIVES: In Brazil's Private Healthcare System, intravenous (IV) oncological drugs approved by the regulatory agency are automatically reimbursed. However, when the treatment involves an oral drug, it must go through the health technology assessment (HTA) process for reimbursement approval. In this context, we conducted a study to evaluate the cost-effectiveness of pembrolizumab (IV) + axitinib (oral) versus nivolumab (IV) + ipilimumab (IV) as first-line treatments in patients with advanced renal cell carcinoma (aRCC) at intermediate/poor IMDC (International Metastatic RCC Database Consortium) risk.

METHODS: A partitioned survival model compared pembrolizumab+axitinib versus nivolumab+ipilimumab over a lifetime horizon in treatment-naïve patients with intermediate/poor IMDC risk aRCC from a Brazilian Private Healthcare System perspective. The simulated cohort could transition through three health states: progression free, progression and death. Each cycle lasted four weeks and a 5% annual discount rate was applied to costs and benefits. Model inputs were obtained from KEYNOTE-426, network meta-analysis, scientific literature, and official price lists. Direct medical costs considered drugs acquisition and administration, subsequent treatments, adverse events, and health state associated costs. The outcomes were cost and quality-adjusted life years (QALYs). Deterministic and probabilistic sensitivity analyses were conducted. For reference, 1.00 Brazilian real (R$) equals approximately 0.19 United States dollar in January 2023.

RESULTS: The deterministic analysis estimated a similar clinical benefit for pembrolizumab+axitinib (2.58 QALYs) and nivolumab+ipilimumab (2.49 QALYs). Pembrolizumab+axitinib (R$ 1,489,180) cost less than nivolumab+ipilimumab (R$ 1,585,160), with estimated savings of R$ 95,979. Thus, pembrolizumab+axitinib dominated nivolumab+ipilimumab in the deterministic scenario. One-way sensitive analysis showed that drug costs and utilities were the parameters with the greatest impact on results. Probabilistic sensitivity analyses estimated that pembrolizumab+axitinib was dominant over nivolumab+ipilimumab in 74.6% of the simulations.

CONCLUSIONS: Pembrolizumab+axitinib is likely cost-effective in comparison to nivolumab+ipilimumab for patients with intermediate/poor prognostic IMDC risk aRCC from a Brazilian Private Healthcare System perspective.