OBJECTIVES: To evaluate the cost-effectiveness of pembrolizumab for the adjuvant treatment of adults with renal cell carcinoma (RCC) at increased risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions, from the French healthcare system perspective.

METHODS: A four-state Markov model (disease-free [DF], locoregional recurrence [LR], distant metastases [DM], and death) was developed to estimate costs, effectiveness, and incremental cost-effectiveness ratio (ICER) of pembrolizumab versus routine surveillance over a 30-year time horizon. Transition probabilities from the DF state were estimated from the KEYNOTE-564 trial, using parametric models to extrapolate the cause-specific hazards of each transition over time. Transitions starting from LR state were obtained from the trial and literature and transitions from DM state from a network meta-analysis. EQ-5D-5L data from KEYNOTE-564 were used for DF and LR states, after a mapping to EQ-5D-3L, to fit with data from KEYNOTE-426 used for DM state. All utilities were weighted by the French value set. Only direct medical costs were considered, based on public sources. Costs and health outcomes were discounted at 2.5% per year. ICER was calculated as cost per quality-adjusted life year (QALY) gained and per life year gained (LYG). Deterministic and probabilistic sensitivity analyses were conducted to assess robustness of results.

RESULTS: Adjuvant treatment with pembrolizumab generated an incremental cost of 28,339 €, 1.21 LYG and 0.97 QALY gained compared with routine surveillance, resulting in an ICER of 29,342 €/QALY. After 5 years, the proportion of patients remaining DF increased by 21% and recurrence decreased by 25% with pembrolizumab versus routine surveillance. At a 67,000 €/QALY threshold, pembrolizumab was cost-effective versus routine surveillance in 80% of probabilistic simulations.

CONCLUSIONS: Receiving pembrolizumab as an adjuvant treatment for patients with RCC who have undergone nephrectomy improves life expectancy, reduces risk of progression to severe stages and appears cost-effective compared to routine surveillance.