Cost-effectiveness of nivolumab combined with chemotherapy as a first-line therapy for patients with unresectable or metastatic urothelial carcinoma

Background: Urothelial carcinoma is a significant health concern in the United States (US), with high mortality and economic burdens. The CheckMate-901 trial showed promising survival benefits for nivolumab combined with gemcitabine and cisplatin followed by nivolumab maintenance therapy (nivolumab-combination) as first-line treatment of unresectable or metastatic urothelial carcinoma (UC), but its cost-effectiveness is unclear. Objectives: This study aimed to evaluate the cost-effectiveness of the nivolumab-combination versus standard chemotherapy (gemcitabine–cisplatin) for advanced UC from the perspective of healthcare payers in the US. Design: A model-based pharmacoeconomic evaluation. Methods: Based on the CheckMate-901 study, a three-state Markov model (progression-free, progression, and death) was developed to evaluate the cost-effectiveness of nivolumab-combination versus gemcitabine–cisplatin as a first-line treatment for unresectable or metastatic UC. The model’s outputs included quality-adjusted life years (QALYs) and costs and were used to calculate the incremental cost-effectiveness ratio (ICER). Costs included drug prices, adverse event management, and healthcare resource utilization from a US healthcare payer’s perspective. State utilities were derived from published literature. One-way sensitivity analysis and probabilistic sensitivity analysis were used to test model robustness. Scenario analyses for drug costs in the UK and Australian health systems were performed. Results: Compared with gemcitabine–cisplatin, the nivolumab-combination resulted in an additional 0.416 QALYs at an incremental cost of $90,523, yielding an ICER of $217,527 per QALY. Sensitivity analyses indicated significant impacts from the cost of nivolumab maintenance therapy. Conclusion: Compared with gemcitabine–cisplatin, nivolumab-combination therapy is not cost-effective for unresectable or metastatic UC at a $100,000 per QALY threshold. High drug prices in the US significantly impact cost-effectiveness, highlighting the need for price negotiations and healthcare policy adjustments to balance innovation incentives and patient affordability.