Cost-effectiveness analysis of belzutifan versus everolimus for previously treated advanced clear cell renal cell carcinoma in the United States and China.

Background: The LITESPARK-005 trial demonstrated the efficacy and safety of belzutifan in patients with previously treated clear cell renal cell carcinoma (ccRCC). This study aims to evaluate the cost-effectiveness of belzutifan compared to everolimus in treating patients with advanced ccRCC who have received at least one systemic therapy from the perspective of the Chinese healthcare system and the US payers.

Objectives: To provide previously treated ccRCC patients with the option of belzutifan and to offer recommendations regarding in China.

Design: The cost-effectiveness analysis.

Methods: A partitioned survival model was constructed based on data from the LITESPARK-005 trial. Patients transitioned through three mutually exclusive health states: progression-free survival (PFS), progressive disease, and death. The model cycle length was set at 28 days, with a lifetime horizon. Direct medical costs and utility values were obtained from published literature and real-world healthcare data. The model estimated total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Price simulations, sensitivity analyses, and scenario analyses were conducted to assess model robustness.

Results: The base-case analysis showed that belzutifan in China generated 2.038 QALYs at a total cost of $102,561.26 and an ICER of $54,430.16/QALY, which exceeded the willingness to pay (WTP) threshold ($39,076.44/QALY). Belzutifan in the United States generated 2.280 QALYs at a total cost of $796,227.28 with an ICER of $270,864.46/QALY, which significantly exceeded the WTP threshold ($150,000/QALY). The PFS utility value and the drug cost of belzutifan were the main factors affecting the change in ICER, whether in China or the United States. At current pricing, belzutifan was unlikely to be cost-effective. Price simulations indicated the belzutifan would be cost-effective when the price of belzutifan remained below $5.524/mg in the United States and $0.779/mg in China.

Conclusion: Compared to everolimus, belzutifan is not cost-effective at its current price for treating previously treated RCC in the United States. In China, the belzutifan group was cost-effective when the price of belzutifan was less than $0.779/mg. This study suggests that reducing the price could substantially improve the economic viability of belzutifan.