OBJECTIVE: This study aims to assess whether immune checkpoint inhibitors (ICIs) -based treatments offer significant survival benefits and economic viability for patients presenting with PD-L1 lower than 1%. METHODS: This exploratory analysis was based on individual patient data from randomized controlled trials (CheckMate-227, CheckMate-9LA, KEYNOTE-189, and KEYNOTE-407). Survival outcomes evaluated for this study were overall survival (OS) and progression-free survival (PFS). Economic evaluations estimated total lifetime costs, incremental cost-effectiveness ratios (ICERs), and incremental net-health benefit (INHB) using the Markov model. The willingness-to-pay (WTP) thresholds were 150, 000/quality-adjusted life-year (QALY) for the USA and 36, 942/QALY for China. RESULTS: Among patients receiving immunotherapy-based treatments (N = 754) relative to those treated with chemotherapy-alone (N = 502), survival outcomes were significantly improved (median OS: 17. 0 vs. 12. 0 months; hazard ratio HR: 0. 69; 95% confidence interval CI: 0. 61-0. 78; median PFS: 6. 5 vs. 5. 5 months; HR: 0. 68; 95% CI: 0. 60-0. 77). The cost-effectiveness analysis found that the ICERs with INHBs for immunotherapy-based versus chemotherapy-alone were 273, 440. 73/QALY with -1. 00 QALYs in the USA and 32, 783. 93/QALY with 0. 89 QALYs in China. Crucially, among ICI-based regimens, nivolumab plus ipilimumab-based (INHB: 0. 09 QALYs) and pembrolizumab plus chemotherapy (INHB: 0. 17 QALYs) were the most cost-effective options in the USA and China, respectively. CONCLUSIONS: First-line immunotherapies-based therapy was cost-effective in metastatic NSCLC with PD-1 lower than 1% in China but not in the USA. Nivolumab plus ipilimumab-based was identified as the most favorable first-line immunotherapy in the USA, while pembrolizumab plus chemotherapy was preferred in China.
Liu et al. (2026) studied this question.