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Randomized trial evaluates survival and cost-effectiveness of immunotherapy in patients with low PD-L1 levels, indicating varying results by region.
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.
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Liu et al. (2026) studied this question.
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