OBJECTIVES: To assess the cost-effectiveness of PD-L1 testing associated with Pembrolizumab for first-line treatment of advanced non-small cell lung cancer (NSCLC) from the perspective of the Chinese healthcare system.

METHODS: A three-state partitioned survival model was developed to assess the cost-effectiveness of PD-L1 testing in guiding treatment with Pembrolizumab compared to no PD-L1 testing. In the PD-L1 testing group, patients were stratified by PD-L1 tumor proportion score (TPS) ≥50%, 1-49%, or <1% and received Pembrolizumab monotherapy, Pembrolizumab plus chemotherapy, or chemotherapy alone, respectively. In the non-PD-L1 testing group, all patients received Pembrolizumab plus chemotherapy. Clinical outcomes were derived from two published randomized trials, KEYNOTE-042 Chinese cohort study and KEYNOTE-189. Direct medical costs and utilities were derived from published literature and a healthcare price database. Costs and outcomes were estimated over a lifetime horizon and discounted at 5% annually. The net monetary benefit (NMB) was estimated for the PD-L1 testing group versus the non-PD-L1 testing group. Deterministic and probabilistic sensitivity analyses, and scenario analyses were conducted to assess the robustness of results. One scenario explored a price reduction for Pembrolizumab, consistent with available patient assistance programs and the other scenario modified the PD-L1 testing group so that patients with PD-L1 TPS <1% received Pembrolizumab plus chemotherapy, in accordance with new clinical guidelines in China.

RESULTS: Using PD-L1 testing to guide treatment in patients with advanced NSCLC led to cost savings of $53,914.71 and a reduction in quality-adjusted life years (QALYs) of 0.234, resulting in a positive NMB of $50,973.98 at a willingness-to-pay (WTP) threshold of $12,551.50/QALY (GDP per capita in China, 2021). Findings were robust across sensitivity and scenario analyses.

CONCLUSIONS: These findings suggest that using PD-L1 testing to guide first-line Pembrolizumab treatment in patients with advanced NSCLC is a cost-effective strategy at a WTP threshold of $12,551.50/QALY for China.