# Cost-effectiveness analysis of pembrolizumab versus chemotherapy in advanced non-small cell lung cancer in China based on real-world studies

## TL;DR

This study evaluates whether pembrolizumab is a cost-effective treatment for advanced lung cancer in China, finding it becomes cost-effective with a drug donation program.

## Contribution

The study provides real-world cost-effectiveness data for pembrolizumab in China, considering the impact of a drug donation program.

## Key findings

- Pembrolizumab improved progression-free survival compared to chemotherapy.
- Without a donation program, pembrolizumab was not cost-effective in China.
- With a donation program, pembrolizumab became cost-effective under certain thresholds.

## Abstract

Although pembrolizumab has been shown to be effective, its high price has prevented it from being widely used. Especially in the real world, the application situation is still uncertain. The purpose of this study was to evaluate the cost-effectiveness of pembrolizumab on the basis of real-world studies, from the perspective of the health care system.

Retrospectively, 630 patients with advanced NSCLC treated with pembrolizumab (monotherapy or combination chemotherapy) versus chemotherapy alone from January 2020 to December 2022 at a large 3 A hospital in China were included. Confounders between groups were eliminated using propensity score matching analysis. A partitioned survival model was developed to evaluate the cost-effectiveness of pembrolizumab versus chemotherapy for the treatment of advanced NSCLC based on progression-free survival, overall survival, and the incidence of adverse effects in the two matched groups (n = 450 patients). The incremental cost-effectiveness ratio was calculated. The impact of a drug donation program on the cost-effectiveness of pembrolizumab was also evaluated.

Pembrolizumab significantly improved median PFS in patients (15.5 months vs. 8.8 months). The median OS in the Pembrolizumab group has not been reached, while it was 26.2 months in the chemotherapy group. When the drug donation program is not considered, the ICER of pembrolizumab is $146,409.07/QALY. Regardless of whether the willingness-to-pay threshold is set at three times the per capita GDP of China ($36,070.2) or three times the per capita GDP of Guangdong Province ($64,523.8), the use of pembrolizumab is not cost-effective. However, after considering the drug donation program, the ICER decreased to $56,127.74/QALY. Under the willingness-to-pay threshold of three times the per capita GDP of Guangzhou in 2022 ($64,523.8), pembrolizumab became a cost-effective choice.

In the treatment of advanced NSCLC in China, pembrolizumab, particularly when considering the drug donation program, offers better survival outcomes and becomes cost-effective. This highlights the importance of such programs in making high-cost treatments accessible in real-world clinical settings.

The online version contains supplementary material available at 10.1007/s00432-025-06242-6.