Cost-effectiveness analysis of pembrolizumab combined with gemcitabine and cisplatin for the first-line treatment for advanced cholangiocarcinoma

<h2>Abstract</h2><h3>Background</h3> This study aims to assess the economic feasibility of incorporating pembrolizumab in combination with gemcitabine and cisplatin (GC) as the first-line therapy for patients with advanced cholangiocarcinoma (CCA) from the perspective of payers in the USA. <h3>Methods</h3> A Markov model was developed to evaluate the cost-effectiveness of KEYNOTE-966. The model incorporated treatment effectiveness, event probabilities, and utilities. Expenses were determined based on data from published sources and nationwide surveys. The primary outcomes were measured in terms of quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were performed to evaluate the uncertainty associated with the model. <h3>Results</h3> The combination of pembrolizumab and GC yielded a significant cost increase of $60,963.53 and an additional 0.090 QALYs compared to GC alone. Consequently, the ICER reached $677,373 per QALY, surpassing the acceptable threshold of $150,000.00/QALY. Within the model, the hazard ratio of OS (0.86; ranging from 0.72 to 0.95) and the price of pembrolizumab ($6914.46, ranging from 5531.57 to 8297.35) emerged as the most influential factors. To achieve cost-effectiveness, the cost of pembrolizumab had to be decreased by 77.9 % to $1531 per cycle. <h3>Conclusion</h3> The utilization of pembrolizumab and GC was not a cost-effective strategy for the first-line systemic treatment of advanced CCA, considering a willingness to pay limit of $150,000.00 per QALY.