Heliyon (Mar 2025)

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

  • Wei Wang,
  • Lijun Xu,
  • Peilin Li,
  • Yongsheng Li,
  • Jing Wu,
  • Jihua Wang,
  • Shanshan Zhao,
  • Guangbing Li,
  • Shifeng Xu,
  • Huaiguang Chen,
  • Yuanwen Zheng

Journal volume & issue
Vol. 11, no. 6
p. e42553

Abstract

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Background: 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. Methods: 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. Results: 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. Conclusion: 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.

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