Cost-effectiveness analysis of pembrolizumab in combination with chemotherapy compared with chemotherapy alone as first-line treatment for patients with advanced biliary tract cancer in China

Abstract

Objective The objective of this study is to evaluate the cost-effectiveness of adding pembrolizumab to the standard first-line therapy of advanced biliary tract cancer (BTC) with gemcitabine and cisplatin from the perspective of the Chinese healthcare system. Methods The partitioned survival model developed from clinical data obtained in The KEYNOTE-966 trial served as the basis for a simulation in the TreeAge Pro 2011 software. The objective of the research was to estimate the 10-year life expectancy and total healthcare costs of patients with BTC, utilizing primary outcomes that evaluated costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). To establish the willingness-to-pay (WTP) threshold, the 2022 Chinese per capita gross domestic product (GDP) of $37304.346/QALY was adopted. Furthermore, sensitivity analysis was conducted to ascertain the study’s results under varying levels of uncertainty. Results Compared to chemotherapy alone, the addition of pembrolizumab to chemotherapy has been shown to yield an incremental gain of 0.184 quality-adjusted life years (QALY) at an additional cost of $103940.706. This translates into an incremental cost-effectiveness ratio (ICER) of $564895.141/QALY, which exceeds the willingness-to-pay (WTP) threshold in China. One-way sensitivity analyses performed on the model recognize the utility of PD, subsequent cost, and the cost of Pembrolizumab 100 mg had a major influence on the outcomes. However, no parameter elicited an ICER lower than the willingness-to-pay (WTP) threshold. Conclusions Based on the perspective of the Chinese healthcare system, the utilization of pembrolizumab in combination with chemotherapy as an first-line treatment option for BTC does not appear to be a cost-effective approach compared to chemotherapy as a standalone therapy.

Evaluation of Overall Survival by Restricted Mean Survival Time of Advanced Biliary Tract Cancer treated with Immunotherapy: A Systematic Review and Meta-Analysis

“…These considerations are not trivial, and in some cases, the time lost in receiving certain treatment regimens should be balanced with the survival gains offered by treatment [31]. Similarly, the contradictory results of different cost-effectiveness analyses further reinforce the potential benefits of assessing clinical benefits in absolute terms, as offered by RMST [32][33][34][35][36][37].…”

The rise of biliary tract cancers and next‐generation population registries

“…16,17 However, both durvalumab and pembrolizumab are cost-prohibitive and studies show these drugs are not cost-effective first-line treatment options for BTCs by most countries' willingnessto-pay thresholds. 18,19 Recently, the SWOG-1815 trial studied the addition of nab-paclitaxel to the standard gemcitabine and cisplatin regimen but its phase 3 double-arm findings were unable to replicate the success found with its phase 2 single-arm study. 20 The increasing rise in iCCA incidence observed in this study should serve as an impetus for clinicians to enroll patients in clinical trials that can potentially impact iCCA-related mortality with greater urgency.…”