ArticleBMJ open2025
Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers.
Article in BMJ open, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04003636 (A Phase 3 Randomized, Double Blind Study of Pembrolizumab Plus Gemcitabine/Cisplatin Versus Placebo Plus Gemcitabine/Cisplatin as First-Line Therapy in Participants With Advanced and/or Unresectable Biliary Tract Carcinoma), which is not on this map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3 Randomized, Double Blind Study of Pembrolizumab Plus Gemcitabine/Cisplatin Versus Placebo Plus Gemcitabine/Cisplatin as First-Line Therapy in Participants With Advanced and/or Unresectable Biliary Tract Carcinoma
Who cites it
3 citing papers in PubMed.
- Cost-effectiveness of pembrolizumab plus trastuzumab and chemotherapy for HER2-positive gastric or gastro-oesophageal junction adenocarcinoma.Human vaccines & immunotherapeutics · 2026Article
- Review
- Design and Optimization of Spiro-Isatin-Thiazolidinone Hybrids with Promising Anticancer Activity.Pharmaceuticals (Basel, Switzerland) · 2025Article
Corrections and comments
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Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe KEYNOTE-966 study demonstrated that pembrolizumab combined with chemotherapy is more effective than chemotherapy alone as first-line treatment for patients with advanced biliary tract cancer (BTC). However, the cost-effectiveness of pembrolizumab combined with chemotherapy in the USA and China remains uncertain.
objectiveThis study aimed to evaluate the cost-effectiveness of pembrolizumab plus chemotherapy compared with placebo plus chemotherapy from the perspective of US and Chinese payers.
designMarkov models with three health states were developed to simulate the process of advanced BTC. Cost data were obtained from available databases and published literature in the US scenario, and from local institutions from the China scenario. Utility values were derived from previous studies. OUTCOME MEASURES: Primary outcomes included quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs).
resultsIn the US scenario, pembrolizumab plus chemotherapy increased costs by US$97,222.13, compared with chemotherapy alone, with a gain of 0.12 QALYs, resulting in an ICER of US$810 184.42 per QALY. In the China scenario, the ICER was $360 933.50 per QALY. Sensitivity analyses indicated the costs of pembrolizumab had the greatest impact on the model in both scenarios. Further analyses suggested that the optimal price of pembrolizumab in the USA would be nearly US$10.33 /mg, while a price reduction of over 90% would be required for the combined therapy to be cost-effective for patients in China.
conclusionBased on the willingness-to-pay threshold set at three times the gross domestic product per capita, pembrolizumab plus chemotherapy is not a cost-effective option for patients with advanced BTC in either the USA or China. Significant price reduction for pembrolizumab may be necessary to achieve an acceptable ICER. TRIAL REGISTRATION NUMBER: NCT04003636; postresults.
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