Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers
Background The 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...
Saved in:
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMJ Publishing Group
2025-04-01
|
| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/15/4/e094047.full |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850042812083470336 |
|---|---|
| author | Can Jiang Pei Shu Kexun Zhou |
| author_facet | Can Jiang Pei Shu Kexun Zhou |
| author_sort | Can Jiang |
| collection | DOAJ |
| description | Background The 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.Objective This study aimed to evaluate the cost-effectiveness of pembrolizumab plus chemotherapy compared with placebo plus chemotherapy from the perspective of US and Chinese payers.Design Markov 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).Results In 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.Conclusion Based 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. |
| format | Article |
| id | doaj-art-ea9f9f8c6b324b1b87a6fefab2d0ae2f |
| institution | DOAJ |
| issn | 2044-6055 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Open |
| spelling | doaj-art-ea9f9f8c6b324b1b87a6fefab2d0ae2f2025-08-20T02:55:25ZengBMJ Publishing GroupBMJ Open2044-60552025-04-0115410.1136/bmjopen-2024-094047Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payersCan Jiang0Pei Shu1Kexun Zhou2Bristol Myers Squibb, Lawrenceville, NJ, USADivision of Thoracic Tumor Multimodality Treatment, Cancer Center, Sichuan University West China Hospital, Chengdu, Sichuan, ChinaDepartment of Immunology, Mayo Clinic in Florida, Jacksonville, Florida, USABackground The 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.Objective This study aimed to evaluate the cost-effectiveness of pembrolizumab plus chemotherapy compared with placebo plus chemotherapy from the perspective of US and Chinese payers.Design Markov 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).Results In 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.Conclusion Based 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.https://bmjopen.bmj.com/content/15/4/e094047.full |
| spellingShingle | Can Jiang Pei Shu Kexun Zhou Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers BMJ Open |
| title | Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers |
| title_full | Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers |
| title_fullStr | Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers |
| title_full_unstemmed | Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers |
| title_short | Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers |
| title_sort | cost effectiveness analysis of pembrolizumab plus chemotherapy as first line treatment for advanced biliary tract cancer perspectives from us and chinese payers |
| url | https://bmjopen.bmj.com/content/15/4/e094047.full |
| work_keys_str_mv | AT canjiang costeffectivenessanalysisofpembrolizumabpluschemotherapyasfirstlinetreatmentforadvancedbiliarytractcancerperspectivesfromusandchinesepayers AT peishu costeffectivenessanalysisofpembrolizumabpluschemotherapyasfirstlinetreatmentforadvancedbiliarytractcancerperspectivesfromusandchinesepayers AT kexunzhou costeffectivenessanalysisofpembrolizumabpluschemotherapyasfirstlinetreatmentforadvancedbiliarytractcancerperspectivesfromusandchinesepayers |