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OBJECTIVE To evaluate the cost-effectiveness of tislelizumab monotherapy in the second-line treatment of advanced or metastatic esophageal squamous cell carcinoma （ESCC），so as to provide reference for rational use of drug in clinic. METHODS A three-state partitioned survival model was constructed from the perspective of China’s health system， based on the data of RATIONALE-302 study，with simulation time limit of 10 years， cycle period of 1 month. The incremental cost-effectiveness ratio （ICER） was calculated with quality-adjusted life year （QALY） as utility index. The cost-effectiveness of tislelizumab monotherapy was compared with that of chemotherapy for second-line treatment of advanced or metastatic ESCC by cost-utility analysis. The stability of basic analysis results was validated through sensitivity analysis and scenario analysis. RESULTS The results of basic analysis showed that compared with chemotherapy group， incremental cost per capita of tislelizumab group was 35 025.32 yuan，and incremental utility per capita was 2.71 QALYs； ICER was 12 892.31 yuan/QALY， which was far lower than the willingness-to-pay （WTP） threshold of 3 times of China’s per capita gross domestic product （GDP） 242 928 yuan in 2021. The results of univariate sensitivity analysis showed that parameters such as the cost of apatinib， the utility value of disease progression status and the cost of adverse reactions in the chemotherapy group had a great impact on the ICER value， but these parameters could not cause the reversal of the basic analysis results. Probabilistic sensitivity analysis showed that WTP threshold was higher than 80 000 yuan/QALY，the probability of tislelizumab monotherapy possessed cost-effectiveness was 100%. Results of scenario analysis showed that in which model simulation time lasted for 5 or 20 years，ICER of tislelizumab was 8 331.00 yuan/QALY and 12 981.00 yuan/QALY， which were less than 3 times of China’s per capita GDP in 2021 as WTP threshold. CONCLUSIONS When three times of China’s GDP per capita in 2021 is taken as the WTP threshold， the second-line treatment of tirelizumab monotherapy for advanced or metastatic ESCC is more cost-effective than chemotherapy.
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OBJECTIVE To evaluate the cost-effectiveness of sugemalimab in patients with stage Ⅲ non-small cell lung cancer （NSCLC） whose disease had not progressed after concurrent or sequential chemoradiotherapy from the perspective of the Chinese healthcare system. METHODS Based on the GEMSTONE-301 clinical trial，a three-health state partitioned survival model （PartSA） was developed to simulate the progression of disease，with model cycle of 3 weeks and a lifetime time range； the main output indicators of the model were total cost，quality-adjusted life year （QALY），and incremental cost-effectiveness ratio （ICER）； the cost and health output were discounted using 5% discount rate. Using 1-3 times China’s per capita gross domestic product （GDP） in 2022 as the willingness-to-pay （WTP） threshold，the cost-utility analysis method was used for analysis，and sensitivity analysis and scenario analysis were conducted to assess model robustness. RESULTS Compared with placebo，ICER for sugemalimab consolidation therapy was 59 872.57 yuan/QALY，which was less than one time China’s per capita GDP in 2022 （85 698 yuan） as the WTP threshold. The scenario analysis results further confirmed the robustness of the model. The results of single factor sensitivity analysis indicated that the cost of drugs （pembrolizumab，sugemalimab，docetaxel） and the utility value of disease progression status had a significant impact on ICER； the results of the probabilistic sensitivity analysis showed that sugemalimab had 67.2% probability of being cost-effective at one time China’s GDP per capita in 2022 as the WTP threshold； when 20 000 yuan （less than 3 times GDP per capita in China of 257 094 yuan） was used as the WTP threshold，the probability of sugemalimab consolidation therapy being cost-effective was greater than 98%. CONCLUSIONS The consolidation therapy with sugemalimab is cost-effective for stage Ⅲ NSCLC whose disease had not progressed after concurrent or sequential chemoradiotherapy.
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OBJECTIVE To evaluate the economics of serplulimab combined with chemotherapy regimens for the first-line treatment of extensive-stage small cell lung cancer （ES-SCLC） from the perspective of health system in China. METHODS A partitioned survival model was constructed based on the ASTRUM-005 clinical trial and related literature data， with a model simulation time frame of 10 years and a 3-week cycle， and both cost and utility values were discounted using a 5% discount rate. The quality-adjusted life year （QALY） was used as a model output indicator and the incremental cost-effectiveness ratio （ICER） was calculated to evaluate the economics of serplulimab combined with chemotherapy regimens （serplulimab group） versus chemotherapy alone regimens （chemotherapy alone group） for the first-line treatment of ES-SCLC. One-way sensitivity analysis and probabilistic sensitivity analysis were used to verify the robustness of the results of the base-case analysis and to conduct a scenario analysis for the serplulimab patient assistance program. RESULTS The results of the base-case analysis showed that compared with chemotherapy alone group， ICER of serplulimab group was 758 690.27 yuan/QALY， which was higher than 3 times China’s per capita gross domestic product （GDP） in 2022 as the willingness-to-pay （WTP） threshold. The results of the scenario analysis showed that compared with chemotherapy alone group， the ICER of serplulimab group was 172 275.74 yuan/QALY， which was below above WTP threshold. The one-way sensitivity analysis showed that the progress-free survival utility value， serplulimab price and so on had a significant impact on the model results. The results of the probabilistic sensitivity analysis showed that the probability of the serplulimab group being economic was 0 when the serplulimab patient assistance program was not considered， but 100% when the patient assistance program was considered. CONCLUSIONS At a WTP threshold of 3 times China’s per capita GDP in 2022， the serplulimab group is no cost-effectiveness compared to the chemotherapy alone group； however， this result is reversed when the patient assistance program is taken into account.
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OBJECTIVE To evaluate the cost-effectiveness of pembrolizumab combined with chemotherapy versus placebo combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma （BTC） from the perspective of China’s health system. METHODS A partitioned survival model was constructed based on the KEYNOTE-966 study data. The simulation period was 21 days， and the simulation time was the patient’s whole life. Using quality-adjusted life year （QALY） as the output indicator， the cost-utility analysis method was used to evaluate the cost-effectiveness of the two schemes mentioned above. Univariate and probabilistic sensitivity analyses were performed to verify the results of the basic analysis， and to explore the cost-effectiveness under the scenario of drug donation scheme. RESULTS The basic analysis showed that both the cost and effectiveness of the pembrolizumab group were higher than those of the placebo group， and the incremental cost-effectiveness ratio （ICER） was 3 909 359.78 yuan/QALY， which was higher than the willingness-to-pay （WTP） threshold of 3 times 2022 gross domestic product （GDP） per capita （257 094 yuan）， indicating no cost-effectiveness. The results of univariate sensitivity analysis showed that the utility discount rate， the utility value of progression-free survival （PFS） status， the cost discount rate， and the cost of pembrolizumab had a great influence on ICER. Probabilistic sensitivity analysis verified the robustness of the results of basic analysis， and concluded that when the WTP threshold was greater than 1 500 000 yuan/QALY， the pembrolizumab group became cost-effective. The results of the scenario analysis showed that considering the drug donation scheme of pembrolizumab for low-income people， although its treatment cost was significantly reduced， it was still not cost-effective. CONCLUSIONS At the WTP threshold of 3 times China’s GDP per capita in 2022， pembrolizumab combined with chemotherapy is not cost-effective compared with placebo combined with chemotherapy for advanced or unresectable BTC.
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OBJECTIVE To evaluate the cost-effectiveness of Ferric carboxymaltose injection in the treatment of iron deficiency anemia in adult Chinese patients. METHODS From the perspective of China’s health system， the partitioned survival model was constructed to simulate the treatment process and outcome of patients with iron deficiency anemia using ferric carboxymaltose and iron sucrose based on the ferric carboxymaltose phase Ⅲ clinical trial in China （NCT03591406）. The study period was 1 year. Total costs and quality-adjusted life years （QALYs） were calculated for both treatment regimens， and incremental cost-effectiveness ratios were calculated. Scenario analysis was performed with different assumptions for efficacy parameters between 9th week and the end of 1 year， and from the perspective of China’s health system and the entire society. The sensitivity analysis was also performed. RESULTS In the basic analysis， compared with iron sucrose， incremental effectiveness of ferric carboxymaltose therapy was 0.007 QALYs， with an additional savings of 1 038 yuan per patient. Ferric carboxymaltose therapy was more effective and less costly， presenting an absolute advantage. The results of the scenario analysis and single-factor sensitivity analysis were consistent with the basic analysis. Probability sensitivity analysis showed that when the willingness-to-pay threshold was 1， 2， 3 times gross domestic product per capita in 2022， the probability of ferric carboxymaltose with a cost- effectiveness advantage was 88.2%，94.5% and 97.6%，respectively. CONCLUSIONS For Chinese adults with iron deficiency anemia， ferric carboxymaltose is a cost-effective treatment for iron deficiency anemia， compared with iron sucrose.
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OBJECTIVE To evaluate the cost-effectiveness of nivolumab combined with ipilimumab in the first-line treatment of unresectable malignant pleural mesothelioma （MPM）. METHODS From the perspective of healthcare system ，a partitioned survival model were developed ，the cost-effectiveness of nivolumab combined with ipilimumab （dual-immunotherapy plan ）versus chemotherapy in the first-line treatment of unresectable MPM by cost-utility analysis. Clinical trial data were collected from CheckMate 743 study. Direct medical cost included drug costs ，disease management cost and cost of treatment of adverse reactions. Costs and utilities were discounted at an annual rate of 5％. The willingness to pay threshold was 3 times of gross domestic product （GDP）per capita in 2021 [242 928 yuan/QALY（quality-adjusted life year ）]. Scenario analysis was used to analyze and compare the two regimens under the scenario of complimentary drug for patients in dual-immunotherapy group. The robustness of the findings was evaluated by one-way sensitivity analysis and probabilistic sensitivity analysis. RESULTS Baseline analysis results showed that total cost of dual-immunotherapy regimen was higher than that of chemotherapy regimen ，and the utility was also better than that of chemotherapy plan ；the incremental cost-effectiveness ratio （ICER）was 417 122.2 yuan/QALY，which was higher than the willingness to pay threshold ；the dual-immunotherapy regimen was not cost-effective compared to the chemotherapy regimen. Under the scenario of complimentary drug ，the cost of dual-immunotherapy was 327 454.5 yuan，ICER was 75 664.1 yuan/QALY，which was lower than the willingness to pay threshold and resulted in a reversal of the baseline analysis. One-way sensitivity analysis showed that under the health states of progression free survival and progressive disease ，utility value and the price of nivolumab had a greater impact on the ICER value. Probabilistic sensitivity analysis showed that the results of baseline analysis were robust. CONCLUSIONS At a 163.com willingness to pay threshold of 3 times of GDP per capita in nivolumab combined with ipilimumab is not cost-effective compared with chemotherapy regimen in the first-line treatment of unresectable MPM. However ，if patients receive complimentary drugs ，the dual-immunotherapy regimen is cost-effective.
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OBJECTIVE To evaluate the econ omical efficiency of daratumumab combined with lenalidomide and dexamethasone （D-Rd） regimen versus lenalidomide and dexamethasone （Rd） regimen alone in the treatment of transplant- ineligible newly diagnosed multiple myeloma （TNE-NDMM）. METHODS From the perspective of China ’s health system ，a partitioned survival model with three health states of progression free survival ，disease progression and death was established by using the published MAIA test data and relevant literature data. The model cycle was 28 days and the simulation time limit was 20 years. The incremental cost-effectiveness ratio （ICER）was calculated using quality-adjusted life years （QALY）as the output index. Sensitivity analysis was performed for key parameters. RESULTS The results of basic analysis showed that the ICER of D-Rd regimen versus Rd regimen was 2 719 038.08 yuan/QALY，far exceeding 3 times of GDP per capita in 2021（242 928 yuan）. The results of single factor sensitivity analysis showed that cost discount rate ，progression-free survival utility value ，utility discount rate，the cost of daratumumab and lenalidomide had a greater impact on ICER. Probabilistic sensitivity analysis suggested that the probability of economic advantage of D-Rd regimen was always 0 within the WTP range of 0-1 200 000 yuan. CONCLUSIONS Compared with Rd regimen ，D-Rd regimen has no cost-effectiveness advantage for the treatment of TNE-NDMM under the WTP of 3 times GDP per capita of China .
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OBJECTIVE To ev aluate the economical efficiency of nivolumab versus everolimus in the second-line treatment of metastatic clear cell renal cell carcinoma. METHODS From the perspective of China ’s health system ，cost-effectiveness analysis of the two therapies was carried out by developing a three-state partitioned survival model. The clinical parameters were from the updated CheckMate 025 study，and the cost and health utility were from relevant websites and published literatures. The model adopted a 2-week cycle and a lifetime research time. The robustness of the results was verified by sensitivity analysis. The economical efficiency of two therapy schemes were evaluated in the scenario of model simulation time of 80 months and charitable drug donation scheme. RESULTS The results of basic analysis showed that compared with everolimus ，the incremental cost-effectiveness ratio （ICER）of nivolumab was 586 982.60 yuan/quality-adjusted life year （QALY），which was far higher than 3 times of China ’s per capita gross domestic product （GDP）in 2020. The results of single-factor sensitivity analysis showed that the 3 parameters that had the greatest impact on the economic evaluation results were the cost of nivolumab ，the utility value of nivolumab group and everolimus group in progressive disease state. The results of probability sensitivity analysis verified the robustness of the basic analysis results. Results of scenario analysis showed that in the first scenario analysis ，in which model simulation time lasted for 80 months，ICER of nivolumab was 417 204.52 yuan/QALY；in the second scenario analysis ，in which nivolumab charitable drug donation program for low-income people was considered ，ICER of nivolumab was 124 988.58 yuan/QALY. CONCLUSIONS Under the threshold of 1-3 times of China ’s per capita GDP in 2020，compared with everolimus ，it is not economical to use nivolumab as the second-line treatment for metastatic clear cell renal cell carcinoma ； nivolumab is economical when considering its charitable drug donation program for low-income people.