1.Pharmacoeconomic studies of transfusion-dependent β thalassemia:systematic review
Junni DU ; Yan LI ; Pingyu CHEN ; Aixia MA
China Pharmacy 2025;36(19):2415-2421
OBJECTIVE To systematically evaluate pharmacoeconomic studies on treatment strategies for transfusion- dependent β thalassemia (TDT) both in China and internationally, to inform clinical decision-making and health resource allocation, and provide methodological references for future domestic research in China. METHODS Databases including PubMed, Embase, Web of Science, the Cochrane Library, CNKI, Wanfang Data, and VIP were searched for pharmacoeconomic studies related to TDT treatments from inception to December 2024. Two researchers independently screened the literature, extracted data, and assessed risk of bias using the Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 2022) checklist. A qualitative systematic review was conducted focusing on model structure, costs, health outcomes, uncertainty analysis, and economic evaluation results. RESULTS A total of 1 685 articles were initially identified, and 13 pharmacoeconomic studies were ultimately included. The overall quality of the included studies was relatively high, covering interventions including blood transfusion and iron chelation therapy (BT-ICT), hematopoietic stem cell transplantation (HSCT), and gene therapy. All studies employed cost-utility analysis from societal, healthcare system, and payer perspectives. Markov models were most frequently used in model design. Most studies adopted a lifetime horizon with a cycle length of one year and a discount rate of 1.5% to 5%. Costs mainly focused on direct medical costs, sourced from national insurance databases and published literature. Utility values were primarily derived from literature or measured using techniques such as time trade-off. Most studies conducted both deterministic and probabilistic sensitivity analyses, identifying the costs of iron chelators and utility values as key influential parameters. Some studies also performed scenario analyses. CONCLUSIONS HSCT was likely more cost-effective than BT-ICT for pediatric/ adolescent patients. BT-ICT demonstrated superior cost-utility for adult patients, with the deferiprone regimen potentially being the most cost-effective option. Current domestic and international pharmacoeconomic evaluations for TDT remain limited. Future research should adopt a societal perspective, use a lifetime horizon, and strictly follow the CHEERS 2022 checklist when conducting pharmacoeconomic evaluations of treatments for TDT.
2.Construction and Application of an Intelligent Health Insurance Development Level Evaluation System Based on the Delphi-Entropy Method
Yuxin YE ; Wenxi TANG ; Shuailong LI ; Qian XING ; Mingyang LI ; Renchang DIAO ; Aixia MA
Chinese Hospital Management 2024;44(2):1-5
Objective It aims to construct an evaluation index system for the development level of intelligent health insurance,which can serve as a reference for health insurance management departments in assessing the develop-ment level of intelligent health insurance and the implementation of health insurance informatization.Methods Key events in intelligent health insurance were identified based on event system theory and text analysis.The evaluation index system was determined through a combination of expert interviews and Delphi expert consultations.The entro-py method was used to calculate the weights of each index,followed by the assessment of the current and ideal de-velopment levels.Results A total of 16 experts were consulted.After two rounds of Delphi expert consultation,two first-level indicators and 18 second-level indicators were finally included in the system.The current development level of intelligent health insurance in China is at the intelligent development stage(2.524 points),while the ideal de-velopment level is at the intelligent improvement stage(4.073 points).The positivity coefficient of both rounds of Del-phi expert consultation was 100%,with an authority coefficient of 0.842,and the degree of expert coordination im-proved with each round.Conclusion The constructed evaluation index system exhibits high scientificity,stability,and generalizability.It can provide an effective evaluation tool for the development of intelligent health insurance in various pooled areas.
3.Research on the Resilience of Local Health Systems Based on Information Platforms:a Case Study of the Epidemic Prevention Material Procurement and Allocation Hall in Nanjing
Yuxin YE ; Wenxi TANG ; Shuailong LI ; Qian XING ; Renchang DIAO ; Aixia MA
Chinese Hospital Management 2024;44(2):11-14
It dified framework of health system resilience analysis.The research integrates practical elements from the case of the online pandemic material procurement and allocation hall in Nanjing,categorizing the resilience-building of local health systems via informatization into two distinct dimensions:static foundation and dynamic endowment.It conducts an in-depth examination of the logical pathways that leverage informatization to bolster resilience,and further investigates the inherent advantages and potential areas for optimization within informatization.The findings suggest that the synergistic empowerment of both static foundation and dynamic endowment effectively amplifies the risk defense capability and resilience of local health systems.
4.A Study on Collaborative Development and Governance Information Mechanism of the Tripartite System Based on the SFIC Model
Yuxin YE ; Wenxi TANG ; Shuailong LI ; Qian XING ; Renchang DIAO ; Aixia MA
Chinese Hospital Management 2024;44(2):15-19
Information collaboration is an important realisation path to deepen the reform of the medical and health system and to promote the collaborative development and governance of the"Three Medicine"during the"14th Five-Year Plan"period.It employs the SFIC model and makes appropriate modifications to it.The analytical framework comprises six elements:"initial conditions-external environment-catalytic leadership-institutional de-sign-collaborative process-results feedback".This framework is used to dissect the current collaborative dilemma in the"Three Medicine"information collaboration and governance.Based on this analysis,an optimized path for infor-mation collaborative governance is proposed:consolidating the foundation of"Three Medicine"information collabora-tion,enhancing the catalytic leadership capability of meta-governance,optimizing the institutional design of informa-tion life-cycle governance,reshaping the information collaboration process,and focusing on the evaluation feedback mechanism.
5.Cost-effectiveness analysis of ferric carboxymaltose for treating iron deficiency anemia in adult Chinese patients
Wentao SUN ; Lingxiang ZHUANG ; Aixia MA
China Pharmacy 2023;34(22):2750-2755
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.
6.Application of pectoralis major flap in complex defects after maxillofacial " frozen neck" surgery
Jingjian WEI ; Hao ZHENG ; Kun HAN ; Xiaodong LI ; Yanhong MA ; Aixia ZHANG ; Jian MENG
Journal of Chinese Physician 2023;25(10):1516-1519
Objective:To summarize the application of pectoralis major flap in complex defects after maxillofacial " frozen neck" surgery.Methods:The clinical data of 7 patients with maxillofacial " frozen neck" admitted to the Department of Stomatology of the Xuzhou Central Hospital from October 2020 to October 2022 were retrospectively analyzed. The pectoralis major flap was used to repair the complex defects after surgery and the treatment effect was observed.Results:All 7 patients had survived flap transplantation with no serious complications. After 3 to 24 months of follow-up, the patients were basically satisfied with the treatment effect.Conclusions:The pectoralis major flap has reliable blood supply, abundant tissue, and can be applied flexibly with a high survival rate and significant repair effect. It is a good choice for repairing complex defects after " frozen neck" surgery, and it has clinical application value.
7.Constructing an evaluation system for hierarchical management of clinical nurse in top three hospitals based on three-dimensional structure theory of medical quality
Yulian SUN ; Yujie MA ; Shuling SI ; Ying GE ; Qianqian YUAN ; Chunlan LIU ; Aixia MA
Chinese Journal of Practical Nursing 2022;38(23):1810-1816
Objective:To provide a reference for the hierarchical management of clinical nurses in top three hospitals, the hierarchical management evaluation system was conducted and evaluated in the present study.Methods:On the context of three-dimensional structure theory of medical quality, the Delphi method was applied to conduct two rounds of correspondence consultation among 23 nursing experts, and then related indexes were established based on the evaluation results and recommendations.Results:An evaluation system for hierarchical management of clinical nurse in top three hospitals was finally constructed, which included 3 first-grade and 19 second-grade indexes. The effective recovery rate of the two rounds of expert questionnaires and authority Cr of two rounds of expert consultation were 100% and 0.915, respectively. The Kendall coefficients of concordance for significance and operability of the first-grade and second-grade indexes were 0.353/0.515 and 0.344/0.469, respectively ( P<0.01). Conclusions:The hierarchical management of clinical nurses in top three hospitals constructed based on three-dimensional structure theory has reliability and feasibility.
8.Pharmacoeconomic evaluation of ensatinib in the first-line treatment of anaplastic lymphoma kinase-positive advanced non-small cell lung cancer
Lei SUN ; Dachuang ZHOU ; Pingyu CHEN ; Aixia MA
China Pharmacy 2022;33(12):1479-1484
OBJECTIVE To evaluate the econo mical efficiency of ensatinib i n the first-line treatment of anaplastic lymphoma kinase(ALK)positive-advanced non-small cell lung cancer (NSCLC),so as to provide reference for China ’s medical insurance decision-making and rational drug use in clinic. METHODS A three-state partitioned survival model was constructed from the perspective of China ’s health system ,based on the data of the international multi-center phase Ⅲ clinical trial (eXalt3 trail),with simulation time limit of 10 years,cycle period of 30 days. The economical efficiency of ensatinib was compared with that of crizotinib(standard treatment )in the first-line treatment of ALK positive-advanced NSCLC. The incremental cost-effectiveness ratio (ICER) was calculated with quality-adjusted life years (QALYs) as utility index. The stability of basic analysis results was validated through uncertainty analysis. RESULTS The basic analysis results showed that compared with crizotinib group , incremental cost per capita of ensatinib group was -343 370.36 yuan,incremental utility per capita was 0.76 QALYs,ICER was -454 292.25 yuan/QALY,which was far lower than the willingness-to-pay (WTP)threshold of 1 time of China ’s per capita gross domestic product (GDP,80 976 yuan)in 2021. The results of univariate sensitivity analysis showed that progression-free survival (PFS)status utility ,progression of disease (PD)status utility and subsequent treatment cost of crizotinib had a greater impact on ICER,but these parameters could not cause the reversal of basic analysis results. Probabilistic sensitivity analysis showed that with 1 time of China ’s per capita GDP in 2021 as the WTP threshold ,the probability of ensatinib group ’s treatment possessed economical efficiency was 100%. In the situational analysis ,ICER obtained by changing the ensatinib group ’s follow-up treatment regimen was -217 671.43 yuan/QALY,which was far below WTP threshold. CONCLUSIONS For Chinese patients with ALK positive-advanced NSCLC ,compared with commonly used the first-line treatment (crizotinib),ensartinib is economical and absolutely dominant.
9.Pharmacoeconomic evaluation of loratinib in the first-line treatment of anaplastic lymphoma kinase-positive advanced non-small cell lung cancer
Lei SUN ; Pingyu CHEN ; Aixia MA
China Pharmacy 2022;33(9):1102-1108
OBJECTIVE To eva luate the economy of loratinib versus crizo tinib in the first-line treatment of anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC)from the perspective of China ’s health system , and to provide reference for the product pricing and related medical decisions of the drug in other regions of China except for Hong Kong. METHODS Markov model and partition survival model both constructed based on the CROWN data (the simulation time limit was 10 years and the cycle period was 4 weeks);the quality adjusted life year (QALY)was used as the outcome index to calculate the incremental cost-effectiveness ratio (ICER). One-way sensitivity analysis ,probability sensitivity analysis and scenario analysis were used to verify the robustness of the results. RESULTS The basic analysis results based on Markov model showed that compared with crizotinib group ,the per capita cost of loratinib group increased by 17 867 588.63 yuan,the per capita utility increased by 1.76 QALYs,and the ICER was 10 152 038.99 yuan/QALY. The basic analysis results based on the partition survival model showed that compared with the crizotinib group ,the Δ 基金项目:江苏省博士后科研资助计划项目(No.2021K496C); per capita cost of loratinib group increased by 18 009 592.54 2020年度高校哲学社会科学研究一般项目(No.2020SJA0070) yuan,the per capita utility increased by 1.74 QALYs,and the *硕士研究生 。研究方向 :药物经济学 、卫生经济与政策 。 E-mail:sunlei_cpu@163.com ICER was 10 350 340.54 yuan/QALY. The results of one-way # 通信作者:教授,博士生导师。研究方向:药物经济学、卫生经 sensitivity analysis of the two models both showed that 济与政策。E-mail:ma86128@sina.com progression-free survival (PFS)state utility v alue,progression- ·1102· China Pharmacy 2022Vol. 33 No. 9 中国药房 2022年第33卷第9期 disease(PD)state utility value and loratinib cost had great influence on the results. The results of probability sensitivity analysis showed that when 1-3 times of China ’s per capita GDP in 2020 was taken as the willingness to pay threshold ,the probability of loratinib being economical was 0. The recommended unit price of loratinib per 100 mg was 657.10-815.60 yuan. CONCLUSIONS For patients with ALK-positive advanced NSCLC ,loratinib is more effective than crizotinib in the first-line treatment ,but it is not economical under the current price ;reasonably lowering the price of loratinib can increase the probability of its economy.
10.Cost-effectiveness analysis of nivolumab in the second-line treatment of metastatic clear cell renal cell carcinoma
Yingdan CAO ; Fenghao SHI ; Rui MENG ; Pingyu CHEN ; Zhengyang FEI ; Xueke ZHANG ; Aixia MA
China Pharmacy 2022;33(10):1233-1239
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.

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