With the continuous progress of national medical insurance strategic purchasing and value-based healthcare, pharmacoeconomic evaluation, serving as a technical tool for assessing the cost-effectiveness of healthcare interventions, has played an important role in policy decision support. Comparative efficacy evidence is the core data source for pharmacoeconomic evaluation, and also the foundation for conducting pharmacoeconomic research. In recent years, the number of innovative drugs approved based on single-arm trial has been increasing. Most existing randomized controlled clinical trials are also placebo-controlled or compared with traditional treatments, unable to directly meet the need for efficacy evidence of comparisons with conventional or standard treatments in pharmacoeconomic evaluations. In the absence of direct comparative efficacy evidence, exploring indirect comparison methods for efficacy has become a cutting-edge direction in pharmacoeconomic evaluation. Through a comprehensive literature review and systematic analysis, this study focuses on five indirect comparison methods based on individual patient data for population adjustment, including match adjusted indirect comparison (MAIC), simulated treatment comparison (STC), propensity score matching (PSM), inverse probability of treatment weighting (IPTW) and network meta regression (NMR), and discussing their basic concepts, advantages and disadvantages and application comparisons. Finally, it provides methodological suggestions on how to choose an indirect comparison method for efficacy, with the aim of promoting the generation of higher-quality indirect comparison evidence for efficacy and advancing pharmacoeconomic evaluation to provide high-quality evidence references for healthcare policy decision-making.
Objective To evaluate the cost-effectiveness of chemotherapy in children with newly diagnosed Hodgkin lymphoma at low-, intermediate-, and high-risk. Methods From the perspective of health system, a decision-tree model was designed for cost-effectiveness analysis. The chemotherapy regimens of low-risk group included OEPA (vincristine, etoposide, prednisone, doxorubicin), AV-PC (doxorubicin, vincristine, prednisone, cyclophosphamide), and ABVD (doxorubicin, bleomycin, vincristine, dacarbazine); intermediate-risk group included OEPA, ABVE-PC (doxorubicin, bleomycin, vincristine, etoposide, prednisone, cyclophosphamide) and ABVD; high-risk group included OEPA, ABVE-PC, ABVD and BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, prednisone). The effectiveness and cost parameters were derived from the event-free survival rate reported in the literature, the drug linked reference price in Sichuan province, and treatment price of medical institutions. Univariate and probabilistic sensitivity analysis were performed to explore the impact of uncertainty. Results In the low-risk group, compared with AV-PC, the incremental cost-effectiveness ratios (ICER) of OEPA and ABVD were 80 700 yuan and 108 799 yuan, respectively. In the intermediate-risk group, compared with OEPA, the ICER of ABVE-PC and ABVD were −17 737 yuan and −4 701 yuan respectively. In the high-risk group, compared with ABVE-PC, the ICER of OEPA, ABVD and BEACOPP were 149 262, 472 090 and 64 652 yuan, respectively. Univariate sensitivity analysis showed that in low-risk group, the most influential factors were cost of OEPA and cost of ABVD; in moderate-risk group were cost of ABVE-PC and cost of OEPA; in the high-risk group were cost of OEPA, cost of ABVD, and cost of BEACOPP, respectively. The results of probabilistic sensitivity analysis are basically consistent with those of the main analysis. Conclusion If China's per capita gross domestic product in 2023 (89 358 yuan) was used as the willingness-to-pay (WTP) threshold, OEPA in the low-risk group, OEPA in the intermediate-risk group and BEACOPP in the high-risk group are cost-effective.
Objective To systematically evaluate the quality of published pharmacoeconomics studies on Chinese patent medicines for neoplasms. Methods Datasets including CNKI, WanFang Data, VIP, SinoMed, PubMed, Web of Science, Cochrane Library, and EMbase were searched to collect pharmacoeconomics studies of Chinese patent medicines in neoplasms from the establishment of the database to September 30, 2022. Consolidated health economic evaluation reporting standards 2022 (CHEERS 2022) and quality of health economics studies (QHES) were used to evaluate the reporting quality and methodological quality. Results A total of 25 studies were included, with an average CHEERS 2022 coincidence rate of 40.09% and an average QHES score of 53.2. Conclusion The quantity and quality of pharmacoeconomics studies on Chinese patent medicines in neoplasms are insufficient and flawed. It is suggested to strenthen the collaboration between scientific research institutions and hospitals and standardize the pharmacoeconomics studies on Chinese patent medicines in neoplasms to provide hygienic decision-making evidence for Chinese patent medicines in neoplasms.
ObjectiveTo introduce economic evaluation methods for anticancer-drugs with basket trial design, and to provide references for related research and decision-making. MethodsA case analysis was conducted on economic evaluation methods for anticancer-drugs with basket trial design, which was issued by Canadian Agency for Drugs and Technologies in Health (CADTH) in the Economic Guidance Report. Moreover, both the advantages and disadvantages of the methods were analyzed in accordance with the characteristics of basket trials. ResultsPooled analysis and tumor-specific analysis were two methods frequently employed in the case analysis. However, great uncertainties were available in both of them. The uncertainty of the former was mainly reflected in the heterogeneity of the targeted population, while the uncertainty of the latter was mainly shown in the insufficient sample size of the subgroup. ConclusionCurrently, economic evaluation methods for anticancer-drugs with basket trial design are immature. Thus, researchers are required to explore the methods of innovation evaluation with lower uncertainty; reimbursement decision-makers should fully consider the uncertainty of evaluation results and enterprises should collect the real-world data for the demands of evaluation to promote the reasonable allocation of healthcare resources in China.
ObjectiveTo systematically review the pharmacoeconomics of high-dose intravenous iron ferric carboxymaltose in the treatment of patients with iron deficiency anemia. MethodsPharmacoeconomic studies of ferric carboxymaltose in the treatment of patients with iron deficiency anemia were searched in PubMed, The Cochrane Library, York University CRD, Web of Science, EBSCO, CNKI, WanFang Data and VIP databases, and relevant health technology assessment websites from inception to September 30th, 2021. A descriptive analysis was performed after two reviewers independently screened the literature, extracted data, and assessed the risk of bias of the included studies. ResultsA total of 11 studies were included, most of them compared the health economics of ferric carboxymaltose with other therapies from a hospital perspective. The main costs included in these studies were costs for iron, infusion, blood transfusion, EPO, hospitalization, and transportation, as well as productivity cost. The ferric carboxymaltose was presumed to be more economical than other intravenous irons. ConclusionIt is suggested that the ferric carboxymaltose be considered in more clinical settings to improve the ischemic condition of patients with iron deficiency anemia, so as to promote the rational utilization of medical resources.
Objective To systematically evaluate the pharmacoeconomic vaule of chemotherapy combined with rituximab for patients with non-Hodgkin’s lymphomas (NHL). Methods A systematic literature search of cost-effectiveness studies on rituximab treating NHL published from 1998 to 2012 was carried out in following databases: PubMed, ScienceDirect, Health Technology Assessment (HTA) and Cochrane Database of Systematic Reviews (CDSR). And the references of included studies were also retrieved manually. The studies were screened according to the pre-designed inclusion and exclusion criteria, and the incremental cost- effectiveness ratio (ICER) in comparison between chemotherapy plus rituximab and chemotherapy alone was systematically evaluated according to the literature evaluation index system. Results The average ICER of Rituximab treating NHL was 16 318/QALY, 17 688/QALY, and 22 461/QALY in the UK, Mainland Europe, and US, respectively. All the reported ICERs in the included studies were below the implemented country-specific thresholds. Conclusion Based on present foreign literature, the integrated therapy of chemotherapy and rituximab for NHL is supposed to be a better cost-effective therapy with ICER below the implemented country-specific thresholds.
ObjectiveTo systematically review the pharmacoeconomics research of coagulation factor Ⅷ for the treatment of hemophilia A. MethodsPubMed, EMbase, Web of Science, The Cochrane Library, CNKI, VIP and WanFang Data databases were electronically searched to collect pharmacoeconomic studies of coagulation factor Ⅷ for the treatment of hemophilia A from inception to February 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, qualitative systematic review was carried out from the aspects of research model, research parameters and uncertainty analysis. ResultsA total of 17 pharmacoeconomic studies were included. The overall quality of the included literature was relatively high, and most of them conformed to the basic framework of pharmacoeconomic research; however, there were still differences and deficiencies in model setting and parameter selection. Most results of the study evaluation showed that prophylaxis of coagulation factor Ⅷ had cost-effectiveness advantages over on-demand treatment. ConclusionCurrent evidence shows that the preventive treatment of coagulation factor Ⅷ may have certain cost-effectiveness advantages compared with on-demand treatment; however, the adaptability of this conclusion to China still needs to be analyzed.
ObjectiveTo systematically evaluate pharmacoeconomic studies on Programmed cell death-1/Programmed cell death-ligand 1 (PD-1/PD-L1) inhibitors for advanced gastric cancer (GC) globally, providing evidence for healthcare policy formulation and clinical decision-making. MethodsWe conducted electronic searches in PubMed, Cochrane Library, Web of Science, ScienceDirect, Embase, CNKI, WanFang Data, and VIP databases, retrieving relevant literature published from inception to February 2025. Two researchers independently screened the literature, extracted data, and assessed study quality using the CHEERS 2022 checklist. We systematically summarized and analyzed the basic characteristics, model structures, methodological approaches, and economic outcomes of the included studies through inductive analysis. ResultsA total of 15 studies were included, with overall good quality. All included studies employed cost-utility analyses, among which 9 utilized partitioned survival models and 6 adopted Markov models. Direct medical costs were used as the cost calculation basis in all studies. Economic evaluations showed that most PD-1/PD-L1 inhibitors combined with chemotherapy were not cost-effective compared with chemotherapy alone. However, in patients with high PD-L1 expression, PD-1/PD-L1 inhibitor-based combination therapy demonstrated cost-effectiveness for advanced gastric cancer treatment. ConclusionThe economic viability of PD-1/PD-L1 inhibitor-based combination therapy for advanced GC varies significantly across regions due to drug pricing, economic status, and national contexts. Although most current regimens are not cost-effective, their clinical efficacy advantages suggest substantial future potential. Reducing drug prices is recommended to enhance drug accessibility in China.
ObjectivesTo survey the systematic reviews of pharmacoeconomic evaluations.MethodsDatabases including The Cochrane Library, PubMed, EMbase (Ovid), NHS EED (Ovid), CENTRAL, Health Technology Assessment (HTA) Database, CNKI, WanFang Data, VIP and CBM were searched from inception to May 2018 to collect systematic reviews of pharmacoeconomic evaluations. Two reviewers independently screened literature and extracted data. Data statistics and frequency analysis were then conducted on the basic characteristics of included literatures, which involves the publication journal type and influencing factors (IF), disease type, quality assessment tool, etc. The amended AMSTAR scale was used to assess the methodological quality of pharm-SR.ResultsOne hundred and forty-three systematic reviews were included in the overview. The UK had a large number of publications (39.8%), which were mostly published in the Health Technology Assessment and Pharmacoeconomics. Among the included literatures, most were evaluated tumor related pharmacoeconomics systematic reviews (20.8%). They searched on average 7.42±4.00 databases. The British Medical Journal checklist (20.15%) and the Drummond checklist (19.40) were the main tools for quality evaluation. The methodological qualities of these studies were not high.ConclusionsThe evidence shows that the number of systematic reviews of pharmacoeconomic is increasing and research methodology is gradually unifying. However, the quality is still required to be further improved.
As an important auxiliary means of pharmacoeconomics evaluation, budget impact analysis can effectively measure the affordability of medical insurance fund, and plays a significant role in the process of medical insurance access negotiation, adjustment of medical insurance reimbursement directory and establishment of payment price. The quality of budget impact analysis data has a great impact on the analysis results and the scientific decision-making. When the existing data cannot meet the requirements of the paper, relevant software is needed to carry out Delphi method to ensure the data accuracy. Infopoll is a powerful, easy-to-use application that designs consultation questionnaires by providing multiple question choices and multiple forms of answer settings, as well as detailed statistical charts for results analysis. This paper introduces how to obtain the data of budget impact analysis based on Delphi method using Infopoll software, and analyzes the main results in detail.