Objective To explore the effects on quality of life (QOL), the targeted rates of metabolic parameters and cost-effectiveness in newly diagnosed type 2 diabetic patients who underwent multifactorial intensive intervention. Methods One hundred and twenty seven cases in an intensive intervention and 125 cases in a conventional intervention group were investigated by using the SF-36 questionnaire. The comparison of QOL and the targeted rates of metabolic parameters between the two groups were made. We assessed the influence factors of QOL by stepwise regression analysis and evaluated the efficiency by pharmacoeconomic cost-effectiveness analysis. Results The targeted rates of blood glucose, blood lipid and blood pressure with intensive policies were significantly higher than those with conventional policy (P＜0.05). The intensive group’s role limitations due to physical problems (RP), general health (GH), vitality (VT), role limitation due to emotional problems (RE) and total scores after 6 months intervention were significantly higher than those of baseline (P＜0.05). The vitality scores and health transition (HT) of the intensive group were better than those of the conventional group after 6 months intervention. But the QOL scores of the conventional group were not improved after intervention. The difference of QOL’s total scores after intervention was related to that of HbA1c. The total cost-effectiveness rate of blood glucose, blood lipid, blood pressure control and the total cost-effectiveness rate of QOL with intensive policy were higher than those with the conventional policy. Conclusions Quality of life and the targeted rates of blood glucose, blood lipid and blood pressure in newly diagnosed type 2 diabetic patients with multifactorial intensive intervention policy are better and more economic than those with conventional policy.
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.
ObjectiveTo systematically review the pharmacoeconomics of hemodialysis and peritoneal dialysis in patients with end-stage renal disease. MethodsCRD, NICE, CADTH, HITAP, NECA, IWIQG, ISPOR, PubMed, EMbase, CNKI, and WanFang Data databases were electronically searched to collect pharmacoeconomic studies on the hemodialysis and peritoneal dialysis in the treatment of end-stage renal disease from inception to December 2020. Two reviewers independently screened literature, extracted data, and assessed the risk of bias of included studies. Then, the conclusions of research models, pharmacoeconomic evaluation results, and sensitivity analysis were summarized. ResultsA total of 15 pharmacoeconomic studies were included, among which 9 studies used the Markov state transition model, and 6 were observational studies. From the perspective of health outcomes, peritoneal dialysis had cost-effectiveness advantages over hemodialysis in the treatment of end-stage renal disease under the condition of a clear threshold. ConclusionsCurrent evidence shows that compared with hemodialysis, peritoneal dialysis has certain cost-effectiveness advantages in the treatment of end-stage renal disease. Due to limited quality and quantity of the included studies, more high-quality studies are required to verify the above conclusions.
Markov model is one of the decision analysis models, which is widely used in pharmacoeconomic evaluation studies. In terms of dealing with changes of disease risks during different times, the transition probabilities among different Markov health states becomes hard to calculate. Nevertheless, survival analysis is an available resolution. In this paper, we introduced how to apply survival analysis in calculation of transition probability in time-dependent model based on cumulative probability with a case analysis on advanced gastric cancer Markov model, and provide more information for researchers to build models.
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.
ObjectivesTo review the pharmacoeconomic evaluation of rheumatoid arthritis patients with an inadequate efficacy or intolerance with conventional synthetic disease modifying antirheumatic drugs (csDMARDs).MethodsCNKI, WanFang Data, VIP, PubMed, EMbase, Web of Science and The Cochrane Library were electronically searched to collect pharmacoeconomic studies about rheumatoid arthritis patients with an inadequate efficacy or intolerance with csDMARDs from inception to February 2019. Two reviewers independently screened literature, extracted data and assessed risk of bias of the included studies, then, descriptive analysis was performed.ResultsA total of 16 studies were included, where most compared the economics of different treatment methods from the perspective of the payer by cohort or individual model. The economic costs in the studies were primarily on direct cost. Sensitivity analyses were used to prove the robustness of the main analysis in each study. Biological disease-modifying antirheumatic drugs (bDMARDs) might be more cost-effective than csDMARDs. In addition, compared with the bDMARDs, new-marketed targeted synthetic disease-modifying antirheumatic drugs (tsDMARDs) might be more cost-effective.ConclusionsIt could be considered to implement more new marketed tsDMARDs to improve patients’ condition to reduce the economic burden and optimize the allocation of health care resources.
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.
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.