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.
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.
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 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.
ObjectiveTo systematically review the pharmacoeconomic studies of negotiated drugs in the Chinese National Reimbursement Drug List (NRDL). MethodsPubMed, EMbase, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect the pharmacoeconomic studies of negotiated drugs in the Chinese NRDL from January 1, 2012 to January 1, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies, then, the qualitative systematic review was performed. ResultsA total of 59 (33.9%) drugs were evaluated in 124 pharmacoeconomic studies. Only 23 (13.2%) drugs were evaluated in 29 (23.3%) pharmacoeconomic studies prior to the negotiation. Besides, 75 (60.5%) of the negotiated drugs were supported by evidence of economic superiority than the comparison. ConclusionThe pharmacoeconomic studies on drugs which listed in the NRDL is insufficient. Future pharmacoeconomic studies should perform to provide more substantial support for the national drug negotiations.
ObjectiveTo systematically evaluate the pharmacoeconomic value of radiofrequency ablation (RFA) versus amiodarone in the treatment of atrial fibrillation (AF), and to provide reference for treatment scheme selection, drug selection and the formulation of drug policy. MethodsWe searched databases including PubMed, The Cochrane Library, CNKI and CBM from 2000 to 2014 to collect pharmacoeconomic studies on RFA versus Amiodarone for treating AF. Two reviewers independently screened literature, extracted data, and assessed the methodological quality of included studies. The cost-effectiveness of RFA and Amiodarone for AF was compared according to the cost, effectiveness, and incremental cost-effectiveness ratio (ICER). ResultsA total of three studies were included. The results of pharmacoeconomic evaluation showed that the ICERs for each study were $7 976 to $29 068, ￡7 763 to ￡27 745, and $59 194, respectively. According to country-specific willingness to pay thresholds, the ICER of each included study was acceptable. ConclusionCompared to Amiodarone, RFA is a cost-effective therapy for AF.
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.
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.
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.