Objective To systematically review the health economic evaluation studies of medicines for the treatment of acute myeloid leukemia (AML). MethodsThe PubMed, EMbase, Cochrane Library, CBM, CNKI, and WanFang Data, as well as the CRD database specifically for health economics were electronically searched from inception to June 2022, and related journals in the field of health economics and the websites of HTA institutions in various countries were manually searched. The quality of the studies was assessed using the CHEERS checklist. The basic characteristics of health economics evaluation publications were summarized, the quality of model structures and methodologies was assessed and economic evaluation results were compared among different treatments. Results A total of 17 studies were included, and cost-effectiveness analyses were conducted from the perspectives of the health system, patients, the whole society, and medical insurance payers. The economic evaluation models were relatively unified, but there were differences in methods and results reporting, and the quality needed to be improved. The research objects were mainly the comparison of hypomethylating agents, targeted medicine and traditional chemotherapy regimens, as well as the comparison of different chemotherapy combinations and different drug dosages. Conclusion Real-world studies are mainly focused on traditional chemotherapy regimens, and model-based health economic evaluations, such as Markov models, are more frequently applied to newly developed targeted drugs and demethylation drugs. Among all treatments, the chemotherapy regimens including cytarabine, midostaurin, and decitabine are found to be more cost-effective.
ObjectiveTo systematically review the economic evaluations of anaplastic lymphoma kinase (ALK)-tyrosine kinase inhibitors (TKIs) in the treatment of ALK-positive non-small cell lung cancer (NSCLC). MethodsPubMed, Web of Science, The Cochrane Library, CNKI, VIP and WanFang Data databases were electronically searched to collect economic evaluations of ALK-TKIs in the treatment of ALK-positive NSCLC from inception to July 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, a systematic review was performed. ResultsA total of 20 studies were included. 18 of the included studies were cost-utility analyses based on the models. The method of survival data extrapolation involved the standard parameter model and the standard parameter model with a hazard ratio adjusting. 10 studies considered or included the disutility value of adverse events. 18 studies performed cost estimation on direct costs. In China, 45% of the included studies were first-line treatment, the results showed that ALK-TKIs were less economical than chemotherapy, and second-/third-generation ALK-TKIs were less economical than crizotinib. Only 1 studies were second-line treatment, the result showed that crizotinib was more economical than chemotherapy. ConclusionThe economic evaluation results of ALK-TKIs in ALK-positive NSCLC vary according to treatment stage and national scenario, and there is also room for optimization of methodological application in this field.
In response to the need for health economics modelers to apply more appropriate complex systems models to address complex challenges in public health, an international team of more than 40 experts in the field of complex systems models and economic evaluation has developed and recently published a guideline on the application of complex systems models to the economic evaluation of public health interventions. This paper introduces the development process and main content of the guidelines, which can provide references to facilitate the application of the guidelines by domestic researchers, aiming to ultimately improve the overall quality of public health research and services and improve the health of the population in China.
Objective To critically appraise and systematically reviewe the economic evaluations of all alternative interventions for hepatitis B in China. Methods We searched MEDLINE and the four largest Chinese electronic databases. The references of eligible studies were also screened. Economic evaluations of any type, which studied interventions for hepatitis B, were eligible for inclusion. A 25-item quality checklist modified from a BMJ checklist was used to appraise the quality of studies. The overall quality score was calculated against 100 points to indicate the risk of bias. Quality appraisal and data extraction were conducted by two independent reviewers. Results Nineteen full economic evaluations and two cost studies were included of which fourteen studies were scored 25-44 points, and seven scored 45-61 points. Most studies adequately documented effectiveness of interventions. However, the costs of interventions were not well reported in over 50% of studies. Many studies inadequately conducted data analysis, particular in sensitivity analysis and discounting. Ten studies compared lamivudine with interferon or conventional therapy for 1-year (or 6-month) effects, which indicated that lamivudine was generally cost-effective. Three evaluations studied 30-year outcomes of interferon compared with conventional therapy, which suggested that interferon usually saved additional costs and years of life. Another three studies compared interferon with less frequently used antiviral agents, however the comparative cost-effectiveness varied. Two cost studies showed the total costs and the percentage of medical costs increased rapidly in proportion to disease severity.Conclusions Of alternative interventions, lamivudine is cost effective for short-term effects. Interferon is superior to conventional therapy for long-term outcomes. However, the long-term economic outcomes cannot be justified by the current evidence. Quality of methods, particularly, that of costing and analytical methods, is a major limitation. There remains a b need to improve the quality of reporting. Careful considerations should be paid before applying the results to decision making.
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.
ObjectivesTo systematically review the methods of pharmacoeconomic evaluation model for hepatitis C therapies and to identify shortcomings of the existing modeling research by comparing the model structure, hypothesis and methodological differences, and to provide suggestions for the construction of high-quality hepatitis C pharmacoeconomic evaluation models.MethodsPubMed, EMbase, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect relevant literatures on the pharmacoeconomic evaluation models for hepatitis C therapies from August 2014 to August 2019. Two reviewers independently screened literature, extracted data, and evaluated the quality of the included studies. Then, the data related to the model structure, methods, and assumptions were compared and summarized.ResultsMost of the 46 studies that finally included used similar modeling methods. Ignoring different modeling elements would cause overestimation or underestimation of the value of hepatitis C therapies. Model structure of all studies were similar and key parameters were from the same source. Forty-five studies measured the cost of drugs and medical cost of health status. All studies used quality-adjusted life years as the outcome and reported incremental cost-effectiveness ratio. Thirty studies conducted one-way sensitivity analysis and probability sensitivity analysis.ConclusionsThe included studies share similar methodological designs and have high quality in general. However, there are some differences and deficiencies in research perspective, model types, model assumptions and model verification. Future pharmacoeconomic evaluation model of hepatitis C therapies should report the results of the whole society, establish dynamic model to consider the impact of transmission, make half-cycle correction for long periods, consider the recurrence after cure, model liver transplantation, and verify the model.
ObjectiveTo assess the health effectiveness, cost and cost effectiveness of different oral anticoagulation (OAT) therapies in China, including warfarin plus international normalized ratio (INR) test in hospital labs (Lab test), warfarin plus patient self-management (PSM) with point of care device, and novel anticoagulant (Dabigatran) alone. MethodsA Markov model containing four states (no complication, hemorrhagic event, thrombotic event and death) was developed to account for long-term cost and outcomes of warfarin/novel anticoagulant users including atrial fibrillation patients and deep venous thrombosis patients. Direct medical cost was taken into consideration, covering expenses of drugs, OAT monitoring and complication management. Both clinical and cost parameters were mainly derived from literatures. ResultsCompared with hospital lab test, the PSM pattern obtained a prolonged 8.48 years and 5.08 QALYs with the larger amount of cost, CNY 47 482. The incremental cost-effectiveness ratio (ICER) of PSM versus hospital lab test came to CNY 19 240 per QALY gained, lower than GDP China per capita in 2014 (CNY 46 628). And the novel anticoagulant pattern was dominated by PSM pattern due to shortened QALYs while increased cost. The sensitivity analysis demonstrated the results were not sensitive to main indicators, including utility in different health status, complication probability, and disease management cost. ConclusionPSM can generate more QALYs by reducing the risk of major thrombotic and bleeding events with acceptable incremental cost, which turns to be the most cost effective way among the 3 patterns and demonstrates promising future in OAT management.
ObjectiveTo systematically review the health economic evaluation studies in which externalities of antibacterial drug uses were identified.MethodsPubMed, EMbase, The Cochrane Library, CNKI, WanFang Data and VIP databases were electronically searched to collect health economic evaluation studies in which externalities of antibacterial drug uses were identified from inception to December 31st, 2020. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies. Descriptive analysis was then performed.ResultsA total of 14 studies were included. Negative externalities and their impacts on costs and/or effectiveness were examined in 13 literature, and positive externalities in terms of an improvement in disease control were included in only one study. No study was found in which both negative and positive externalities were included. The methods used to quantify negative externalities included: only costs associated with drug resistance per prescription or per unit were calculated; both costs and health impacts associated with the second/third line treatments followed a treatment failure (due to drug resistance) were calculated using a decision tree. In one study in which positive externalities were measured, both health gain and cost reduction from an improvement in disease control (as a benefit of antibacterial drug uses) were calculated by constructing a dynamic model at the population level.ConclusionsWe propose that both the positive and negative externalities should be included in health economic evaluation. This can be achieved by measuring the relevant costs and health impacts in a broader perspective, using a disease-transmission dynamic model. In addition, to achieve an improved health utility measurement, disability-adjusted-life years rather than quality-adjusted-life years should be encouraged for use. Finally, both costs and effectiveness should be discounted.
The application of economic tools to evaluate the cost and health benefits and screen out more cost-effective drugs and technologies is an important measure to improve efficiency of medical resource allocation in China. Given the inherent differences between strict clinical trials and clinical routine practice, using trial-based economic evaluations to guide relevant medical decisions may lead to a certain risk of value deviation. Recent development of real-world data provides opportunities to assess the cost-effectiveness of drugs under the practical utilization, and has gradually become a new research hotspot. However, the complexity of the actual clinical environment also puts higher demands on researchers and decision makers to construct, understand and apply real-world evidence. In order to further prompt the normalization of economic evaluation based on real-world data and promote the scientific application of real-world evidence in medical and health decision-making, this project aims at the crucial issues including scope, research design and quality evaluation, to clarify the key considerations on the using of real-world evidence in medical decision-making. Combined with the international guidelines, the latest advancement of relevant research areas and the advice and opinions from multidisciplinary experts, we aim to provide technical references and guidance for researchers and decision makers, and to strengthen the evidence base of management policies.
It is potential for N-of-1 trials to evaluate economics of health care, however, it is still in the exploratory stage. With the advantage of accurate estimation of costs and effects, it is beneficial to promote the application of N-of-1 trials for economic evaluation in the era of precision medicine. In this study, we introduce the necessity, feasibility, selection, calculation of indicators and influence factors of N-of-1 trials for economic evaluation, and in order to provide references for researchers to perform related studies.