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.
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 systematically review the pharmacoeconomic evaluation related to relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL), and to summarize its model structure, parameter inclusion and other methodological parts for future r/r B-ALL-related interventions, and to provide references for conducting pharmacoeconomic evaluations. Methods PubMed, EMbase, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect relevant literature on the pharmacoeconomic evaluation model of r/r B-ALL from inception to August 6th, 2021. Two reviewers independently screened literature, extracted data, and assessed the quality of the included studies. The data on the model structure, methods, and parameter inclusion were then summarized. Results A total of 10 studies using different modeling methods were included. Due to the lack of head-to-head trials, most of the efficacy parameters for the intervention and control groups were derived from different clinical trials and compared indirectly. All studies used quality-adjusted life years (QALYs) as output indicators, and some used life years (LYs) as output indicators and reported the incremental cost effectiveness ratio (ICER). All studies measured the cost of treatment and hematopoietic stem cell transplantation; a few studies also conducted subgroup analysis. Conclusion The number of studies on the economic evaluation of r/r B-ALL is relatively small, and there are large differences in model types, health status, and parameter inclusion. It is suggested that researchers should guarantee the integrity of the report format and normative according to available data choice drug economics evaluation model and establish the reasonable hypothesis under the condition of the patient population heterogeneity uncertainty, perform subgroup analysis especially on the subgroup which did not receive salvage therapy. In the absence of head-to-head clinical trials, appropriate indirect comparison methods are adopted according to the data obtained to reduce methodological differences and improve the quality of relevant pharmacoeconomic research in China.
Medicine is a very important health resource in China. Although numerous efforts are paid to pre-marketed medicines, little is done to address practical problems in marketed medicines. The rational use and allocation of marketed medicines remain a major concern for decision-makers in China. It has been recognized that economic evaluation is an efficient tool for prioritizing the choice, and optimizing the use of medicines. This paper has explored the methods and principles for conducting economic evaluation of marketed medicines. Different strategies will be adopted for economic evidence for marketed medicines in terms of adequacy and sufficiency.However, a standard study pathway should be applied in economic evaluation of marketed medicines. Besides, the aspects for developing economic framework and the methods for reviewing existing economic evidence are also introduced in this paper, particularly, for marketed medicines within the same therapeutic group.
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.
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.
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.
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.
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.
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.
