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.
Primary economic evaluations are needed in the case that the systematic review of existing economic evidence is not capable of informing economic profiles of marketed medicines. Following the first section of this programs, we presented the principles of designing a study, measuring costs and outcomes, and performing sensitivity analyses. Generally, four designs of economic evaluations are available to perform economic evaluations, each of which has specific strengths and weaknesses. Valuation of outcomes and costs may differ in methods, mainly based on the requirements and applicability of study. The possible factors leading to variation of results should be analyzed using analytic methods with different techniques.
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.
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.
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.
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.
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.
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.
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.
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.
