质量调整生命年

质量调整生命年理论在国外发展已有30余年,但自身存有一定的缺点并面临诸多争议。本文介绍质量调整生命年的概念与计算,具有的优缺点及面临的争议,为国内相关研究和卫生决策提供参考。     

质量调整生命年：基础知识

一、现状 本文旨在对质量调整生命年（QALY）的概念进行回顾。质量调整生命年是一种广泛应用的健康改善的测量方法,用于指导医疗资源配置的决策。最初,QALY是作为成本-效果分析中健康效果的测量工具而被开发的,成本-效果分析旨在帮助决策者将稀缺资源在竞争性医疗项目之间进行分配。     

成本-效用分析系列介绍之四 质量调整生命年(QALYs)的定义和计算

在前几次的讲解中,我们介绍了效用和偏好的基本概念,以及偏好的多种测量方法。在这一讲中我们将涉及成本-效用分析中最核心的技术——质量调整生命年(QALY)的计算。而QALY的计算将运用到上两讲中所测量出的偏好值。QALY计算的完成意味着所有成本-效用分析的关键技术环节的计算完成。     

维格列汀与二甲双胍合用治疗糖尿病的药物经济学研究

目的比较维格列汀、吡格列酮、格列美脲分别与二甲双胍合用治疗2型糖尿病时的效果、成本及成本-效果。方法运用Markov模型对三种治疗方案的终身治疗成本及效用（期望寿命、质量调整寿命年）进行经济学评价,通过文献资料和专家问卷咨询获得临床、生命质量、并发症年治疗成本等数据,进行敏感度分析。结果维格列汀、吡格列酮、格列美脲分别与二甲双胍合用治疗2型糖尿病分别延长11.02、10.96、10.90个质量调整生命年,而生命周期中三种治疗方案的治疗费用分别为124892元、134135元、126010元。敏感度分析证明了结果的可靠性。结论与吡格列酮、格列美脲合并二甲双胍质量相比,维格列汀合并二甲双胍治疗获得的健康效果更好,而治疗成本更低。     

塞来昔布用于治疗中国骨关节炎疼痛的短期成本-效用

目的探讨塞来昔布、布洛芬和萘普生在中国骨关节炎(osteoarthritis, OA)疼痛治疗中的短期成本-效用。方法基于医保支付方的角度,采用已公开发表的基于英国国家卫生和临床技术优化研究所(NICE)骨关节炎指南研发的半马尔科夫模型,模拟为期1年塞来昔布、布洛芬和萘普生在OA治疗中的健康产出和成本。模型中转移概率来源于PRECISION随机对照临床研究,成本数据包括公开的中国本土药品费用、不良反应事件治疗费用及后续治疗费用数据。结果在1年的OA患者治疗中,相比于布洛芬,塞来昔布生命年和质量调整生命年分别提高了0.001年、0.001QALYs,直接医疗费用、药费、3种不良反应事件(AEs)治疗及其后续治疗成本分别降低了576.43元、424.72元、138.09元、13.62元,增量成本-效用比(ICUR)值为-419 182元/QALY。与萘普生比较,OA患者接受为期一年的塞来昔布治疗生命年和质量调整生命年分别提高了0.001年、0.001 QALYs,直接医疗费用、药费、3种AEs治疗及其后续治疗成本分别降低了490.09元、433.52元、53.41元、3.17元,ICUR值为-568 039元/QALY。因此相比于布洛芬与萘普生,塞来昔布在短期治疗OA为绝对优势方案。单因素敏感性分析表明基础分析结果稳健。结论在中国OA患者为期1年治疗中,与布洛芬和萘普生比较,塞来昔布能延长OA患者的生命年,提高生命质量并节约成本,是具有成本-效用的方案。     

健康寿命研究的介绍与评述

随着人口迅速老龄化,一些国家都出现了在平均预期寿命提高的同时,人口健康状况下降的情况,即人活得长,并不一定活得健康。人们开始认识到:健康可以导致长寿,但长寿不一定就健康。因此,在测量生命长度(即数量)的同时,还要能够测量生命质量。健康寿命正是这样一个既可以测量生命数量也可以测量生命质量的指标。主要讨论测量健康寿命的意义;健康寿命的涵义和概念;有关健康寿命的理论假说;健康寿命的测量和计算方法;国际和国内健康寿命研究状况;以及健康寿命研究中存在的问题。     

社会支持对老年人健康相关生命质量影响研究——基于南通的实证

应用欧洲五维健康量表(EQ-5D)和社会支持量表(SSRS)分别评价南通市老年人健康相关生命质量和社会支持现状,实证分析社会支持对老年人健康相关生命质量的影响。数据来源于2010年南通市老年人健康与居家养老服务课题调查,包括了377名农村老年人和383名城市老年人。用OLS回归模型估计社会支持对老年人EQ-5D指数得分影响,用Logistic回归模型估计社会支持对老年人生命质量各维度的影响。社会支持显著提高了老年人健康相关生命质量指数得分。政府应该加强老年人社会支持体系的建立,尤其是农村,提高老年人健康相关生命质量,实现健康老龄化。     

化工企业质量管理战略实施探析

随着城市建设发展和能源结构的调整，化工产品使用范围越来越广泛，重要性也不言而喻，由于化学产品的特殊属性。一旦发生意外或者是产品质量不合格，对人民的生命、财产以及周边的环境造成的损失将不可逆转。可见化学产品质量问题的重要性。本文就此分析了化工企业质量管理存在的主要问题，阐述了化工企业应如何全面实施质量管理的战略。     

生命质量与商品质量的协同性分析

商品质量与生命质量休戚相关,生命质量是个内涵丰富的概念,丰富的商品虽然可以对生命质量产生积极的作用,但在商品生产与消费过程中产生的副作用也在损害着生命质量.研究与了解两者的内在联系,有利于实践以人为本的科学发展观.科学发展观是全面、协调、可持续的综合发展观,其中全面发展是基础,协同发展是关键,可持续发展是目的.论证了以人为本是科学发展观的实质所在,提高人类的生命质量是科学发展观的归宿.     

西洛他唑预防脑梗塞的药物经济学分析

背景抗血小板制剂西洛他唑(Cilostazol)可以降低脑梗塞二次发作的风险。然而,该治疗方案与阿司匹林相比较的成本-效果研究还未进行过。方法与结果我们对65岁以上年龄每天服用西洛他唑200mg和每天服用阿司匹林81mg的两组患者进行了对比。我们建立了马尔科夫模型来计算健康产出和相关成本。西洛他唑更有效,但是也比阿司匹林更昂贵。西洛他唑可以延长0.64个质量生命调整年(QALYs),与此相应的成本是大约110万日元。西洛他唑与阿司匹林相比的增量成本效果比例估计是每QALYs 180万日元。结论使用西洛他唑预防脑梗塞的二次发作是具有成本-效果优势的。     

Cost-effectiveness of continuous subcutaneous apomorphine in the treatment of Parkinson’s disease in the UK and Germany

Abstract

Background:

Parkinson’s disease (PD) is the second most common neurodegenerative disease, affecting ～5.2 million people worldwide. Continuous subcutaneous apomorphine (CSAI) represents an alternative treatment option for advanced PD with motor fluctuation. The purpose of this analysis was to estimate the cost-effectiveness of CSAI compared with Levodopa/carbidopa intestinal gel (LCIG), Deep-Brain-Stimulation (DBS) and Standard-of-care (SOC).

Methods:

A multi-country Markov-Model to simulate the long-term consequences, disease progression (Hoehn & Yahr stages 3–5, percentage of waking-time in the OFF-state), complications, and adverse events was developed. Monte-Carlo simulation accounted for uncertainty. Probabilities were derived from RCT and open-label studies. Costs were estimated from the UK and German healthcare provider’s perspective. QALYs, life-years (LYs), and costs were projected over a life-time horizon.

Results:

UK lifetime costs associated with CSAI amounts to £78,251.49 and generates 2.85 QALYs and 6.28 LYs (€104,500.08, 2.92 QALYs and 6.49 LYs for Germany). Costs associated with LCIG are £130,011.34, achieves 3.06 QALYs and 6.93 LYs (€175,004.43, 3.18 QALYs and 7.18 LYs for Germany). The incremental-cost per QALY gained (ICER) was £244,684.69 (€272,914.58). Costs for DBS are £87,730.22, associated with 2.75 QALYs and 6.38 LYs (€105,737.08, 2.85 QALYs and 6.61 LYs for Germany). CSAI dominates DBS. SOC associated UK costs are £76,793.49; 2.62 QALYs and 5.76 LYs were reached (€90,011.91, 2.73 QALYs and 6 LYs for Germany).

Conclusions:

From a health economic perspective, CSAI is a cost-effective therapy and could be seen as an alternative treatment to LCIG or DBS for patients with advanced PD.     

贝前列腺素钠对比西洛他唑和沙格雷酯治疗下肢外周动脉闭塞的成本-效用

目的评价贝前列腺素钠分别对比西洛他唑和沙格雷酯在治疗外周动脉疾病(PAD)方面的经济效益,为PAD患者的治疗及医保支付提供循证决策依据。方法利用决策树模型,模拟6个月用药周期内的健康经济产出。模型结果产出包括直接医疗成本、生命质量调整年(QALYs),以及增量成本-效用比(ICUR)。临床疗效数据和患者医疗成本数据通过15位分布全国的PAD领域的临床专家访谈得到,其职称为副主任及以上;效用数据和药品价格分别通过已发表的文献和药品招标采购价格数据库获得;严重不良事件的分支概率进行了网状Meta分析和合理假设。最终对所有的模型参数进行单因素敏感分析和概率敏感性分析,以检测模型的稳健性。结果对于PAD患者,与西洛他唑比较,6个月贝前列腺素钠的治疗方案将会产生0.026 QALYs的健康获益,对应减少806元成本,即ICUR为-31 247元/QALYs;与沙格雷酯比较,贝前列腺素钠将会产生0.017 QALYs的健康获益,对应额外479元的成本产出,即ICUR结果为28 778元/QALYs。单因素敏感性分析显示,模型产出的ICUR值对药物的成本和严重不良事件处理成本最为敏感。概率敏感性分析结果显示,2000次迭代的ICUR均值与确定性结果误差小于5%;成本-效用可接受曲线显示,在一倍中国人均GDP的支付意愿阈值内,贝前列腺素钠的成本-效用有100%的概率占优。结论从中国医保支付的角度,与西洛他唑和沙格雷酯比较,贝前列腺素钠在治疗PAD上具有成本-效用优势。     

Cost-effectiveness of grass pollen subcutaneous immunotherapy (SCIT) compared to sublingual immunotherapy (SLIT) and symptomatic treatment in Austria,Spain, and Switzerland

Background: While specific immunotherapy (SIT) has been proven to be cost-effective for the treatment of allergic rhinitis compared to symptomatic treatment, there is a lack of European studies in which sublingual (SLIT) and subcutaneous (SCIT) immunotherapy were compared. The present analysis is focused on the cost-effectiveness of SCIT compared to SLIT and symptomatic treatment of grass pollen allergy in Austria, Spain, and Switzerland. It will address specific properties of the underlying healthcare systems.

Methods: The investigation is based on a previously published health economic model calculation. This was designed as a Markov model with pre-defined health stages and a duration of 9 years covering specific preparations for SCIT (Allergovit) and SLIT (Oralair). The effectiveness was assessed as symptom-score based quality-adjusted life years (QALYs). Additionally, total cost has been determined as well as the cost-effectiveness of SCIT. The robustness of model results was proved in further sensitivity analyses.

Results: With regard to the effectiveness of both SCIT and SLIT, preparations were dominant compared to pharmacological symptomatic therapy. Both strategies were associated with additional cost, but, combined with the results on effectiveness, both have to be regarded as cost-effective. A direct comparison of the SCIT (Allergovit) and SLIT (Oralair) showed lower total costs of SCIT vs SLIT for Austria, Spain, and Switzerland (€1,368 vs €2,012, €2,229 vs €2,547, and €1,901 vs €2,220) and superior effectiveness (SCIT =8.02 QALYs; SLIT =7.98 QALYs; and symptomatic therapy =7.90 QALYs).

Conclusion: In patients with allergic rhinitis, SIT offers cost-effective treatment options compared to symptomatic treatment. When comparing SCIT (Allergovit) and SLIT (Oralair), SCIT was dominant in terms of QALYs as well as costs, in particular due to a slightly higher patient compliance and lower drug costs.     

Cost-effectiveness analysis of ramucirumab treatment for patients with hepatocellular carcinoma who progressed on sorafenib with α-fetoprotein concentrations of at least 400 ng/ml

Abstract

Objective: This study aimed to compare the cost-effectiveness of ramucirumab versus placebo for patients with hepatocellular carcinoma who progressed on sorafenib with α-fetoprotein concentrations (AFP) of at least 400?ng/ml in the United States.

Methods: A Markov model was constructed to assess the cost-effectiveness of ramucirumab. Health outcomes were measured as quality-adjusted life years (QALYs). With TreeAge software, the disease process was modeled as three health states: progression-free survival (PFS), progressive disease (PD), and death. Costs were extracted from the REACH-2 trial, and utility was derived from published literature. Incremental cost-effectiveness ratios (ICERs) were calculated to compare ramucirumab with placebo. Probabilistic sensitivity analyses were developed to examine the robustness of the results.

Results: In the base case analysis, ramucirumab therapy had a cost of $55,508.41 and generated 0.54 QALYs, while placebo therapy had a cost of $761.09 and generated 0.47 QALYs, leading to an additional $54,747.32 in costs and 0.07 QALYs. The ICER was $782,104.57 per QALY, which was much higher than the willingness-to-pay threshold of $100,000 per QALY. According to sensitivity analyses, the utility of PD in the two groups was the dominant parameter influencing the ICER.

Conclusion: Although ramucirumab was associated with prolonged survival for patients with advanced hepatocellular carcinoma who progressed on sorafenib treatment with an AFP of at least 400?ng/ml, it is not a cost-effective treatment from a United States payer perspective.     

Heart rate variability testing: could it change spending for rheumatoid arthritis patients in the United States? An exploratory economic analysis

Background: Autonomic nervous system (ANS) testing with heart rate variability (HRV) has been shown in early research to predict 52-week outcomes in rheumatoid arthritis (RA). HRV testing could be combined with putative ANS biologic pathways to improve treatment response for RA patients. This study explored potential costs and health outcomes of introducing HRV testing into RA treatment, without and with ANS optimization.

Methods: A decision tree exploratory economic model compared HRV testing to standard care in moderate-to-severe biologic-eligible patients over a 10-year time horizon. HRV data was derived from an observational study of RA patients (n?=?33). Patients were stratified into treatment groups based on HRV test scores indicating “low probability of response” and “moderate to high probability of response”. This study explored adding ANS optimization based on HRV score followed by clinically-appropriate treatment. Costs and quality-adjusted life-years (QALYs) for the US population were estimated.

Results: HRV testing in biologic-eligible patients decreased non-effective biologic use, reducing US healthcare costs by $34.6 billion over 10 years with QALYs unchanged. When combined with ANS optimization in biologic-eligible patients, HRV testing could increase costs by $3.6 billion over 10 years but save over 350,000 QALYs. Among all RA patients, HRV testing with ANS optimization could save over $8 billion and over 100,000 QALYs over 10 years, depending on the positive predictive value (PPV) of the HRV test.

Conclusions: The potential economic impact of introducing HRV testing and ANS optimization into RA treatment appears substantial and cost-effective based on the exploratory analysis. Additional rigorous studies are warranted in larger patient samples to better inform decision-making.     

心房颤动患者卒中防治药物利伐沙班与华法林的药物经济学分析

目的对心房颤动患者卒中防治药物利伐沙班与华法林进行药物经济学评价。方法本研究对心房颤动卒中的患者制订了两种治疗方案,华法林片(A组)、利伐沙班片(B组),运用药物经济学知识(成本-效果分析及敏感性分析)对利伐沙班和华法林预防心房颤动相关卒中进行药物经济学评价。结果利伐沙班组与华法林组比较增量成本-效果比(ICER)是508565元/QALYs元,即每多获得1个QALYs需要多花508565元。结论我国大部分地区华法林用于预防心房颤动卒中与利伐沙班比较具备成本优势。     

Valuing Environmental Health Effects – An Economic Perspective

This paper provides an overview of some of the issues involved in comparing benefit-cost analysis and cost-effectiveness analysis based on quality-adjusted life-years as alternative approaches to assessing environmental policies that affect human health. It concludes that: (i) although QALYs have the advantage of reflecting policy impacts on both health status and longevity in a single scalar measure, they are not consistent with utility theory unless individuals’ preferences satisfy some restrictive conditions; (ii) they do not capture other important aspects of the valuation of changes in mortality and morbidity; (iii) cost-effectiveness analysis based on QALYs as a measure of effectiveness omits non-health related effects of environmental policy; and (iv) it leaves unanswered the important question of what level of environmental regulation is appropriate.     

Implementing QALYs in the Analysis of Air Pollution Regulations

In recent years, there has been growing interest in cost-effectiveness analysis for environmental regulations using quality-adjusted life years as the measure of effectiveness. This paper explores the implications of the QALY approach for measuring the impacts of air pollution regulations, with an example using the U.S. Environmental Protection Agency’s Heavy Duty Engine/Diesel Fuel regulations. The paper also examines the issues surrounding the potential use of QALY measures in cost-benefit analysis for air pollution regulations. Key findings are that, compared with a cost-benefit approach, the QALY framework gives more weight to reductions in incidence of chronic disease relative to reductions in premature mortality risk, especially when the mortality risk reductions occur in older populations. In addition, use of monetized QALYs in cost-benefit analysis is not recommended, due to fundamental differences in the theoretical grounding of the different measures. However, application of monetized QALYs based on age-specific willingness to pay (WTP) for mortality risk reductions gives very similar results to typical cost-benefit analysis for mortality risk reductions, as opposed to using values for QALYs based on non-age specific WTP. The paper concludes that in cases where mortality provide the majority of a regulation’s impacts, QALY based cost-effectiveness analysis and WTP based cost-benefit analysis may not differ in their conclusions. However, in cases where morbidity or non-health outcomes are significant, cost-effectiveness and cost-benefit analysis may result in different evaulations of the efficiency of the regulation.     

Cost-effectiveness of delayed-release dimethyl fumarate for the treatment of relapsing forms of multiple sclerosis in the United States

Objective:

To assess the cost-effectiveness of delayed-release dimethyl fumarate (DMF, also known as gastro-resistant DMF), an effective therapy for relapsing forms of multiple sclerosis (MS), compared with glatiramer acetate and fingolimod, commonly used treatments in the US.

Methods:

A Markov model was developed comparing delayed-release DMF to glatiramer acetate and fingolimod using a US payer perspective and 20-year time horizon. A cohort of patients, mean age 38 years, with relapsing-remitting MS and Kurtzke Expanded Disability Status Scale (EDSS) scores between 0–6 entered the model. Efficacy and safety were estimated by mixed-treatment comparison of data from the DEFINE and CONFIRM trials and clinical trials of other disease-modifying therapies. Data from published studies were used to derive resource use, cost, and utility inputs. Key outcomes included costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. Alternative scenarios tested in a sensitivity analysis included drug efficacy, EDSS-related or relapse-related costs, alternative perspectives, drug acquisition costs, and utility.

Results:

Base-case results with a 20-year time horizon indicated that delayed-release DMF increased QALYs +0.450 or +0.359 compared with glatiramer acetate or fingolimod, respectively. Reductions in 20-year costs with delayed-release DMF were ?$70,644 compared with once-daily glatiramer acetate and ?$32,958 compared with fingolimod. In an analysis comparing delayed-release DMF to three-times-weekly glatiramer acetate and assuming similar efficacy and safety to the once-daily formulation, 20-year costs with delayed-release DMF were increased by $15,806 and cost per QALY gained was $35,142. The differences in costs were most sensitive to acquisition cost and inclusion of informal care costs and productivity losses. The differences in QALYs were most sensitive to the impact of delayed-release DMF on disease progression and the EDSS utility weights.

Conclusion:

Delayed-release DMF is likely to increase QALYs for patients with relapsing forms of MS and be cost-effective compared with fingolimod and glatiramer acetate.     

Cost-effectiveness of Access to Critical Cerebral Emergency Support Services (ACCESS): a neuro-emergent telemedicine consultation program

Aims: Access to Critical Cerebral Emergency Support Services (ACCESS) was developed as a low-cost solution to providing neuro-emergent consultations to rural hospitals in New Mexico that do not offer comprehensive stroke care. ACCESS is a two-way audio-visual program linking remote emergency department physicians and their patients to stroke specialists. ACCESS also has an education component in which hospitals receive training from stroke specialists on the triage and treatment of patients. This study assessed the clinical and economic outcomes of the ACCESS program in providing services to rural New Mexico from a healthcare payer perspective.

Methods: A decision tree model was constructed using findings from the ACCESS program and existing literature, the likelihood that a patient will receive a tissue plasminogen activator (tPA), cost of care, and resulting quality adjusted life years (QALYs). Data from the ACCESS program includes emergency room patients in rural New Mexico from May 2015 to August 2016. Outcomes and costs have been estimated for patients who were taken to a hospital providing neurological telecare and patients who were not.

Results: The use of ACCESS decreased neuro-emergent stroke patient transfers from rural hospitals to urban settings from 85% to 5% (no tPA) and 90% to 23% (tPA), while stroke specialist reading of patient CT/MRI imaging within 3?h of onset of stroke symptoms increased from 2% to 22%. Results indicate that use of ACCESS has the potential to save $4,241 ($3,952–$4,438) per patient and increase QALYs by 0.20 (0.14–0.22). This increase in QALYs equates to ～73 more days of life at full health. The cost savings and QALYs are expected to increase when moving from a 90-day model to a lifetime model.

Conclusion: The analysis demonstrates potential savings and improved quality-of-life associated with the use of ACCESS for patients presenting to rural hospitals with acute ischemic stroke (AIS).