盐酸安罗替尼用于晚期非小细胞肺癌治疗的成本效用分析

目的:明确盐酸安罗替尼用于晚期非小细胞肺癌治疗的成本效用。方法:基于三期临床试验ALTER0303数据及医疗成本,建立决策树模型。结果:基础分析结果显示,安罗替尼相比安慰剂的增量成本为52839.72元,增量效用为0.067 QALY,增量成本-效用比为784860.34元/QALY,超出了WTP阈值。结论:在我国目前的经济形势下,安罗替尼相比安慰剂用于晚期非小细胞肺癌患者三线治疗暂不具有经济学优势。     

丙肝病毒载量测试指导治疗的经济学评价

目的:分析慢性丙肝患者在病毒载量测试指导下,使用聚乙二醇a-2a干扰素联合利巴韦林治疗的健康效果、成本及其成本效果。方法:结合丙肝治疗路线图,建立COBAS Taqman HCV RNA检测及国产试剂检测的决策树模型,并代入短期和长期结果,比较两种试剂的成本效果。结果:COBAS Taqman HCV RNA检测的长期结果为期望生存年30.57岁,质量调整生存年19.11QALY,人均治疗费用为7 0287.91元,国产HCV RNA检测的长期结果为期望生存年30.34岁,质量调整生存年18.89QALY,治疗费用为7 4151.83元;相比于国产试剂及24周治疗方案,COBAS Taqman检测试剂可多获得0.23个生命年,0.23个质量调整生命年,而总治疗费用降低3 863.9元;并且模型对关键变量不敏感。结论:从短期角度,COBAS Taqman检测可以准确判定RVR和EVR,决定更恰当的疗程;长期而言,COBAS Taqman检测可延长患者的生存年数,提高患者的生命质量,并减少疾病进展导致的治疗费用。     

高血压前期用药干预的成本效果的Markov预测模型分析

目的分析和比较高血压前期不同用药方案的成本效果,为决策的制定提供依据。方法建立包含健康、高血压前期、高血压、因病死亡和衰老死亡等高血压前期相关状态的Markov模型,采用Monte Carlo模拟的方法,应用R 1.12.0软件进行数据模拟,预测和比较仅服用利尿剂和仅服用替米沙坦两种用药方案的效果。结果对高血压前期患者用利尿剂进行干预,平均可以延长1.7年的质量调整生命年(quality adjusted life year,QALY),延长0.6年的寿命,减少1.82%因病死亡率,节省7 704元的经济负担;用替米沙坦进行干预,平均可以延长1.8年的QALY,延长0.7年的寿命,减少2.33%因病死亡率,节省5 547元的经济负担。替米沙坦相对于利尿剂的成本效果比为21 570元/QALY,即每多花21 570元可以减少1个QALY。结论利尿剂和替米沙坦都可以延长患者QALY并且减少经济负担,相比较而言利尿剂更加经济,替米沙坦效果更好。     

青岛地区儿童青少年1型糖尿病持续皮下胰岛素输注与每日多次注射胰岛素治疗的成本效果比较

目的比较持续皮下胰岛素输注(continuous subcutaneous insulin infusion,CSII)与每日多次注射(multiple daily injection,MDI)胰岛素治疗0～18岁儿童青少年1型糖尿病(type 1 diabetes mellitus,T1DM)的卫生经济学获益。方法构建符合中国实际情况的卫生经济学评估模型,使用CORE糖尿病模型预测1型糖尿病患者的长期疾病转归。将2015年1月1日—2019年3月31日青岛市应用CSII和MDI治疗儿童青少年T1DM的基线数据代入CORE糖尿病模型进行模拟,获得患者在不同干预条件下的预期寿命、质量调整生命年、直接医疗成本等。结果对于青岛地区患T1DM的儿童青少年:CSII组的直接医疗成本比MDI组高67 137元;CSII组的预期寿命、质量调整生命年均比MDI组高0.41年;CSII组的增量成本效果比为163 749元/质量调整生命年,低于2019年青岛市人均地区/国内生产总值(gross domestic product,GDP)的1.5倍(186 423元);CSII治疗具有性价比。结论对于青岛地区患T1DM的儿童青少年,与MDI相比,CSII能更好地提高患者的生命质量,具有更好的成本效果。     

乳腺癌多基因面板测序的成本效果分析

目的:以英国德系犹太妇女人群为背景,用决策模型评估多基因面板检测在乳腺癌与卵巢癌筛检上的成本效果。方法:结合英国人口流行病及成本数据,建立决策树模型,基于付费者的角度,从多个文献中获取相关概率和成本数据,进行成本效果分析。成本以2014年价格为基准,采用3.5%的贴现率进行贴现。通过单因素敏感性分析和概率敏感性分析测试模型的稳健性。结果:与无基因检测相比,多基因面板检测降低了1.1%的癌症发生率,挽救了601例乳腺癌和283例卵巢癌患者,延长了0.87个生命年和0.89个质量调整生命年,增量成本效果比为￡6 766/QALY。敏感性分析显示模型稳健性较好,乳腺癌易感基因突变率是影响模型的重要因素。在￡20 000/QALY~￡30 000/QALY的阈值范围内多基因检测有90%以上的概率具有成本效果。结论:与无基因检测相比,乳腺癌二代面板测序在突变率较高的英国德系犹太妇女人群中具有成本效果。     

蒽环类药物联用右雷佐生治疗乳腺癌的药物经济学评价

目的:对蒽环类药物联用右雷佐生(又称右丙亚胺)治疗乳腺癌进行药物经济学评价。方法:从支付方角度出发,采用Markov模型,对于蒽环类药物联用右雷佐生(试验组)和不联用右雷佐生(对照组)两种乳腺癌治疗方案进行成本效用分析。成本参数来源于广东地区的医院抽样数据,疾病概率和效用参数来源于文献。结果:试验组和对照组的治疗效果指标为质量调整生命年,分别为12.86QALYs和12.35QALYs,相应的终身直接医疗费用分别为15.51万元和14.83万元。试验组的效果更好,同时费用更高,增量成本效果比(ICER)为13 155元/QALY,显著低于2015年中国的3倍人均GDP(148 053元)。敏感性分析显示了研究结果的稳健性。结论:从长期来看,对于乳腺癌患者,相对单用蒽环类药物,蒽环类药物联用右雷佐生治疗乳腺癌更具有成本效果优势。     

广西壮族自治区专项示范区HIV筛查项目成本效果分析

目的 对2013年广西壮族自治区3个国家科技重大专项示范县开展的HIV筛查项目进行成本效果分析.方法 计算广西HIV筛查项目实施期间执行的经费,统计项目检出的HIV/AIDS和抗病毒治疗数据,建立马尔科夫(Markov)5树模型,评估该项目获得的质量调整生命年(QALY),分析该项目的成本效果.结果 2013年度广西3个示范县区共投入经费1 920.5万元用于HIV筛查项目,检出HIV/AIDS阳性1 218例,3个示范县HIV/AIDS阳性检出的平均成本为1.456万元/例、1.842万元/例和1.404万元/例,通过检出HIV/AIDS获得的QALY平均数分别为12.736、8.523和8.321个,挽回的QALY总数分别为5 973.184个、3 613.752个和2 704.325个;项目整体成本效果为0.156万元/QALY,各示范县分别为0.114万元/QALY、0.216万元/QALY和0.169万元/QALY,A县成本效果指标优于B、C县.结论 广西示范县HIV筛查项目成本效益较好,但HIV/AIDS阳性检出的平均成本较高,加强HIV/AIDS的抗病毒治疗工作有利于提高该项目的成本效益.     

原发性帕金森病给予左旋多巴联合普拉克索治疗的临床分析

目的:探讨选择原发性帕金森病对左旋多巴+普拉克索进行治疗后获得的临床效果.方法:选择我院2014年09月~2016年09月收治的原发性帕金森病患者63例作为本次实验对比观察对象;分组依据为原发性帕金森病保守治疗药物选择的不同;观察组(33例):左旋多巴+普拉克索;对照组(30例):左旋多巴;通过对比UPDRSⅡ评分以及UPDRSⅢ评分等,以突出左旋多巴+普拉克索的临床应用价值.结果:在UPDRSⅡ评分以及UPDRSⅢ评分方面,两组原发性帕金森病患者之间凸显差异(P<0.05);在保守治疗药物不良反应方面,两组原发性帕金森病患者之间未凸显差异(P>0.05).结论:对于原发性帕金森病患者,临床治疗方法选择左旋多巴+普拉克索,可以成功改善患者的UPDRSⅡ评分以及UPDRSⅢ评分,最终获得显著的原发性帕金森病疗效.     

尼洛替尼纳入医保用于伊马替尼耐药或不耐受的髓性白血病患者治疗的预算影响分析

目的:通过评估二代酪氨酸激酶抑制剂靶向药物(Tyrosine Kinase Inhibitors, TKIs)尼洛替尼纳入医保对医保预算产生的影响,为医保目录药品的遴选决策、医保药品谈判及确定医保支付价格提供借鉴和参考。方法:构建预算影响分析模型,测算将尼洛替尼纳入医保报销系统后,未来5年对医保总预算的影响。结果:在全国范围内,TKIs特药、住院费用、门诊费用以及总费用,每年支出各减少1 575.2万元、3 484.2万元、51.4万元及5 110.7万元,每千万人口中伊马替尼耐药性或不耐受患者的慢性髓性白血病患者生存期内(QALY1)多获得35.0个质量调整生命年(quality-adjusted life year,QALY)。结论:相比伊马替尼原研药,尼洛替尼纳入医保后,具有明显的经济性,然而将伊马替尼仿制药纳入医保后,尼洛替尼在不同的价格变动下,一定程度上会对医保基金造成经济负担。     

吗替麦考酚酯和麦考酚钠用于肾移植后免疫抑制的药物经济学评价

目的:对吗替麦考酚酯和麦考酚钠用于肾移植后免疫抑制进行药物经济学评价.方法:基于社会视角,来用二次文献研究和模型研究方法,根据接受肾移植后患者健康状态发展规律,构建Markov模型,以成本效果比、成本效用比作为评价指标,并对结果进行敏感度分析.结果:在10个模型周期内,EC-MPS组肾移植患者获得的生命年和质量调整生命年分别为8.4年和6.1年,MMF组为7.5年和5.0年.EC-MPS组肾移植患者每获得一个生命年和质量调整生命年需付出的年医疗费用分别为47936元和66011元,MMF组为63513元和95270元.结论:EC-MPS用于肾移植后免疫抑制较MMF更具有成本效果.     

社区戒毒康复人员毛发毒品检测模式的效果与成本效用分析

目的 评价在社区戒毒社区康复人员中应用毛发毒品检测模式的效果与成本效用。方法 将社区戒毒人员分为尿液检测模式组和毛发检测模式组,利用Markov模型评价30年后2种毒品检测模式的累积复吸率、总成本和总质量调整寿命年（quality-adjusted life years,QALYs）,并对模型进行单变量敏感度分析。结果 相较于尿液检测模式,在社区戒毒社区康复人员中应用毛发检测模式的累积复吸率降低了3.9%（82.6% vs 86.5%）,国家支付总成本降低了13 978.22元/人（186 578.96元/人vs 200 557.18元/人）,总QALY提高了0.14QALY/人（13.77QALY/人vs 13.63QALY/人）,即每获得1个QALY,国家可以节省101 465.12元/人。敏感度分析表明,这种成本效用结果具有稳定性。结论 相对于尿液检测模式,对社区戒毒社区康复人员使用毛发检测其吸毒行为降低了累积复吸率,且极具有成本效用,增加了QALY而节省了成本。     

Pramipexole v. levodopa as initial treatment for Parkinson's disease: a randomized clinical-economic trial.

PURPOSE: To determine the 2-year incremental cost effectiveness of initial pramipexole treatment compared with initial levodopa treatment in patients with early Parkinson's disease (PD). METHODS: 301 subjects with early PD were randomized to either pramipexole or levodopa and followed every 3 months over a 2-year period. Costs were assigned to patient collected health utilization data using a variety of methods. Health state preferences were estimated using the EuroQol. RESULTS: Pramipexole strategy was an estimated 2,138 dollars (SE = 1,182 dollars) more expensive than levodopa strategy. The incremental cost-effectiveness of pramipexole compared with levodopa was 106,900 dollars/QALY (EQ-5D), compared with pramipexole being dominated by levodopa using the EQVAS. CONCLUSIONS: Although considerable uncertainty exists in the 2-year cost-effectiveness of initial pramipexole compared with initial levodopa in the treatment of early PD, our estimates suggest that pramipexole may not be welfare enhancing during the first 2 years of treatment. If initial pramipexole results in long-term improvements in quality of life, its cost-effectiveness will become more favorable.     

我国消除丙型肝炎的普通人群HCV检测策略的成本效果分析

目的 分析我国消除丙型肝炎（丙肝）的普通人群HCV检测策略的成本效果,明确最佳成本效果的HCV检测年龄。方法 运用TreeAge pro 2019软件构建决策树马尔科夫模型,以1年为周期,模拟10万名20~59岁各年龄组人群HCV检测和治疗结果,以全社会角度分析策略间比较的成本效果和效益。效果指标为增量成本效果比（ICER）,效益指标为净货币效益（NMB）,以我国2022年人均国内生产总值（85 698元）为意愿支付阈值。通过单因素敏感性分析和概率敏感性分析评估结果可靠性。结果 在20~59岁人群HCV检测有成本效果,在40~49岁年龄组进行HCV检测成本效果最佳。20~59岁年龄组人群HCV检测策略与未HCV检测策略比较,增量成本为161.24元/人,增量效用为0.003 6质量调整寿命年（QALYs）/人,ICER为45 197.26元/QALY,ICER小于意愿支付阈值,具有成本效果。各年龄组人群HCV检测策略与未HCV检测策略比较,ICER为42 055.06~53 249.43元/QALY,NMB为96.52~169.86元/人,其中40~49岁年龄组的ICER最低,NMB最高。单因素敏感性分析结果显示,贴现率、丙肝抗体（抗-HCV）检测成本、人群抗-HCV阳性率和直接抗病毒药物治疗成本对经济学评价影响较大,但改变参数取值,结论不变。概率敏感性分析结果表明模型分析结果稳定。结论 医疗机构探索动员20~59岁普通人群进行HCV检测具有较好的成本效果,以40~49岁年龄组人群的HCV检测成本效果最佳。在我国普通人群中实施HCV检测的“愿检尽检”策略,能降低人群丙肝疾病负担。     

Determining the cost-effectiveness of a computer-based smoking cessation intervention in primary care

PURPOSE: To evaluate the incremental effectiveness and cost-effectiveness of a staged-based, computerized smoking cessation intervention relative to standard care in an urban managed care network of primary care physicians. DESIGN: Decision-analytic model based on results of a randomized clinical trial. METHODOLOGY: Patient outcomes and cost estimates were derived from clinical trial data. Effectiveness was measured in terms of 7-day point-prevalence abstinence at 6 months post-intervention. Quality-adjusted life years (QALYs) and cost-effectiveness (CE) were calculated, with CE measured as cost per patient per life year saved and per quality-adjusted life years saved. CE estimates were adjusted to account for partial behavior change as measured in terms of progression in stage of readiness to quit. Sensitivity analyses were conducted to evaluate the robustness of key model assumptions. PRINCIPAL FINDINGS: Intervention patients were 1.77 times more likely to be smoke-free at 6 months follow-up than those in standard care (p=.078). The intervention generated an additional 3.24 quitters per year. Annualized incremental costs were $5,570 per primary care practice, and $40.83 per smoker. The mean incremental cost-effectiveness ratio was $1,174 per life year saved ($869 per QALY). When the intervention impact on progression in stage of readiness to quit was also considered, the mean incremental cost-effectiveness ratio declined to $999 per life year saved ($739 per QALY). CONCLUSIONS: From a physician's practice perspective, the stage-based computer tailored intervention was cost-effective relative to standard care. Incorporation of partial behavior change into the model further enhanced favorability of the cost-effectiveness ratio.     

Diabetes nephropathy in the Netherlands: a cost effectiveness analysis of national clinical guidelines

BACKGROUND: In the Netherlands a program on quality assurance in medical care has started in 1996. Clinical professionals, patient organizations and health services researchers formulate evidence based guidelines with a concomitant cost-effectiveness analysis. OBJECTIVES: To examine the cost-effectiveness of guideline recommendations for prevention of nephropathy in diabetes mellitus type 1 and 2. RESEARCH DESIGN: A semi-Markov compartment model was developed. Data from international publications on epidemiological surveys and randomized trials, together with national data on health care use and costs, were used to feed the model. A cohort of diabetes patients without renal disease enters the model. MEASURES: Complication (end-stage renal disease) free years, QALY's, and life-time medical costs per patient treated according to guideline recommendations or current anti-diabetic strategy. RESULTS: Guideline treatment for type 1 diabetes yields 4.2 complication free life years, at a cost-effectiveness ratio of 13 500 (Dutch guilders) NLG per QALY. Type 2 diabetes patients gain 0.2 complication free life years at a cost-effectiveness ratio of 31 000 NLG per QALY. CONCLUSION: Guideline development for diabetes nephropathy, with concomitant cost-effectiveness calculations, has resulted in a transparent guideline with explicit information on long-term cost and effects. The project has brought health care providers and health services researchers together.     

Cost-effectiveness of pediatric norovirus vaccination in daycare settings

ObjectiveNoroviruses are the leading cause of acute gastroenteritis in the United States and outbreaks frequently occur in daycare settings. Results of norovirus vaccine trials have been promising, however there are open questions as to whether vaccination of daycare children would be cost-effective. We investigated the incremental cost-effectiveness of a hypothetical norovirus vaccination for children in daycare settings compared to no vaccination.MethodsWe conducted a model-based cost-effectiveness analysis using a disease transmission model of children attending daycare. Vaccination with a 90% coverage rate in addition to the observed standard of care (exclusion of symptomatic children from daycare) was compared to the observed standard of care. The main outcomes measures were infections and deaths averted, quality-adjusted life years (QALYs), costs, and incremental cost-effectiveness ratio (ICER). Cost-effectiveness was analyzed from a societal perspective, including medical costs to children as well as productivity losses of parents, over a two-year time horizon. Data sources included outbreak surveillance data and published literature.ResultsA 50% efficacious norovirus vaccine averts 571.83 norovirus cases and 0.003 norovirus-related deaths per 10,000 children compared to the observed standard of care. A $200 norovirus vaccine that is 50% efficacious has a net cost increase of $178.10 per child and 0.025 more QALYs, resulting in an ICER of $7,028/QALY. Based on the probabilistic sensitivity analysis, we estimated that a $200 vaccination with 50% efficacy was 94.0% likely to be cost-effective at a willingness-to-pay of $100,000/QALY threshold and 95.3% likely at a $150,000/QALY threshold.ConclusionDue to the large disease burden associated with norovirus, it is likely that vaccinating children in daycares could be cost-effective, even with modest vaccine efficacy and a high per-child cost of vaccination. Norovirus vaccination of children in daycare has a cost-effectiveness ratio similar to other commonly recommended childhood vaccines.     

Modeling the Cost-Effectiveness of Galantamine for Mild to Moderately Severe Alzheimer's Disease in Korea

Objective:  The aim of the study was to determine the cost-effectiveness, from the third-party payer viewpoint, of galantamine compared with usual care in the treatment of mild to moderately severe Alzheimer's disease (AD).
Methods:  An existing Markov model was adapted to Korea to predict long-term outcomes over a 5-year time horizon and to estimate the cost-effectiveness of galantamine for the treatment of AD. The model structure is informed by a review of national and international literature on the clinical and cost-effectiveness of galantamine and on the costs and outcomes associated with treatment for AD. The main outcome measure used was the cost per quality-adjusted life year (QALY) gained. All costs were indexed to US$ (2007 value). Multivariate probabilistic sensitivity analysis and scenario analysis were undertaken to assess uncertainty in the results.
Results:  The study findings indicate that the clinical benefits on AD progression from galantamine treatment resulted in an incremental cost per QALY gained of US$4939 over 5 years (vs. usual care). Probabilistic sensitivity analysis and cost-effectiveness acceptability curve suggest that the probability of galantamine treatment having an incremental cost per QALY over US$6740 is zero. Incremental cost per QALY gained according to scenario analyses ranged from US$2271 to US$8335.
Conclusion:  These findings suggest that the use of galantamine may be a cost-effective use of Korean national health-care resources, considering the gross domestic product per capita of US$21,695 in 2007.     

Cost-effectiveness of inactivated influenza vaccination in children with medical risk conditions in the Netherlands

BackgroundIn many countries, annual immunization with inactivated influenza vaccine (IIV) is recommended for children with medical risk conditions. Prior cost-effectiveness analyses found such immunization to be cost saving, but assumed effectiveness against non-severe influenza outcomes and a higher effectiveness against severe influenza outcomes than recent studies would suggest. However, recent vaccine studies do not indicate any reduction in community or outpatient disease episodes in IIV immunized individuals. We therefore evaluated cost-effectiveness of IIV immunization in children with medical risk conditions in the Netherlands, assuming that IIV reduces influenza-related hospitalization and death, but has no meaningful impact on non-severe health outcomes.MethodsA health economic decision tree model was developed to evaluate health effects and costs of annual IIV immunization versus no immunization. Model inputs were based on our study on influenza-related primary care visits and other literature. Immunization was considered cost effective if associated costs were less than €20,000 per quality-adjusted life year (QALY) gained. Probabilistic sensitivity analyses were performed to assess robustness of results, and one-way sensitivity analyses and scenario analyses were done to assess the influence of individual parameters.ResultsAnnual IIV prevents an average of 1.59 influenza-related hospitalizations and 0.02 deaths per 1,000 children with medical risk conditions. This results in an expected QALY gain of 0.43 at incremental costs of €21,564 per 1,000 children, corresponding to an incremental cost-effectiveness ratio (ICER) of €50,297/QALY compared to no immunization. Under base case assumptions, immunization had a 5% probability of being cost effective. Results were most influenced by vaccine efficacy against fatal influenza, QALY loss due to death, and mortality rate.ConclusionsIf IIV only reduces severe disease outcomes, as current evidence suggests, annual immunization of medical risk children is unlikely to be cost effective. Results should however be interpreted with caution as cost-effectiveness is largely dependent on incidence and QALY losses for fatal influenza, for which evidence is scarce.     

The benefits and costs of tamoxifen for breast cancer prevention

OBJECTIVE: To estimate the effects of key uncertainties on the effectiveness and cost-effectiveness of breast cancer prevention with tamoxifen. METHODS: The incremental cost-effectiveness ratio of tamoxifen therapy relative to placebo was estimated using decision analysis with Markov modelling of health states, outcomes and costs for a simulated cohort of women at high risk for breast cancer. Relative effects of tamoxifen's benefits and harms were estimated from meta-analyses of randomised controlled trials. Cost estimates were based on Australian treatment patterns and costs. The main outcome measure was cost per quality-adjusted life year (QALY) gained with costs and effects discounted at a 5% annual rate. RESULTS: Tamoxifen therapy over five years reduces the incidence of breast cancer by approximately 1.4%, which is offset by an increase in endometrial cancer of 0.7% and pulmonary embolism of 0.2%. If the reduction is permanent (preventing new breast cancers emerging over five years and no further treatment effect thereafter), the model estimates an increase in life expectancy of 0.057 QALYs and an extra cost of $2,193; or $38,271/QALY gained. A model assuming further treatment effects of tamoxifen preventing new breast cancers emerging for up to 10 years results in an incremental cost of $19,354/QALY. However, if five years of tamoxifen therapy merely delays when these breast cancers appear (such that by 10 years there is no longer a reduced incidence), the incremental cost per QALY saved is estimated to be $199,149. CONCLUSIONS: Tamoxifen is potentially cost-effective in preventing breast cancer in women at high risk. However, its cost-effectiveness as a preventive therapy is highly sensitive to whether these cancers are permanently prevented or their clinical presentation is only delayed. Long-term follow-up in randomised controlled trials is therefore crucial in forming health policy.