我国结直肠癌筛查卫生经济学证据系统更新：2009－2018

目的 系统更新中国大陆结直肠癌筛查的卫生经济学评价证据。方法 基于2015年发表的系统综述（2004-2014年），扩大检索数据库范围（PubMed、EMbase、The Cochrane Library、Web of Science、中国知网、万方数据知识服务平台、维普中文科技期刊数据库和中国生物医学文献数据库），延展时间至2018年12月，重点呈现近10年证据（2009-2018年）。系统摘录研究基本特征及主要结果。成本数据采用医疗保健类居民消费价格指数均贴现至2017年，计算增量成本效果比（ICER）与对应年份全国人均GDP的比值。结果 最终纳入12篇文献（新增8篇），其中9篇基于人群（均为横断面研究），3篇基于模型。起始年龄多为40岁（7篇），筛查频率多为终生1次（11篇）。筛查技术涉及问卷评估、免疫法粪便隐血试验和结肠镜。经济学评价指标以每检出1例结直肠癌的成本最为常见，中位数（范围，筛查方案数）为52 307元（12 967~3 769 801，n=20）；每检出1例腺瘤的成本为9 220元（1 859~40 535，n=10）。3篇文献报告了与不筛查相比，每挽救1个生命年的成本，其ICER与GDP比值为0.673（-0.013~2.459，n=11），是WHO认为的非常经济有效；不同筛查技术间及不同频率间该比值的范围重叠较大，但起始年龄50岁（0.002，-0.013~0.015，n=3）比40岁（0.781，0.321~2.459，n=8）筛查方案更经济有效。结论 人群研究提示腺瘤检出成本仅为癌症检出成本的1/6，有限的ICER证据提示在我国人群开展结直肠癌筛查经济有效；尽管最优初筛技术无法定论，但初步提示筛查起始年龄50岁优于40岁。未见随机对照试验评价等高级别证据。     

全球肺癌筛查卫生经济学研究的系统评价

目的 了解全球肺癌筛查的卫生经济学评价研究进展,为我国相关研究和筛查工作开展提供参考。方法 系统检索PubMed、EMbase、The Cochrane Library、中国知网及万方数据知识服务平台自建库至2018年6月30日间肺癌筛查卫生经济学研究相关文献,摘录其经济学评价方法及结果等信息,并进行质量评价。成本统一货币单位后计算增量成本效果比（ICER）后计算与当年当地人均GDP比值。结果 共纳入23项研究（1项基于人群随机对照试验,20项完全基于模型评价）,整体质量较好;22项来自发达国家,11项筛查起始年龄为55岁,18项目标人群考虑了吸烟史;评价的筛查技术全部涉及低剂量螺旋CT（LDCT）,筛查频率以每年1次（17项）和终生1次（7项）居多。22项研究可获得与未筛查相比的ICER,其中17项研究报道的ICER低于3倍当年当地人均GDP。各有15项和7项研究可获得每年1次和终生1次的ICER,其中各有12项和7项支持其经济有效,且终生1次略优于每年1次;不同筛查起始年龄和吸烟包年的经济有效性优劣差异不明显。结论 发达国家多开展基于模型LDCT肺癌筛查卫生经济学评价,并结合年龄和吸烟史进行高危人群选择,初步提示该方案经济有效;可为证据有限的欠发达地区提供参考,但实施需结合当地卫生资源现状;预算有限时低频次LDCT筛查更佳,而筛查起始年龄和吸烟史等细节确定需结合人群特征进行精准评价。     

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。     

乳腺X线摄影技术应用于人群筛查准确性的Meta分析

目的 对全球X线摄影技术运用于乳腺癌筛查的准确性研究进行Meta分析。方法 在Medline、Embase、Cochrane和中国学术期刊网全文数据库（CNKI）文献库中，通过疾病名称、筛查干预、结果指标等关键词整合进行检索。截至2015年6月4日，共检索获得1 167篇文献。根据纳入和排除标准筛选，主要摘录筛查试验中的真阳性、假阳性、假阴性、真阴性的“四格表”数据。采用QUADAS量表进行文献质量评价。利用综合受试者工作特征（SROC）分析方法判断研究间的阈值效应并计算曲线下面积（AUC），采用双变量混合效应模型对X线摄影技术在所有人群和乳腺致密的亚组人群中筛查的灵敏度及特异度进行Meta分析；对样本量大于10万的亚组进行敏感性分析。利用Q检验和I2统计量分析文献异质性，以漏斗图和线性回归方法检验发表偏倚。结果 最终纳入文献48篇（欧洲地区及美国38篇、亚洲地区8篇、大洋洲地区2篇），总样本量为8 551 873例，筛查开展时间为1975-2013年，对象起止筛查年龄大部分在40～75岁。分析得出，X线摄影技术用于乳腺癌筛查的AUC为0.95（95% CI：0.93～0.97），总体灵敏度为0.81（95% CI：0.77～0.84），总体特异度为0.96（95% CI：0.94～0.96），敏感性分析提示该结果稳定。其中对乳腺致密人群的亚组分析显示，X线摄影技术的合并灵敏度和特异度分别为0.74（95% CI：0.61～0.83）和0.93（95% CI：0.89～0.96）。漏斗图和线性回归结果显示纳入研究不存在发表偏倚。结论 X线摄影作为乳腺癌筛查技术具有较高灵敏度和特异度，但对乳腺致密女性的筛查准确性降低。     

乳腺癌自然史模型研究系统评价:现状及构建中国人群特异性模型面临的挑战

目的 系统评价全球乳腺癌自然史模型研究及相关参数,为构建我国人群特异性模型提供参考。方法 检索PubMed和Cochrane Library数据库1980-2015年乳腺癌自然史模型研究,根据纳入排除标准分别开展文献筛选及资料提取,归纳乳腺癌自然史状态及相关参数。结果 共纳入文献36篇,首篇文献发表于1990年;研究集中于欧美国家,仅2篇涉及中国人群;其中筛查项目评价有32篇,方法多采用为Markov模型（32篇）。模型设置状态包括"健康"（36篇）、乳腺导管原位癌（DCIS,17篇）、浸润癌（36篇）和死亡（27篇）。明确报道浸润癌分类系统主要有肿瘤大小（9篇）和TNM分期（9篇,其中3篇报道有1年进展概率）：DCIS发展为Ⅰ期、Ⅰ~Ⅱ期、Ⅱ~Ⅲ期、Ⅲ~Ⅳ期乳腺癌的概率M值（范围）依次是0.279（0.259~0.299）、0.150（0.069~0.430）、0.100（0.060~0.128）和0.210（0.010~0.625）。有15篇报道乳腺癌从临床前期到临床出现症状的平均时间为1.95~4.70年,且随年龄增加逐渐延长;7篇报道了DCIS对应时长。结论 目前乳腺癌疾病自然史模型研究渐受关注,但未见不典型增生等癌前病变的报道;导管原位癌出现症状时长参数有待合理转化;采用浸润癌分类系统多样但进展概率参数有限,对构建我国人群特异性模型有一定参考但也提出挑战。     

HIV疫苗接种策略经济学评价的综合分析

目的 概述全球HIV疫苗接种策略相关的经济学评价研究，为HIV疫苗接种有关决策和研究提供参考。方法 中文以“艾滋病或获得性免疫缺陷综合征”和“疫苗”和“经济学评价或成本效果分析或成本效用分析或成本效益分析”，英文以“Human immunodeficiency virus（HIV） or Acquired immunodeficiency syndrome（AIDS）”和“vaccine or vaccination”和“economic evaluation or cost-effectiveness analysis or cost-utility analysis or cost-benefit analysis or Health technology assessment（HTA）”3组检索词分别组合，在万方数据知识服务平台（万方）、中国医院知识仓库（CHKD）和PubMed数据库，检索截至2022年7月31日的HIV疫苗接种策略经济学评价相关文献，并对文献进行质量评估和综合分析。结果 共纳入17篇质量评估良好的文献，综合分析结果提示，无论是艾滋病重点人群或全人群，接种HIV疫苗是节省成本或具有成本效果的策略，可有效减少新发感染并提高人群生命质量。疫苗的有效率、覆盖率、价格和接种后风险性行为的变化等因素影响不同目标人群的接种效果。结论 目前HIV疫苗接种策略经济学评价的高质量研究数据较少，研究者可基于真实世界证据开展深入研究。     

乙肝疫苗接种成本-效用现状分析

应用成本-效用分析方法对我国乙肝疫苗接种方面应用研究进展作一分析。在知网/万方/维普平台对"乙肝/卫生经济学评价/成本效用/成本效果/成本效益"进行"主题"检索。从研究角度、研究区域、成本测算、效用指标、研究方法、敏感性与阈值等方面对文献进行对比分析。结果显示,本研究中纳入的文献共17篇,其中6篇文献提出了明确的研究角度;8个省份(直辖市)开展了乙肝疫苗接种的成本效用分析;绝大多数仅对疫苗接种费用进行了测算,14篇文献对成本进行3%的贴现;10篇文献使用了数学模型进行分析;9篇文献选取了失能调整生命年(DALY)),10篇选取了质量调整生命年(QALY)作为效用指标,其中13篇进行了效用值贴现;共14篇文章对结果进行了敏感性分析;3篇文献提出了阈值。国内尚未出现系统的乙肝疫苗接种的成本-效用分析研究,缺乏全面的地区性的研究。应加强乙肝疫苗接种方面的调查,完善研究规范与方法,为决策者选取经济有效,提高人群生命质量的策略提出可参考意见。     

疾病筛查方法和策略的计量评价研究

目的 从成本效果、成本效用角度评价疾病的筛查方法和筛查策略.方法 以宫颈癌筛查为例,用VIA/VILI方法模拟对10万名妇女分别终身筛查一次,每1年、3年、5年、10年筛查一次,利用Markov模型预测从现在开始至20年后的宫颈癌的患病率与死亡率,增加的QALY、减少的DALY等.结果 用VIA/VILI方法筛查在20年内可以降低宫颈癌的患病率以及死亡率.即使每1年筛查一次,增加1个QALY的费用仅为532元,减少1个DALY的费用为490元.每3年和每5年筛查一次增加1个QALY的费用相差不大,分别为223元和221元,减少1个DALY的费用都为208元.结论 VIA/VILI方法筛查宫颈癌具有较好的成本效果和效用,在经济不发达地区每3~5年筛查一次,效果较好.     

HIV自检模式在男男性行为者中的成本效果分析

目的 分析珠海市MSM中HIV自我检测（HIVST）模式和现场HIV快速检测（HIV-RDT）模式的成本效果和支付意愿,为政府合理配置卫生资源提供参考依据。方法 以卫生服务提供者的视角,收集珠海市在2019年1-9月MSM参与两种HIV检测模式的成本投入和效果产出,采用TreeAge Pro 2019软件构建10 000名MSM队列决策树模型,测算成本效果比（CER）和增量成本效果比（ICER）,以敏感性分析模型中各参数的不确定性,绘制成本效果可支付曲线评价策略的可支付性。结果 珠海市男同社会组织通过互联网+社交媒体动员参与HIVST和现场HIV-RDT的MSM人次数为2 303 vs.816,发现HIV筛查阳性者人数为33 vs.35,筛查阳性率为1.7% vs.4.3%。每筛查1例的成本为60.45元vs.240.43元,每发现1例筛查阳性的成本为4 218元vs.5 606元。决策树模型运行结果显示,每检测1例MSM的平均费用为44.67元vs.148.42元,ICER为负值。当发现1例HIV筛查阳性支付意愿低于6 528元时,HIVST更具成本效果的选择;当投入高于该阈值时,现场HIV-RDT是更具成本效果的选择。结论 珠海市现行的HIVST模式是具有经济学价值的公共卫生项目,决策者应加大社会组织扶持力度,推广HIVST在MSM中的应用。     

我国女性乳腺癌所致DALY疾病负担：2000-2050年分析与预测

目的 基于全球疾病负担项目2019年最新开放数据,分析我国女性人群因乳腺癌所致伤残调整寿命年（DALY）负担的现况、既往与未来并行国际比较。方法 摘录描述DALY总数、世界标化率值及不同亚组构成,分析我国女性乳腺癌2000-2019年趋势、2019年现况与国际现况比较;利用Joinpoint行2050年预测,主要指标为平均年度变化百分比（AAPC）。结果 2000-2019年,我国女性乳腺癌所致的DALY数在所有女性癌种中的顺位由第四位升至第二位,DALY总数增长了48.4%,其中伤残损失寿命年的占比从4.8%增至8.8%;标化DALY率仅有略微下降（AAPC=-0.3%,其中2016-2019年转为上升,AAPC=1.6%）。2019年,我国女性乳腺癌所致标化DALY率为278.0/10万,DALY数为287.7万人年（占全球乳腺癌的14.2%,占我国女性全部癌种负担的12.1%）,其中26.5%有明确归因（以超重与肥胖最多,为33.6万人年;月经、生育等常见乳腺癌影响因素相关数据在平台未见）;预测提示,2050年,我国女性乳腺癌所致DALY总数将达380.0万人年~516.2万人年,较2019年增加32.1%~79.4%。年龄分布方面,2000-2019年,年龄别DALY数和DALY率峰值均后移,年龄≥ 65岁者的DALY数较<65岁者增长更快（AAPC分别为4.8%和1.3%）;2019年的45~74岁（中国女性乳腺癌筛查与早诊早治指南推荐筛查起始年龄）女性贡献了全部DALY负担的74.3%。结论 近20年我国女性乳腺癌所致DALY率基本未变,近年甚至有增加;若无持续扩大的有效干预,伴随人口老龄化的放大作用,乳腺癌所致DALY在我国女性人群的负担将会加重。乳腺癌主要危险因素相关DALY负担归因数据报道仍有限。     

Probabilistic cost-utility analysis and expected value of perfect information for the Oncotype multigenic test: a discrete event simulation model

ObjectiveTo carry out a cost-utility analysis of the application of the Oncotype genomic test to inform the decision to use or not to use chemotherapy in the Basque Country (Spain).MethodThe cost-utility study was carried out using a discrete event simulation model representing the natural history of breast cancer. The decision of treatment with chemotherapy based on Oncotype was compared with the standard of treatment based on clinical-pathological criteria. The model included clinical data from Basque hospitals and the literature and was processed by deterministic and probabilistic analysis to calculate the incremental cost-effectiveness ratio (ICER), the cost-effectiveness plane, the acceptability curve and the expected value of perfect information. The study adopted both a health and societal perspective.ResultsFrom a health perspective, the deterministic analysis estimated an ICER for Oncotype of 17,453 euros/quality-adjusted life year (QALY), discount included, and 9,613 euros/QALY without the discount. Eighty five percent (85%) of the simulations were below the efficiency threshold for Spain. The parametric variability associated with the Oncotype results was the main uncertainty factor in the decision.ConclusionsOncotype is a cost-effective intervention from a health system perspective since each QALY gained costs less than 25,000 euros. From a societal perspective, it is dominant since it provides greater health and is accompanied by cost savings.     

Economic Evaluation of Cognitive Behavioral Therapy for Depression: A Systematic Review

ObjectivesThis study aimed to conduct a systematic review of cost-utility studies of internet-based and face-to-face cognitive behavioral therapy (CBT) for depression from childhood to adulthood and to examine their reporting and methodological quality.MethodsA structured search for cost-utility studies concerning CBT for depression was performed in 7 comprehensive databases from their inception to July 2020. Two reviewers independently screened the literature, abstracted data, and assessed quality using the Consolidated Health Economic Evaluation Reporting Standards and Quality of Health Economic Studies checklists. The primary outcome was the incremental cost-effectiveness ratio (ICER) across all studies. To make a relevant comparison of the ICERs across the identified studies, cost data were inflated to the year 2020 and converted into US dollars.ResultsThirty-eight studies were included in this review, of which 26 studies (68%) were deemed of high methodological quality and 12 studies (32%) of fair quality. Despite differences in study designs and settings, the conclusions of most included studies for adult depression were general agreement; they showed that face-to-face CBT monotherapy or combination therapy compared with antidepressants and usual care for adult depression were cost-effective from the societal, health system, or payer perspective (ICER ?$241 212.4/quality-adjusted life-year [QALY] to $33 032.47/QALY, time horizon 12-60 months). Internet-based CBT regardless of guided or unguided also has a significant cost-effectiveness advantage (ICER ?$37 717.52/QALY to $73 841.34/QALY, time horizon 3-36 months). In addition, CBT was cost-effective in preventing depression (ICER ?$23 932.07/QALY to $26 092.02/QALY, time horizon 9-60 months). Nevertheless, the evidence for the cost-effectiveness of CBT for children and adolescents was still ambiguous.ConclusionsFair or high-quality evidence showed that CBT monotherapy or combination therapy for adult depression was cost-effective; whether CBT-related therapy was cost-effective for children and adolescents depression remains inconclusive.     

Cost-Effectiveness of Lung Cancer Screening Using Low-Dose Computed Tomography Based on Start Age and Interval in China: Modeling Study

BackgroundLung cancer is the most commonly diagnosed cancer and the leading cause of cancer-related death in China. The effectiveness of screening for lung cancer has been reported to reduce lung cancer–specific and overall mortality, although the cost-effectiveness, optimal start age, and screening interval remain unclear.ObjectiveThis study aimed to assess the cost-effectiveness of lung cancer screening among heavy smokers in China by incorporating start age and screening interval.MethodsA Markov state-transition model was used to assess the cost-effectiveness of a lung cancer screening program in China. The evaluated screening strategies were based on a screening start age of 50-74 years and a screening interval of once or annually. Transition probabilities were obtained from the literature and validated, while cost parameters were derived from databases of local medical insurance bureaus. A societal perspective was adopted. The outputs of the model included costs, quality-adjusted life years (QALYs), and lung cancer–specific mortality, with future costs and outcomes discounted by 5%. A currency exchange rate of 1 CNY=0.1557 USD is applicable. The incremental cost-effectiveness ratio (ICER) was calculated for different screening strategies relative to nonscreening.ResultsThe proposed model suggested that screening led to a gain of 0.001-0.042 QALYs per person as compared with the findings in the nonscreening cohort. Meanwhile, one-time and annual screenings were associated with reductions in lung cancer–related mortality of 0.004%-1.171% and 6.189%-15.819%, respectively. The ICER ranged from 119,974.08 to 614,167.75 CNY per QALY gained relative to nonscreening. Using the World Health Organization threshold of 212,676 CNY per QALY gained, annual screening from a start age of 55 years and one-time screening from the age of 65 years can be considered as cost-effective in China. Deterministic and probabilistic sensitivity analyses were conducted.ConclusionsThis economic evaluation revealed that a population-based lung cancer screening program in China for heavy smokers using low-dose computed tomography was cost-effective for annual screening of smokers aged 55-74 years and one-time screening of those aged 65-74 years. Moreover, annual lung cancer screening should be promoted in China to realize the benefits of a guideline-recommended screening program.     

Cost-effectiveness of screening smokers and ex-smokers for lung cancer in the Netherlands in different age groups

Objective

We aimed to assess the cost-effectiveness of screening smokers and ex-smokers for lung cancer in the Netherlands.

Methods

A Markov model was used to evaluate the health effects and costs of lung cancer screening from the healthcare perspective. The effects and costs of ten screening scenarios with different start and stop ages of screening were examined across a lifetime horizon in a cohort of 100,000 smokers and ex- smokers 50 years and older.

Results

The incremental cost-effectiveness ratios (ICERs) of screening smokers and ex-smokers aged 50–60 years, 50–70 years, and 50 years and older are below the cost-effectiveness threshold of € 20,000 per quality adjusted life year (QALY) gained. Screening 50–60-year-old smokers and ex-smokers was the most cost-effective scenario with an ICER of € 14,094 per QALY gained. However, screening smokers and ex-smokers 50 years and older yielded the highest QALYs and resulted in an ICER of € 16,594 per QALY, which is below the threshold of € 20,000 per QALY. All screening scenarios compared to no screening resulted in CERs between the € 14,000 and € 16,000 per QALY gained. The efficiency frontier showed that screening smokers and ex-smokers in the age groups 70 years and older, 60–70 years, 60 years and older are excluded by extended dominance by no screening, screening smokers and ex-smokers aged 50–60 years and 50–70 years.

Conclusion

This study showed that lung cancer screening is cost-effective in the Netherlands.

     

Cost-Effectiveness of Targeted Genetic Testing for Breast and Ovarian Cancer: A Systematic Review

BackgroundTargeted genetic testing is a tool to identify women at increased risk of gynaecological cancer.ObjectiveThis systematic review evaluates the results and quality of cost-effectiveness modeling studies that assessed targeted genetic-based screen-and-treat strategies to prevent breast and ovarian cancer.MethodsUsing MEDLINE and databases of the Centre for Reviews and Dissemination, we searched for health economic modeling evaluations of targeted genetic-based screen-and-treat strategies to prevent inheritable breast and ovarian cancer (until August 2020). The incremental cost-effectiveness ratios (ICERs) were compared. Methodological variations were addressed by evaluating the model conceptualizations, the modeling techniques, parameter estimation and uncertainty, and transparency and validation of the models. Additionally, the reporting quality of each study was assessed.ResultsEighteen studies met our inclusion criteria. From a payer perspective, the ICERs of (1) BRCA screening for high-risk women without cancer ranged from dominating the no test strategy to an ICER of $21 700/quality-adjusted life years (QALY). In studies that evaluated (2) BRCA cascade screening (ie, screening of women with cancer plus their unaffected relatives) compared with no test, the ICERs were between $6500/QALY and $50 200/QALY. Compared with BRCA alone, (3) multigene testing in women without cancer had an ICER of $51 800/QALY (one study), while for (4) multigene-cascade screening the ICERs were $15 600/QALY, $56.500/QALY, and $69 600/QALY for women in the United Kingdom, Norway, and the United States, respectively (2 studies). More recently published studies showed a higher methodological and reporting quality.ConclusionsTargeted BRCA or multiple gene screening is likely to be cost-effective. Methodological variations could be decreased by the development of a reference model, which may serve as a tool for validation of present and future cost-effectiveness models.     

The Economic Value of Innovative Treatments over the Product Life Cycle: The Case of Targeted Trastuzumab Therapy for Breast Cancer

ObjectivePharmacoeconomic analyses typically project the expected cost-effectiveness of a new product for a specific indication. This analysis develops a dynamic life-cycle model to conduct a multiindication evaluation using the case of trastuzumab licensed in the United States for both early-stage and metastatic (or late-stage) human epidermal growth factor receptor 2 (HER2)-positive breast cancer therapy (early breast cancer [EBC]; metastatic breast cancer [MBC]), approved in 2006 and 1998, respectively.MethodsThis dynamic model combined information on expected incremental cost-utility ratios for specific indications with an epidemiologically based projection of utilization by indication over the product life cycle—from 1998 to 2016. Net economic value was estimated as the cumulative quality-adjusted life years (QALYs) gained over the life cycle multiplied by a societal valuation of health gains ($/QALY) minus cumulative net direct treatment costs. Sensitivity analyses were performed under a range of assumptions.ResultsWe projected that the annual number of EBC patients receiving trastuzumab will be more than three times that of MBC by 2016, in part because adjuvant treatment reduces the future incidence of MBC. Over this life cycle, the estimated overall incremental cost-effectiveness ratio (ICER) was $35,590/QALY with a total of 432,547 discounted QALYs gained. Under sensitivity analyses, the overall ICER varied from $21,000 to $53,000/QALY, and the projected net economic value resulting from trastuzumab treatment ranged from $6.2 billion to $49.5 billion.ConclusionsAverage ICERs for multiindication compounds can increase or decrease over the product life cycle. In this example, the projected overall life-cycle ICER for trastuzumab was less than one half of that in the initial indication. This dynamic perspective—versus the usual static one—highlights the interdependence of drug development decisions and investment incentives, raising important reimbursement policy issues.     

Economic Evaluation of Palliative Management versus Peritoneal Dialysis and Hemodialysis for End-Stage Renal Disease: Evidence for Coverage Decisions in Thailand

OBJECTIVE: To examine the value for money of including peritoneal dialysis (PD) or hemodialysis (HD) into the universal health insurance scheme of Thailand. METHODS: A probabilistic Markov model applied to end-stage renal disease (ESRD) patients aged 20 to 70 years was developed to examine the incremental cost-effectiveness ratio (ICER) of palliative care versus 1) providing PD as an initial treatment followed by HD if complications/switching occur; and 2) providing HD followed by PD if complications/switching occur. Input parameters were extracted from a national cohort, the Thailand Renal Replacement Therapy Registry, and systematic reviews, where possible. The study explored the effects of uncertainty around input parameters, presented as cost-effectiveness acceptability frontier, as well as the value of obtaining further information on chosen parameters, i.e., partial expected value of perfect information. RESULTS: Using a societal perspective, the average ICER of initial treatment with PD and the average ICER of initial treatment with HD were 672,000 and 806,000 Baht per quality-adjusted life-year (QALY) gained (52,000 and 63,000 purchasing power parity [PPP] US$/QALY) compared with palliative care. Providing treatments for younger ESRD patients resulted in a significant improvement of survival and gain of QALYs compared with the older aged group. The cost-effectiveness and cost-utility ratios of both options for the older age group were relatively similar. CONCLUSIONS: The results suggest that offering PD as initial treatment was a better choice than offering HD, but it would only be considered a cost-effective strategy if the social willingness-to-pay threshold was at or higher than 700,000 Baht per QALY (54,000 PPP US$/QALY) for the age 20 group and 750,000 Baht per QALY (58,000 PPP US$/QALY) for age 70 years.     

Economic Evaluation of Cinacalcet in the United States: The EVOLVE Trial

BackgroundPrevious economic evaluations of cinacalcet in patients with secondary hyperparathyroidism (sHPT) relied on the combination of surrogate end points in clinical trials and epidemiologic studies.ObjectivesThe objective was to conduct an economic evaluation of cinacalcet on the basis of the EValuation Of Cinacalcet HCl Therapy to Lower CardioVascular Events (EVOLVE) trial from a US payer perspective.MethodsWe developed a semi-Markov model to assess the cost-effectiveness of cinacalcet in addition to conventional therapy, compared with conventional therapy alone, in patients with moderate-to-severe sHPT receiving hemodialysis. We used treatment effect estimates from the unadjusted intent-to-treat (ITT) analysis and prespecified covariate-adjusted ITT analysis as our main analyses. We assessed model sensitivity to variations in individual inputs and overall decision uncertainty through probabilistic sensitivity analyses.ResultsThe incremental cost-effectiveness ratio (ICER) for cinacalcet was $61,705 per life-year and $79,562 per quality-adjusted life-year (QALY) gained using the covariate-adjusted ITT analysis. Probabilistic sensitivity analysis suggested a 73.2% chance of the ICER being below a willingness-to-pay threshold of $100,000. Treatment effects from unadjusted ITT analysis yielded an ICER of $115,876 per QALY. The model was most sensitive to the treatment effect on mortality.ConclusionsIn the unadjusted ITT analysis, cinacalcet does not represent a cost- effective use of health care resources when applying a willingness-to-pay threshold of $100,000 per QALY. When using the covariate-adjusted ITT treatment effect, which represents the least biased estimate, however, cinacalcet is a cost-effective therapy for patients with moderate-to-severe sHPT on hemodialysis.     

Assessing adolescent immunization options for pertussis in Canada: A cost-utility analysis

BackgroundAdolescent tetanus, diphtheria and pertussis (Tdap) immunization helps prevent pertussis infection. Timing of Tdap receipt represents an important facet of successful adolescent pertussis immunization. Potential strategies for timing of vaccine administration are each associated with different benefits – including disease prevention – and costs. The objective of this study was to assess the cost-utility of adolescent pertussis immunization strategies in Canada.MethodsA cost-utility analysis was conducted using a pertussis disease history-simulating Markov model, with adolescents (beginning at age 10 years) as the cohort of interest. The model assessed three Tdap vaccination strategies: (1) immunization of 10 year olds, (2) removal of adolescent vaccination, and (3) immunization of 14 year olds (status quo). The analysis was conducted from a healthcare payer perspective and used a lifetime time horizon. Primary outcomes included life years, quality-adjusted life years (QALYs), health system costs, and an incremental cost-effectiveness ratio (ICER). Costs and outcomes were discounted at 1.5 percent annually. Deterministic and probabilistic sensitivity analyses were performed to assess parameter uncertainty.ResultsThe current recommended adolescent immunization strategy (at age 14) resulted in an average of 40.4432 expected QALYs and $26.28 per individual. This strategy was dominated by immunization at 10 years and no immunization. Compared to no immunization, immunizing adolescents at age 10 had an ICER of $74,899 per QALY. Results were most sensitive to the incidence of pertussis and the utility of moderate or severe pertussis. At a cost-effectiveness threshold of $50,000/QALY, removal of adolescent vaccination represented the most cost-effective strategy in 78% of simulations.ConclusionAnalysis assumes a policy context where immunization of pregnant women is recommended. Findings suggest that alternate adolescent Tdap vaccine strategies – either immunization of 10 year olds, or removal of the adolescent vaccine – are more cost-effective than the current practice of immunizing 14 year olds.