Average cost-effectiveness ratio with censored data

In cost-effectiveness analysis, interest could lie foremost in the incremental cost-effectiveness ratio (ICER), which is the ratio of the incremental cost to the incremental benefit of two competing interventions. The average cost-effectiveness ratio (ACER) is the ratio of the cost to benefit of an intervention without reference to a comparator. A vast literature is available for statistical inference of the ICERs, but limited methods have been developed for the ACERs, particularly in the presence of censoring. Censoring is a common feature in prospective studies, and valid analyses should properly adjust for censoring in cost as well as in effectiveness. In this article, we propose statistical methods for constructing a confidence interval for the ACER from censored data. Different methods-Fieller, Taylor, bootstrap-are proposed, and through simulation studies and data analysis, we address the performance characteristics of these methods.     

Cost-Effectiveness Analysis: A Proposal of New Reporting Standards in Statistical Analysis

Cost-effectiveness analysis (CEA) is a method for evaluating the outcomes and costs of competing strategies designed to improve health, and has been applied to a variety of different scientific fields. Yet there are inherent complexities in cost estimation and CEA from statistical perspectives (e.g., skewness, bidimensionality, and censoring). The incremental cost-effectiveness ratio that represents the additional cost per unit of outcome gained by a new strategy has served as the most widely accepted methodology in the CEA. In this article, we call for expanded perspectives and reporting standards reflecting a more comprehensive analysis that can elucidate different aspects of available data. Specifically, we propose that mean- and median-based incremental cost-effectiveness ratios and average cost-effectiveness ratios be reported together, along with relevant summary and inferential statistics, as complementary measures for informed decision making.     

厄贝沙坦和贝那普利治疗轻中度原发性高血压的成本-效果分析

目的：评价应用厄贝沙坦和贝那普利治疗轻中度原发性高血压的成本-效果。方法：根据文献资料，运用药物经济学的成本-效果分析方法，对厄贝沙坦和贝那普利治疗轻中度原发性高血压进行药物经济学评价。结果和结论：两组方案对于治疗轻中度原发性高血压的疗效比较无显著性差异，其治疗成本分别为998．68元，923．08元，贝那普利的成本-效果比优于厄贝沙坦。     

马来酸依那普利叶酸片治疗H型高血压的成本—效果分析

目的：比较马来酸依那普利叶酸片单用及与5种钙离子拮抗剂联用治疗H型高血压的成本—效果。方法：选择2012年1月—2013年12月我院收治住院的H型高血压患者144例,根据治疗方案的不同分为6个治疗组：马来酸依那普利叶酸片单用组20例(A组)、马来酸依那普利叶酸片 甲磺酸氨氯地平片27例(B组)、马来酸依那普利叶酸片 苯磺酸氨氯地平片28例(C组)、马来酸依那普利叶酸片 马来酸左旋氨氯地平片22例(D组)、马来酸依那普利叶酸片 非洛地平缓释片23例(E组)、马来酸依那普利叶酸片 硝苯地平控释片24例(F组),运用药物经济学中的成本—效果分析方法,对前述治疗H型高血压药物进行评价,并做相应的敏感性分析。结果：A、B、C、D、E、F组有效率分别为60.0％、88.9％、89.3％、86.4％、87.0％、87.5％;与A组相比,B、C、D、E、F组的增量成本-效果比分别为2.01、4.55、3.00、3.07、3.88;结论：成本效果分析结果认为马来酸依那普利叶酸片与甲磺酸氨氯地平片联用治疗H型高血压是较安全、有效、经济的方案,对临床具有很好的指导意义。     

4种原发性高血压治疗方案的成本—效果分析

目的：探讨血管紧张素转换酶抑制剂和钙拮抗剂对原发性高血压所产生的经济效果。方法：本文对232例原发性高血压病人所用的药物贝那普利，培哚普利，氨氯地平，尼群地平进行回顾性分析评价，用成本－效果分析方法进行比较。结果：培哚普利为最佳降压药物（无论是对于收缩压还是舒张压）。结论：通过分析说明了药物经济学在指导合理用药，控制药品费用上涨，减轻病人负担，提高社会经济效益方面具有重要作用。同时，药物经济学研究是实现卫生资源优化配置，保障基本医疗保险制度健康运行和不断完善的必要条件和关键措施之一，对指导开发疗效高、副作用小、价格合理的新药具有重要指导作用。     

阿奇霉素序贯疗法治疗支原体肺炎成本-效果分析

目的:对阿奇霉素序贯疗法治疗支原体肺炎的成本-效果进行探讨。方法:96例支原体肺炎(MP)患者随机分为序贯疗法治疗组(A)和全程静脉注射给药组(B),运用药物经济学的成本-效果分析方法进行比较。结果:两组治愈率分别为81.5%和83.3%(P>0.05);成本分别为(1354.4±131.8)元(、2536.9±315.1)元;成本-效果比分别为16.2±1.6、30.5±3.9。结论:阿奇霉素序贯疗法治疗MP,在相同疗效情况下,能明显缩短住院天数,显著降低总医疗费用,值得临床推荐。     

2002—2006年我院抗高血压药物应用及经济学分析

目的了解我院口服抗高血压药物的临床应用情况，并对3种抗高血压联合用药方案进行成本-效果分析。方法统计分析我院2002—2006年口服抗高血压药物的销售金额、用药频度、日均费用等数据；并且将93份出院病历分成3组，厄贝沙坦150mg＋氢氯噻嗪12．5mg（A组）、苯磺酸氨氯地平5mg＋氢氯噻嗪12．5mg（B组）、盐酸阿罗洛尔10nag＋氢氯噻嗪12．5mg（C组），观察各组疗效及不良反应，采用成本-效果分析进行评价。结果抗高血压物药销售金额占第一位的始终是苯磺酸氨氯地平。3组疗效和不良反应发生率无统计学差异（P〉0．05），C组方案成本、成本-效果比较其他组低。结论我院抗高血压药物的应用情况与国内外总体用药情况基本相符，C组方案较佳。     

西酞普兰与万拉法新治疗抑郁症的循证药物经济学评价

目的评价西酞普兰与万拉法新治疗抑郁症的成本与效果。方法采用循证医学方法收集西酞普兰与万拉法新治疗抑郁症的临床资料,应用药物经济学的成本-效果分析法进行分析。结果西酞普兰与万拉法新治疗抑郁症的治愈率分别为41.11%、52.22%（P〉0.05）;成本-效果比分别为1425.13、2037.34。结论西酞普兰治疗抑郁症的成果-效果优于万拉法新。     

三种治疗方案用于乳腺癌化疗的成本—效果分析

OBJECTIVETo analyze and evaluate three chemotherapeutic programs for mammary gland carcinoma for rational use of drugs. METHODSUsing pharmacoeconomic cost-effectiveness analysis. RESULTSAmong three programs,I,e PP,FAC,IFAF, the effective rate of PP was 80     

3种药物组合方案治疗小儿肺炎的成本-效果分析

目的探讨3种方案治疗小儿肺炎的成本-效果比率，为临床治疗提供依据。方法对2007年1月-2008年1月间住院的96例小儿肺炎采用的3种治疗方案进行成本-效果分析。结果A组总有效率为90.63％，B组总有效率为87.50％，C组总有效率为81.25％，3组总有效率比较差异无统计学意义（P〉0.05）；不良反应发生率A组为3.13％，B组为6.25％，C组为6.25％，3组不良反应发生率比较差异无统计学意义（P〉0.05）；C组C／E、△C／△E为3组中最低。结论C组方案为较佳治疗方案。     

基于HPLC-DAD与LC-MS/MS法检测拉莫三嗪血药浓度结果分析及其经济学比较

目的：通过比较高效液相色谱法（HPLC-DAD,以下简称HPLC）和高效液相串联质谱法（LC-MS/MS）测定的拉莫三嗪（LTG）血药浓度值,考察2种方法检测值的一致性,并进行经济学评价。方法：采用HPLC法和LC-MS/MS法分别测定117份癫痫患者体内拉莫三嗪血清样本,用Passing-Bablok回归法和Bland-Altman偏差图分析检测结果的一致性和差异性,并计算2种方法的成本-效益。结果：2种方法得到的拉莫三嗪血药浓度值比较无显著性差异（P=0.707）,Passing-Bablok回归方程为LTG_HPLC=1.074×LTG_LC-MS/MS+0.071,Pearson相关系数为0.997。HPLC法检测结果比LC-MS/MS法偏高（0.216±0.347）μg·mL^-1 （95% CI：-0.465~0.897）。当样本量 > 9例/d时,HPLC法取得收益 > 56.04元/d;样本量 > 16例时,LC-MS/MS法取得收益 > 36.46元/d,HPLC法收益 > 653.77元/d。当HPLC法达到最大检测量（58例/d）,LC-MS/MS法检测量为66例/d时,2种方法产生的日收益相近（4 240.20元 vs 4305.43元）。当检测量 > 85例/d时,LC-MS/MS法的收益 > 5 927.64元/d,远高于HPLC法。结论：HPLC法和LC-MS/MS法在检测拉莫三嗪血药浓度时具有良好的一致性;HPLC法仪器成本较低,适用于检测量 < 58例/d的情况;LC-MS/MS法仪器成本高,单位时间内检测量为HPLC法的3~4倍,日检测量 > 85例时,检测效益优于HPLC法。     

The cost-effectiveness issue of adjuvant trastuzumab in early breast cancer

Trastuzumab has shown activity in patients with early breast cancer that overexpresses HER-2, and this drug has been employed in the adjuvant setting. Significant resources have been allocated to finance this new therapy. To support decision makers in their allocation of resources, cost-effectiveness models are constructed to compare the costs and outcomes of anticancer therapy. This survey focuses on studies exploring adjuvant trastuzumab therapy in early-stage breast cancer that have been published since 2003, which report on efficacy, benefit and/or cost data in this setting. The paper summarises the results, focuses on the level of evidence of these studies, compares the calculated cost-effectiveness ratios and makes recommendations for future cost-effectiveness analyses.     

A cost-effectiveness model of smoking cessation based on a randomised controlled trial of varenicline versus placebo in patients with chronic obstructive pulmonary disease

Objectives: Smoking is an important risk factor in chronic obstructive pulmonary disease (COPD). A recent clinical trial demonstrated the efficacy of varenicline versus placebo as an aid to smoking cessation in patients with COPD. This study examines the cost-effectiveness of varenicline from the perspective of the healthcare systems of Spain (base case), the UK, France, Germany, Greece and Italy.

Methods: A Markov model was developed to determine the cost-effectiveness of varenicline as an aid to smoking cessation, compared to a placebo, in a COPD population. Cost-effectiveness was determined by the incremental cost per quality-adjusted life year (QALY) gained.

Results: In the Spanish base case varenicline had an incremental cost of €1021/person for an average of 0.24 life years (0.17 QALYs), gained over the lifetime of a cohort of COPD patients, resulting in an incremental cost-effectiveness ratio (ICER) of €5,566. In the other European countries, the ICER varied between €4,519 (UK) and €10,167 (Italy). Probabilistic sensitivity analysis suggested varenicline had a high probability (>95%) of being cost-effective at a threshold of €30,000/QALY.

Conclusions: Varenicline is expected to be a cost-effective aid to smoking cessation in COPD patients in all of the countries studied.     

银杏达莫与脉络宁治疗急性脑梗死的临床疗效及成本-效果分析

目的 评价银杏达莫注射液与脉络宁注射液治疗急性脑梗死（ACI）的临床疗效,并对其成本-效果进行分析.方法 ACI患者120例,随机分为银杏达莫组及脉络宁组,每组60例.2组均给予常规抗血小板聚集及一般脑保护治疗,银杏达莫组在常规治疗基础上加用银杏达莫注射液25 ml,脉络宁组在常规治疗基础上加用脉络宁注射液20 ml,均加入5%葡萄糖注射液500 ml中静脉滴注,2次/d,均治疗14 d.2组治疗前后进行血液流变学指标观察、神经功能缺损程度评分（NDS）及C-反应蛋白（CRP）水平检测,并以成本-效果分析法对2种治疗方法进行分析.结果 银杏达莫组总有效率（95.0%）较脉络宁组（81.6%）高（P＜0.05）;2组治疗后血液流变学指标、CRP水平及NDS评分均较治疗前明显降低（P＜0.05）,但银杏达莫组治疗后血液流变学指标及CRP较脉络宁组降低更明显（P＜0.05）;银杏达莫组成本-效果比较脉络宁组低.结论 银杏达莫注射液治疗ACI起效快,疗程短,单位效果成本较脉络宁注射液低,不良反应少,为ACI的理想用药.     

药物经济学成本-效果在临床路径给药方案中的作用探究

目的:探究药物经济学成本-效果评价方法在临床路径给药方案的实践应用。正确认识药物经济学评价方法对临床路径的建立和应用的重要意义。方法:采用回顾法对2013年1月-2014年12月入住呼吸科第一诊断为"社区获得性肺炎"的患者。其中实施前(路径前组)100例,实施后药师参与(路径后组)100例,比较2组在给药方案药物费用、住院总费用、住院时间、药占比(药物费用/住院总费用)、抗菌药物占比(抗菌药物费用/住院药物费用)、检查费用、治疗效果、安全性等方面的指标。结果:2组患者在药物费用、住院总费用、成本-效果、住院时间方面统计学上有显著性差异(P<0.05),在治疗效果、安全性、检查费、药占比、抗菌药物占比方面无显著性差异(P>0.05)。结论:药师应用药物经济学成本-效果评价方法,协同医师制订给药方案,规范了合理用药,降低了药品费用和住院总费用,同时缩短了住院时间,节约了医疗资源,减轻了个人和社会负担,提高了医疗质量和患者满意度。     

KRAS基因检测干预西妥昔单抗治疗转移性结直肠癌的成本-效果探讨

目的：通过评价KRAS基因检测用于西妥昔单抗治疗转移性结直肠癌（metastatic colorectal cancer,mCRC）的国内外药物经济学研究,探讨其临床疗效及成本效果。方法：从Pubmed、CNKI等大型数据库对2006—2016年期间国内外公开发表的有关KRAS基因检测干预西妥昔单抗治疗的药物经济学评价文献进行检索,对研究内容及结果进行分析及综述。结果：纳入分析的7篇文献表明,与西妥昔单抗（无KRAS检测）相比,KRAS基因检测干预西妥昔单抗治疗mCRC可显著降低成本或提高质量调整生命年（quality adjusted life year,QALY）,改善成本效果,具有优势;且额外进行BRAF及RAS基因检测可进一步改善KRAS检测干预西妥昔单抗治疗的成本效果。结论：KRAS等基因突变检测干预西妥昔单抗治疗相比无KRAS检测为具有优势,且对于指导西妥昔单抗治疗方案的合理选择及实现肿瘤精准医疗有重大实践意义,或将成为未来肿瘤学研究趋势。     

Evaluation of the drug treatment regimens for pulmonary tuberculosis and their cost-effectiveness

According to the World Health Organization (WHO), in 1995 up to one-third of the total global population were estimated to be infected with the tubercle bacilli with nearly 90% of cases occurring in the developing countries. In addition, the 1999 WHO report on tuberculosis (TB) estimated the total number of new sputum-positive cases to have been just over 3.5 m globally in 1997. The incorrect usage of the available drugs has lead to drug-resistant forms of the bacteria which has further complicated the treatment needs and the costs imposed on healthcare services. Faced with this scenario it is important that a comprehensive policy is adopted to make best use of the existing drugs and to do so in a cost-effective way. This article considers the studies conducted on drug treatment regimens for pulmonary TB and their cost-effectiveness in the developing world.     

A tolerance interval approach for assessment of agreement with left censored data

We describe a tolerance interval approach for assessing agreement in method comparison data that may be left censored. We model the data using a mixed model and discuss a Bayesian and a frequentist methodology for inference. A simulation study suggests that the Bayesian approach with noninformative priors provides a good alternative to the frequentist one for moderate sample sizes as the latter tends to be liberal. Both may be used for sample sizes 100 or more, with the Bayesian one being slightly conservative. The proposed methods are illustrated with real data involving comparison of two assays for quantifying viral load in HIV patients.     

Modelling the cost-effectiveness of tofacitinib for the treatment of rheumatoid arthritis in the United States

Background and objectives: Rheumatoid arthritis (RA) is a chronic, debilitating disease affecting an estimated 1.5 million patients in the US. The condition is associated with a substantial health and economic burden. An economic model was developed to evaluate the cost-effectiveness of tofacitinib (a novel oral Janus kinase inhibitor) versus biologic therapies commonly prescribed in the US for the treatment of RA.

Methods: A cost–utility model was developed whereby sequences of treatments were evaluated. Response to treatment was modeled by HAQ change, and informed by a network meta-analysis. Mortality, resource use and quality of life were captured in the model using published regression analyses based on HAQ score. Treatment discontinuation was linked to response to treatment and to adverse events. Patients were modeled as having had an inadequate response to methotrexate (MTX-IR), or to a first biologic therapy (TNFi-IR).

Results: The tofacitinib strategy was associated with cost savings compared with alternative treatment sequences across all modeled scenarios (i.e. in both the MTX-IR and TNFi-IR scenarios), with lifetime cost savings per patient ranging from $65,205 to $93,959 (2015 costs). Cost savings arose due to improved functioning and the resulting savings in healthcare expenditure, and lower drug and administration costs. The tofacitinib strategies all resulted in an increase in quality-adjusted life years (QALYs), with additional QALYs per patient ranging from 0.01 to 0.22.

Conclusions: Tofacitinib as a second-line therapy following methotrexate failure and as a third-line therapy following a biologic failure produces lower costs and improved quality of life compared with the current pathway of care.     

标准一线化疗联用贝伐珠单抗在晚期非小细胞肺癌患者治疗中的成本-效果分析

目的从我国现阶段医疗卫生角度出发,评价四川地区一线化疗联用贝伐珠单抗治疗晚期非小细胞癌的成本-效果性。方法依据NCT00021060临床试验数据,结合四川地区医疗费用水平,采用成本-效果分析法,以增量成本-效果比为评价指标,评价标准一线化疗联用贝伐珠单抗在晚期非小细胞癌治疗方案的优劣,并分析结果敏感度。结果与标准一线化疗组相比,贝伐珠单抗组ICER为1 909 117.8元/年;敏感度分析结果提示,贝伐珠单抗使用成本和医疗检查成本对总成本影响最大,其他参数影响较小。结论从我国现阶段医疗卫生角度出发,与标准一线化疗相比,四川地区晚期非小细胞肺癌一线化疗联用贝伐珠单抗,不具有成本效果优势。