五价轮状病毒疫苗纳入新西兰儿童免疫接种计划的预算影响与成本-效果分析

目的：预测（1）新西兰5岁以下儿童轮状病毒疾病负担（rotavirus disease burden）;（2）五价轮状病毒疫苗（pentavalent rotavirus vaccine,PRV）纳入国家免疫计划的健康效益、预算影响和成本-效果。方法：建立静态均衡模型评价5年连续出生队列（five successive birth cohorts）接种PRV[三剂的覆盖率85%,每剂50新西兰元（新元）]的健康效益与预算影响,第5年从社会角度进行项目的成本-效果评价,未来健康效益贴现率为3.5%。结果：5岁前,每5人中就有1人因轮状病毒肠胃炎就医,每43人中有1人住院。2009年,预计住院病例为1506人（476人/10万人;95%CI,451～502）,不需要住院的轮状病毒肠胃炎急诊（Emergency Department,ED）病例3086人,仅需初级保健护理的10 120人。年社会成本707万新元,其中住院成本占41%,看护者收入损失占25%。对连续出生队列免疫5年,健康效益将提高,疾病成本可降低78%。到第5年,轮状病毒肠胃炎住院病例为1191人,ED病例2442人,需初级保健护理的9762人,可避免0.8例死亡。避免1例初级保健护理的轮状病毒肠胃炎需要对6个儿童进行免疫接种;避免1例住院治疗需要免疫接种49人;避免1例死亡需要免疫接种73357人。增量成本为299万新元,每剂疫苗保本价格（break-even price）为32.39新元（以2006年价格计）。避免1例住院的成本为2509新元,避免1例寻求医疗帮助的成本为305新元。第5年获得的每个生命年（life-year）成本为143079新元,每个质量调整生命年（QALY）成本为46092新元（26774美元）。每个QALY成本对发病率、疫苗价格和效果、儿童生命质量损失、病死率及看护者收入损失敏感。结论：从社会角度来看,将PRV纳入新西兰儿童免疫计划,会以低廉成本获得QALY,具有重大临床效益。     

疫苗药物经济学评价中有关成本和效果的测算方法探讨

与一般药物相比,疫苗具有外部性、保护率和作用时间不确定等特性,因此疫苗的经济学评价应当有其特殊性.本文回顾了近十几年世界有关疫苗经济学评价的研究报道,综合概括了疫苗经济学评价方法的最新进展.本文讨论的主要内容包括间接成本法、边际成本法、选择合适流行病学模型测量健康结果、成本和效果的贴现等.     

成本-效果象限方法的演变及其对净成本收益的解释

目的：寻找成本-效果象限分析工具用于成本-效果、成本-效益方法的理论与方法的意义。方法：通过梳理成本-效果象限方法的研究进展,提出本论文设想的效益成本象限分析工具,从而剖析两种分析工具所具有的理论与方法意义。结论：成本-效果象限与效益成本象限均是边际分析思想的运用,是理解成本-效果分析、成本-效益分析以及增量分析法的重要途径。成本-效果象限解决了成本-效果分析发展中的ICER为负值的解释,以及不同象限的备选方案的比较等问题。效益成本象限方法是其再发展,重点是为了解决效益成本比指标和净收益指标相矛盾的难题,并建立了增量分析法的图形化推导逻辑。     

3种第二代抗组胺药治疗儿童过敏性鼻炎的成本-效果

目的运用药物经济学评价方法,对氯雷他定、西替利嗪、左西替利嗪3种第二代抗组胺药物治疗儿童过敏性鼻炎进行成本-效果分析,为临床治疗儿童过敏性鼻炎用药提供参考和建议。方法通过单组率Meta分析获得效果指标,于山东省药品招标采购平台,结合儿童各年龄段的服用剂量和疗程获得药品成本,对3种第二代抗组胺药物治疗儿童过敏性鼻炎进行成本-效果分析,并进行单因素敏感性分析、概率敏感性分析及基于药品成本变化的情境假设分析。结果对于治疗2~6岁儿童过敏性鼻炎,氯雷他定为绝对劣势方案被排除,西替利嗪相比于左西替利嗪的增量成本-效果比(ICER)为19.34元;对于治疗6~11岁儿童过敏性鼻炎,氯雷他定为绝对劣势方案被排除,西替利嗪相比于左西替利嗪的ICER为38.69元;对于治疗≥12岁儿童过敏性鼻炎,左西替利嗪、氯雷他定成本较高为绝对劣势方案被排除,西替利嗪为优势方案。敏感性分析与基础分析结果基本一致。结论第二代抗组胺药物治疗各年龄段儿童过敏性鼻炎,氯雷他定为绝对劣势方案;临床治疗2~11岁儿童过敏性鼻炎时,可以根据患儿的意愿支付(WTP)阈值选择最优方案,若WTP低于ICER,则优先使用左西替利嗪,若WTP高于ICER,则优先使用西替利嗪;临床治疗≥12岁儿童过敏性鼻炎时,西替利嗪为绝对优势方案。     

成本-效果分析与决策实践间的桥梁

成本-效果分析（广义,包括成本-效用分析）是应用较为广泛的药物经济学评价方法,在评价实践中占有重要的地位。然而,由于成本-效果分析评价标准的缺失导致其评价结果ICER值无法直接应用到报销、定价、合理用药、新药研发等决策实践中。为此,经济学家们在ICER值与决策实践间构建了一些桥梁,这些桥梁是连接ICER值与决策制定的重要工具,可使药物经济学评价结果ICER值更好地为决策实践服务。药物经济学评价在各领域中的应用方法大同小异,本文主要以在报销领域中的决策实践为例进行阐述。     

基于决策树模型的芪龙胶囊治疗缺血性脑卒中的成本-效果分析

目的 评价芪龙胶囊治疗缺血性脑卒中的经济性,以期为临床合理用药、卫生资源合理配置提供参考依据。方法 本研究采用Treeage Pro软件对芪龙胶囊+常规疗法VS常规疗法干预缺血性脑卒中进行成本-效果分析,成本参数来源于米内网数据库和最新发表的相关文献,同时根据一项已发表的随机对照研究得出效果参数（临床有效率）评价两种治疗方案短期内的经济性,模拟的时间跨度为12周。通过单因素（旋风图）、概率（成本-效果可接受曲线、成本-效果散点图）敏感性分析对本研究增量成本-效果比（ICER）值的稳健性进行验证。结果 基础分析结果显示,芪龙胶囊组与对照组比较的ICER为6 827.59元,即相比于单用常规疗法,使用芪龙胶囊联合常规疗法治疗缺血性脑卒中每多治疗1例有效的缺血性脑卒中患者需多花费6 827.59元,此时若患者的意愿支付价格高于6 827.59元,芪龙胶囊联合常规疗法更具经济性,否则常规疗法更具经济性。敏感性分析结果显示本研究的ICER较为稳健。结论 假设人均可支配收入（2020年）作为患者意愿支付阈值,缺血性脑卒中患者使用芪龙胶囊联合常规治疗方案比单用常规治疗方案更具经济性。     

成本-效果可接受曲线:不确定状态下的医疗决策方法

医疗领域的决策制定不可避免地要在考虑医疗干预的效果和成本的不确定性下进行。成本-效果可接受曲线是在不确定状态下的一种医疗决策方法。这种技术比增量成本-效果比率的置信区间估计具有优势,它直接解决了决策制定问题。本文提供了一个基于净效益方法的成本-效果可接受曲线的定义,并详细阐述了它的内涵。成本-效果可接受曲线可以通过参数和非参数的方法计算。对于这两种计算方法,在成本-效果分析中,我们在成本-效果可接受曲线和统计推断(基于置信区间和P值)之间建立正式的联系。     

成本-效果分析中药物成本测量的良好研究规范：管理式医疗角度——ISPOR药物成本工作组报告之三

目的：本报告的目的是就管理式医疗机构（managed care organization,MCO）角度下如何测量成本一效果分析（cost Effectivenss Analyse,CEA）中的药物成本这一问题提供指导和建议。方法：国际药物经济学和结果研究会（ISPOR）的专项计划——成本一效果分析中药物成本测量的良好研究规范工作组（DCTF）由ISPOR董事会指定。工作组成员均为富有经验的CEA模型开发或使用者。在制定报告草案前DCTF已召开了会议并制定了报告的核心假设和纲要,而且就报告草案征求了外部评审人员的意见,并在ISPOR会议和ISPRO网站上征求了ISPOR会员的意见。结果：对于MCO而言,药物成本等于它向药剂师支付的药品成本和调剂费用之和,再减去患者共付部分和来自制药厂商的任何折扣。MCO对这些部分的报销程度随药物类型（单一来源还是多重来源）、配送方式（零售还是邮寄药品）、给药场所（自我药疗还是在医生诊所给药）等多种因素的不同会存在很大区别。由于成本部分的价值取决于个人和保密合同,因此难以对其进行准确测算。结论：MCO角度下药物成本的测算应包括药费和处方费,再加上扣除共付金额、折扣和其他原因减少的药物价格。考虑到药物价格的自然属性,ISPOR应建立一个网站,以便出现新信息之后在该网站上对DCTF的成本建议进行更新。     

剖宫产产妇抗菌药物使用疗程的成本-效果分析

目的:寻找广州市妇婴医院剖宫产产妇术后预防性使用抗菌药物的最佳疗程。方法:采用回顾性研究,收集和整理该院2006年6月至2007年5月493例剖宫产产妇相关资料,利用成本-效果分析方法对剖宫产产妇术后预防性使用抗菌药的不同疗程进行分析。结果:剖宫产产妇预防性使用抗菌药的疗程A组((d<3)天)、B组((3≤d<4)天)、C组((4≤d<5)天)、D组((d≥5)天)的有效率分别为:66.67%、85.12%、89.5%、85.19%;以抗菌药费用作为成本计算的成本-效果比分别为:9.21、7.22、8.62、14.32;以西药费作为成本计算的成本-效果比分别为:13.92、15.29、16.89、21.89;以住院费作为成本计算的成本-效果比分别为:61.15、67.09、66.78、79.96。结论:剖宫产产妇术后预防性使用抗菌药物,最经济、有效、合理的使用时间为术后3天(B组),增加用药天数并不能增加治疗效果,而费用却明显增加。     

儿泻康贴膜治疗小儿腹泻的成本-效果

目的 探讨儿泻康贴膜治疗小儿寒性腹泻的经济性。方法 基于4家中心开展的儿泻康贴膜治疗小儿腹泻的随机对照试验,共纳入观察组123例,对照组121例。对两组治疗5 d的疾病疗效（愈显率）、单项症状（大便次数、粪便性质、腹痛、食欲不振、肠鸣、发热恶寒、鼻塞流涕、咳嗽咽痒、异常舌质、异常舌苔、异常脉象或指纹）消失情况,从医疗机构角度出发,进行成本-效果分析,并进行敏感性分析。结果 观察组疾病疗效（愈显率）、异常大便次数消失率、异常粪便性质消失率、食欲不振消失率高于对照组,差异有统计学意义（P<0.05）。相比于对照组,观察组每提高1%的疾病疗效（愈显率）、异常大便次数消失率、异常粪便性质消失率、食欲不振消失率,分别需要多花费5.33、5.26、6.74、7.55元。敏感性分析结果与成本-效果分析结果一致。结论 对于改善疾病疗效、异常大便次数、异常粪便性质、食欲不振症状方面,在效果较好的情况下,所增加的成本是值得的,与对照组相比,具有经济学优势。     

Cost-effectiveness analysis of second-line tyrosine kinase inhibitor treatment for chronic myelogenous leukemia

Aim:

A cost-effectiveness analysis was performed for sequential treatments of chronic myelogenous leukemia (CML) with tyrosine kinase inhibitors (TKIs) after failure of 1st line imatinib, from a commercial payer perspective in the US.

Methods:

A Markov model was developed to simulate lifetime treatment costs and health outcomes for TKI sequences for treatment of patients resistant or intolerant to 1st-line imatinib. Five health states were included, chronic phase 2nd-line TKI, chronic phase 3rd-line TKI, chronic phase post-TKI, advanced phases, and death. Efficacy (response achievement, loss of response, transformation, death) and safety (adverse events incidence, discontinuation) data are based on clinical trials. Resource utilization, costs, and utilities were based on product labels and publically available data. Uncertainty analyses were conducted for key inputs.

Results:

In patients failing imatinib, dasatinib-initiating treatment sequences provide the most survival (ΔLYs?=?0.2–2.0), QALYs (ΔQALYs?=?0.2–1.9), and accrue highest CML-related costs (ΔCosts?=?$64,000–$222,000). The average ICER per QALY for dasatinib- vs imatinib-initiating sequences is $100,000 for an imatinib-resistant population. The average ICER per QALY for dasatinib- vs nilotinib-initiating sequences is $170,000 for an imatinib-resistant population, and $160,000 for an imatinib-intolerant population.

Conclusions:

This analysis suggests that dasatinib is associated with increased survival and quality of life compared to high dose imatinib and to a smaller extent with nilotinib, among patients resistant or intolerant to 1st-line imatinib, primarily based on higher cytogenetic response rates observed in clinical studies of dasatinib. Head-to-head studies of sequential use of dasatinib and nilotinib are needed to validate the model findings of improved survival (LYs) with better quality-of-life (QALYs) for patients initiating dasatinib in 2nd-line. However, the model findings (in light of higher cytogenetic response rates with dasatinib) are supported by other studies showing improved quality-of-life for responders, and improved survival for patients achieving cytogenetic response.     

Cost-effectiveness of combination daclatasvir-sofosbuvir for treatment of genotype 3 chronic hepatitis C infection in the United States

Background: A phase III trial evaluated the efficacy and safety of Daklinza (daclatasvir or DCV) in combination with sofosbuvir (SOF) for treatment of genotype (GT) 3 hepatitis C virus (HCV) patients.

Aim: This study evaluated the cost-effectiveness of DCV?+?SOF vs SOF in combination with ribavirin (RBV) over a 20-year time horizon from the perspective of a United States (US) payer.

Methods: A published Markov model was adapted to reflect US demographic characteristics, treatment patterns, costs of drug acquisition, monitoring, disease and adverse event management, and mortality risks. Clinical inputs came from the ALLY-3 and VALENCE trials. The primary outcome was the incremental cost-utility ratio. Life-years, incidence of complications, number of patients achieving sustained virological response (SVR), and the total cost per SVR were secondary outcomes. Costs (2014 USD) and quality-adjusted life years (QALYs) were discounted at 3% per year. Deterministic, probabilistic, and scenario sensitivity analyses were conducted.

Results: DCV?+?SOF was associated with lower costs and better effectiveness than SOF?+?RBV in the base case and in almost all scenarios (i.e. treatment-experienced, non-cirrhotic, time horizons of 5, 10, and 80 years). DCV?+?SOF was less costly, but also slightly less effective than SOF?+?RBV in the cirrhotic and treatment-naïve population scenarios. Results were sensitive to variations in the probability of achieving SVR for both treatment arms. DCV?+?SOF costs less than $50,000 per QALY gained in 79% of all probabilistic iterations compared with SOF?+?RBV.

Conclusion: DCV?+?SOF is a dominant option compared with SOF?+?RBV in the US for the overall GT 3 HCV patient population.     

Cost-effectiveness of omalizumab for uncontrolled allergic asthma in the Netherlands

Abstract

Background:

Omalizumab, licensed for patients with uncontrolled persistent allergic (IgE mediated) asthma, was found to be cost-effective based upon its clinical trial data. Observational studies have been undertaken to determine the real life outcomes of using omalizumab in the community.

Objective:

To determine the cost-effectiveness of omalizumab based upon observational data from the Netherlands and compare to its cost-effectiveness using clinical trial data.

Methods:

An observational study (eXpeRience) recruited allergic asthma patients eligible for Omalizumab therapy and followed them while on treatment. At 1 year, data from the Dutch patients enrolled in eXpeRience were examined to estimate the number of exacerbations and resource use while on omalizumab therapy compared to the year prior to omalizumab use. Observational data were used in a Markov model to calculate the lifetime cost-effectiveness ratios.

Results:

In the 1 year prior to omalizumab therapy the per-person rate of exacerbations was 3.39 compared to 1.07 in the year taking omalizumab. The discounted incremental lifetime additional costs for omalizumab were €55,865 for 1.46 additional quality-adjusted life years (QALY), resulting in €38,371/QALY. Using the INNOVATE clinical trial outcomes and current resource use, the prior ratio was €34,911/QALY, similar to the observational ratio. As in all observational studies, the main limitation is obtaining complete and accurate data. Patients with missing exacerbation or response data were excluded from this analysis.

Conclusion:

Non-clinical trial experience with omalizumab supported the finding of fewer exacerbations in the allergic asthma population while treated with omalizumab, and therapy was found to continue to have an attractive cost-effectiveness ratio.     

Cost-effectiveness of ranibizumab in the treatment of visual impairment due to diabetic macular edema

Objective Ranibizumab, an anti-vascular endothelial growth factor designed for ocular use, has been deemed cost-effective in multiple indications by several Health Technology Assessment bodies. This study assessed the cost-effectiveness of ranibizumab monotherapy or combination therapy (ranibizumab plus laser photocoagulation) compared with laser monotherapy for the treatment of visual impairment due to diabetic macular edema (DME).

Methods A Markov model was developed in which patients moved between health states defined by best-corrected visual acuity (BCVA) intervals and an absorbing ‘death’ state. The population of interest was patients with DME due to type 1 or type 2 diabetes mellitus. Baseline characteristics were based on those of participants in the RESTORE study. Main outputs were costs (in 2013 CA$) and health outcomes (in quality-adjusted life-years [QALYs]) and the incremental cost-effectiveness ratio (ICER) was calculated. This cost-utility analysis was conducted from healthcare system and societal perspectives in Quebec.

Results From a healthcare system perspective, the ICERs for ranibizumab monotherapy and combination therapy vs laser monotherapy were CA$24 494 and CA$36 414 per QALY gained, respectively. The incremental costs per year without legal blindness for ranibizumab monotherapy and combination therapy vs laser monotherapy were CA$15 822 and CA$20 616, respectively. Based on the generally accepted Canadian ICER threshold of CA$50 000 per QALY gained, ranibizumab monotherapy and combination therapy were found to be cost-effective compared with laser monotherapy. From a societal perspective, ranibizumab monotherapy and combination therapy provided greater benefits at lower costs than laser monotherapy (ranibizumab therapy dominated laser therapy).

Conclusions Ranibizumab monotherapy and combination therapy resulted in increased quality-adjusted survival and time without legal blindness and lower costs from a societal perspective compared with laser monotherapy.     

双金连合剂治疗风热型感冒患者的成本-效果分析

目的比较双金连合剂与双黄连口服液治疗风热型感冒患者的成本-效果,为相关部门提供决策依据。方法根据两个不同临床终点(退热、所有症状消退),建立两个决策树模型(M1和M2),用成本-效果比值(ICER)比较两方案的经济学,并进行敏感性分析来验证结果稳定性。结果在以退热为终点的主要情境下,以24 h退热有效率为效果指标(M1)时的ICER为0.31元,以质量调整生命年(QALY)为效果指标(M2)时,ICER为12015.02元;在以所有症状消退为终点的主要情境下,以3 d所有症状消退率为效果指标(M1)时的ICER为2.17元,以QALY为效果指标(M2)时,ICER为21245.01元。敏感性分析中各因素对研究结论无明显影响。结论双金连合剂对比双黄连口服液治疗风热型感冒在以退热有效率和所有症状消退为主要终点情境下,ICER值低于各自的阈值,结果具有成本-效果。     

Cost-effectiveness of dedicated dietitians for hyperphosphatemia management among hemodialysis patients in Lebanon: results from the Nutrition Education for Management of Osteodystrophy trial

Aim: To assess the cost-effectiveness of nutrition education by dedicated dietitians (DD) for hyperphosphatemia management among hemodialysis patients.

Materials and methods: This was a trial-based economic evaluation in 12 Lebanese hospital-based units. In total, 545 prevalent patients were cluster randomized to DD, trained hospital dietitian (THD), and existing practice (EP) groups. During Phase I (6 months), DD (n?=?116) received intensive education by DD trained on renal nutrition, THD (n?=?299) received care from trained hospital dietitians, and EP (n?=?130) received usual care from untrained hospital dietitians. Patients were followed-up during Phase II (6 months).

Results: At baseline, EP had the lowest weekly hemodialysis time, and DD had the highest serum phosphorus and malnutrition-inflammation score. The additional costs of the intervention were low compared with the societal costs (DD: $76.7, $21,007.7; EP: $4.6, $18,675.4; THD: $17.4, $20,078.6, respectively). Between Phases I and II, DD showed the greatest decline in services use and societal costs (DD: –$2,364.0; EP: –$1,727.7; THD: –$1,105.7). At endline, DD experienced the highest decrease in adjusted serum phosphorus (DD: –0.32; EP: +0.16; THD: +0.04?mg/dL), no difference in quality-adjusted life-years (QALY), and the highest societal costs. DD had a cost-effectiveness ratio of $7,853.6 per 1?mg decrease in phosphorus, compared with EP; and was dominated by THD. Regarding QALY, DD was dominated by EP and THD. The results were sensitive to changes in key parameters.

Limitations: The analysis depended on numerous assumptions. Interpreting the results is limited by the significant baseline differences in key parameters, suggestive of higher baseline societal costs in DD.

Conclusions: DD yielded the greatest effectiveness and decrease in societal costs, but did not affect QALY. Regarding serum phosphorus, DD was likely to be cost-effective compared with EP, but had a low cost-effectiveness probability compared with THD. Regarding QALY, DD was not likely to be cost-effective. Assessing the long-term cost-effectiveness of DD, on similar groups, is recommended.     

Cost-effectiveness of renal denervation therapy for the treatment of resistant hypertension in The Netherlands

Abstract

Objectives:

Safety and efficacy data for catheter-based renal denervation (RDN) in the treatment of resistant hypertension have been used to estimate the cost-effectiveness of this approach. However, there are no Dutch-specific analyses. This study examined the cost-effectiveness of RDN from the perspective of the healthcare payer in The Netherlands.

Methods:

A previously constructed Markov state-transition model was adapted and updated with costs and utilities relevant to the Dutch setting. The cost-effectiveness of RDN was compared with standard of care (SoC) for patients with resistant hypertension. The efficacy of RDN treatment was modeled as a reduction in the risk of cardiovascular events associated with a lower systolic blood pressure (SBP).

Results:

Treatment with RDN compared to SoC gave an incremental quality-adjusted life year (QALY) gain of 0.89 at an additional cost of €1315 over a patient’s lifetime, resulting in a base case incremental cost-effectiveness ratio (ICER) of €1474. Deterministic and probabilistic sensitivity analyses (PSA) showed that treatment with RDN therapy was cost-effective at conventional willingness-to-pay thresholds (€10,000–80,000/QALY).

Conclusion:

RDN is a cost-effective intervention for patients with resistant hypertension in The Netherlands.     

Cost-effectiveness of rivaroxaban versus warfarin for stroke prevention in non-valvular atrial fibrillation in the Japanese healthcare setting

Abstract

Aims: This article aimed to examine the cost-effectiveness of rivaroxaban in comparison to warfarin for stroke prevention in Japanese patients with non-valvular atrial fibrillation (NVAF), from a public healthcare payer’s perspective.

Materials and methods: Baseline event risks were obtained from the J-ROCKET AF trial and the treatment effect data were taken from a network meta-analysis. The other model inputs were extracted from the literature and official Japanese sources. The outcomes included the number of ischaemic strokes, myocardial infarctions, systemic embolisms and bleedings avoided, life-years, quality-adjusted life-years (QALYs), incremental costs and incremental cost-effectiveness ratio (ICER). The scenario analysis considered treatment effect data from the same network meta-analysis.

Results: In comparison with warfarin, rivaroxaban was estimated to avoid 0.284 ischaemic strokes per patient, to increase the number of QALYs by 0.535 per patient and to decrease the total costs by ¥118,892 (€1,011.11) per patient (1 JPY = 0.00850638 EUR; XE.com, 7 October 2019). Consequently, rivaroxaban treatment was found to be dominant compared to warfarin. In the scenario analysis, the ICER of rivaroxaban versus warfarin was ¥2,873,499 (€24,446.42) per QALY.

Limitations: The various sources of data used resulted in the heterogeneity of the cost-effectiveness analysis results. Although, rivaroxaban was cost-effective in the majority of cases.

Conclusion: Rivaroxaban is cost-effective against warfarin for stroke prevention in Japanese patients with NVAF, giving the payer WTP of 5,000,000 JPY.     

Cost-effectiveness of partially-hydrolyzed formula for prevention of atopic dermatitis in Australia

Abstract

Objective:

To perform an economic evaluation of a specific brand of partially hydrolyzed infant formula (PHF-W) in the prevention of atopic dermatitis (AD) among Australian infants.

Methods:

A cost-effectiveness analysis was undertaken from the perspectives of the Department of Health and Aging (DHA), of the family of the affected subject and of society as a whole in Australia, based on a decision-analytic model following a hypothetical representative cohort of Australian newborns who are not exclusively breastfed and who have a familial history of allergic disease (i.e., are deemed ‘at risk’). Costs, consequences, and incremental cost-effectiveness ratios (ICER) were calculated for PHF-W vs standard cow’s milk based infant formula (SF), and, in a secondary analysis, vs extensively hydrolyzed infant formula (EHF-Whey), when the latter was used for the prevention of AD.

Results:

From a representative starting cohort of 87,724 ‘at risk’ newborns in Australia in 2009, the expected ICERs for PHF-W vs SF were AU$496 from the perspective of the DHA and savings of AUD1739 and AU$1243 from the family and societal perspectives, respectively. When compared to EHF-Whey, PHF-W was associated with savings for the cohort of AU$5,183,474 and AU$6,736,513 from the DHA and societal perspectives.

Limitations:

The generalizability and transferability of results to other settings, populations, or brands of infant formula should be made with caution. Whenever possible, a conservative approach directing bias against PHF-W rather than its comparators was applied in the base case analysis. Assumptions were verified in one-way and probabilistic sensitivity analyses, which confirmed the robustness of the model.

Conclusions:

PHF-W appears to be cost-effective when compared to SF from the DHA perspective, dominant over SF from the other perspectives, and dominant over EHF-Whey from all perspectives, in the prevention of AD in ‘at risk’ infants not exclusively breastfed, in Australia.     

Cost-effectiveness of natalizumab versus fingolimod for the treatment of relapsing multiple sclerosis

Abstract

Background:

With the addition of new agents for the treatment of multiple sclerosis (MS) (e.g., fingolimod), there is a need to evaluate the relative value of newer therapies in terms of cost and effectiveness, given healthcare resource constraints in the United States.

Objective:

To assess the cost-effectiveness of natalizumab vs fingolimod in patients with relapsing MS.

Methods:

A decision analytic model was developed to estimate the incremental cost per relapse avoided of natalizumab and fingolimod from a US managed care payer perspective. Two-year costs of treating patients with MS included drug acquisition costs, administration and monitoring costs, and costs of treating MS relapses. Effectiveness was measured in terms of MS relapses avoided (data from AFFIRM and FREEDOMS trials). One-way and probabilistic sensitivity analyses were conducted to assess uncertainty.

Results:

Mean 2-year estimated treatment costs were $86,461 (natalizumab) and $98,748 (fingolimod). Patients receiving natalizumab had a mean of 0.74 relapses avoided per 2 years vs 0.59 for fingolimod. Natalizumab dominated fingolimod in the incremental cost-effectiveness analysis, as it was less costly and more effective in reducing relapses. One-way sensitivity analysis showed the results of the model were robust to changes in drug acquisition costs, administration costs, and costs of treating MS relapses. Probabilistic sensitivity analysis showed natalizumab was cost-effective 95.1% of the time, at a willingness-to-pay (WTP) threshold of $0 per relapse avoided, increasing to 96.3% of the time at a WTP threshold of $50,000 per relapse avoided.

Limitations:

Absence of data from direct head-to-head studies comparing natalizumab and fingolimod, use of relapse rate reduction rather than sustained disability progression as primary model outcome, assumption of 100% adherence to MS treatment, and not capturing adverse event costs in the model.

Conclusions:

Natalizumab dominates fingolimod in terms of incremental cost per relapse avoided, as it is less costly and more effective.