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The Importance of the Pharmacoeconomic Analyses in Drug Negotiation. A Farewell Editorial 药物经济学分析在药品谈判中的重要性。告别社论
Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2023-10-04 DOI: 10.7175/fe.v24i1.1547
Mario Eandi
Decision analysis is considered an essential tool that every responsible decision-maker should use to make rational, informed choices that are ideally optimal according to predefined and possibly shared criteria. In the management of public healthcare, decision analysis tends to propose more cost-effective choices for the benefit of the community, operating in a specific context of limited healthcare resources that do not allow satisfying all patient needs. The use of decision analysis in the public healthcare context inevitably refers to the bioethical value of health equity: the most cost-effective choice aims to satisfy the maximum number of patients with the scarce available resources, adopting a utilitarian interpretation of the equity criterion. Unfortunately, within the management of the public healthcare system, the use of decision analysis has faced challenges due to cultural, social, and organizational reasons. In Italy, the management of the National Healthcare System (SSN) and regions is structured into separate compartments (silos): pharmaceutical spending is managed separately from hospital and community care. Even today, managers of the pharmaceutical service must pursue the primary objective of keeping pharmaceutical spending within a predetermined maximum limit. During the early years of AIFA, this criterion hindered or at least discouraged the use of traditional pharmacoeconomic analyses in preparing Price and Reimbursement (P&R) dossiers for new drugs to be negotiated. The fundamental economic analysis required by AIFA was a simulated estimation of the budget impact of pharmaceutical spending in the first three years after the introduction of a new drug to the market. However, pharmacoeconomists from pharmaceutical companies progressively started submitting dossiers with BIA prepared according to international standards, which require evaluating the overall impact of the new drug not only on pharmaceutical spending but also on all types of healthcare costs (e.g., hospital costs, home care, etc.). Only in recent years, AIFA has explicitly valued cost-effectiveness analyses as a tool to support its decisions on drug prices and reimbursement. The recent AIFA Monitoring Report 2022, prepared by the Economic Evaluations Office (Table I), highlights that in recent years, there has been a progressive increase in the percentage of P&R dossiers including pharmacoeconomic analyses. In 2022, one hundred six dossiers, equivalent to 62% of the dossiers submitted to AIFA, were accompanied by a pharmacoeconomic study: 105/106 (99%) included Budget Impact Analysis (BIA), 73/106 (69%) also featured a Cost-Effectiveness (CEA) or Cost-Utility (CUA) analysis, and only 1/106 (1%) exclusively included a CEA. In summary, AIFA still considers BIA fundamental for negotiating the P&R of a drug intended for the Italian market. However, the percentage of dossiers including CEA studies is now significant and growing, particularly important for the negotiat
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引用次数: 0
[Measurement-Based Procurement Approach for Biosimilars in Italy: A Position Paper] [意大利基于测量的生物仿制药采购方法:立场文件]
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2023-03-29 DOI: 10.7175/fe.v24i1s.1544
Arturo Cavaliere, F. Mennini, M. Pani, Paolo Gennaro Torrico, S. Torrisi
The full potential of biosimilars has not been achieved yet, and this document has been written with the aim of methodizing an effective and measurable procurement system that ensures wider, simplified, and economically sustainable access of biosimilars to patients. The economic and financial sustainability generated by the increased use of biosimilars for the benefit of patients and society has been estimated and confirmed in several observational studies and probabilistic projections conducted in Europe. Despite differences in procurement policies, significant reductions in spending for some biosimilars have been recorded in several European countries between 2015 and 2018 compared with the period preceding the patent’s expiry. An accurate evaluation of the economic outcomes resulting from an extended use of biosimilars has also been conducted in Italy, with the settled purpose of calculating the combined effects that various biosimilars with patent expired have generated and will generate in terms of overall spending reduction. Even with the limitations typically associated with probabilistic models, it is also evident that an expanded use of biosimilars is an effective tool to govern the expenditure and channel resources to support the intrinsic balance of the health system (i.e., sustainability) or to ensure the introduction of innovative drugs or diagnostic procedures (i.e., accessibility). The economic sustainability of the health system must also be achieved through the maintenance of a competitive procurement system. This requires a serious reconsideration of the purchasing criteria. The principle of competitiveness can contribute to the governance, containment, reduction or streamlining of public expenditure on biosimilars, but it is appropriate to establish which factors can feed and support a lawful competition, actually. Competition merely based on price reduction between biosimilar and biosimilar, excluding, undervaluing, or taking for granted that between originator and biosimilar, leads to a price erosion and this condition could have unpredictable and undesirable consequences. The market must have its own balance to be of interest to manufacturing companies and ensure the competitiveness. In the field of biosimilars, as in other areas, the need to move from purchasing criteria based on cost exclusively to those based on overall economic benefit by combining price with quality is generally shared. This approach allows for a balanced competition on multiple parameters and above all enables the best service to be provided to patients and the National Health Service. Hence, what are the quality criteria to be considered in relation to price? The areas relevant to the procurement code regulations, the determination of needs, the technical-market knowledge, the encoding of quality criteria, and their specific parameterization to the contingent dynamics of the market must certainly be identified and examined: this is what the present do
即使存在通常与概率模型相关的局限性,也很明显,扩大生物仿制药的使用是管理支出和渠道资源以支持卫生系统的内在平衡(即可持续性)或确保引进创新药物或诊断程序(即可及性)的有效工具。保健系统的经济可持续性也必须通过维持竞争性采购制度来实现。这需要对采购标准进行认真的重新考虑。竞争力原则有助于治理、遏制、减少或精简生物仿制药方面的公共支出,但确定哪些因素实际上可以促进和支持合法竞争是适当的。仅仅基于生物仿制药和生物仿制药之间价格降低的竞争,排除、低估或理所当然地认为原创者和生物仿制药之间的竞争,会导致价格侵蚀,这种情况可能会产生不可预测的不良后果。市场必须有自己的平衡,以确保制造企业的利益和竞争力。与其他领域一样,在生物仿制药领域,一般都需要从完全以成本为基础的采购标准转向以价格与质量相结合的总体经济效益为基础的标准。这种方法可以在多个参数上进行平衡竞争,最重要的是能够向患者和国民保健服务体系提供最好的服务。因此,与价格相关的质量标准是什么?当然必须查明和审查与采购守则条例、确定需要、技术市场知识、质量标准的编码以及它们对市场偶然动态的具体参数化有关的领域:这就是本文件的目的。目的是提供一个指南,通过评估经济上最有利的报价来促进可奖励招标的实施,并使我们能够衡量“新”采购系统的效果,比较目前记录的情况与在意大利引入基于质量-价格比的生物仿制药招标后阶段检测到的情况之间的差异。总之,通过该领域专家和专家的共同工作,本立场文件确定了评估生物仿制药的质量标准,并根据确定的标准提出了技术规范,从而为中央采购机构和机构付款人提供了在区域和国家层面实际实施以质量-价格比为重点的采购方法的战略。
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引用次数: 0
[Burden of Short Bowel Syndrome in Italy: Direct and Indirect Costs and Quality of Life] [意大利短肠综合征负担:直接和间接成本与生活质量]
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2023-03-24 DOI: 10.7175/fe.v24i1.1545
F. Mennini, C. Bini, M. Paoletti, A. Marcellusi, P. Sciattella, R. Viti, L. Amoroso, L. Fioravanti, G. Di Martino, A. Ragonese, R. Caccialanza, L. Norsa, A. Diamanti, L. Pironi
OBJECTIVE: The Short Bowel Syndrome (SBS) is the most common cause of chronic intestinal failure (CIF) due to benign disease. The prevalence in Europe was estimated about 1.4 cases per million and few information are available for Italy. Home parenteral nutrition (HPN) is the primary and lifesaving treatment for patients with CIF. The parenteral nutrition (PN) has a great impact on the quality of life (QoL) of patients and the role of the caregiver is crucial for the disease management. The aim of this study was to evaluate the economic burden of SBS in Italy and to assess the impact of the disease and the parenteral nutrition on the quality of life of patients.METHODS: The total burden of SBS in Italy was assessed both in terms of costs and QoL using different tools. A prevalence-based cost of illness (COI) model was developed to estimate direct costs (PN cost, central venous catheter insertion cost, monitoring cost, hospitalization cost) and indirect costs (absenteeism, presenteeism, unemployment, abandonment of work due to the disease and economic benefits granted by the National Social Security Institute) associated with patient with SBS in Italy. The total annual costs were calculated considering a micro-costing approach, thus associating the average costs per patient with the prevalence of the disease. A systematic literature review (SLR) was conducted to collect epidemiological and direct cost data related to the patients with SBS. Hospital costs were estimated using the national Hospital Information System. Indirect costs were estimated using a human capital approach; therefore, the productivity loss was estimated both for patients and caregivers. A survey was conducted to obtain data about productivity loss and quality of life of patients and caregivers. The questionnaires were completed by clinicians, who were asked to indirectly report the experience of patients with SBS in parenteral nutrition currently being treated at their referral center. In a subsequent phase, a focus group was conducted to collect further information on QoL for patients and caregivers based on the experience of the KOLs involved. The QoL was evaluated considering a Likert scale.RESULTS: The prevalence of patients with gastrointestinal disease in HPN was estimated equal to 9.4 and 2.3 patients per million inhabitants for adults (age >18) and pediatric (age 0-18 years) patients, respectively. Knowing that SBS is the main cause of CIF due to benign disease, constituting 75% among adults and 56% among children, the number of adults with SBS in HPN present in Italy were 420, while the number of children with SBS in HPN were 77. Regarding direct costs, the mean total annual cost associated with adult and pediatric patient with SBS in Italy was estimated equal to € 36,434 and € 46,682, respectively. Parenteral nutrition accounted for 91% of the mean total cost estimated for the adult and for 87% of the mean total cost estimated for pediatric patient. Concerning indirec
目的:短肠综合征(SBS)是由良性疾病引起的慢性肠衰竭(CIF)的最常见原因。据估计,欧洲的流行率约为每百万人1.4例,关于意大利的信息很少。家庭肠外营养(HPN)是CIF患者的主要和挽救生命的治疗。肠外营养(PN)对患者的生活质量(QoL)有很大的影响,护理人员的作用对疾病的管理至关重要。本研究的目的是评估意大利SBS的经济负担,并评估该疾病和肠外营养对患者生活质量的影响。方法:采用不同的工具对意大利SBS患者的总负担进行成本和生活质量评估。建立了一种基于患病率的疾病成本(COI)模型,用于估计意大利SBS患者的直接成本(PN成本、中心静脉导管插入成本、监测成本、住院成本)和间接成本(旷工、出勤、失业、因病放弃工作和国家社会保障研究所授予的经济效益)。考虑微观成本计算方法计算年度总成本,从而将每位患者的平均成本与疾病患病率联系起来。采用系统文献综述(SLR)收集与SBS患者相关的流行病学和直接费用资料。使用国家医院信息系统估算医院费用。使用人力资本方法估算间接成本;因此,对患者和护理人员的生产力损失进行了估计。进行了一项调查,以获得有关患者和护理人员的生产力损失和生活质量的数据。问卷由临床医生完成,他们被要求间接报告目前在转诊中心接受肠外营养治疗的SBS患者的经历。在随后的阶段,进行了一个焦点小组,根据所涉及的kol的经验,收集有关患者和护理人员生活质量的进一步信息。采用李克特量表评估生活质量。结果:HPN中胃肠道疾病患者的患病率估计为成人(>18岁)和儿科(0-18岁)患者分别为每百万居民9.4例和2.3例。知道SBS是良性疾病导致CIF的主要原因,在成人中占75%,在儿童中占56%,目前意大利HPN中患有SBS的成人人数为420人,而患有HPN的儿童人数为77人。关于直接成本,意大利成人和儿童SBS患者的平均年总成本估计分别为36,434欧元和46,682欧元。肠外营养占成人估计平均总成本的91%,占儿科患者估计平均总成本的87%。关于间接成本,每位成年患者的平均年度总成本估计为51,093欧元(81%与因疾病放弃工作而导致的生产力损失有关),而每位儿科患者的平均总成本估计为3,201欧元(60%与护理人员出勤有关,40%归因于护理人员缺勤)。最后,意大利每位成年SBS患者的年平均总成本估计为87,527欧元(占直接成本的42%和间接成本的58%),而儿科患者的年平均总成本估计为49,882欧元(占直接成本的94%和间接成本的6%)。总体而言,意大利SBS患者的加权平均成本估计为81,712欧元(47%的直接成本和53%的间接成本)。对SBS患者的生活质量进行的分析表明,生活质量被认为很低(平均值等于5)。对生活质量的最大影响是由于疾病(平均值等于9),而PN似乎影响较小(平均值等于6)。结论:该分析从成本和生活质量两方面估计了意大利SBS患者的总负担。与肠外营养相关的费用和间接费用是意大利SBS患者估计总费用的主要驱动因素。根据本研究涉及的KOL的经验,我们也发现疾病对这些患者的生活质量有很大的影响。
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引用次数: 0
Cost-effectiveness of Empagliflozin, in Addition to Metformin, in Patients with Type 2 Diabetes in Italy 在意大利,恩格列净和二甲双胍在2型糖尿病患者中的成本效益
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-12-23 DOI: 10.7175/fe.v23i1.1539
E. Mannucci, G. Ghetti
BACKGROUND: Cardiovascular diseases represent the main cause of mortality and morbidity in type 2 diabetes mellitus (T2DM) patients. Empagliflozin is used as a treatment for T2DM because of its association with reduced risk of hospitalization for heart failure (hHF). Recently oral semaglutide, in association with metformin, has shown better results. This study analyzes the cost-effectiveness of empagliflozin versus oral semaglutide, in addition to metformin, in patients with T2DM who are inadequately controlled on metformin alone in Italy.METHODS: This analysis was conducted from the Italian National Health Service (SSN) perspective using the IQVIA Core Diabetes Model. For the base case analysis, a 50-year time horizon was chosen to capture the complications, their associated costs, and the final impact on life-years (LYs) and quality-adjusted life-years (QALYs) gained. Cohort baseline characteristics and efficacy data, were mainly sourced from the PIONEER 2 study. Health-state utilities and event disutilities were based on published sources. Drug acquisition and administration costs and patient management inputs were sourced from Italian-specific data. A sensitivity analysis and a range of scenario analyses were carried out.RESULTS: In the base case analysis treatment cost of empagliflozin plus metformin were significantly lower compared to oral semaglutide plus metformin both including and excluding the effect of empagliflozin on hHF (€-13.371/€-13.580; LYs -0.004/0.109 and QALYs -0.037/0.038). The sensitivity analysis confirmed the robustness of the model with empagliflozin plus metformin that was dominant in 63% and in 42% of simulations considering and non-considering the treatment effect on hHF, respectively.CONCLUSIONS: Empagliflozin 25 mg plus metformin is a cost-effective option versus oral semaglutide 14 mg plus metformin for patients with T2DM uncontrolled on metformin alone in Italy
背景:心血管疾病是2型糖尿病(T2DM)患者死亡和发病的主要原因。恩帕列净被用于治疗2型糖尿病,因为它与降低心力衰竭住院风险(hHF)有关。最近口服西马鲁肽联合二甲双胍显示出更好的效果。本研究分析了在意大利单独使用二甲双胍控制不充分的T2DM患者中,恩格列净与口服semaglutide以及二甲双胍的成本-效果。方法:采用IQVIA核心糖尿病模型,从意大利国家卫生服务(SSN)的角度进行分析。对于基本案例分析,选择50年的时间范围来捕获并发症、相关成本以及对获得的生命年(LYs)和质量调整生命年(QALYs)的最终影响。队列基线特征和疗效数据主要来自PIONEER 2研究。运行状况实用程序和事件不实用程序基于已发布的来源。药品采购和管理费用以及患者管理投入来自意大利特定数据。进行了敏感性分析和一系列情景分析。结果:在基本病例分析中,恩帕列净加二甲双胍的治疗成本显著低于口服西马鲁肽加二甲双胍,包括和不包括恩帕列净对hHF的影响(€-13.371/€-13.580;LYs -0.004/0.109和QALYs -0.037/0.038)。敏感性分析证实了恩格列净加二甲双胍模型的稳健性,在考虑和不考虑hHF治疗效果的模拟中,恩格列净加二甲双胍分别占63%和42%的优势。结论:在意大利,恩帕列净25mg +二甲双胍与口服semaglutide 14mg +二甲双胍相比,对于单独使用二甲双胍控制的2型糖尿病患者而言,是一种更具成本效益的选择
{"title":"Cost-effectiveness of Empagliflozin, in Addition to Metformin, in Patients with Type 2 Diabetes in Italy","authors":"E. Mannucci, G. Ghetti","doi":"10.7175/fe.v23i1.1539","DOIUrl":"https://doi.org/10.7175/fe.v23i1.1539","url":null,"abstract":"BACKGROUND: Cardiovascular diseases represent the main cause of mortality and morbidity in type 2 diabetes mellitus (T2DM) patients. Empagliflozin is used as a treatment for T2DM because of its association with reduced risk of hospitalization for heart failure (hHF). Recently oral semaglutide, in association with metformin, has shown better results. This study analyzes the cost-effectiveness of empagliflozin versus oral semaglutide, in addition to metformin, in patients with T2DM who are inadequately controlled on metformin alone in Italy.METHODS: This analysis was conducted from the Italian National Health Service (SSN) perspective using the IQVIA Core Diabetes Model. For the base case analysis, a 50-year time horizon was chosen to capture the complications, their associated costs, and the final impact on life-years (LYs) and quality-adjusted life-years (QALYs) gained. Cohort baseline characteristics and efficacy data, were mainly sourced from the PIONEER 2 study. Health-state utilities and event disutilities were based on published sources. Drug acquisition and administration costs and patient management inputs were sourced from Italian-specific data. A sensitivity analysis and a range of scenario analyses were carried out.RESULTS: In the base case analysis treatment cost of empagliflozin plus metformin were significantly lower compared to oral semaglutide plus metformin both including and excluding the effect of empagliflozin on hHF (€-13.371/€-13.580; LYs -0.004/0.109 and QALYs -0.037/0.038). The sensitivity analysis confirmed the robustness of the model with empagliflozin plus metformin that was dominant in 63% and in 42% of simulations considering and non-considering the treatment effect on hHF, respectively.CONCLUSIONS: Empagliflozin 25 mg plus metformin is a cost-effective option versus oral semaglutide 14 mg plus metformin for patients with T2DM uncontrolled on metformin alone in Italy","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"22 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2022-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"83515140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Budget Impact Analysis of the Adjuvanted Quadrivalent Influenza Vaccine in the Elderly in Italy] [意大利老年人佐剂四价流感疫苗的预算影响分析]
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-11-30 DOI: 10.7175/fe.v23i1.1538
Vincenzo Baldo, M. Bellone
BACKGROUND: Vaccination is the most effective way to prevent influenza and its complications. The MF59-adjuvanted quadrivalent (aQIV) and the high-dose quadrivalent (QIV-HD) influenza vaccines have been specifically developed to protect subjects aged ≥65 years. The aim of this study was to evaluate the economic consequences associated with the use of aQIV in the elderly population in Italy.METHODS: An Excel-based budget impact model was developed to estimate the costs of aQIV for the prevention of influenza and its complications in elderly subjects, from the perspective of the Italian National Health Service. In the base-case analysis a current scenario (with aQIV) was compared with a past scenario (without aQIV), in which only the standard quadrivalent influenza vaccine (QIV-STD) was available. In the scenario analysis, a current scenario was compared with a future (hypothetical) scenario, in which the market share of aQIV grows. Efficacy (or effectiveness) data of vaccines, in terms of reduction of influenza-related deaths and hospitalizations for influenza/pneumonia, respiratory, and cardiac complications, were obtained from the scientific literature. Relative effectiveness of aQIV and QIV-HD vs. QIV-STD in preventing laboratory-confirmed influenza cases came from two meta-analysis of real-world studies. Epidemiological data and unit costs are collected from Italian published sources.RESULTS: In the base-case analysis the introduction of aQIV e QIV-STD was associated with a reduction of influenza and influenza-like illness – ILI (-93,171) cases, hospitalization for influenza, respiratory and cardiovascular complications (-6,823), and deaths (-1,841) and a consequent saving of € 1.9 million and € 24 million related to events and hospital admissions avoided, respectively. In the scenario analysis, the growth of the market shares of aQIV was associated with 13,602 fewer cases of influenza and ILI, 996 fewer hospitalizations, and an overall saving equal to € 3.8 million.CONCLUSIONS: aQIV reduces the number of cases of influenza and ILI, hospitalizations for influenza and respiratory or cardiac complications, and deaths in the Italian population aged ≥65 years.
背景:疫苗接种是预防流感及其并发症最有效的方法。mf59佐剂四价(aQIV)和高剂量四价(QIV-HD)流感疫苗是专门为保护年龄≥65岁的受试者而开发的。本研究的目的是评估在意大利老年人中使用aQIV的相关经济后果。方法:建立基于excel的预算影响模型,从意大利国家卫生服务的角度估计aQIV预防老年人流感及其并发症的成本。在基础病例分析中,将当前的情况(有aQIV)与过去的情况(没有aQIV)进行比较,在过去的情况下,只有标准的四价流感疫苗(QIV-STD)可用。在情景分析中,将当前情景与未来(假设)情景进行比较,在未来情景中,aQIV的市场份额将增长。疫苗在减少流感相关死亡和因流感/肺炎、呼吸道和心脏并发症住院方面的功效(或有效性)数据是从科学文献中获得的。aQIV和QIV-HD与QIV-STD在预防实验室确诊流感病例中的相对有效性来自对现实世界研究的两项荟萃分析。流行病学数据和单位费用从意大利出版的资料中收集。结果:在基础病例分析中,引入aQIV和QIV-STD与流感和流感样疾病——ILI(- 93171例)、流感、呼吸系统和心血管并发症住院(- 6823例)和死亡(- 1841例)的减少有关,并因此分别节省190万欧元和2400万欧元的相关事件和住院费用。在情景分析中,aQIV市场份额的增长与减少13602例流感和ILI病例,减少996例住院治疗相关,总体节省相当于380万欧元。结论:aQIV减少了意大利≥65岁人群中流感和ILI病例、流感和呼吸或心脏并发症住院人数以及死亡人数。
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引用次数: 0
Economic Consequences of Administering Obinutuzumab as a Short Duration Infusion in Italian Patients with Advanced Follicular Lymphoma: A Cost Analysis 意大利晚期滤泡性淋巴瘤患者短期输注Obinutuzumab的经济后果:成本分析
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-11-07 DOI: 10.7175/fe.v23i1.1532
M. Bellone, L. Pradelli, R. Tavarozzi, M. Ladetto, G. Nepoti, E. Guardalben, D. Ghislieri
OBJECTIVE: To assess time- and cost-savings in relation to active time of health care professional (HCP) and resource consumption of administering obinutuzumab as a short duration infusion (SDI) in patients in treatment for Follicular Lymphoma (FL).METHODS: A cost-minimization model was developed to compare resource consumption and cost of the obinutuzumab SDI relative to obinutuzumab regular infusion rate (RIR) for the previously untreated and rituximab-refractory FL. Monetary valuation of resource and time allocated to treatment as a whole was carried out from the Italian Hospital and the societal perspective. Direct costs included HCP costs for drug preparation and administration activities, non-drug consumable costs, drug acquisition costs, and formal care costs. Indirect costs included the lost productivity of patients and informal caregivers. All costs (updated to 2021-value) were estimated by multiplying resource use by the unit cost of each resource. Evidence on resource use and unit costs were retrieved from scientific literature and standard Italian tariffs. A deterministic sensitivity analysis was used to test the results.RESULTS: The administration time of obinutuzumab SDI is shorter than with obinutuzumab RIR, with a difference of 102 minutes per patient and for every cycle of administration beyond the first one. On average, the cost of HCP time invested in the preparation and administration of obinutuzumab RIR is € 92 during cycle 2 and from cycle 2 onwards, compared to € 54 per cycle of obinutuzumab SDI. Overall, the cost from the societal perspective is estimated to be € 38,698 for obinutuzumab RIR and € 37,692 for obinutuzumab SDI, resulting in a cost-saving per patient of € 1,007 (2.6%).CONCLUSIONS: The application of obinutuzumab SDI schedule allows substantial reduction of hospital stay, improving quality of life of patient and caregiver and reducing costs and health care system burden. The time-savings with obinutuzumab SDI may improve clinical unit capacity by optimizing chair utilization and/or allowing rearrangements of the nurse residual time into valuable supplementary activities, spanning from more patient-centered clinical support to research and learning activity
目的:评估在治疗滤泡性淋巴瘤(FL)的患者中,短时间输注(SDI)给予obinutuzumab治疗的时间和成本节约与卫生保健专业人员(HCP)的活动时间和资源消耗有关。方法:开发成本最小化模型,比较先前未治疗和利妥昔单抗难治性FL的obinutuzumab SDI相对于obinutuzumab常规输注率(RIR)的资源消耗和成本。从意大利医院和社会角度对分配给治疗的资源和时间进行整体货币评估。直接成本包括药物制备和管理活动的HCP成本、非药物耗材成本、药物获取成本和正式护理成本。间接成本包括病人和非正式护理人员的生产力损失。所有成本(更新到2021年的值)通过将资源使用量乘以每种资源的单位成本来估算。有关资源利用和单位成本的证据是从科学文献和意大利标准关税中检索的。采用确定性敏感性分析对结果进行检验。结果:奥比妥珠单抗SDI给药时间比奥比妥珠单抗RIR给药时间短,每个患者和第一个给药周期后的每个给药周期的差异为102分钟。平均而言,在第2周期和第2周期以后,用于制备和给药obinutuzumab RIR的HCP时间成本为92欧元,而obinutuzumab SDI每个周期为54欧元。总体而言,从社会角度来看,obinutuzumab RIR的成本估计为38,698欧元,obinutuzumab SDI的成本估计为37,692欧元,导致每位患者节省成本1,007欧元(2.6%)。结论:应用obinutuzumab SDI方案可大幅减少住院时间,改善患者和护理人员的生活质量,降低成本和卫生保健系统负担。obinutuzumab SDI节省的时间可以通过优化椅子的利用和/或允许护士将剩余时间重新安排为有价值的补充活动(从更多以患者为中心的临床支持到研究和学习活动)来提高临床单位的能力
{"title":"Economic Consequences of Administering Obinutuzumab as a Short Duration Infusion in Italian Patients with Advanced Follicular Lymphoma: A Cost Analysis","authors":"M. Bellone, L. Pradelli, R. Tavarozzi, M. Ladetto, G. Nepoti, E. Guardalben, D. Ghislieri","doi":"10.7175/fe.v23i1.1532","DOIUrl":"https://doi.org/10.7175/fe.v23i1.1532","url":null,"abstract":"OBJECTIVE: To assess time- and cost-savings in relation to active time of health care professional (HCP) and resource consumption of administering obinutuzumab as a short duration infusion (SDI) in patients in treatment for Follicular Lymphoma (FL).METHODS: A cost-minimization model was developed to compare resource consumption and cost of the obinutuzumab SDI relative to obinutuzumab regular infusion rate (RIR) for the previously untreated and rituximab-refractory FL. Monetary valuation of resource and time allocated to treatment as a whole was carried out from the Italian Hospital and the societal perspective. Direct costs included HCP costs for drug preparation and administration activities, non-drug consumable costs, drug acquisition costs, and formal care costs. Indirect costs included the lost productivity of patients and informal caregivers. All costs (updated to 2021-value) were estimated by multiplying resource use by the unit cost of each resource. Evidence on resource use and unit costs were retrieved from scientific literature and standard Italian tariffs. A deterministic sensitivity analysis was used to test the results.RESULTS: The administration time of obinutuzumab SDI is shorter than with obinutuzumab RIR, with a difference of 102 minutes per patient and for every cycle of administration beyond the first one. On average, the cost of HCP time invested in the preparation and administration of obinutuzumab RIR is € 92 during cycle 2 and from cycle 2 onwards, compared to € 54 per cycle of obinutuzumab SDI. Overall, the cost from the societal perspective is estimated to be € 38,698 for obinutuzumab RIR and € 37,692 for obinutuzumab SDI, resulting in a cost-saving per patient of € 1,007 (2.6%).CONCLUSIONS: The application of obinutuzumab SDI schedule allows substantial reduction of hospital stay, improving quality of life of patient and caregiver and reducing costs and health care system burden. The time-savings with obinutuzumab SDI may improve clinical unit capacity by optimizing chair utilization and/or allowing rearrangements of the nurse residual time into valuable supplementary activities, spanning from more patient-centered clinical support to research and learning activity","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"22 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2022-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"87551069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Cost-Utility Analysis of Dupilumab for the Treatment of Severe Atopic Dermatitis in Children and Adolescents in Italy] 【Dupilumab治疗意大利儿童和青少年严重特应性皮炎的成本-效用分析】
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-06-22 DOI: 10.7175/fe.v23i1.1527
E. Galli, M. Pedone, Miryana Dobreva, R. Bitonti, R. Di Turi
BACKGROUND AND OBJECTIVE: Atopic dermatitis (AD) is a chronic, multifactorial, inflammatory condition characterized by a significant impact on patients’ quality of life. Dupilumab is reimbursed by the Italian Medicines Agency (AIFA) for the treatment of adolescent and adult patients with severe AD (according to AIFA registry criteria). Recently, dupilumab has been reimbursed in the treatment of children with severe AD. The objective of this analysis was to estimate the incremental cost-utility ratio (ICUR) of dupilumab compared to current supportive care (SC), for the treatment of severe AD in children (6-11 years) and adolescents (12-17 years) in Italy.MATERIALS AND METHODS: Cost-effectiveness analysis was conducted using a 1-year decision tree followed by a Markov model over a lifetime period. The base case analysis was performed on the overall population of the LIBERTY AD ADOL (NCT03054428) and LIBERTY AD PEDS (NCT03345914) studies, adopting the National Health Service (NHS) perspective. The following costs were considered: acquisition of treatment, management of disease, adverse events and complications. The robustness of the model was tested through sensitivity analysis. In addition, a scenario analysis adopting the social perspective was performed.RESULTS: In the base case, over a lifetime, dupilumab was more effective than SC in both children and adolescents (+2.44 and +1.62 quality-adjusted life years—QALYs, respectively). The introduction of dupilumab generated an increase in treatment costs (+€ 64,800 and +€ 52,853 € for children and adolescents, respectively), partially offset by a decrease in the costs of disease management and complications. Incremental cost-utility ratios (ICURs) were € 21,189 per QALY gained, for children, and € 26,569 per QALY gained, for adolescents. In both cases, the ICUR was lower than the willingness to pay threshold considered in Italy (€ 50,000 per QALY gained). Both the deterministic and probabilistic sensitivity analysis confirmed the robustness of the base case results. Finally, the scenario analysis, adopting the social perspective, showed coherent results compared to the base case.DISCUSSION: Dupilumab is a cost-effective option for the treatment of children and adolescents with severe AD eligible for systemic treatment in Italy compared to SC, from both the NHS and social perspective, confirming the results obtained in the adult population.
背景与目的:特应性皮炎(AD)是一种慢性、多因素的炎症性疾病,其特点是对患者的生活质量有显著影响。Dupilumab由意大利药品管理局(AIFA)报销,用于治疗青少年和成人严重AD患者(根据AIFA注册标准)。最近,dupilumab已被报销用于治疗严重AD患儿。本分析的目的是评估dupilumab与当前支持治疗(SC)相比的增量成本效用比(ICUR),用于治疗意大利儿童(6-11岁)和青少年(12-17岁)的严重AD。材料和方法:成本-效果分析采用1年的决策树和马尔可夫模型进行。采用国家卫生服务(NHS)的观点,对LIBERTY AD ADOL (NCT03054428)和LIBERTY AD PEDS (NCT03345914)研究的总体人群进行基本病例分析。考虑了以下费用:获得治疗、疾病管理、不良事件和并发症。通过敏感性分析检验模型的稳健性。此外,采用社会视角进行情景分析。结果:在基本情况下,在儿童和青少年的一生中,dupilumab比SC更有效(分别为+2.44和+1.62质量调整生命年)。dupilumab的引入导致了治疗费用的增加(儿童和青少年分别为+ 64,800欧元和+ 52,853欧元),部分被疾病管理和并发症费用的减少所抵消。儿童的增量成本效用比(ICURs)为21,189欧元/ QALY,青少年的增量成本效用比为26,569欧元/ QALY。在这两种情况下,ICUR都低于意大利考虑的支付意愿门槛(每获得QALY 50,000欧元)。确定性和概率敏感性分析均证实了基本情况结果的稳健性。最后,采用社会视角的情景分析显示了与基本情况相一致的结果。讨论:从NHS和社会的角度来看,与SC相比,Dupilumab是治疗在意大利有资格接受全身治疗的严重AD儿童和青少年的一种成本效益选择,证实了在成人人群中获得的结果。
{"title":"[Cost-Utility Analysis of Dupilumab for the Treatment of Severe Atopic Dermatitis in Children and Adolescents in Italy]","authors":"E. Galli, M. Pedone, Miryana Dobreva, R. Bitonti, R. Di Turi","doi":"10.7175/fe.v23i1.1527","DOIUrl":"https://doi.org/10.7175/fe.v23i1.1527","url":null,"abstract":"BACKGROUND AND OBJECTIVE: Atopic dermatitis (AD) is a chronic, multifactorial, inflammatory condition characterized by a significant impact on patients’ quality of life. Dupilumab is reimbursed by the Italian Medicines Agency (AIFA) for the treatment of adolescent and adult patients with severe AD (according to AIFA registry criteria). Recently, dupilumab has been reimbursed in the treatment of children with severe AD. The objective of this analysis was to estimate the incremental cost-utility ratio (ICUR) of dupilumab compared to current supportive care (SC), for the treatment of severe AD in children (6-11 years) and adolescents (12-17 years) in Italy.MATERIALS AND METHODS: Cost-effectiveness analysis was conducted using a 1-year decision tree followed by a Markov model over a lifetime period. The base case analysis was performed on the overall population of the LIBERTY AD ADOL (NCT03054428) and LIBERTY AD PEDS (NCT03345914) studies, adopting the National Health Service (NHS) perspective. The following costs were considered: acquisition of treatment, management of disease, adverse events and complications. The robustness of the model was tested through sensitivity analysis. In addition, a scenario analysis adopting the social perspective was performed.RESULTS: In the base case, over a lifetime, dupilumab was more effective than SC in both children and adolescents (+2.44 and +1.62 quality-adjusted life years—QALYs, respectively). The introduction of dupilumab generated an increase in treatment costs (+€ 64,800 and +€ 52,853 € for children and adolescents, respectively), partially offset by a decrease in the costs of disease management and complications. Incremental cost-utility ratios (ICURs) were € 21,189 per QALY gained, for children, and € 26,569 per QALY gained, for adolescents. In both cases, the ICUR was lower than the willingness to pay threshold considered in Italy (€ 50,000 per QALY gained). Both the deterministic and probabilistic sensitivity analysis confirmed the robustness of the base case results. Finally, the scenario analysis, adopting the social perspective, showed coherent results compared to the base case.DISCUSSION: Dupilumab is a cost-effective option for the treatment of children and adolescents with severe AD eligible for systemic treatment in Italy compared to SC, from both the NHS and social perspective, confirming the results obtained in the adult population.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"140 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2022-06-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78131589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Artificial Urinary Sphincters as a Treatment for Post-Prostatectomy Severe Urinary Incontinence in Italy: A Cost-Utility Analysis 人工尿道括约肌治疗意大利前列腺切除术后严重尿失禁:成本-效用分析
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-05-19 DOI: 10.7175/fe.v23i1.1525
F. Mennini, D. Rossi, A. Marcellusi
Objective: This study aimed at evaluating the cost-utility of artificial urinary sphincter (AUS) in men affected by postprostatectomy severe urinary incontinence and identifying the most cost-effective alternative among the various devices analyzed in Italy.Methods: A 5-year cycles Markov model was developed to simulate the disease evolution. The analysis compared conservative therapy, ZSI 375®, single-cuff (SC) AMS 800TM, and double-cuff (DC) AMS 800TM. A Probabilistic Sensitivity Analysis (PSA) was performed. One thousand Monte Carlo simulations were conducted to generate the Cost-Effectiveness Acceptability Curve for each intervention strategy. A sensitivity analysis on the price of the device was conducted.Results: From the Italian National Health Service perspective, DC AMS 800TM was the most cost-effective alternative in comparison with conservative therapy, with an Incremental Cost-Effectiveness Ratio (ICER) value equal to € 12,893. From the NHS + patient perspective, both the AMS 800TM devices (SC and DC) were dominant in comparison with conservative therapy. From the societal perspective, ICER was dominant for all the alternatives considered in terms of cost-effectiveness. The PSA showed that DC AMS 800TM had a greater probability to be cost-effective with respect to the other strategies considered in the analysis. The sensitivity analysis on the price of the device showed that in all the cases analyzed the incremental cost per QALY gained would be below € 25,000.Conclusions: This cost-utility analysis confirms that AUSs are cost-effective options in the Italian context with respect to conservative therapy. Among AUSs, DC AMS 800TM has the greatest probability to be cost-effective.
目的:本研究旨在评估人工尿道括约肌(AUS)用于前列腺切除术后严重尿失禁男性的成本效益,并在意大利分析的各种装置中确定最具成本效益的替代方案。方法:建立5年周期马尔可夫模型,模拟疾病演变。分析比较了保守治疗、ZSI 375®、单袖带(SC) AMS 800TM和双袖带(DC) AMS 800TM。进行概率敏感性分析(PSA)。进行了1000次蒙特卡罗模拟,以生成每种干预策略的成本-效益可接受度曲线。对该设备的价格进行了敏感性分析。结果:从意大利国家卫生服务的角度来看,与保守治疗相比,DC AMS 800TM是最具成本效益的替代方案,增量成本-效果比(ICER)值为12,893欧元。从NHS +患者的角度来看,与保守治疗相比,AMS 800TM设备(SC和DC)都占主导地位。从社会角度来看,ICER在所有考虑成本效益的替代方案中占主导地位。PSA表明,与分析中考虑的其他策略相比,DC AMS 800TM具有更大的成本效益。对设备价格的敏感性分析表明,在所分析的所有案例中,每个QALY获得的增量成本将低于25,000欧元。结论:该成本效用分析证实,在意大利背景下,相对于保守治疗,AUSs是具有成本效益的选择。在aus中,DC AMS 800TM最有可能具有成本效益。
{"title":"Artificial Urinary Sphincters as a Treatment for Post-Prostatectomy Severe Urinary Incontinence in Italy: A Cost-Utility Analysis","authors":"F. Mennini, D. Rossi, A. Marcellusi","doi":"10.7175/fe.v23i1.1525","DOIUrl":"https://doi.org/10.7175/fe.v23i1.1525","url":null,"abstract":"Objective: This study aimed at evaluating the cost-utility of artificial urinary sphincter (AUS) in men affected by postprostatectomy severe urinary incontinence and identifying the most cost-effective alternative among the various devices analyzed in Italy.Methods: A 5-year cycles Markov model was developed to simulate the disease evolution. The analysis compared conservative therapy, ZSI 375®, single-cuff (SC) AMS 800TM, and double-cuff (DC) AMS 800TM. A Probabilistic Sensitivity Analysis (PSA) was performed. One thousand Monte Carlo simulations were conducted to generate the Cost-Effectiveness Acceptability Curve for each intervention strategy. A sensitivity analysis on the price of the device was conducted.Results: From the Italian National Health Service perspective, DC AMS 800TM was the most cost-effective alternative in comparison with conservative therapy, with an Incremental Cost-Effectiveness Ratio (ICER) value equal to € 12,893. From the NHS + patient perspective, both the AMS 800TM devices (SC and DC) were dominant in comparison with conservative therapy. From the societal perspective, ICER was dominant for all the alternatives considered in terms of cost-effectiveness. The PSA showed that DC AMS 800TM had a greater probability to be cost-effective with respect to the other strategies considered in the analysis. The sensitivity analysis on the price of the device showed that in all the cases analyzed the incremental cost per QALY gained would be below € 25,000.Conclusions: This cost-utility analysis confirms that AUSs are cost-effective options in the Italian context with respect to conservative therapy. Among AUSs, DC AMS 800TM has the greatest probability to be cost-effective.","PeriodicalId":41585,"journal":{"name":"Farmeconomia-Health Economics and Therapeutic Pathways","volume":"1997 1","pages":""},"PeriodicalIF":0.5,"publicationDate":"2022-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82510842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Importance of Real-World Evidence in Understanding Influenza Vaccine Effectiveness 真实世界证据对了解流感疫苗有效性的重要性
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-03-03 DOI: 10.7175/fe.v23i1.1522
P. Ferrara, L. Mantovani
© 2022 The Authors. Published by SEEd srl. This is an open access article under the CC BY-NC 4.0 license (https://creativecommons.org/licenses/by-nc/4.0) BACKGROUND Seasonal influenza is an acute viral respiratory disease which circulates globally with constantly evolving epidemiology [1]. Influenza viruses can infect up to 20% of the global population each year, resulting in more than 650,000 annual deaths [2]. Seasonal influenza is therefore a major cause of illness, associated with substantial health and economic burdens, due to its impact on healthcare – with increased medical resource utilization and costs, and on society – with enormous death toll and loss of production [3]. Effective vaccines and timely vaccination programs are crucial strategies for the control of seasonal influenza, the spread and severity of which is unpredictable. Influenza viruses are constantly changing due to different types of mutation in viral genes that result in altered surface glycoproteins (hemagglutinin and neuraminidase), which enables viruses to escape the antigen-specific immunity that is induced by prior infections and/or vaccination [1]. Changes in antigenic sites result from either common point mutations during viral replication, which cause small changes in surface proteins (antigenic drift), or from less frequent significant genetic reassortment and the consequent emergence of novel virus subtypes or clades (antigenic shift) [4]. Antigenic evolution and regional variations in environmental strain / clade dominance require that the antigen composition of seasonal influenza vaccines is changed annually [1,4]. Annual vaccination is the best way to prevent influenza disease in people aged 6 months and older [3]. Influenza vaccines are proven to be economically favorable in specific populations including older adults, people with chronic medical problems, pregnant women, and children – from both healthcare and societal perspectives [3]. Therefore, the appropriate evaluation of influenza vaccines based on epidemiological and economic data is of the utmost importance for accurately informing policymakers and allocating resources for seasonal influenza vaccination programs [2,3].
©2022作者。由SEEd出版社出版。这是一篇基于CC BY-NC 4.0许可的开放获取文章(https://creativecommons.org/licenses/by-nc/4.0)背景季节性流感是一种急性病毒性呼吸道疾病,在全球范围内流行,流行病学不断演变[1]。流感病毒每年可感染全球20%的人口,每年造成65万多人死亡[2]。因此,季节性流感是疾病的主要原因,由于其对医疗保健的影响(增加医疗资源的利用和成本)和对社会的影响(造成巨大的死亡人数和生产损失),与重大的健康和经济负担相关[3]。有效的疫苗和及时的疫苗接种规划是控制季节性流感的关键战略,季节性流感的传播和严重程度是不可预测的。由于病毒基因的不同类型突变导致表面糖蛋白(血凝素和神经氨酸酶)发生改变,流感病毒不断发生变化,这使得病毒能够逃脱由先前感染和/或疫苗接种引起的抗原特异性免疫[1]。抗原位点的变化可能是由于病毒复制过程中的共同点突变引起表面蛋白的微小变化(抗原漂移),也可能是由于不太频繁的显著基因重组以及由此产生的新型病毒亚型或分支(抗原漂移)[4]。抗原进化和环境毒株/进化支系优势的区域差异要求季节性流感疫苗的抗原组成每年发生变化[1,4]。每年接种疫苗是6个月及以上人群预防流感疾病的最佳途径[3]。从医疗保健和社会的角度来看,流感疫苗已被证明对特定人群具有经济效益,包括老年人、慢性疾病患者、孕妇和儿童[3]。因此,基于流行病学和经济数据对流感疫苗进行适当评估,对于准确告知决策者并为季节性流感疫苗接种规划分配资源至关重要[2,3]。
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引用次数: 4
Cost-Effectiveness of Dimethyl Fumarate Compared to Teriflunomide for Relapsing Remitting Multiple Sclerosis Patients in Italy 意大利富马酸二甲酯与特立氟米特治疗复发缓解型多发性硬化症患者的成本-效果比较
IF 0.5 Q4 HEALTH CARE SCIENCES & SERVICES Pub Date : 2022-02-07 DOI: 10.7175/fe.v23i1.1510
L. Mantovani, P. Cortesi, Alessandra Cardillo, L. Santoni, L. Prosperini
BACKGROUND: The objective of this economic analysis was to compare the cost-effectiveness of dimethyl fumarate vs teriflunomide for the treatment of adult patients with relapsing-remitting multiple sclerosis (RRMS) in the Italian setting. Additionally, the cost-effectiveness analysis was used to predict some patient-relevant outcomes such as burden of relapses and survival with disability over time.METHODS: A Markov model was used to conduct the cost-effectiveness analysis. The model measured health outcomes and costs of RRMS patients treated with either dimethyl fumarate or teriflunomide. Data from a published mixed treatment comparison were used for efficacy and safety input. Local economic data were used to calculate costs. A supplementary analysis was carried out to assess ICER variability over time from the Italian National Healthcare Service (NHS) and societal perspectives. Further analyses were conducted to compare clinical effectiveness of the alternatives over time, in terms of incidence of relapses, proportion of patients with EDSS (Expanded Disability Status Scale) score ≤3 and EDSS score ≥6.RESULTS: In the base-case analysis (lifetime horizon; societal perspective) dimethyl fumarate was dominant over teriflunomide (6.526 vs 5.953 QALYs – quality-adjusted life-years; € 1.01 M vs € 1.03 M). The most relevant cost savings (per-patient) with dimethyl fumarate were related to relapses (-€ 5,096), inpatient care (-€ 5,767), informal care (-€ 9,603), long-term absence/early retirement (-€ 14,187). The additional analysis of ICER by time horizon shows that dimethyl fumarate is cost-effective vs teriflunomide (i.e., ICER <€ 50,000 per QALY gained) at already 6 years and at 15 years in societal or NHS perspectives, respectively. Results favoured dimethyl fumarate vs teriflunomide also for: cumulative burden of relapses (-0.23 and -1.37 relapses saved per patient already at 1 year and 10 years, respectively), proportion of patients with mild disability (+4.0% at 10 years), proportion of patients with severe disability (-4.0% at 10 years).CONCLUSIONS: Dimethyl fumarate is dominant (societal perspective), or cost-effective (NHS perspective), referring to a threshold of € 50,000 per QALY gained, vs teriflunomide for the first-line treatment of RRMS, in the Italian setting.
背景:本经济分析的目的是比较富马酸二甲酯与特立氟米特治疗意大利复发-缓解型多发性硬化症(RRMS)成人患者的成本-效果。此外,成本-效果分析用于预测一些与患者相关的结果,如复发负担和残疾生存率。方法:采用马尔可夫模型进行成本-效果分析。该模型测量了用富马酸二甲酯或特立氟米特治疗的RRMS患者的健康结果和成本。来自已发表的混合治疗比较的数据用于疗效和安全性输入。当地经济数据被用来计算成本。从意大利国家医疗保健服务(NHS)和社会角度评估ICER随时间变化的补充分析。在复发率、EDSS(扩展残疾状态量表)评分≤3分和EDSS评分≥6分的患者比例方面,进一步分析比较两种替代方案随时间的临床疗效。结果:在基本情况分析中(生命周期;社会视角)富马酸二甲酯优于特立氟米特(6.526 vs 5.953);富马酸二甲酯最相关的成本节约(每位患者)与复发(- 5,096欧元)、住院护理(- 5,767欧元)、非正式护理(- 9,603欧元)、长期缺勤/提前退休(- 14,187欧元)相关。按时间范围对ICER进行的额外分析表明,从社会或NHS的角度来看,富马酸二甲酯与特立氟米特相比,在已经6年和15年的情况下具有成本效益(即,ICER < 50,000欧元/每获得的QALY)。结果显示富马酸二甲酯和特立氟米特在以下方面均优于富马酸二甲酯和特立氟米特:累积复发负担(1年和10年分别为每位患者减少0.23次和-1.37次复发),轻度残疾患者比例(10年+4.0%),严重残疾患者比例(10年-4.0%)。结论:在意大利,富马酸二甲酯是主要的(社会观点),或具有成本效益(NHS观点),指的是每个QALY获得50,000欧元的阈值,而特立氟米特用于RRMS的一线治疗。
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Farmeconomia-Health Economics and Therapeutic Pathways
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