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Erratum: Article Correction: We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It? 文章更正:我们正处于突破1型糖尿病治疗的边缘,但200万美国人是谁?
IF 2.3 Q2 ECONOMICS Pub Date : 2025-04-18 eCollection Date: 2025-01-01 DOI: 10.36469/001c.134140
Rebecca Smith, Samara Eisenberg, Aaron Turner-Phifer, Jacqueline LeGrand, Sarah Pincus, Yousra Omer, Fei Wang, Bruce Pyenson

[This corrects the article DOI: 10.36469/jheor.2024.124604.].

[这更正了文章DOI: 10.36469/jheor.2024.124604.]。
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引用次数: 0
Unveiling the Public Economic Burden of Migraine in Argentina. 揭示阿根廷偏头痛的公共经济负担。
IF 2.3 Q2 ECONOMICS Pub Date : 2025-04-09 eCollection Date: 2025-01-01 DOI: 10.36469/001c.133639
Zrinka Orlović, Lucila Rey-Ares, María Florencia Viozzi, Rui Martins, Juliana Villarreal Ramírez, Santiago Veiga, Mark P Connolly

Background: Migraine is a prevalent, underdiagnosed, highly debilitating neurological condition that affects individuals' quality of life and often negatively influences normal daily activities. Objectives: The study objective is to estimate the economic burden of migraine to the Argentine government by assessing the impact of the disease on tax revenue, absenteeism, and social support transfers. Methods: The analysis combines a cross-sectional model utilizing national demographic data and published migraine prevalence rates to estimate the annual burden for the entire migraine-affected cohort, and a longitudinal model assessing the average burden per individual from the age of 40, over a 20-year horizon. A fiscal framework based on generational accounting evaluated the impact of migraine on government finances. Sources of revenue such as direct and indirect taxes were weighted against elements of public expenditure (public sector absenteeism, healthcare expenses, and financial support) and compared with the general population. The effect of migraine on occupational outcomes was sourced from peer-reviewed publications, and costs were sourced from national databases. Results were reported as incremental fiscal consequences (2023 US dollars) and were discounted at 3% annually. Results: The fiscal burden of migraine in Argentina was estimated to be 6505 p e r i n d i v i d u a l a n d 1237 million across the entire migraine population. Annually, 29% of government costs were due to public sector absenteeism, 39% related to healthcare costs, 19% to foregone direct and indirect tax revenue, and 12% to foregone corporation taxes. Additional government transfers represented a minor contribution to the overall fiscal impact of migraine in Argentina. Discussion: The high rate of informal employment is likely to undermine disease burden estimates. Gender disparities were notable, with women bearing 76% of the burden, highlighting the need for gender-specific interventions. Conclusions: This study reveals a significant economic burden of migraine to the Argentinian government, primarily driven by absenteeism, healthcare costs, and foregone tax contributions. Targeted, gender-responsive healthcare and labor policies, especially for sectors with high informal employment, could help reduce these fiscal impacts.

背景:偏头痛是一种普遍的、未被诊断的、高度衰弱的神经系统疾病,它会影响个体的生活质量,并经常对正常的日常活动产生负面影响。目的:研究目的是通过评估偏头痛对税收、缺勤和社会支持转移的影响,估计偏头痛给阿根廷政府带来的经济负担。方法:该分析结合了一个利用国家人口统计数据和公布的偏头痛患病率的横断面模型来估计整个偏头痛影响队列的年负担,以及一个纵向模型来评估从40岁开始20年的人均负担。一个基于代际会计的财政框架评估了偏头痛对政府财政的影响。将直接税和间接税等收入来源与公共支出要素(公共部门缺勤、医疗保健费用和财政支助)进行加权,并与一般人口进行比较。偏头痛对职业结果的影响来源于同行评审的出版物,成本来源于国家数据库。结果报告为增量财政结果(2023美元),并按每年3%的折现率计算。结果:阿根廷偏头痛的财政负担估计为每年6505英镑,而整个偏头痛人群的财政负担为1237亿英镑。每年,29%的政府成本是由于公共部门的旷工,39%与医疗保健费用有关,19%是由于放弃的直接和间接税收,12%是由于放弃的公司税。额外的政府转移支付对阿根廷偏头痛的总体财政影响贡献不大。讨论:非正规就业的高比率可能破坏疾病负担的估计。性别差异明显,妇女承担了76%的负担,突出表明需要采取针对性别的干预措施。结论:本研究揭示了偏头痛给阿根廷政府带来的重大经济负担,主要是由缺勤、医疗保健成本和放弃的税收贡献造成的。有针对性的、注重性别平等的保健和劳工政策,特别是针对非正规就业高的部门,可以帮助减少这些财政影响。
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引用次数: 0
People and Families Affected by Glycogen Storage Disease Type Ia: An Analysis of Narrative Accounts Written by Individuals Living with GSDIa and Their Caregivers. 受Ia型糖原储存病影响的人和家庭:GSDIa患者及其照护者所写的叙述分析
IF 2.3 Q2 ECONOMICS Pub Date : 2025-04-03 eCollection Date: 2025-01-01 DOI: 10.36469/001c.131811
Eliza Kruger, Hayley M de Freitas, Iris Ferrecchia, Millie Gaydon, Andrew Lloyd

Background: Glycogen storage disease type Ia (GSDIa) is a rare inherited disorder that can lead to renal and hepatic complications, brain damage, and death. There is a lack of qualitative research describing the impact of GSDIa on individuals and their caregivers. Objective: This study was designed to describe the individual experiences of those affected by GSDIa through qualitative methods. Methods: Individuals with GSDIa and caregivers were recruited through two patient associations (US, UK). Participants were asked to write narratives about their experiences with GSDIa in their own words, with no set questions or topics. The resulting narrative accounts were analyzed thematically using a primarily inductive approach. Themes describing the impact of GSDIa on individuals and caregivers were identified. Results: Eight caregivers, three of whom had lost a child to GSDIa, and 11 individuals living with GSDIa provided written summaries of their experiences of life with the condition. Participants described symptoms, the burden of managing frequent cornstarch feeds, and the broader impacts of GSDIa. These included effects on daily activities, family, emotional and social functioning, romantic relationships, and educational/professional impacts. Participants also described challenges related to obtaining diagnosis and support, and the lack of GSDIa knowledge from healthcare professionals. Discussion: This study provided novel insights into the impact of GSDIa on individuals living with the condition and their caregivers. Participants reported a wide range of issues in their narratives and openly shared sensitive information about their experiences, which may not have been captured if they had taken part in a semi-structured interview format. Conclusion: The narrative method yielded a rich data set that provided details of the varied and individual experiences of GSDIa not captured by traditional quantitative methods or structured interviews. The narratives highlighted the unmet needs of both caregivers of and patients with GSDIa around cornstarch management, obstacles to proper treatment from healthcare professionals, and the absence of effective treatments. The open-ended narrative approach had advantages over standard qualitative methods to capture greater insight into individual experiences directly from participants in their own words.

背景:Ia型糖原储存病(GSDIa)是一种罕见的遗传性疾病,可导致肾脏和肝脏并发症、脑损伤和死亡。目前缺乏描述GSDIa对个体及其照顾者影响的定性研究。目的:本研究旨在通过定性方法描述GSDIa患者的个体体验。方法:通过两个患者协会(美国、英国)招募GSDIa患者和护理人员。参与者被要求用自己的话描述他们在GSDIa的经历,没有固定的问题或主题。由此产生的叙事叙述使用主要归纳方法进行主题分析。确定了描述GSDIa对个人和照顾者影响的主题。结果:8名照顾者(其中3人因GSDIa失去了孩子)和11名患有GSDIa的人提供了他们患有这种疾病的生活经历的书面总结。与会者描述了症状、管理频繁玉米淀粉饲料的负担以及GSDIa的更广泛影响。这些影响包括对日常活动、家庭、情感和社会功能、恋爱关系以及教育/职业的影响。与会者还描述了与获得诊断和支持有关的挑战,以及卫生保健专业人员缺乏GSDIa知识。讨论:本研究为GSDIa对患者及其护理者的影响提供了新的见解。参与者在他们的叙述中报告了广泛的问题,并公开分享了关于他们经历的敏感信息,如果他们参加半结构化的采访形式,这些信息可能不会被捕捉到。结论:叙述方法产生了丰富的数据集,提供了传统定量方法或结构化访谈无法捕获的GSDIa的各种个人经历的细节。这些叙述强调了GSDIa护理人员和患者在玉米淀粉管理方面的未满足需求,卫生保健专业人员对适当治疗的障碍,以及缺乏有效治疗。开放式叙述方法比标准定性方法更有优势,可以直接从参与者自己的话中获得更深入的个人体验。
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引用次数: 0
Cost-Effectiveness Analysis of a Heart Failure Management System in the United States. 美国心力衰竭管理系统的成本-效果分析。
IF 2.3 Q2 ECONOMICS Pub Date : 2025-03-17 eCollection Date: 2025-01-01 DOI: 10.36469/001c.130066
Antonia Bosworth Smith, Ubong Silas, Alex Veloz, Peter Mallow, Barbara Pisani, Diana Bonderman, Rhodri Saunders

Background: The disease burden of heart failure is mainly driven by high hospital readmission rates. Remote monitoring devices can be used to assess the status of patients after discharge and identify early signs of worsening symptoms. Initial studies indicated that Heart Failure Management System (HFMS), a novel monitoring device, can prevent hospital readmission. Objective: To determine the cost effectiveness of HFMS compared with standard of care (SOC) in the United States. Methods: A Markov model was developed to follow patients after their discharge from index hospitalization for heart failure. The costs and outcomes were estimated for 5 years. The patient cohort was initially in "outpatient care," where they are at risk of an emergency room visit or hospital readmission. If hospitalized, patients returned to a second outpatient care health state. An "escalation of care" (eg, surgical intervention) may have removed patients from the intervention. The model took the payer perspective with costs in 2022 US dollars. The incremental cost-effectiveness ratio measured effectiveness through hospital readmissions. The willingness-to-pay threshold was set to the published cost of a heart failure rehospitalization ($10 737). Sensitivity and scenario analyses explored the robustness of the model to changes in inputs. Results: Compared with SOC, HFMS reduced the mean cost of care by 6723 ( 155 122 vs $161 846) over the 5-year period. The mean number of hospital readmissions was reduced to 1.075 with HFMS from 1.201 with SOC (-0.126 events). The incremental cost-effectiveness ratio showed that HFMS was a dominant strategy compared with SOC, leading to reduced costs and hospital readmissions in 93.4% of the 1000 Monte Carlo simulations; 94.1% of the simulations fell below the willingness-topay threshold. Savings with HFMS emerged from the third month. Discussion: The results indicated the cost-effectiveness of HFMS compared with SOC. The sensitivity analyses supported this finding. Reducing costly hospital readmissions may help to alleviate the burden of heart failure. Longer-term data on HFMS are encouraged to confirm or contest the model outcomes. Conclusions: The use of HFMS is expected to save costs and reduce hospitalizations over a 5-year period compared with the current SOC.

背景:心力衰竭的疾病负担主要是由高再入院率引起的。远程监测设备可用于评估患者出院后的状态,并识别症状恶化的早期迹象。初步研究表明,心衰管理系统(HFMS)是一种新型的监测设备,可以预防再入院。目的:比较美国HFMS与标准护理(SOC)的成本效益。方法:建立马尔可夫模型,对心力衰竭住院患者出院后进行随访。估计了5年的费用和结果。患者队列最初是在“门诊护理”,在那里他们有去急诊室或再次住院的风险。如果住院,患者返回到第二个门诊护理健康状态。“护理升级”(如手术干预)可能使患者脱离干预。该模型采用付款人视角,成本以2022年美元计算。增量成本效益比通过再入院率来衡量有效性。支付意愿门槛设定为公布的心力衰竭再住院费用(10737美元)。敏感性和情景分析探讨了模型对输入变化的鲁棒性。结果:与SOC相比,HFMS在5年期间降低了平均护理成本6723美元(155 122美元对161 846美元)。HFMS患者的平均再入院次数从SOC患者的1.201例减少到1.075例(-0.126例)。增量成本-效果比表明,与SOC相比,HFMS是一种优势策略,在1000个蒙特卡罗模拟中,93.4%的HFMS降低了成本和住院再入院率;94.1%的模拟低于愿意支付的阈值。从第三个月开始,HFMS出现了节省。讨论:结果表明HFMS与SOC的成本-效果比较。敏感性分析支持这一发现。减少昂贵的再入院可能有助于减轻心力衰竭的负担。鼓励HFMS的长期数据来证实或质疑模型的结果。结论:与目前的SOC相比,使用HFMS有望在5年内节省成本并减少住院次数。
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引用次数: 0
Costs of Adverse Event Management Associated with First-Line Cetuximab or Panitumumab in Metastatic Colorectal Cancer Patients in Saudi Arabia. 沙特阿拉伯转移性结直肠癌患者一线西妥昔单抗或帕尼单抗相关的不良事件管理成本
IF 2.3 Q2 ECONOMICS Pub Date : 2025-03-07 eCollection Date: 2025-01-01 DOI: 10.36469/001c.130878
Gihan Elsisi, Hana Abdul Kareem, Abdelaziz Alaseiri, Abdullah Alsharm, Mohamed Al Garni, Hajer Al-Mudaiheem, Fouad Alnagar, Hazem Lotfy, Mohamed Ouda, Ahmed Elshehri

Introduction: In Saudi Arabia, patients with metastatic colorectal cancer (mCRC) with wild-type RAS mutations may be treated with either cetuximab plus chemotherapy (CET + CT) or panitumumab plus chemotherapy (PAN + CT), which are epidermal growth factor receptor (EGFR) antibodies. This study calculated the costs of adverse event (AE) management linked to anti-EGFR treatment in Saudi Arabia's national health budget from payer and societal perspectives. Methods: An adaptation of a global model developed in Microsoft Excel® was performed to estimate the costs of AE management associated with the first-line treatment (CET + CT and PAN + CT) of RAS wild-type mCRC patients in Saudi healthcare settings. The frequencies of common and highly common AEs were sourced from the summaries of product characteristics of CET and PAN, whereas AE severity was captured from a meta-analysis. Unit costs in Saudi Riyal (SAR) were obtained from the National Guard of Health Affairs and Ministry of Health 2024 price lists. The model assumptions, inputs, and results were validated using a local Delphi panel. Results: Within the Saudi payer perspective model, treatment with CET + CT vs PAN + CT resulted in average population cost savings of SAR 9 246 133 (4 741 606 international dollars [Intl ] ) a n d p e r - p a t i e n t c o s t s a v i n g s o f S A R 20 321 ( I n t l 10 421) for severe AEs, and average population cost savings of SAR 16 039 427 (Intl 8 225 347 ) a n d p e r - p a t i e n t c o s t s a v i n g s o f S A R 35 251 ( I n t l 18 077) for all-grade AEs. On the other hand, within the Saudi societal perspective model, treatment with CET + CT vs PAN + CT resulted in average population cost savings of SAR 11 386 314 (Intl 5 839 135 ) a n d p e r - p a t i e n t c o s t s a v i n g s o f
简介:在沙特阿拉伯,RAS野生型突变的转移性结直肠癌(mCRC)患者可以使用西妥昔单抗联合化疗(CET + CT)或帕尼单抗联合化疗(PAN + CT)进行治疗,这两种药物都是表皮生长因子受体(EGFR)抗体。本研究从支付方和社会角度计算了沙特阿拉伯国家卫生预算中与抗表皮生长因子受体治疗相关的不良事件(AE)管理成本。方法:对 Microsoft Excel® 中开发的全球模型进行了调整,以估算沙特医疗环境中 RAS 野生型 mCRC 患者一线治疗(CET + CT 和 PAN + CT)的相关 AE 管理成本。常见和高发 AE 的频率来自 CET 和 PAN 的产品特征概要,而 AE 的严重程度则来自一项荟萃分析。以沙特里亚尔(SAR)为单位的单位成本来自国家卫生事务卫队和卫生部 2024 年价目表。模型假设、输入和结果由当地德尔菲小组进行验证。结果:在沙特支付方视角模型中,使用 CET + CT 与 PAN + CT 相比,治疗严重 AE 的平均人群成本可节省 9 246 133 沙特里亚尔(4 741 606 美元 [Intl ]),而治疗严重 AE 的平均人群成本可节省 S A R 20 321(I n t l 10 421)、对于所有等级的急性呼吸道感染,平均每人可节省 16 039 427 里亚尔(8 225 347 英镑)的费用,平均每人可节省 35 251 里亚尔(18 077 英镑)的费用。另一方面,在沙特社会视角模型中,使用 CET + CT 与 PAN + CT 相比,治疗严重 AE 平均可节省 11 386 314 沙特里亚尔(5 839 135 英镑)的人口成本,而治疗严重 AE 平均可节省 S A R 25 025(12 833 英镑)的人口成本、同时,所有等级的急性呼吸道感染平均可节约 18 179 608 里亚尔(9 322 875 英镑)的人口成本和 39 955 里亚尔(20 489 英镑)的医疗费用。结论:在治疗未经治疗的 RAS 野生型 mCRC 患者时,CET + CT 方案的 AE 发生率低于 PAN + CT 方案,因此从沙特支付方和社会角度来看,都能节省 AE 管理成本。这些可观的成本节约可减轻沙特医疗机构对 mCRC 的经济负担。
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引用次数: 0
Cost-Effectiveness and Budget Impact Analysis of the Use of Faricimab in Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration in Colombia. 法利昔单抗治疗哥伦比亚糖尿病性黄斑水肿和新生血管性年龄相关性黄斑变性的成本-效果和预算影响分析
IF 2.3 Q2 ECONOMICS Pub Date : 2025-03-04 eCollection Date: 2025-01-01 DOI: 10.36469/001c.129832
Daniel Samacá-Samacá, Antonio Robles, Hugo Ocampo, Francisco J Rodríguez, Carolina Sardi-Correa, Laura Prieto-Pinto, Christian Bührer, Camilo Tamayo, David Rodríguez, Mauricio Hernández-Quintana
<p><p><b>Background:</b> Retinal diseases are major contributors to disability, significantly affecting patients' quality of life. Diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) represent a significant disease and economic burden in Colombia. Assessing the economic evaluation of available treatments is essential for the efficient allocation of healthcare resources. <b>Objective:</b> To estimate the cost-effectiveness and budget impact of using faricimab for the treatment of patients with DME and nAMD within the Colombian health system. <b>Methods:</b> This study used a 25-year Markov cohort model to estimate the cost-effectiveness of faricimab vs aflibercept, ranibizumab, and brolucizumab. We used the methodological guidelines of the local health technology assessment agency for conducting the cost-effectiveness and budget impact analysis. Transition probabilities and injection frequencies were derived from the literature. Drug prices were retrieved from public local databases. Quality-adjusted life years (QALYs) were assessed. The potential patient population for the budget impact analysis was estimated based on disease prevalence and expert consultations. <b>Results:</b> Faricimab treat-and-extend (T&E) was dominant vs aflibercept T&E (+0.22 QALYs), ranibizumab T&E (+0.55 QALYs), and brolucizumab for 8 to 12 weeks (+0.06 QALYs) in DME, generating cost savings (in US dollars) of <math><mn>3849</mn> <mo>,</mo></math> 1375, and <math><mn>2824</mn> <mo>,</mo> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>p</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>e</mi> <mi>l</mi> <mi>y</mi> <mo>.</mo> <mi>I</mi> <mi>n</mi> <mi>n</mi> <mi>A</mi> <mi>M</mi> <mi>D</mi> <mo>,</mo> <mi>f</mi> <mi>a</mi> <mi>r</mi> <mi>i</mi> <mi>c</mi> <mi>i</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mi>a</mi> <mi>l</mi> <mi>s</mi> <mi>o</mi> <mi>s</mi> <mi>h</mi> <mi>o</mi> <mi>w</mi> <mi>e</mi> <mi>d</mi> <mi>d</mi> <mi>o</mi> <mi>m</mi> <mi>i</mi> <mi>n</mi> <mi>a</mi> <mi>n</mi> <mi>c</mi> <mi>e</mi> <mi>v</mi> <mi>s</mi> <mi>a</mi> <mi>f</mi> <mi>l</mi> <mi>i</mi> <mi>b</mi> <mi>e</mi> <mi>r</mi> <mi>c</mi> <mi>e</mi> <mi>p</mi> <mi>t</mi> <mi>a</mi> <mi>s</mi> <mi>n</mi> <mi>e</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>d</mi> <mo>(</mo> <mo>+</mo> <mn>0.12</mn> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>s</mi> <mo>)</mo> <mo>,</mo> <mi>r</mi> <mi>a</mi> <mi>n</mi> <mi>i</mi> <mi>b</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mi>a</mi> <mi>s</mi> <mi>n</mi> <mi>e</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>d</mi> <mo>(</mo> <mo>+</mo> <mn>0.05</mn> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>s</mi> <mo>)</mo> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>b</mi> <mi>r</mi> <mi>o</mi> <mi>l</mi> <mi>u</mi> <mi>c</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mn>8</mn> <mi>t</mi> <mi>o</mi> <mn>12</mn> <mi>w</mi> <mi>e</mi> <mi>e</mi> <mi>k</mi> <mi>s</mi> <mo>(</mo> <mo>+</mo>
背景:视网膜疾病是导致残疾的主要原因,严重影响患者的生活质量。糖尿病性黄斑水肿(DME)和新生血管性年龄相关性黄斑变性(nAMD)是哥伦比亚的一个重要疾病和经济负担。评估现有治疗方法的经济评价对于有效分配医疗资源至关重要。目的:评估在哥伦比亚卫生系统内使用法利昔单抗治疗二甲醚和nAMD患者的成本效益和预算影响。方法:本研究采用25年Markov队列模型来评估faricimab与阿非利西贝、雷尼单抗和brolucizumab的成本-效果。我们使用当地卫生技术评估机构的方法指导方针进行成本效益和预算影响分析。过渡概率和注入频率由文献推导。药品价格从当地公共数据库检索。评估质量调整生命年(QALYs)。预算影响分析的潜在患者群体是根据患病率和专家咨询估计的。结果:Faricimab治疗延长(T&E)在DME治疗中占主导地位,而afliberept T&E (+0.22 QALYs)、ranibizumab T&E (+0.55 QALYs)和brolucizumab治疗8 - 12周(+0.06 QALYs),节省成本(以美元计)分别为3849、1375和2824。我n n M D, f r我c I M b l s o s h o w e D D o M I n n c e v s f l I b e r c e p t s n e e D e D (+ 0.12 Q l Y s), r n我b z u M b s n e e D e D (+ 0.05 Q l Y s), n D b r o l u c我z M b 8 t o 12 w e e k s (+ 0.12 Q l Y s) w我t h s v我n g s n(美国)7223,5792,6798 n D,分别。假设二甲醚的市场份额每年增长15%,nAMD的市场份额每年增长13%,那么哥伦比亚卫生系统可以在2011年节省1.44亿美元,而在2010年,哥伦比亚卫生系统可以节省3亿美元。其中,1.227亿美元归因于药品费用,2130万美元归因于医疗费用,其中1亿美元归因于医疗费用,1亿美元归因于医疗费用,1亿美元归因于医疗费用,1亿美元归因于医疗费用。结论:考虑到每个额外的QALY愿意支付5988美元的门槛,faricimab是哥伦比亚医疗保健系统中DME和nAMD的成本效益替代方案,显示出其他抗血管内皮生长因子药物的优势。与其他治疗方法相比,Faricimab以更低的成本提供了更好的健康结果。
{"title":"Cost-Effectiveness and Budget Impact Analysis of the Use of Faricimab in Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration in Colombia.","authors":"Daniel Samacá-Samacá, Antonio Robles, Hugo Ocampo, Francisco J Rodríguez, Carolina Sardi-Correa, Laura Prieto-Pinto, Christian Bührer, Camilo Tamayo, David Rodríguez, Mauricio Hernández-Quintana","doi":"10.36469/001c.129832","DOIUrl":"10.36469/001c.129832","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Background:&lt;/b&gt; Retinal diseases are major contributors to disability, significantly affecting patients' quality of life. Diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) represent a significant disease and economic burden in Colombia. Assessing the economic evaluation of available treatments is essential for the efficient allocation of healthcare resources. &lt;b&gt;Objective:&lt;/b&gt; To estimate the cost-effectiveness and budget impact of using faricimab for the treatment of patients with DME and nAMD within the Colombian health system. &lt;b&gt;Methods:&lt;/b&gt; This study used a 25-year Markov cohort model to estimate the cost-effectiveness of faricimab vs aflibercept, ranibizumab, and brolucizumab. We used the methodological guidelines of the local health technology assessment agency for conducting the cost-effectiveness and budget impact analysis. Transition probabilities and injection frequencies were derived from the literature. Drug prices were retrieved from public local databases. Quality-adjusted life years (QALYs) were assessed. The potential patient population for the budget impact analysis was estimated based on disease prevalence and expert consultations. &lt;b&gt;Results:&lt;/b&gt; Faricimab treat-and-extend (T&E) was dominant vs aflibercept T&E (+0.22 QALYs), ranibizumab T&E (+0.55 QALYs), and brolucizumab for 8 to 12 weeks (+0.06 QALYs) in DME, generating cost savings (in US dollars) of &lt;math&gt;&lt;mn&gt;3849&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt;&lt;/math&gt; 1375, and &lt;math&gt;&lt;mn&gt;2824&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mo&gt;.&lt;/mo&gt; &lt;mi&gt;I&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;M&lt;/mi&gt; &lt;mi&gt;D&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mo&gt;+&lt;/mo&gt; &lt;mn&gt;0.12&lt;/mn&gt; &lt;mi&gt;Q&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;Y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;z&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mo&gt;+&lt;/mo&gt; &lt;mn&gt;0.05&lt;/mn&gt; &lt;mi&gt;Q&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;Y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;z&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mn&gt;8&lt;/mn&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mn&gt;12&lt;/mn&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;k&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mo&gt;+&lt;/mo&gt;","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"97-105"},"PeriodicalIF":2.3,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11884430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Effect of Dobbs v. Jackson Women's Health Organization on Clinical Diagnosis of Postpartum Depression. Dobbs诉Jackson妇女健康组织对产后抑郁症临床诊断的影响。
IF 2.3 Q2 ECONOMICS Pub Date : 2025-02-26 eCollection Date: 2025-01-01 DOI: 10.36469/001c.129633
Onur Baser, Yuanqing Lu, Facundo Sepulveda, Ariani Alemzadeh, Amy Endrizal

Background: The 2022 US Supreme Court decision in Dobbs v. Jackson Women's Health Organization eliminated the constitutional right to abortion and activated trigger laws in 21 states, either banning or significantly restricting abortion access. This study estimated changes in postpartum depression (PPD) diagnoses after Dobbs in states with trigger laws vs those without. Methods: Medicaid data from Kythera Labs spanning December 2019 to June 2024 were utilized. Difference-in-difference models assessed changes in PPD diagnosis rates post-Dobbs (21 trigger states, 29 non-trigger states). Results: Women in trigger states were younger (mean, 26.53 vs 27.98 years), more likely to reside in low socioeconomic status areas (41.28% vs 24.42%) and less likely to have obstetrical complications (66.06% vs 77.36%), maternal complications (16.41% vs 18.9%), and lifestyle risk factors (13.58% vs 21.17%). Baseline PPD diagnosis rates were 8.51% in trigger states and 12.66% in non-trigger states. Post-Dobbs, PPD diagnosis rates were 10.20% in trigger states and 14.34% in non-trigger states. Conclusions: Overall, women in states with abortion trigger laws experienced a small positive but statistically insignificant increase in PPD diagnoses following Dobbs compared with those in non-trigger states.

背景:2022 年美国最高法院在多布斯诉杰克逊妇女健康组织案中的判决取消了宪法规定的堕胎权,并在 21 个州启动了触发法,禁止或大幅限制堕胎。本研究估计了在多布斯案之后,有触发法的州与没有触发法的州产后抑郁症(PPD)诊断率的变化。研究方法采用 Kythera 实验室提供的医疗补助数据,时间跨度为 2019 年 12 月至 2024 年 6 月。差异模型评估了多布斯事件后 PPD 诊断率的变化(21 个触发州,29 个非触发州)。结果:触发州的妇女更年轻(平均 26.53 岁 vs 27.98 岁),更有可能居住在社会经济地位较低的地区(41.28% vs 24.42%),不太可能出现产科并发症(66.06% vs 77.36%)、产妇并发症(16.41% vs 18.9%)和生活方式风险因素(13.58% vs 21.17%)。基线 PPD 诊断率在触发状态下为 8.51%,在非触发状态下为 12.66%。多布斯后,触发状态下的 PPD 诊断率为 10.20%,非触发状态下为 14.34%。结论:总体而言,与非触发州的妇女相比,触发堕胎法的州的妇女在多布斯事件后的 PPD 诊断率略有增加,但在统计学上并不显著。
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引用次数: 0
Cost-effectiveness Analysis of Nirmatrelvir/Ritonavir Compared with Molnupiravir in Patients at High Risk for Progression to Severe COVID-19 in Japan. 日本尼马特利韦/利托那韦与莫努匹拉韦在进展为严重COVID-19高风险患者中的成本-效果分析
IF 2.3 Q2 ECONOMICS Pub Date : 2025-02-24 eCollection Date: 2025-01-01 DOI: 10.36469/001c.129067
Akira Yuasa, Hiroyuki Matsuda, Yosuke Fujii, Tendai Mugwagwa, Yuki Kado, Mizuki Yoshida, Koichi Murata, Yoshiaki Gu

Background: Nirmatrelvir/ritonavir (NMV/r) and molnupiravir are oral antiviral drugs approved for the treatment of early symptomatic patients with mild to moderate COVID-19 at high risk of progression to severe disease in Japan. Objective: This study evaluated, from a Japanese payer perspective, the cost-effectiveness of NMV/r compared with molnupiravir among patients with COVID-19. Methods: This cost-effectiveness model describes the COVID-19 disease history and the impact of antiviral treatment on short-term and long-term outcomes. Nirmatrelvir/ritonavir was compared with molnupiravir, and in the scenario analysis NMV/r was compared with standard of care over a lifetime horizon. Results: When compared with molnupiravir, NMV/r showed higher quality-adjusted life years (QALYs) (15.752 vs 15.739) and higher total cost (¥6 248 014 vs ¥6 245 829 [US 44 136.86 v s 44 121.42]). The incremental cost-effectiveness ratio was ¥164 934 (US 1165.12 ) p e r Q A L Y g a i n e d , w h i c h w a s l o w e r t h a n t h e w i l l i n g n e s s - t o - p a y ( W T P ) t h r e s h o l d i n J a p a n ( ¥ 5 000 000 / Q A L Y [ U S 35 320.71/QALY]). In the scenario analysis, the incremental cost-effectiveness ratio was ¥3 646 821 (US 25 761.66 ) p e r Q A L Y g a i n e d . O n e - w a y s e n s i t i v i t y a n a l y s i s a n d p r o b a b i l i s t i c s e n s i t i v i
背景:Nirmatrelvir/ritonavir (NMV/r)和molnupiravir是日本批准用于治疗轻至中度早期症状且进展为严重疾病高风险的COVID-19患者的口服抗病毒药物。目的:本研究从日本支付者的角度,评价NMV/r与莫诺匹拉韦在COVID-19患者中的成本-效果。方法:该成本-效果模型描述了COVID-19疾病史以及抗病毒治疗对短期和长期预后的影响。将Nirmatrelvir/ritonavir与molnupiravir进行比较,并在情景分析中将NMV/r与生命周期内的标准护理进行比较。结果:与莫诺匹拉韦相比,NMV/r具有更高的质量调整生命年(QALYs) (15.752 vs 15.739)和更高的总成本(6 248 014 vs 6 245 829 [US 44 136.86 vs s 44 121.42])。增量成本效益比率¥164 934(合1165.12)p e r L Y g i n e d w h i w c h s L o w e r t h n t h e w我L L n g n e s s - t o - p Y (w t p) t h r e s h o L d i n J p n(¥5 000 000 / Q L Y [U s 35 320.71 / QALY])。在情景分析中,增量成本-效果比为3 646 821日元(25 761.66美元)/ Q / A / L / d / d。O n e - w y s e n s我t v t y一个l n y s i s n d p r O b b我l s t c s e n s我t v t y一个l n y s i s s h O w e t h t n M v d s c O s t / r w - e f f e c t i v e c O M p r e d w i t h M O l n u p ir i r n d s t n d r d O f c r e c O n s i s t e n t l y。l l r e s u l t s o f o n e - w y s e n s我t v t y一个l n y s i s s h o w e d t h t t h e n c r e m e n t l c o s e t - f f e c t i v e n e s s r t i o s w e r e b e l o w t h e w t P t h r e s h o P l d i n J n。T h e r e s u l T o f T h e c o s e T - f f e c T i v e n e s s c c e p T b我l T y c u r e s h o w e d T h T T h e p r o b b我l T y T h T T h e n c r e m e n T l c o s e T - f f e c T i v e n e s s r T i o w s b e l o w T h e p w T o f¥5 000 000 / Q l y (u s 35 320.71 / QALY)为100.00%。结论:从日本支付者的角度来看,与莫努匹拉韦和标准护理相比,尼马特利韦/利托那韦具有成本效益。本研究为NMV/r治疗COVID-19患者的成本效益提供了证据。
{"title":"Cost-effectiveness Analysis of Nirmatrelvir/Ritonavir Compared with Molnupiravir in Patients at High Risk for Progression to Severe COVID-19 in Japan.","authors":"Akira Yuasa, Hiroyuki Matsuda, Yosuke Fujii, Tendai Mugwagwa, Yuki Kado, Mizuki Yoshida, Koichi Murata, Yoshiaki Gu","doi":"10.36469/001c.129067","DOIUrl":"10.36469/001c.129067","url":null,"abstract":"<p><p><b>Background:</b> Nirmatrelvir/ritonavir (NMV/r) and molnupiravir are oral antiviral drugs approved for the treatment of early symptomatic patients with mild to moderate COVID-19 at high risk of progression to severe disease in Japan. <b>Objective:</b> This study evaluated, from a Japanese payer perspective, the cost-effectiveness of NMV/r compared with molnupiravir among patients with COVID-19. <b>Methods:</b> This cost-effectiveness model describes the COVID-19 disease history and the impact of antiviral treatment on short-term and long-term outcomes. Nirmatrelvir/ritonavir was compared with molnupiravir, and in the scenario analysis NMV/r was compared with standard of care over a lifetime horizon. <b>Results:</b> When compared with molnupiravir, NMV/r showed higher quality-adjusted life years (QALYs) (15.752 vs 15.739) and higher total cost (¥6 248 014 vs ¥6 245 829 [US <math><mn>44</mn> <mrow><mo> </mo></mrow> <mn>136.86</mn> <mi>v</mi> <mi>s</mi></math> 44 121.42]). The incremental cost-effectiveness ratio was ¥164 934 (US <math><mn>1165.12</mn> <mo>)</mo> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>g</mi> <mi>a</mi> <mi>i</mi> <mi>n</mi> <mi>e</mi> <mi>d</mi> <mo>,</mo> <mi>w</mi> <mi>h</mi> <mi>i</mi> <mi>c</mi> <mi>h</mi> <mi>w</mi> <mi>a</mi> <mi>s</mi> <mi>l</mi> <mi>o</mi> <mi>w</mi> <mi>e</mi> <mi>r</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>n</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>w</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>n</mi> <mi>e</mi> <mi>s</mi> <mi>s</mi> <mo>-</mo> <mi>t</mi> <mi>o</mi> <mo>-</mo> <mi>p</mi> <mi>a</mi> <mi>y</mi> <mo>(</mo> <mi>W</mi> <mi>T</mi> <mi>P</mi> <mo>)</mo> <mi>t</mi> <mi>h</mi> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>h</mi> <mi>o</mi> <mi>l</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>J</mi> <mi>a</mi> <mi>p</mi> <mi>a</mi> <mi>n</mi> <mo>(</mo> <mrow><mo>¥</mo></mrow> <mn>5</mn> <mrow><mo> </mo></mrow> <mn>000</mn> <mrow><mo> </mo></mrow> <mn>000</mn> <mrow><mo>/</mo></mrow> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mo>[</mo> <mi>U</mi> <mi>S</mi></math> 35 320.71/QALY]). In the scenario analysis, the incremental cost-effectiveness ratio was ¥3 646 821 (US <math><mn>25</mn> <mrow><mo> </mo></mrow> <mn>761.66</mn> <mo>)</mo> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>Q</mi> <mi>A</mi> <mi>L</mi> <mi>Y</mi> <mi>g</mi> <mi>a</mi> <mi>i</mi> <mi>n</mi> <mi>e</mi> <mi>d</mi> <mo>.</mo> <mi>O</mi> <mi>n</mi> <mi>e</mi> <mo>-</mo> <mi>w</mi> <mi>a</mi> <mi>y</mi> <mi>s</mi> <mi>e</mi> <mi>n</mi> <mi>s</mi> <mi>i</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>i</mi> <mi>t</mi> <mi>y</mi> <mi>a</mi> <mi>n</mi> <mi>a</mi> <mi>l</mi> <mi>y</mi> <mi>s</mi> <mi>i</mi> <mi>s</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>p</mi> <mi>r</mi> <mi>o</mi> <mi>b</mi> <mi>a</mi> <mi>b</mi> <mi>i</mi> <mi>l</mi> <mi>i</mi> <mi>s</mi> <mi>t</mi> <mi>i</mi> <mi>c</mi> <mi>s</mi> <mi>e</mi> <mi>n</mi> <mi>s</mi> <mi>i</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>i</","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"75-85"},"PeriodicalIF":2.3,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864595/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143516031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clinical and Economic Outcomes in Patients With Alpha-1 Antitrypsin Deficiency in a US Medicare Advantage Population. 美国医疗保险优势人群中α -1抗胰蛋白酶缺乏症患者的临床和经济结果
IF 2.3 Q2 ECONOMICS Pub Date : 2025-02-20 eCollection Date: 2025-01-01 DOI: 10.36469/001c.127446
Nikhil Khandelwal, Jimmy Hinson, Trinh Nguyen, Alexjandro Daviano, Yihua Xu, Brandon T Suehs, Sally Higgins, Marie Sanchirico, J Michael Wells

Background: Alpha-1 antitrypsin deficiency (AATD) testing rates and associated clinical and economic outcomes data in the US Medicare population are limited. Objective: To characterize individuals with AATD, describe clinical outcomes/healthcare research utilization (HCRU) among individuals with chronic obstructive pulmonary disease (COPD) with or without AATD, and identify AATD testing rates among individuals newly diagnosed with COPD. Methods: This retrospective, observational analysis of claims data included individuals from the Humana Research Database (aged 18-89 years) enrolled in Medicare Advantage Prescription Drug plans. Three cohorts included individuals with evidence of AATD; individuals with COPD + AATD matched to individuals with COPD; and individuals with newly diagnosed COPD. AATD health-related outcomes, such as pulmonary and extrapulmonary conditions or events, and economic outcomes, including inpatient admissions, emergency department visits, and physician visits, were examined independently during the pre-index and post-index periods and compared between those with ATTD and without AATD. Results: We identified 1103 individuals with AATD (aged 67.2 ± 10.0 years, 56.3% women, 94.5% White); overall, 22.2% had exacerbations, respiratory distress, and respiratory failure. Individuals with COPD and AATD (n = 742) were matched to individuals with COPD (n = 7420), based on age (68 ± 9 years), sex (55.0% women), and race (97.2% White). The AATD group had a higher proportion of emphysema (47.4% vs 18.7%), COPD exacerbations (40.6% vs 24.7%), and cirrhosis (4.0% vs 1.3%) than the non-AATD group. All-cause inpatient admissions (31.7% vs 27.3%), COPD-specific inpatient admissions (7.4% vs 4.3%), and COPD-specific emergency department visits (19.5% vs 10.8%) were higher in individuals who had ATTD than in those without AATD. AATD testing rates among individuals with newly diagnosed COPD increased slightly over time (2015: 1.07%; 2020: 1.49%). Individuals with COPD and AATD had more comorbidities and higher HCRU. Testing rates increased slightly but remained low. Discussion: Further research is needed to assess the impact of improved AATD testing on those with COPD. Conclusion: Increased awareness, earlier testing, and treatment may reduce the healthcare burden of AATD in the US Medicare population.

背景:美国医疗保险人群中α -1抗胰蛋白酶缺乏症(AATD)检测率和相关临床和经济结果数据有限。目的:描述AATD患者的特征,描述伴有或不伴有AATD的慢性阻塞性肺疾病(COPD)患者的临床结果/医疗保健研究利用(HCRU),并确定新诊断为COPD患者的AATD检测率。方法:回顾性观察性分析来自Humana研究数据库(年龄在18-89岁)参加医疗保险优势处方药计划的索赔数据。三个队列包括有AATD证据的个体;COPD + AATD患者与COPD患者匹配;以及新诊断的慢性阻塞性肺病患者。AATD与健康相关的结果,如肺和肺外状况或事件,以及经济结果,包括住院、急诊科就诊和医生就诊,在指数前和指数后独立检查,并比较ATTD患者和非AATD患者。结果:我们发现1103例AATD患者(年龄67.2±10.0岁,女性56.3%,白人94.5%);总体而言,22.2%的患者有急性发作、呼吸窘迫和呼吸衰竭。COPD和AATD患者(n = 742)与COPD患者(n = 7420)匹配,基于年龄(68±9岁)、性别(55.0%为女性)和种族(97.2%为白人)。与非AATD组相比,AATD组肺气肿(47.4% vs 18.7%)、COPD加重(40.6% vs 24.7%)和肝硬化(4.0% vs 1.3%)的比例更高。ATTD患者的全因住院率(31.7% vs 27.3%)、copd特异性住院率(7.4% vs 4.3%)和copd特异性急诊科就诊率(19.5% vs 10.8%)高于无AATD患者。新诊断的COPD患者的AATD检测率随着时间的推移略有增加(2015年:1.07%;2020年:1.49%)。COPD和AATD患者有更多的合并症和更高的HCRU。检测率略有上升,但仍然很低。讨论:需要进一步的研究来评估改进的AATD检测对COPD患者的影响。结论:提高对AATD的认识,早期检测和治疗可以减轻美国医疗保险人群中AATD的医疗负担。
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引用次数: 0
A Vignette Study to Derive Health-Related Quality-of-Life Weights for Individuals with Steroid Refractory Chronic Graft-versus-Host Disease Receiving Third-Line Therapy in the United Kingdom. 在英国,一项获得接受三线治疗的类固醇难治性慢性移植物抗宿主病患者健康相关生活质量权重的小研究
IF 2.3 Q2 ECONOMICS Pub Date : 2025-02-19 eCollection Date: 2025-01-01 DOI: 10.36469/001c.125546
Emma Williams, Luke Skinner, Richard Hudson, Arunesh Sil, Katharina Ecsy, Elisheva Lew, Abdul Jabbar Omar Alsaleh, Elin Gruffydd, Andrew Lloyd, Daniele Avenoso

Background: Chronic graft-versus-host disease (cGvHD) - a potentially debilitating complication of allogeneic hematopoietic stem cell transplantation - is a rare condition. Objectives: This vignette-based study aimed to generate utility values to inform an economic model via an online survey wherein cGvHD health state (HS) vignettes were valued by the general UK population using the EQ-5D-5L and the EQ-5D-visual analog scale (EQ-5D VAS). Methods: This non-interventional health-related quality of life (HRQoL) study was conducted in 3 stages across the UK: the development, validation, and valuation of HS vignettes to generate utility values for cGvHD. Four HS for cGvHD were defined based on an economic model partitioning different treatment level responses in patients with cGvHD receiving third-line (3L) therapy (HS1: complete response, HS2: partial response, HS3: lack of response, and HS4: recurrent cGvHD). Draft vignettes were developed for each HS based on 4 previously published GvHD vignettes. The contents of the draft vignettes were reviewed for all aspects of cGvHD symptoms and functional impact and validated through semistructured interviews with 5 clinical experts. The 4 finalized HS vignettes were valued by 300 participants from the UK general population using EQ-5D-5L and EQ-5D VAS. Results: Previously published vignettes were used to develop the vignettes for the current study that described GvHD in the context of blood cancer and other rare blood disorders (n = 2 each) and included symptoms, functioning, and quality of life for a patient in the HS. The highest and lowest mean EQ-5D-5L utility scores were observed for HS1 (mean [95% CI]: 0.577 [0.558-0.595]) and HS4 (0.061 [0.034-0.088]), respectively. The EQ-5D-VAS showed the highest and lowest mean utility scores for HS1 (46.8 [44.9-48.6]) and HS4 (25.6 [23.4-27.7]), respectively. Conclusion: This study generated utility values for HS vignettes describing symptoms, functioning, and HRQoL for patients with cGvHD receiving 3L therapy. The utility values highlighted a substantial burden of cGvHD and HRQoL impact associated with the treatment response level. However, assessing concordance between utility estimates derived from the vignette-based method in a general population and those from patients with cGvHD is further warranted.

背景:慢性移植物抗宿主病(cGvHD)是一种罕见的疾病,是同种异体造血干细胞移植的一种潜在的衰弱并发症。目的:本研究旨在通过在线调查产生实用价值,为经济模型提供信息,其中英国普通人群使用EQ-5D- 5l和EQ-5D视觉模拟量表(EQ-5D VAS)对cGvHD健康状态(HS)小量表进行评估。方法:这项非介入性健康相关生活质量(HRQoL)研究在英国分三个阶段进行:HS量表的开发、验证和评估,以产生cGvHD的实用价值。根据经济模型划分接受三线(3L)治疗的cGvHD患者的不同治疗水平反应,定义了cGvHD的四种HS (HS1:完全缓解,HS2:部分缓解,HS3:缺乏缓解,HS4:复发性cGvHD)。基于先前发布的4个GvHD小片段,为每个HS开发了草稿小片段。从cGvHD症状和功能影响的各个方面对草稿内容进行了审查,并通过与5位临床专家的半结构化访谈进行了验证。来自英国普通人群的300名参与者使用EQ-5D- 5l和EQ-5D VAS对最终确定的4个HS小片段进行评估。结果:先前发表的小片段被用于开发当前研究的小片段,该研究描述了血癌和其他罕见血液疾病背景下的GvHD(每种n = 2),包括HS患者的症状、功能和生活质量。HS1(平均[95% CI]: 0.577[0.558-0.595])和HS4(0.061[0.034-0.088])的平均EQ-5D-5L效用得分最高和最低。EQ-5D-VAS分别显示HS1(46.8[44.9-48.6])和HS4(25.6[23.4-27.7])的平均效用得分最高和最低。结论:本研究为描述接受3L治疗的cGvHD患者的症状、功能和HRQoL的HS小片段提供了实用价值。效用值强调了与治疗反应水平相关的cGvHD和HRQoL影响的实质性负担。然而,评估在普通人群和cGvHD患者中基于小插曲的方法得出的效用估计之间的一致性是进一步必要的。
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引用次数: 0
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Journal of Health Economics and Outcomes Research
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