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The Hidden Economic Consequences of Migraine to the UK Government: Burden-of-Disease Analysis Using a Fiscal Framework 偏头痛对英国政府的潜在经济后果:使用财政框架的疾病负担分析
Q3 Medicine Pub Date : 2023-10-03 DOI: 10.36469/jheor.2023.87790
Rui Martins, Samuel Large, Rachel Russell, Gary Surmay, Mark Connolly
Background: Migraine is a highly prevalent and incapacitating neurological disorder associated with the highest global disability burden in people aged 15 to 49 years. Europe has the fourth-highest prevalence of migraine, after North America, South America, and Central America, and above Asia and Africa. Migraine leads to relatively modest direct healthcare expenditure but has substantial indirect costs due to reduced productivity. Methods: The economic burden of migraine was estimated in comparison with the general population of the United Kingdom (UK) using an analytical fiscal modeling framework applying the government cost perspective. Published measures of migraine’s impact on labor participation were applied to rates of economic activity/inactivity of the general population. The model estimates lifetime changes to earnings from employment, direct and indirect taxes paid, and financial support requirements over the life course. Incremental differences between those affected and unaffected by migraine are reported as net fiscal consequences to public accounts. Fiscal costs are reported as the discounted average per capita over a 20-year time horizon and for the entire annual UK cohort with prevalent migraine. Results: People affected by migraine are more likely to be absent from work, unemployed, and disabled, and to retire early. A 44-year-old individual affected by migraine was associated with £19 823 in excess fiscal costs to the UK government, £1379 per year living with the condition, compared with someone not affected by the disease. Annually, migraine was estimated to represent £12.20 billion to the public economy, approximately £130.63 per migraine episode. The model predicted annual productivity losses in the health and social care workforce to be £2.05 billion and total annual productivity losses to be over £5.81 billion. Conclusions: This fiscal analysis monetizes the occupational consequences of migraine to the UK government, both in terms of lost tax revenue and transfer payments. The findings are substantial and useful to characterize disease severity and to inform the body of evidence considered by decision makers appraising the cost-effectiveness of health technologies.
背景:偏头痛是一种高度流行和致残的神经系统疾病,与全球15至49岁人群中最高的残疾负担相关。欧洲是偏头痛发病率第四高的地区,仅次于北美、南美和中美洲,高于亚洲和非洲。偏头痛导致相对适度的直接医疗保健支出,但由于生产力降低而产生大量间接成本。方法:采用政府成本视角的分析财政模型框架,与英国普通人群进行比较,估计偏头痛的经济负担。公布的偏头痛对劳动参与率影响的测量方法应用于一般人群的经济活动/不活动比率。该模型估计了一生中就业收入的变化、所缴纳的直接和间接税以及一生中所需的财政支持。受偏头痛影响和未受偏头痛影响的人之间的增量差异被报告为公共账户的净财政后果。财政成本报告为20年时间范围内的人均折现平均值和整个英国年度偏头痛流行队列。结果:受偏头痛影响的人更有可能缺勤、失业、残疾,并提前退休。一名44岁的偏头痛患者与未患偏头痛的人相比,英国政府的额外财政支出为19823英镑,患有偏头痛的人每年的财政支出为1379英镑。据估计,偏头痛每年给公共经济带来122亿英镑的损失,每次偏头痛发作约为130.63英镑。该模型预测,卫生和社会保健劳动力的年度生产力损失将达到20.5亿英镑,年度生产力损失总额将超过58.1亿英镑。结论:这一财政分析将偏头痛对英国政府的职业后果货币化,包括税收损失和转移支付。这些发现是实质性的和有用的,可以描述疾病的严重程度,并为决策者评估卫生技术的成本效益所考虑的证据提供信息。
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引用次数: 0
The Hidden Economic Consequences of Migraine to the UK Government: Burden-of-Disease Analysis Using a Fiscal Framework. 偏头痛对英国政府的潜在经济后果:使用财政框架进行疾病负担分析。
Q3 Medicine Pub Date : 2023-10-03 eCollection Date: 2023-01-01 DOI: 10.36469/001c.87790
Rui Martins, Samuel Large, Rachel Russell, Gary Surmay, Mark P Connolly

Background: Migraine is a highly prevalent and incapacitating neurological disorder associated with the highest global disability burden in people aged 15 to 49 years. Europe has the fourth-highest prevalence of migraine, after North America, South America, and Central America, and above Asia and Africa. Migraine leads to relatively modest direct healthcare expenditure but has substantial indirect costs due to reduced productivity. Methods: The economic burden of migraine was estimated in comparison with the general population of the United Kingdom (UK) using an analytical fiscal modeling framework applying the government cost perspective. Published measures of migraine's impact on labor participation were applied to rates of economic activity/inactivity of the general population. The model estimates lifetime changes to earnings from employment, direct and indirect taxes paid, and financial support requirements over the life course. Incremental differences between those affected and unaffected by migraine are reported as net fiscal consequences to public accounts. Fiscal costs are reported as the discounted average per capita over a 20-year time horizon and for the entire annual UK cohort with prevalent migraine. Results: People affected by migraine are more likely to be absent from work, unemployed, and disabled, and to retire early. A 44-year-old individual affected by migraine was associated with £19 823 in excess fiscal costs to the UK government, £1379 per year living with the condition, compared with someone not affected by the disease. Annually, migraine was estimated to represent £12.20 billion to the public economy, approximately £130.63 per migraine episode. The model predicted annual productivity losses in the health and social care workforce to be £2.05 billion and total annual productivity losses to be over £5.81 billion. Conclusions: This fiscal analysis monetizes the occupational consequences of migraine to the UK government, both in terms of lost tax revenue and transfer payments. The findings are substantial and useful to characterize disease severity and to inform the body of evidence considered by decision makers appraising the cost-effectiveness of health technologies.

背景:偏头痛是一种高度流行的致残性神经系统疾病,在15至49岁的人群中,与全球最高的残疾负担有关。欧洲的偏头痛患病率排名第四,仅次于北美、南美和中美洲,高于亚洲和非洲。偏头痛导致相对温和的直接医疗支出,但由于生产力下降,有大量的间接成本。方法:采用政府成本视角的分析性财政建模框架,与英国普通人群相比,估计偏头痛的经济负担。已公布的偏头痛对劳动参与影响的测量方法适用于普通人群的经济活动/不活动率。该模型估计了终身就业收入、缴纳的直接和间接税以及终身财政支持需求的变化。受偏头痛影响和未受偏头痛影响的人之间的增量差异被报告为公共账户的净财政后果。财政成本被报告为20年内的人均贴现平均值,以及英国全年患有偏头痛的人群。结果:受偏头痛影响的人更有可能缺席工作、失业和残疾,并提前退休。一名44岁的偏头痛患者与19英镑相关 英国政府的财政成本为823英镑,与未受疾病影响的人相比,每年有1379英镑患有这种疾病。据估计,每年偏头痛对公共经济的贡献为122亿英镑,每发作一次偏头痛约为130.63英镑。该模型预测,卫生和社会护理劳动力的年生产力损失将达到20.5亿英镑,年生产力损失总额将超过58.1亿英镑。结论:这项财政分析将偏头痛对英国政府的职业后果货币化,包括税收损失和转移支付。这些发现具有实质性,有助于描述疾病的严重程度,并为决策者评估卫生技术成本效益所考虑的证据提供信息。
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引用次数: 0
Global Surgeon Opinion on the Impact of Surgical Access When Using Endocutters Across Specialties 全球外科医生对跨专业使用内腔器时手术通道影响的意见
Q3 Medicine Pub Date : 2023-09-20 DOI: 10.36469/jheor.2023.87644
Marina Gutierrez, Nadine Jamous, William Petraiuolo, Sanjoy Roy
Background: Despite design enhancements in endocutters, key challenges related to limited surgical access and space can impact stapling and, potentially, surgical outcomes. Objectives: This study aimed to develop consensus statements outlining the clinical value of precise articulation and greater anatomical access in minimally invasive surgery performed by bariatric, colorectal, and thoracic surgeons. Methods: Colorectal, bariatric, and thoracic surgeons from Japan, the United States, United Kingdom, and France participated in a 2-round modified Delphi panel. Round 1 included binary, Likert scale–type, multiple-response, and open-ended questions. These were converted to affirmative statements for round 2 if sufficient agreement was reached. Consensus was set at a predefined threshold of at least 90% of panelists across all surgical specialties and regions selecting the same option (“agree” or “disagree”) for the affirmative statements. Results: Of the 49 statements in the round 2 questionnaire, panelists (n=135) reached consensus that (1) tissue slippage outside stapler jaws can occur due to limited access and space; (2) greater jaw aperture could help to manipulate thick or fragile tissue more easily; (3) articulation of an endocutter is clinically important in laparoscopic surgeries; (4) improved access to hard-to-reach targets and in limited space would improve safety; and (5) an endocutter with improved access through greater articulation would become common use. Discussion: By understanding user-specific challenges and needs from both specialty- and region-wide perspectives, endoscopic stapling devices can continue to be refined. In this study, improved articulation and greater jaw aperture were the key design features examined. Improved articulation and greater jaw aperture were key stapler design features identified in this study that may mitigate the risk of instrument clashes and intraoperative complications such as anastomotic leaks. Conclusions: This study gained insights into surgeons’ perspective across a variety of specialties and from 3 distinct geographies. Participating surgeons reached consensus that an endocutter with greater jaw aperture and articulation may improve surgical access and has potential to improve surgical outcomes.
背景:尽管植入器的设计得到了改进,但与有限的手术通道和空间相关的关键挑战会影响吻合术,并可能影响手术结果。目的:本研究旨在达成共识,概述精确关节和更大解剖通路在减肥外科、结直肠外科和胸外科微创手术中的临床价值。方法:来自日本、美国、英国和法国的结直肠、肥胖和胸外科医生参加了2轮改进的德尔菲小组。第一轮包括二元、李克特量表型、多重回答和开放式问题。如果达成充分的协议,这些将在第二轮转为肯定的陈述。所有外科专业和地区的专家至少有90%的人选择相同的选项(“同意”或“不同意”)作为肯定陈述的共识设定为预定义的阈值。结果:在第2轮问卷的49个陈述中,小组成员(n=135)达成了以下共识:(1)由于通道和空间有限,订书机颚外可能发生组织滑移;(2)较大的颌口孔径有利于较厚或脆弱组织的操作;(3)在腹腔镜手术中,内肠器的关节是临床上重要的;(4)在有限的空间内改善对难以到达的目标的访问将提高安全性;(5)通过更大的清晰度改善访问的内夹将成为普遍使用。讨论:通过从专业和区域的角度了解用户特定的挑战和需求,内窥镜吻合器可以继续改进。在这项研究中,改进的关节和更大的颚孔是主要的设计特征。改善的关节和更大的颚孔是本研究确定的订书机设计的关键特征,可以减轻器械碰撞和术中并发症(如吻合口泄漏)的风险。结论:本研究从3个不同地区的不同专科获得了外科医生视角的见解。与会的外科医生一致认为,具有更大颌口和关节的内肠器可以改善手术通路,并有可能改善手术结果。
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引用次数: 0
Global Surgeon Opinion on the Impact of Surgical Access When Using Endocutters Across Specialties. 全球外科医生对跨专业使用内分泌时手术途径影响的看法。
Q3 Medicine Pub Date : 2023-09-20 eCollection Date: 2023-01-01 DOI: 10.36469/001c.87644
Marina Gutierrez, Nadine Jamous, William Petraiuolo, Sanjoy Roy

Background: Despite design enhancements in endocutters, key challenges related to limited surgical access and space can impact stapling and, potentially, surgical outcomes. Objectives: This study aimed to develop consensus statements outlining the clinical value of precise articulation and greater anatomical access in minimally invasive surgery performed by bariatric, colorectal, and thoracic surgeons. Methods: Colorectal, bariatric, and thoracic surgeons from Japan, the United States, United Kingdom, and France participated in a 2-round modified Delphi panel. Round 1 included binary, Likert scale-type, multiple-response, and open-ended questions. These were converted to affirmative statements for round 2 if sufficient agreement was reached. Consensus was set at a predefined threshold of at least 90% of panelists across all surgical specialties and regions selecting the same option ("agree" or "disagree") for the affirmative statements. Results: Of the 49 statements in the round 2 questionnaire, panelists (n=135) reached consensus that (1) tissue slippage outside stapler jaws can occur due to limited access and space; (2) greater jaw aperture could help to manipulate thick or fragile tissue more easily; (3) articulation of an endocutter is clinically important in laparoscopic surgeries; (4) improved access to hard-to-reach targets and in limited space would improve safety; and (5) an endocutter with improved access through greater articulation would become common use. Discussion: By understanding user-specific challenges and needs from both specialty- and region-wide perspectives, endoscopic stapling devices can continue to be refined. In this study, improved articulation and greater jaw aperture were the key design features examined. Improved articulation and greater jaw aperture were key stapler design features identified in this study that may mitigate the risk of instrument clashes and intraoperative complications such as anastomotic leaks. Conclusions: This study gained insights into surgeons' perspective across a variety of specialties and from 3 distinct geographies. Participating surgeons reached consensus that an endocutter with greater jaw aperture and articulation may improve surgical access and has potential to improve surgical outcomes.

背景:尽管内口的设计有所改进,但与有限的手术途径和空间相关的关键挑战可能会影响缝合,并可能影响手术结果。目的:本研究旨在达成共识,概述精确关节连接和更大的解剖通路在减肥、结直肠和胸部外科医生进行的微创手术中的临床价值。方法:来自日本、美国、英国和法国的结直肠、减肥和胸外科医生参加了一个两轮改良的德尔菲小组。第一轮包括二元、Likert量表类型、多重回答和开放式问题。如果达成了充分的协议,这些声明将转化为第二轮的肯定声明。共识被设定在一个预定义的阈值上,即所有外科专业和地区的至少90%的小组成员选择相同的选项(“同意”或“不同意”)进行肯定性陈述。结果:在第二轮问卷中的49项陈述中,小组成员(n=135)达成共识:(1)由于进入和空间有限,缝合器钳口外可能发生组织滑动;(2) 更大的颌骨开口可以帮助更容易地操作厚组织或脆弱组织;(3) 内口的关节在腹腔镜手术中具有重要的临床意义;(4) 在有限的空间内更好地接近难以达到的目标将提高安全性;以及(5)通过更大的关节连接改善通路的内口器将成为常用。讨论:通过从专业和区域角度了解用户的具体挑战和需求,可以继续改进内窥镜缝合设备。在这项研究中,改善的关节和更大的下颌开口是检查的关键设计特征。本研究中确定的关键缝合器设计特点是改善关节和扩大颌骨孔径,这可能会降低器械碰撞和吻合口瘘等术中并发症的风险。结论:本研究深入了解了外科医生在不同专业和3个不同地区的观点。参与的外科医生达成了共识,即具有更大颌骨孔径和关节的内口钳可以改善手术途径,并有可能改善手术结果。
{"title":"Global Surgeon Opinion on the Impact of Surgical Access When Using Endocutters Across Specialties.","authors":"Marina Gutierrez, Nadine Jamous, William Petraiuolo, Sanjoy Roy","doi":"10.36469/001c.87644","DOIUrl":"10.36469/001c.87644","url":null,"abstract":"<p><p><b>Background:</b> Despite design enhancements in endocutters, key challenges related to limited surgical access and space can impact stapling and, potentially, surgical outcomes. <b>Objectives:</b> This study aimed to develop consensus statements outlining the clinical value of precise articulation and greater anatomical access in minimally invasive surgery performed by bariatric, colorectal, and thoracic surgeons. <b>Methods:</b> Colorectal, bariatric, and thoracic surgeons from Japan, the United States, United Kingdom, and France participated in a 2-round modified Delphi panel. Round 1 included binary, Likert scale-type, multiple-response, and open-ended questions. These were converted to affirmative statements for round 2 if sufficient agreement was reached. Consensus was set at a predefined threshold of at least 90% of panelists across all surgical specialties and regions selecting the same option (\"agree\" or \"disagree\") for the affirmative statements. <b>Results:</b> Of the 49 statements in the round 2 questionnaire, panelists (n=135) reached consensus that (1) tissue slippage outside stapler jaws can occur due to limited access and space; (2) greater jaw aperture could help to manipulate thick or fragile tissue more easily; (3) articulation of an endocutter is clinically important in laparoscopic surgeries; (4) improved access to hard-to-reach targets and in limited space would improve safety; and (5) an endocutter with improved access through greater articulation would become common use. <b>Discussion:</b> By understanding user-specific challenges and needs from both specialty- and region-wide perspectives, endoscopic stapling devices can continue to be refined. In this study, improved articulation and greater jaw aperture were the key design features examined. Improved articulation and greater jaw aperture were key stapler design features identified in this study that may mitigate the risk of instrument clashes and intraoperative complications such as anastomotic leaks. <b>Conclusions:</b> This study gained insights into surgeons' perspective across a variety of specialties and from 3 distinct geographies. Participating surgeons reached consensus that an endocutter with greater jaw aperture and articulation may improve surgical access and has potential to improve surgical outcomes.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10515882/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41135499","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
Outcomes for Dostarlimab and Real-World Treatments in Post-platinum Patients With Advanced/Recurrent Endometrial Cancer: The GARNET Trial Versus a US Electronic Health Record-Based External Control Arm 多斯塔利单抗和真实世界治疗在铂后晚期/复发子宫内膜癌患者中的结果:GARNET试验与基于美国电子健康记录的外部控制臂
Q3 Medicine Pub Date : 2023-09-08 DOI: 10.36469/jheor.2023.77484
Scott Goulden, Qin Shen, Robert Coleman, Cara Mathews, Matthias Hunger, Ankit Pahwa, Rene Schade
Background: Patients with advanced or recurrent endometrial cancer (EC) have limited treatment options following platinum-based chemotherapy and poor prognosis. The single-arm, Phase I GARNET trial (NCT02715284) previously reported dostarlimab efficacy in mismatch repair–deficient/microsatellite instability–high advanced or recurrent EC. Objectives: The objective of this study was to compare overall survival (OS) and describe time to treatment discontinuation (TTD) for dostarlimab (GARNET Cohort A1 safety population) with an equivalent real-world external control arm receiving non-anti-programmed death (PD)-1/PD-ligand (L)1/2 treatments (constructed using data from a nationwide electronic health record–derived de-identified database and applied GARNET eligibility criteria). Methods: Propensity scores constructed from prognostic factors, identified by literature review and clinical experts, were used for inverse probability of treatment weighting (IPTW). Kaplan-Meier curves were constructed and OS/TTD was estimated (Cox regression model was used to estimate the OS-adjusted hazard ratio). Results: Dostarlimab was associated with a 52% lower risk of death vs real-world treatments (hazard ratio, 0.48; 95% confidence interval [CI], 0.35-0.66). IPTW-adjusted median OS for dostarlimab (N=143) was not estimable (95% CI, 19.4–not estimable) versus 13.1 months (95% CI, 8.3-15.9) for real-world treatments (N = 185). Median TTD was 11.7 months (95% CI, 6.0-38.7) for dostarlimab and 5.3 months (95% CI, 4.1-6.0) for the real-world cohort. Discussion: Consistent with previous analyses, patients treated with dostarlimab had significantly longer OS than patients in the US real-world cohort after adjusting for the lack of randomization using stabilized IPTW. Additionally, patients had a long TTD when treated with dostarlimab, suggesting a favorable tolerability profile. Conclusion: Patients with advanced or recurrent EC receiving dostarlimab in GARNET had significantly lower risk of death than those receiving real-world non-anti-PD-(L)1/2 treatments.
背景:晚期或复发子宫内膜癌(EC)患者在铂基化疗后治疗选择有限,预后较差。单臂I期GARNET试验(NCT02715284)先前报道了dostarlimumab对错配修复缺陷/微卫星不稳定性-高度晚期或复发性EC的疗效。目的:本研究的目的是比较dostarlimumab (GARNET A1队列安全人群)与接受非抗程序性死亡(PD)-1/PD配体(L)1/2治疗的等效现实世界外部对照组的总生存期(OS)和描述治疗停止时间(TTD)(使用来自全国电子健康记录衍生的去识别数据库的数据构建,并应用GARNET资格标准)。方法:根据文献回顾和临床专家确定的预后因素构建倾向评分,用于治疗加权逆概率(IPTW)。构建Kaplan-Meier曲线,估计OS/TTD(采用Cox回归模型估计OS校正后的风险比)。结果:与现实治疗相比,dostarlimumab与52%的死亡风险降低相关(风险比,0.48;95%可信区间[CI], 0.35-0.66)。dostarlimumab经iptws调整后的中位OS (N=143)不可估计(95% CI, 19.4 -不可估计),而现实世界治疗(N= 185)的中位OS为13.1个月(95% CI, 8.3-15.9)。dostarlimumab组的中位TTD为11.7个月(95% CI, 6.0-38.7),真实队列组的中位TTD为5.3个月(95% CI, 4.1-6.0)。讨论:与先前的分析一致,在使用稳定IPTW调整缺乏随机化后,接受dostarlimab治疗的患者的生存期明显长于美国现实世界队列中的患者。此外,患者在接受多司达单抗治疗时,TTD时间较长,这表明患者具有良好的耐受性。结论:晚期或复发性EC患者在GARNET中接受dostarlimumab治疗的死亡风险明显低于接受现实世界非抗pd -(L)1/2治疗的患者。
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引用次数: 0
Outcomes for Dostarlimab and Real-World Treatments in Post-platinum Patients With Advanced/Recurrent Endometrial Cancer: The GARNET Trial Versus a US Electronic Health Record-Based External Control Arm. 铂后晚期/复发性子宫内膜癌患者接受多斯他利单抗和真实世界治疗的结果:GARNET试验与基于美国电子健康记录的外部对照组的对比。
Q3 Medicine Pub Date : 2023-09-08 eCollection Date: 2023-01-01 DOI: 10.36469/001c.77484
Scott Goulden, Qin Shen, Robert L Coleman, Cara Mathews, Matthias Hunger, Ankit Pahwa, Rene Schade

Background: Patients with advanced or recurrent endometrial cancer (EC) have limited treatment options following platinum-based chemotherapy and poor prognosis. The single-arm, Phase I GARNET trial (NCT02715284) previously reported dostarlimab efficacy in mismatch repair-deficient/microsatellite instability-high advanced or recurrent EC. Objectives: The objective of this study was to compare overall survival (OS) and describe time to treatment discontinuation (TTD) for dostarlimab (GARNET Cohort A1 safety population) with an equivalent real-world external control arm receiving non-anti-programmed death (PD)-1/PD-ligand (L)1/2 treatments (constructed using data from a nationwide electronic health record-derived de-identified database and applied GARNET eligibility criteria). Methods: Propensity scores constructed from prognostic factors, identified by literature review and clinical experts, were used for inverse probability of treatment weighting (IPTW). Kaplan-Meier curves were constructed and OS/TTD was estimated (Cox regression model was used to estimate the OS-adjusted hazard ratio). Results: Dostarlimab was associated with a 52% lower risk of death vs real-world treatments (hazard ratio, 0.48; 95% confidence interval [CI], 0.35-0.66). IPTW-adjusted median OS for dostarlimab (N=143) was not estimable (95% CI, 19.4-not estimable) versus 13.1 months (95% CI, 8.3-15.9) for real-world treatments (N = 185). Median TTD was 11.7 months (95% CI, 6.0-38.7) for dostarlimab and 5.3 months (95% CI, 4.1-6.0) for the real-world cohort. Discussion: Consistent with previous analyses, patients treated with dostarlimab had significantly longer OS than patients in the US real-world cohort after adjusting for the lack of randomization using stabilized IPTW. Additionally, patients had a long TTD when treated with dostarlimab, suggesting a favorable tolerability profile. Conclusion: Patients with advanced or recurrent EC receiving dostarlimab in GARNET had significantly lower risk of death than those receiving real-world non-anti-PD-(L)1/2 treatments.

背景:晚期或复发性子宫内膜癌(EC)患者在铂类化疗后的治疗选择有限,且预后较差。单臂 I 期 GARNET 试验(NCT02715284)曾报道了多司他利单抗对错配修复缺陷/微卫星不稳定性高的晚期或复发性子宫内膜癌的疗效。研究目的本研究旨在比较多司他利单抗(GARNET队列A1安全人群)与接受非抗程序性死亡(PD)-1/PD配体(L)1/2治疗的等效真实世界外部对照组的总生存期(OS),并描述治疗终止时间(TTD)(使用来自全国性电子健康记录的去身份化数据库的数据并应用GARNET资格标准构建)。方法:根据文献综述和临床专家确定的预后因素构建倾向分数,用于反向治疗概率加权(IPTW)。构建 Kaplan-Meier 曲线并估算 OS/TTD (使用 Cox 回归模型估算 OS 调整后的危险比)。结果与真实世界的治疗相比,多斯他利单抗的死亡风险降低了52%(危险比为0.48;95%置信区间[CI]为0.35-0.66)。多司他利单抗(N=143)的IPTW调整后中位OS无法估计(95% CI,19.4-无法估计),而实际治疗(N=185)的中位OS为13.1个月(95% CI,8.3-15.9)。多司他利单抗的中位TTD为11.7个月(95% CI,6.0-38.7),而真实世界队列的中位TTD为5.3个月(95% CI,4.1-6.0)。讨论情况与之前的分析一致,在使用稳定的IPTW调整随机化的缺失后,接受多司他利单抗治疗的患者的OS明显长于美国真实世界队列中的患者。此外,接受多司他利单抗治疗的患者TTD较长,这表明多司他利单抗具有良好的耐受性。结论在GARNET中接受多司他利单抗治疗的晚期或复发性EC患者的死亡风险明显低于接受真实世界非抗PD-(L)1/2治疗的患者。
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引用次数: 0
Use of Open Claims vs Closed Claims in Health Outcomes Research. 开放声明与封闭声明在健康结果研究中的应用。
IF 2.3 Q2 ECONOMICS Pub Date : 2023-09-05 eCollection Date: 2023-01-01 DOI: 10.36469/001c.87538
Onur Baser, Gabriela Samayoa, Nehir Yapar, Erdem Baser, Fatih Mete

Background: Closed claims are frequently used in outcomes research studies. Lately, the availability of open claims has increased the possibility of obtaining information faster and on a larger scale. However, because of the possibility of missing claims and duplications, these data sets have not been highly utilized in medical research. Objective: To compare frequently used healthcare utilization measures between closed claims and open claims to analyze if the possibility of missing claims in open claims data creates a downward bias in the estimates. Methods: We identified 18 different diseases using 2022 data from 2 closed claims data sets (MarketScan® and PharMetrics® Plus) and 1 open claims database (Kythera). After applying an algorithm that removes possible duplications from open claims data, we compared healthcare utilizations such as inpatient, emergency department, and outpatient use and length of stay among these 3 data sets. We applied standardized differences to compare the medians for each outcome. Results: The sample size of the open claims data sets was 10 to 65 times larger than closed claims data sets depending on disease type. For each disease, the estimates of healthcare utilization were similar between the open claims and closed claims data. The difference was statistically insignificant. Conclusions: Open claims data with a bigger sample size and more current available information provide essential advantages for healthcare outcomes research studies. Therefore, especially for new medications and rare diseases, open claims data can provide information much earlier than closed claims, which usually have a time lag of 6 to 8 months.

背景:封闭式索赔经常用于结果研究。最近,公开索赔的可用性增加了更快、更大规模地获取信息的可能性。然而,由于索赔可能缺失和重复,这些数据集在医学研究中没有得到充分利用。目的:比较已结案索赔和未结案索赔之间常用的医疗保健利用率指标,以分析未结案索赔数据中遗漏索赔的可能性是否会造成估计值的向下偏差。方法:我们使用来自2个封闭索赔数据集(MarketScan®和PharMetrics®Plus)和1个开放索赔数据库(Kythera)的2022年数据,确定了18种不同的疾病。在应用了一种从开放索赔数据中消除可能重复的算法后,我们比较了这3个数据集的医疗利用率,如住院、急诊和门诊使用率以及住院时间。我们采用标准化差异来比较每种结果的中位数。结果:根据疾病类型,开放索赔数据集的样本量是封闭索赔数据集样本量的10到65倍。对于每种疾病,公开索赔和非公开索赔数据对医疗利用率的估计是相似的。这一差异在统计学上并不显著。结论:具有更大样本量和更多最新可用信息的开放索赔数据为医疗保健结果研究提供了基本优势。因此,特别是对于新药和罕见病,公开索赔数据可以比封闭索赔更早地提供信息,封闭索赔通常有6到8个月的时间滞后。
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引用次数: 0
Use of Open Claims vs Closed Claims in Health Outcomes Research 开放式索赔与封闭式索赔在健康结果研究中的应用
Q3 Medicine Pub Date : 2023-09-05 DOI: 10.36469/jheor.2023.87538
Onur Baser, Gabriela Samayoa, Nehir Yapar, Erdem Baser, Fatih Mete
Background: Closed claims are frequently used in outcomes research studies. Lately, the availability of open claims has increased the possibility of obtaining information faster and on a larger scale. However, because of the possibility of missing claims and duplications, these data sets have not been highly utilized in medical research. Objective: To compare frequently used healthcare utilization measures between closed claims and open claims to analyze if the possibility of missing claims in open claims data creates a downward bias in the estimates. Methods: We identified 18 different diseases using 2022 data from 2 closed claims data sets (MarketScan® and PharMetrics® Plus) and 1 open claims database (Kythera). After applying an algorithm that removes possible duplications from open claims data, we compared healthcare utilizations such as inpatient, emergency department, and outpatient use and length of stay among these 3 data sets. We applied standardized differences to compare the medians for each outcome. Results: The sample size of the open claims data sets was 10 to 65 times larger than closed claims data sets depending on disease type. For each disease, the estimates of healthcare utilization were similar between the open claims and closed claims data. The difference was statistically insignificant. Conclusions: Open claims data with a bigger sample size and more current available information provide essential advantages for healthcare outcomes research studies. Therefore, especially for new medications and rare diseases, open claims data can provide information much earlier than closed claims, which usually have a time lag of 6 to 8 months.
背景:封闭式索赔常用于结果研究。最近,公开索赔的可得性增加了更快更大规模地获得信息的可能性。然而,由于可能出现索赔缺失和重复,这些数据集在医学研究中没有得到充分利用。目的:比较封闭索赔和开放索赔之间常用的医疗保健利用措施,以分析开放索赔数据中缺失索赔的可能性是否会导致估计中的向下偏差。方法:我们使用来自2个封闭索赔数据集(MarketScan®和PharMetrics®Plus)和1个开放索赔数据库(Kythera)的2022个数据,确定了18种不同的疾病。在应用一种算法从公开索赔数据中删除可能的重复后,我们比较了这3个数据集的医疗保健利用情况,如住院、急诊和门诊的使用情况和住院时间。我们应用标准化差异来比较每个结果的中位数。结果:根据疾病类型,开放索赔数据集的样本量比封闭索赔数据集大10 ~ 65倍。对于每种疾病,公开索赔和封闭索赔数据之间的医疗保健利用估计是相似的。差异在统计上不显著。结论:具有更大样本量和更多当前可用信息的公开索赔数据为医疗保健结果研究提供了必不可少的优势。因此,特别是对于新药和罕见疾病,公开索赔数据可以比封闭索赔更早地提供信息,后者通常有6至8个月的时间滞后。
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引用次数: 0
Health Economic Analysis of Two-Layer Bandage System for Treatment of Chronic Venous Insufficiency 双层绷带系统治疗慢性静脉功能不全的卫生经济学分析
Q3 Medicine Pub Date : 2023-08-25 DOI: 10.36469/jheor.2023.82159
Peter Mallow
Background: Compression therapy is the gold standard for the treatment of chronic venous insufficiency (CVI). Two-layer bandage (2LB) systems have been shown to be a safe and effective treatment option. Objective: To estimate the total cost per response (CPR) for the resolution of edema and wounds in patients with CVI treated with a 2LB system as part of their overall wound healing regimen. Methods: A probabilistic decision tree model was developed to estimate the incremental CPR for a 2LB system. The model simulated 10 000 patients to estimate the CPR for the resolution of edema and wound healing. The analysis was performed using clinical data from a published single-arm, multicenter prospective study of CVI indicated for compression therapy. The response outcomes of interest were resolution of edema and rate of wound healing. The follow-up time was a maximum of 6 weeks, and the perspective of the study was a US outpatient treatment center. Economic data for compression therapy were based on the public prices of a 2LB system. Dressing changes occurred per manufacturer instructions for use. Results: The study comprised 702 patients (56% female), with a total of 414 wounds. The median duration of the wounds was 42 days, and the median size at the initial visit was 3.5 cm2. The average pain reduction fell by 67% using a visual analog score. Bandages were typically changed once or twice a week (51.7%). Wound healing occurred in 128 of the 414 wounds (30.9%). The expected incremental CPR of a 2LB system for the resolution of edema was $65.67 (range, $16.67-$124.32). The expected incremental CPR of a 2LB system for the healing of a wound was $138.71 (range, $35.71-$273.53). Conclusion: This economic evaluation complements previous clinical effectiveness and safety studies of 2LB systems for the treatment of CVI. The results demonstrate that the costs of incorporating 2LB into standard wound-healing protocols are negligible compared with overall treatment costs. Two-layer bandages may be considered a cost-effective first-line system for the treatment of wounds caused by CVI.
背景:压迫疗法是治疗慢性静脉功能不全(CVI)的金标准。双层绷带(2LB)系统已被证明是一种安全有效的治疗选择。目的:评估2LB系统作为CVI患者整体伤口愈合方案的一部分治疗水肿和伤口的每次缓解总成本(CPR)。方法:采用概率决策树模型估计2LB系统的增量CPR。该模型模拟了1万例患者,以估计心肺复苏术对水肿和伤口愈合的解决效果。该分析是根据一项已发表的单臂、多中心前瞻性CVI临床数据进行的,该研究表明CVI需要进行压迫治疗。我们感兴趣的反应结果是水肿的消退和伤口愈合的速度。随访时间最长为6周,研究视角为美国门诊治疗中心。压缩疗法的经济数据基于2LB系统的公开价格。根据制造商的使用说明进行敷料更换。结果:本研究纳入702例患者(56%为女性),共414处伤口。伤口的中位持续时间为42天,初次就诊时的中位面积为3.5平方厘米。使用视觉模拟评分,疼痛平均减轻了67%。通常每周更换1 - 2次绷带(51.7%)。414例创面中有128例创面愈合(30.9%)。2LB系统解决水肿的预期增量CPR费用为65.67美元(范围16.67- 124.32美元)。2LB系统对伤口愈合的预期增量CPR为138.71美元(范围35.71- 273.53美元)。结论:这项经济评估补充了之前2LB系统治疗CVI的临床有效性和安全性研究。结果表明,与总体治疗成本相比,将2LB纳入标准伤口愈合方案的成本可以忽略不计。双层绷带可能被认为是治疗CVI引起的伤口的一种具有成本效益的一线系统。
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引用次数: 0
Health Economic Analysis of Two-Layer Bandage System for Treatment of Chronic Venous Insufficiency. 双层包扎系统治疗慢性静脉功能不全的健康经济分析。
IF 2.3 Q2 ECONOMICS Pub Date : 2023-08-25 eCollection Date: 2023-01-01 DOI: 10.36469/001c.82159
Peter J Mallow

Background: Compression therapy is the gold standard for the treatment of chronic venous insufficiency (CVI). Two-layer bandage (2LB) systems have been shown to be a safe and effective treatment option. Objective: To estimate the total cost per response (CPR) for the resolution of edema and wounds in patients with CVI treated with a 2LB system as part of their overall wound healing regimen. Methods: A probabilistic decision tree model was developed to estimate the incremental CPR for a 2LB system. The model simulated 10 000 patients to estimate the CPR for the resolution of edema and wound healing. The analysis was performed using clinical data from a published single-arm, multicenter prospective study of CVI indicated for compression therapy. The response outcomes of interest were resolution of edema and rate of wound healing. The follow-up time was a maximum of 6 weeks, and the perspective of the study was a US outpatient treatment center. Economic data for compression therapy were based on the public prices of a 2LB system. Dressing changes occurred per manufacturer instructions for use. Results: The study comprised 702 patients (56% female), with a total of 414 wounds. The median duration of the wounds was 42 days, and the median size at the initial visit was 3.5 cm2. The average pain reduction fell by 67% using a visual analog score. Bandages were typically changed once or twice a week (51.7%). Wound healing occurred in 128 of the 414 wounds (30.9%). The expected incremental CPR of a 2LB system for the resolution of edema was $65.67 (range, $16.67-$124.32). The expected incremental CPR of a 2LB system for the healing of a wound was $138.71 (range, $35.71-$273.53). Conclusion: This economic evaluation complements previous clinical effectiveness and safety studies of 2LB systems for the treatment of CVI. The results demonstrate that the costs of incorporating 2LB into standard wound-healing protocols are negligible compared with overall treatment costs. Two-layer bandages may be considered a cost-effective first-line system for the treatment of wounds caused by CVI.

背景:压迫疗法是治疗慢性静脉功能不全(CVI)的金标准。双层绷带(2LB)系统已被证明是一种安全有效的治疗选择。目的:作为整体伤口愈合方案的一部分,评估用2LB系统治疗CVI患者水肿和伤口的每次反应总成本(CPR)。方法:建立概率决策树模型来估计2LB系统的增量CPR。该模型模拟了10 000名患者,以估计用于水肿和伤口愈合的CPR。该分析使用了一项已发表的用于压迫治疗的CVI单臂、多中心前瞻性研究的临床数据。感兴趣的反应结果是水肿的消退和伤口愈合率。随访时间最长为6周,研究对象为美国门诊治疗中心。压缩治疗的经济数据基于2LB系统的公开价格。按照制造商的使用说明更换了敷料。结果:该研究包括702名患者(56%为女性),共414处伤口。伤口的中位持续时间为42天,初次就诊时的中位大小为3.5 cm2。使用视觉模拟评分,疼痛的平均减轻程度下降了67%。绷带通常每周更换一到两次(51.7%)。414个伤口中有128个伤口愈合(30.9%)。2LB系统用于解决水肿的预期增量CPR为65.67美元(范围16.67美元至124.32美元)。2L B系统用于伤口愈合的预期增量心肺复苏为138.71美元(范围35.71美元至273.53美元)。结论:本经济评估补充了之前的评估2LB系统治疗CVI的临床有效性和安全性研究。结果表明,与整体治疗成本相比,将2LB纳入标准伤口愈合方案的成本可以忽略不计。双层绷带可以被认为是治疗CVI引起的伤口的具有成本效益的一线系统。
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引用次数: 0
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Journal of Health Economics and Outcomes Research
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