Value in Health最新文献_第9页

Prevalence of Immature Survival Data for Anticancer Drugs Presented to the National Institute for Health and Care Excellence Between 2018 and 2022 2018-2022年期间提交给国家健康与护理卓越研究所的抗癌药物未成熟生存数据的患病率

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-24 DOI: 10.1016/j.jval.2024.11.013

Cara L. Gibbons MSc , Nicholas R. Latimer PhD

Objectives

Between 2015 and 2017, 41% of National Institute for Health and Care Excellence (NICE) cancer single-technology appraisal (STA) decisions relied upon immature survival data. This occurs when clinical trials that form the evidence base in support of new or existing technologies suffer from limited follow-up. During this period, NICE did not negatively recommend any cancer technologies that used immature data. This suggests a potential incentive to submit to NICE with immature data to avoid rejection. Using immature survival data in cost-effectiveness evaluations has resulted in significantly different conclusions compared with cost-effectiveness reestimations using matured data. We assessed the reliance on immature survival data in NICE decision making of cancer treatments, appraised after 2017.

Methods

A structured literature review of NICE cancer STAs published between 2018 and 2022 was conducted. The relationship between data maturity and NICE recommendations was assessed, and the extent to which past decisions were later reviewed was explored.

Results

56% (n = 57) of NICE’s cancer recommendations relied upon immature survival data. Fifty-four percent (n = 31) of these received a positive recommendation, 39% (n = 22) were placed into the Cancer Drugs Fund (CDF), and 7% (n = 4) received a negative recommendation. STAs with mature data received a similar proportion of negative recommendations. Only 1 non-CDF recommendation based on immature data was reappraised using updated survival data.

Conclusion

The majority of NICE cancer technology decisions are based on immature survival data and receive positive recommendations. Non-CDF decisions are unlikely to be reappraised. Consequently, many technologies could receive an inappropriate recommendation based on immature data and not be subsequently rectified.

在2015年至2017年期间，41%的NICE癌症单一技术评估（STA）决策依赖于不成熟的生存数据。当形成支持新技术或现有技术的证据基础的临床试验缺乏随访时，就会出现这种情况。在此期间，NICE没有负面推荐任何使用不成熟数据的癌症技术。这暗示了向NICE提交不成熟数据以避免被拒的潜在动机。与使用成熟数据进行成本效益重新评估相比，在成本效益评估中使用不成熟生存数据得出了重要的不同结论。我们评估了2017年后评估的NICE癌症治疗决策对未成熟生存数据的依赖。方法：对2018年至2022年发表的NICE癌症sta进行结构化文献综述。评估了数据成熟度和NICE建议之间的关系，并探讨了对过去的决定进行审查的程度。结果：56% （n=57）的NICE癌症建议依赖于未成熟生存数据。其中54% （n=31）被推荐为阳性，39% （n=22）被纳入癌症药物基金（CDF）， 7% （n=4）被推荐为阴性。数据成熟的sta收到的负面推荐比例相似。只有一个基于未成熟数据的非cdf推荐使用更新的生存数据重新评估。结论：大多数NICE癌症技术决策是基于未成熟的生存数据，并得到了积极的建议。非cdf决策不太可能被重新评估。因此，许多技术可能会根据不成熟的数据得到不适当的建议，而随后不加以纠正。

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引用次数: 0

The Estimation of Health State Utility Values for Psychological Distress in Australia: Implications for Future Economic Evaluations 澳大利亚心理困扰的健康状态效用值的估计：对未来经济评估的影响。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-24 DOI: 10.1016/j.jval.2024.12.002

Syed Afroz Keramat PhD , Tracy Comans PhD , Rabeya Basri MSS , Daniel Bailey PhD , Deborah Brooks PhD , Nadeeka N. Dissanayaka PhD

Objectives

Psychological distress is a state of emotional suffering and discomfort that often manifests as anxiety, depression, or other mental health symptoms, impairing daily functioning and hindering concentration, relationships, and work or school performance. We aimed to examine the disutility associated with psychological distress.

Methods

We used longitudinal data obtained from the Household, Income, and Labour Dynamics in Australia Survey. We measured health state utility values (HSUVs) using the Short-Form 6-Dimension (SF-6D) Utility Index and psychological distress using the Kessler Psychological Distress Scale. We used longitudinal fixed-effects regression model to examine the effects of psychological distress on HSUVs.

Results

The results from fixed-effects panel regression models indicate a negative effect of psychological distress on HSUVs. We found that moderate psychological distress (β = −0.057, 95% CI −0.059 to −0.055) and high psychological distress (β = −0.123, 95% CI −0.126 to −0.121) led to a significant reduction in HSUVs. These findings hold across different subsamples, such as age, gender, and race.

Conclusions

By quantifying the reduction in HSUVs due to psychological distress, our study provides valuable data for future economic evaluations of healthcare interventions. The evidence generated from future economic evaluations will assist policymakers in making informed decisions about the cost-effective interventions for treating psychological distress.

目的：心理困扰是一种情绪痛苦和不适的状态，通常表现为焦虑、抑郁或其他心理健康症状，损害日常功能，妨碍注意力集中、人际关系和工作或学习表现。我们的目的是研究与心理困扰相关的负效用。方法：我们利用从澳大利亚家庭、收入和劳动力动态（HILDA）调查中获得的纵向数据。我们使用短格式六维效用指数（SF-6D）测量健康状态效用值（hsuv），使用凯斯勒心理困扰量表（K10）测量心理困扰。我们采用纵向固定效应回归模型来检验心理困扰对hsuv的影响。结果：固定效应面板回归模型的结果表明，心理困扰对hsuv有负向影响。我们发现，中度心理困扰（β = -0.057, 95% CI = -0.059, -0.055）和重度心理困扰（β = -0.123, 95% CI = -0.126, -0.121）导致hsuv的显著减少。这些发现适用于不同的子样本，如年龄、性别和种族。结论：通过量化心理困扰导致的hsuv减少，我们的研究为未来医疗干预的经济评估提供了有价值的数据。未来经济评估产生的证据将有助于决策者就治疗心理困扰的最有效干预措施做出明智的决定。

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引用次数: 0

A Systematic Literature Review of Important and Meaningful Differences in the EQ-5D Index and Visual Analog Scale Scores EQ-5D指数与视觉模拟量表评分重要且有意义差异的系统文献综述。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-16 DOI: 10.1016/j.jval.2024.11.006

Fatima Al Sayah PhD , Xuejing Jin PhD , Hilary Short MSc , Nathan S. McClure PhD , Arto Ohinmaa PhD , Jeffrey A. Johnson PhD

Objectives

We aimed to provide a comprehensive summary, synthesis, and appraisal of minimally important difference (MID) estimates for EQ-5D instruments.

Methods

We conducted a systematic search using relevant terms related to “minimally/clinically, meaningful/ important difference/change” and “EQ-5D” in 6 major databases, including MEDLINE, Embase, PsycINFO, CINAHL, Scopus, and Cochrane Library (up to January 2023). We included studies that provided at least 1 original MID estimate for the EQ-5D.

Results

A total of 90 studies reporting 840 MID estimates were included. MID estimates for the EQ-5D-3L index score ranged from 0.075 to 0.8 using distribution-based approaches (239 estimates; 20 studies), from 0.003 to 0.72 using anchor-based approaches (189 estimates; 43 studies), and from 0.038 to 0.082 using instrument-defined approaches (4 estimates; 1 study). For the EQ-5D-5L, MID estimates ranged from 0.023 to 0.115 using distribution-based approaches (17 estimates; 12 studies), from 0.01 to 0.41 using anchor-based approaches (97 estimates; 15 studies), and from 0.037 to 0.101 using instrument-defined approaches (62 estimates; 8 studies). For the EQ visual analog scale, MID estimates ranged from 0.96 to 16.6 using distribution-based approaches (87 estimates; 14 studies) and from 0.42 to 51.0 using anchor-based approaches (84 estimates; 24 studies). MID estimates varied by underlying clinical conditions, baseline scores, and direction of change.

Conclusions

A wide range of MID estimates for EQ-5D instruments were identified, highlighting the variability of MID across populations, estimation methods, direction of change, baseline scores, and EQ-5D versions. These factors should be carefully considered when selecting an appropriate MID for interpreting EQ-5D scores.

目的：我们旨在对EQ-5D仪器的最小重要差异（MID）估计提供全面的总结、综合和评估。方法：系统检索MEDLINE、EMBASE、PsycINFO、CINAHL、Scopus、Cochrane Library等6个主要数据库（截至2023年1月）中“最小/临床、有意义/重要差异/变化”和“EQ-5D”相关术语。我们纳入了为EQ-5D提供至少一个原始MID估计的研究。结果：共纳入90项研究报告840例MID估计。使用基于分布的方法，EQ-5D-3L指数评分的MID估计范围为0.075至0.8(239个估计；使用基于锚点的方法，从0.003到0.72(189个估计；43项研究)，使用工具定义的方法从0.038到0.082(4项估计；1研究)。对于EQ-5D-5L，使用基于分布的方法，MID估计范围为0.023至0.115(17个估计；12项研究)，使用基于锚点的方法，从0.01到0.41(97项估计；15项研究)，使用工具定义方法从0.037到0.101(62项估计；8研究)。对于EQ视觉模拟量表（VAS），使用基于分布的方法，MID估计范围为0.96至16.6(87个估计；14项研究)，使用基于锚点的方法从0.42到51.0(84项估计；24研究)。MID的估计因潜在的临床状况、基线评分和变化方向而异。结论：确定了EQ-5D仪器的广泛MID估计，突出了人群、估计方法、变化方向、基线分数和EQ-5D版本之间MID的可变性。在选择合适的MID来解释EQ-5D分数时，应仔细考虑这些因素。

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引用次数: 0

How Do Bundled Payment Initiatives Account for Differences in Patient Risk Profiles? A Systematic Review. 捆绑支付计划如何解释患者风险概况的差异？系统评价。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-16 DOI: 10.1016/j.jval.2024.11.011

Celine M R Hendriks, Fiona Koster, Daniëlle Cattel, Marc R Kok, Angelique E A M Weel-Koenders, Deirisa Lopes Barreto, Frank Eijkenaar

Objectives: Bundled payments (BPs) are increasingly being adopted to enable the delivery of high-value care. For BPs to reach their goals, accounting for differences in patient risk profiles (PRPs) predictive of spending is crucial. However, insight is lacking into how this is done in practice. This study aims to fill this gap.

Methods: We conducted a systematic review of literature published until February 2024, focusing on BP initiatives in the Organization for Economic Cooperation and Development countries. We collected data on initiatives' general characteristics, details on the (stated reasons for) approaches used to account for PRP, and suggested improvements. Patterns within and across initiatives were analyzed using extraction tables and thematic analysis.

Results: We included 95 documents about 17 initiatives covering various conditions and procedures. Across these initiatives, patient exclusion (n = 14) and risk adjustment (n = 12) of bundle prices were the most applied methods, whereas risk stratification was less common (n = 3). Most authors stated mitigating perverse incentives as the primary reason for PRP accounting. Commonly used risk factors included comorbidities and sociodemographic and condition/procedure-specific characteristics. Our findings show that, despite increasingly sophisticated approaches over time, key areas for improvement included better alignment with value and equity goals, and enhanced data availability for more comprehensive corrections for relevant risk factors.

Conclusions: BP initiatives use various approaches to account for PRP differences. Despite a trend toward more sophisticated approaches, most remain basic with room for improvement. To enable cross-initiative comparisons and learning, it is important that stakeholders involved in BPs be transparent about the (reasons for) design choices made.

目标：越来越多地采用捆绑支付（bp）来提供高价值的医疗服务。对于bp来说，考虑患者风险概况（PRP）预测支出的差异是至关重要的。然而，缺乏对如何在实践中做到这一点的洞察力。本研究旨在填补这一空白。方法：我们对截至2024年2月发表的文献进行了系统回顾，重点关注经合组织国家的BP计划。我们收集了关于计划的一般特征的数据，关于用于解释PRP的方法的细节（说明的原因），以及建议的改进。使用抽取表和专题分析分析了活动内部和跨活动的模式。结果：我们纳入了91份文件，涉及17项措施，涵盖了各种条件和程序。在这些举措中，患者排除（n=14）和捆绑价格风险调整（n=12）是应用最多的方法，而风险分层不太常见（n=3）。大多数作者表示，减轻不当激励是PRP会计的主要原因。常用的危险因素包括合并症、社会人口学和病情/手术特异性特征。我们的研究结果表明，尽管随着时间的推移，方法越来越复杂，但需要改进的关键领域包括更好地与价值和公平目标保持一致，以及增强数据的可用性，以便对相关风险因素进行更全面的修正。结论：BP计划使用各种方法来解释PRP差异。尽管有向更复杂的方法发展的趋势，但大多数仍然是基本的，有改进的余地。为了实现跨计划的比较和学习，参与bp的涉众必须对所做的设计选择（原因）保持透明。

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引用次数: 0

Does Regulatory and Reimbursement Parallel Processing Provide Swifter Funded Access to Medicines for Patients? Evaluation of Cancer Medicines Using the Australian Parallel Process 监管和报销并行处理是否为患者提供更快的资金获取药物？使用澳大利亚平行程序评估癌症药物。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-16 DOI: 10.1016/j.jval.2024.11.008

Yuan Gao MPH , Mah Laka PhD , Tracy Merlin PhD

Objectives

To assess the impact of a parallel regulatory and reimbursement process on (1) direction of funding decisions, (2) time lag until funding recommendation, and (3) type of evidence submitted, for cancer medicines.

Methods

Public regulatory and reimbursement decision documents were reviewed for cancer medicines considered by the Pharmaceutical Benefits Advisory Committee since the introduction of parallel processing. Medicine-indication pairs were identified from these documents and data extracted on the type and quality of evidence submitted, funding decisions, and timelines, by type of review process. Associations were explored using univariate and multivariable logistic regression analysis.

Results

A total of 182 cases were selected from the 1590 screened. Compared with sequential evaluation, a higher proportion of parallel review submissions presented a cost-effectiveness/cost-utility analysis (67.0% vs 79.7% P < .01) and used the same pivotal evidence as used for market authorization (77.5% vs 90.0%, P = .08). There was no difference in the quality of supporting evidence and other decision-relevant predictors used for either process. Submissions undergoing parallel review were not more likely to receive a negative public funding decision (adjusted odds ratio 0.46; 95% CI 0.14–1.39; P = .17). Under parallel processing, the lag between drug registration and Pharmaceutical Benefits Advisory Committee funding recommendation was drastically reduced (67 weeks) when compared with the sequential process.

Conclusions

The type of evidence supplied to regulators and health technology assessment bodies and the funding decisions that ensue have not been affected by the introduction of parallel processing in Australia. The parallel process has, however, drastically reduced the time taken to the initial funding decision.

目的：评估平行监管和报销流程对(1)资助决策方向、(2)资助建议前的时间滞后以及(3)癌症药物提交证据类型的影响。方法：对药品福利咨询委员会（PBAC）自引入并行处理以来审议的癌症药物的公共监管和报销决定文件进行审查。从这些文件和按审查过程类型提取的关于提交证据的类型和质量、供资决定和时间表的数据中确定了药物适应证对。使用单变量和多变量逻辑回归分析探讨关联。结果：从1590例中筛选出182例。与顺序评估相比，更高比例的平行审查提交了成本-效果/成本-效用分析（67.0% vs 79.7% p < 0.01），并使用了与市场批准相同的关键证据（77.5% vs 90.0%, p = 0.08）。支持证据的质量和用于两种过程的其他决策相关预测指标没有差异。接受平行审查的提交不太可能获得负面的公共资金决定(ORadj =0.46, 95%CI 0.14, 1.39；P = 0.17)。在并行处理下，与顺序处理相比，药物注册和PBAC资助建议之间的滞后时间大大缩短（67周）。结论：向监管机构和HTA机构提供的证据类型，以及随后的资助决定，并没有受到澳大利亚引入并行处理的影响。但是，并行进程大大缩短了作出初步供资决定所需的时间。

{"title":"Does Regulatory and Reimbursement Parallel Processing Provide Swifter Funded Access to Medicines for Patients? Evaluation of Cancer Medicines Using the Australian Parallel Process","authors":"Yuan Gao MPH , Mah Laka PhD , Tracy Merlin PhD","doi":"10.1016/j.jval.2024.11.008","DOIUrl":"10.1016/j.jval.2024.11.008","url":null,"abstract":"<div><h3>Objectives</h3><div>To assess the impact of a parallel regulatory and reimbursement process on (1) direction of funding decisions, (2) time lag until funding recommendation, and (3) type of evidence submitted, for cancer medicines.</div></div><div><h3>Methods</h3><div>Public regulatory and reimbursement decision documents were reviewed for cancer medicines considered by the Pharmaceutical Benefits Advisory Committee since the introduction of parallel processing. Medicine-indication pairs were identified from these documents and data extracted on the type and quality of evidence submitted, funding decisions, and timelines, by type of review process. Associations were explored using univariate and multivariable logistic regression analysis.</div></div><div><h3>Results</h3><div>A total of 182 cases were selected from the 1590 screened. Compared with sequential evaluation, a higher proportion of parallel review submissions presented a cost-effectiveness/cost-utility analysis (67.0% vs 79.7% <em>P</em> < .01) and used the same pivotal evidence as used for market authorization (77.5% vs 90.0%, <em>P</em> = .08). There was no difference in the quality of supporting evidence and other decision-relevant predictors used for either process. Submissions undergoing parallel review were not more likely to receive a negative public funding decision (adjusted odds ratio 0.46; 95% CI 0.14–1.39; <em>P</em> = .17). Under parallel processing, the lag between drug registration and Pharmaceutical Benefits Advisory Committee funding recommendation was drastically reduced (67 weeks) when compared with the sequential process.</div></div><div><h3>Conclusions</h3><div>The type of evidence supplied to regulators and health technology assessment bodies and the funding decisions that ensue have not been affected by the introduction of parallel processing in Australia. The parallel process has, however, drastically reduced the time taken to the initial funding decision.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 3","pages":"Pages 431-440"},"PeriodicalIF":4.9,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A Causal Model for Primary Prevention of Cardiovascular Disease: The Health Economic Model for the Primary Prevention of Cardiovascular Disease 心血管疾病一级预防因果模型：心血管疾病一级预防卫生经济模型（HEM-PPCVD）。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-01 DOI: 10.1016/j.jval.2024.07.010

Jedidiah I. Morton PhD , Danny Liew PhD , Zanfina Ademi PhD

Objectives

Our objective was to design and develop an open-source model capable of simulating interventions for primary prevention of cardiovascular disease (CVD) that incorporated the cumulative effects of risk factors (eg, cholesterol years or blood-pressure years) to enhance health economic modeling in settings which clinical trials are not possible.

Methods

We reviewed the literature to design the model structure by selecting the most important causal risk factors for CVD—low-density lipoprotein-cholesterol (LDL-C), systolic blood pressure (SBP), smoking, diabetes, and lipoprotein (a) (Lp(a))—and most common CVDs—myocardial infarction and stroke. The epidemiological basis of the model involves the simulation of risk factor trajectories, which are used to modify CVD risk via causal effect estimates derived from Mendelian randomization. LDL-C, SBP, Lp(a), and smoking all have cumulative impacts on CVD risk, which were incorporated into the health economic model. The data for the model were primarily sourced from the UK Biobank study. We calibrated the model using clinical trial data and validated the model against the observed UK Biobank data. Finally, we performed an example health economic analysis to demonstrate the utility of the model. The model is open source.

Results

The model performed well in all validation tests. It was able to produce interpretable and plausible (consistent with expectations of the existing literature) results from an example health economic analysis.

Conclusions

We have constructed an open-source health economic model capable of incorporating the cumulative effect of LDL-C (ie, cholesterol years), SBP (SBP-years), Lp(a), and smoking on lifetime CVD risk.

目标：我们的目标是设计并开发一个开源模型，该模型能够模拟心血管疾病（CVD）一级预防的干预措施，其中包含风险因素（如胆固醇年或血压年）的累积效应，从而在无法进行临床试验的情况下加强卫生经济建模：我们查阅了相关文献，选择了心血管疾病最重要的致病风险因素--低密度脂蛋白胆固醇（LDL-C）、收缩压（SBP）、吸烟、糖尿病和脂蛋白（a）（Lp（a）），以及最常见的心血管疾病--心肌梗死和中风，从而设计了模型结构。该模型的流行病学基础包括模拟风险因素轨迹，通过孟德尔随机化得出的因果效应估计值来改变心血管疾病风险。低密度脂蛋白胆固醇（LDL-C）、血压（SBP）、脂蛋白（a）和吸烟都会对心血管疾病风险产生累积影响，这些影响都被纳入了健康经济模型。模型的数据主要来自英国生物库研究。我们使用临床试验数据对模型进行了校准，并根据观察到的英国生物库数据对模型进行了验证。最后，我们进行了一次健康经济分析，以展示该模型的实用性。该模型开源：该模型在所有验证测试中均表现良好。结果：该模型在所有验证测试中均表现良好，能够从一个健康经济分析示例中得出可解释且合理的结果（符合现有文献的预期）：我们构建了一个开源健康经济模型，该模型能够纳入低密度脂蛋白胆固醇（LDL-C）（即胆固醇-年）、血压（SBP-年）、脂蛋白（a）和吸烟对终生心血管疾病风险的累积效应。

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引用次数: 0

Cost-Effectiveness Analysis of Bevacizumab Biosimilars Versus Originator Bevacizumab for Metastatic Colorectal Cancer: A Comparative Study Using Real-World Data 贝伐珠单抗生物仿制药与原研贝伐珠单抗治疗转移性结直肠癌的成本效益分析：一项使用真实世界数据的比较研究。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-01 DOI: 10.1016/j.jval.2024.07.018

Brandon Lu BSc , Erind Dvorani MSc , Lena Nguyen MSc , Jaclyn M. Beca MSc , Rebecca E. Mercer PhD , Andrea Adamic BA , Caroline Muñoz MSc , Kelvin K.W. Chan MD, MSc, PhD

Objectives

MVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC.

Methods

We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer’s perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis.

Results

The matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of −$6379 (95%CI: −9417, −3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: −0.02, 0.02) LY and −0.01 (95% CI: −0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses.

Conclusion

Bevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.

目的：MVASI（安进公司）和Zirabev（辉瑞公司）是最早获准用于转移性结直肠癌（mCRC）一线治疗的两种贝伐珠单抗生物仿制药。我们的目的是确认和量化 MVASI 和 Zirabev 相对于原研贝伐珠单抗治疗 mCRC 患者的实际成本节约和成本效益：我们在加拿大安大略省开展了一项基于人群的回顾性队列研究，该省的原研药和生物仿制药贝伐珠单抗均由政府资助。对 2008 年 1 月至 2019 年 8 月期间接受原研贝伐珠单抗治疗或 2019 年 8 月至 2021 年 3 月期间接受生物类似物贝伐珠单抗治疗的所有 mCRC 患者进行倾向评分匹配（1:4），以调整基线差异。从公共卫生支付方的角度计算了1年患者层面的总成本（CAD）和效果（生命年（LY）和质量调整生命年（QALY））。主要结果包括增量净货币效益（INMB）和增量净健康效益（INHB）。敏感性分析包括按生物类似药类型（MVASI/Zirabev）进行的亚组分析和两年分析：匹配队列包括 747 个生物类似药病例和 2,945 个比较者。贝伐珠单抗生物仿制药的增量成本为-6,379美元（95%CI：-9,417, -3,537）（即节省成本），增量效果为0.0（95%CI：-0.02, 0.02）LY和-0.01（95%CI：-0.03, 0）QALY。在 50,000 美元/LYG 的支付意愿阈值下，INMB 和 INHB 的估计值分别为 6,331 美元（95% CI：6,245, 6,417）和 0.127 LY（95% CI：0.125, 0.128），所有估计值均表明生物类似药贝伐珠单抗具有成本效益。在生物类似药品牌亚组和2年敏感性分析中，成本效益保持一致：贝伐珠单抗生物仿制药在提供与原研药贝伐珠单抗相似的生存获益的同时，还显示出了实际的成本节约效果，证实了最初对其实施的预期，并支持了医疗系统的可持续性。

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引用次数: 0

Practicality, Validity, and Responsiveness of Using the Proxy Version of the Child Health Utility–9 Dimensions With Children Aged 2 to 5 Years 对 2-5 岁儿童使用 CHU-9D 代理版本的实用性、有效性和响应性。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-01 DOI: 10.1016/j.jval.2024.08.010

Tracey H. Sach PhD , Hywel C. Williams DSc

Objectives

This study aimed to assess the practicality, validity, and responsiveness of the proxy Child Health Utility–9 Dimensions (CHU9D) in children aged 2 to 5 years.

Methods

We used data from the Barrier Enhancement for Eczema Prevention trial, a UK randomized controlled trial testing whether daily emollients in infancy could prevent eczema in high-risk infants. The main parent/carer completed the proxy CHU9D using developers’ additional guidance for completion in those younger than 5 years and the Patient-Oriented Eczema Measure (POEM) at ages 2, 3, 4, and 5 years. Practicality was assessed by completion rates. Construct validity assessed whether CHU9D could discriminate between those with/without eczema and between eczema severity levels on POEM. Responsiveness was determined by ability to discriminate between 3 groups: (1) those whose POEM score deteriorated ≥3 points, (2) those whose change was not clinically important (−2.9 to 2.9 points), and (3) those whose POEM score improved ≥3 points. Analysis was conducted in Stata 17.

Results

Of 1394 children participating in the Barrier Enhancement for Eczema Prevention trial, study questionnaires were completed by 1212 (87%), 981 (70%), 990 (71%), and 976 (70%) at 2, 3, 4, and 5 years. Of these the CHU9D was completed by 1066 (88.0%), 685 (69.8%), 925 (93.4%), and 923 (94.6%), respectively. Mean utility at all time points was approximately 0.934 (range 0.443-1). For construct validity, very small differences in the CHU9D between known groups were observed (P < .01). A total of 801 participants had responsiveness data: 13% deteriorated, 72% had nonclinically important change, and 15% improved. Mean utility change (standardized response mean) for these groups was −0.0198 (0.21), 0.0041 (0.05), and 0.0175 (0.21) showing small change and small responsiveness.

Conclusions

Proxy CHU9D in 2- to 5-year-old children shows potential but further research is needed.

目的：评估代用 CHU-9D 在 2-5 岁儿童中的实用性、有效性和响应性：在 2-5 岁儿童中评估代理 CHU-9D 的实用性、有效性和响应性：我们使用了英国随机对照试验 BEEP 的数据，该试验测试了婴儿期每日使用润肤剂能否预防高风险婴儿湿疹。主要家长/照护者使用开发人员为 5 岁以下儿童提供的附加指导填写了代用 CHU-9D，并在 2、3、4 和 5 岁时填写了以患者为中心的湿疹测量 (POEM)。根据完成率评估实用性。结构效度评估 CHU-9D 是否能区分湿疹患者/非湿疹患者以及 POEM 中的湿疹严重程度。响应性通过区分以下三组的能力来确定：POEM 评分 i) 恶化≥3 分；ii) 无临床意义的变化（-2.9 至 2.9 分）；iii) 改善≥3 分。分析采用 STATA 17.Results：在参与 BEEP 的 1394 名儿童中，1212 名（87%）、981 名（70%）、990 名（71%）和 976 名（70%）分别在 2、3、4 和 5 年时完成了研究问卷。其中，分别有 1,066 人（88.0%）、685 人（69.8%）、925 人（93.4%）和 923 人（94.6%）完成了 CHU-9D。所有时间点的平均效用值约为 0.934（范围为 0.443-1）。在构建效度方面，已知组别之间的 CHU-9D 差异非常小（P 结论：CHU-9D 的构建效度为 0.934（范围 0.443-1））：2-5岁儿童的CHU-9D替代值显示出潜力，但仍需进一步研究。

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引用次数: 0

Cost-Effectiveness of an Extended-Role General Practitioner Clinic for Persistent Physical Symptoms: Results From the Multiple Symptoms Study 3 Pragmatic Randomized Controlled Trial 针对顽固性身体症状的全科医生扩展角色诊所的成本效益：多重症状研究 3 (MSS3) 实用随机对照试验的结果。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-01 DOI: 10.1016/j.jval.2024.09.015

Aileen R. Neilson MSc , Cara Mooney MSc , Laura Sutton PhD , David White MPH , Jeremy Dawson PhD , Gillian Rowlands MD , Ruth E. Thomas PhD , Jonathan Woodward MSc , Vincent Deary PhD , Christopher Burton PhD

Objectives

This study aimed to evaluate the cost-effectiveness of an extended-role general practitioner symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as medically unexplained symptoms).

Methods

This was a 52-week within-trial cost-utility analysis of a pragmatic multicenter randomized controlled trial comparing SC + UC (n = 178) with UC alone (n = 176), conducted from the primary perspective of the UK National Health Service and personal and social services (PSS). Base-case quality-adjusted life-years (QALYs) were measured using EQ-5D-5L. Missing data were imputed using multiple imputation. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net monetary benefits. Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 nonparametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and ICECAP-A capability measure for adults outcomes to estimate QALYs and years of full capability, respectively, varying intervention costs, missing data mechanism assumptions).

Results

Multiple imputation analysis showed that compared with UC alone, SC + UC was more expensive (adjusted mean cost difference: 704; 95% CI £605-£807) and more effective (adjusted mean QALY difference: 0.0447; 95% CI 0.0067-0.0826), yielding an incremental cost-effectiveness ratio of £15 765/QALY, incremental net monetary benefit of £189.22 (95% CI −£573.62 to £948.28) and a 69% probability of the SC + UC intervention arm being cost-effective at a threshold of £20 000 per QALY. Results were robust to most sensitivity analyses but sensitive to missing data assumptions (2 of the 8 scenarios investigated), SF-6D, and ICECAP_A capability measure for adults quality-of-life outcomes.

Conclusions

A symptoms clinic is likely to be a potentially cost-effective treatment for patients with persistent physical symptoms.

目的评估在常规护理（UC）的基础上增加全科医生（GP）症状门诊（SC）的成本效益，以治疗具有多种持续性身体症状（有时称为 "医学上无法解释的症状"）的患者：方法：从英国国家医疗服务系统（NHS）和个人与社会服务系统（PSS）的角度出发，对一项实用性多中心随机对照试验进行了为期 52 周的试验成本效用分析，该试验比较了症状门诊+UC（178 人）与单纯 UC（176 人）。基础病例质量调整生命年（QALYs）采用 EQ-5D-5L 进行测量。缺失数据采用多重估算法（MI）进行估算。成本效益结果以增量成本效益比（ICER）和增量净货币效益（INMB）表示。使用成本效益可接受性曲线（使用 1000 个非参数引导样本）和敏感性分析（包括社会成本，使用 SF-6D 和能力 ICECAP-A 结果分别估算 QALYs 和完全能力年数 (YFC)，改变干预成本、缺失数据机制假设）对不确定性进行了探讨：多重估算分析表明，与单独使用 UC 相比，SC+UC 更昂贵[（调整后的平均成本差异：704；95% CI：605 英镑，807 英镑）]，更有效[（调整后的平均 QALY 差异：0.0447（95% CI：0.0067，0.0826）]，ICER 为 15,765 英镑/QALY，INMB 为 189.22 英镑（95% CI：-573.62 英镑，948.28 英镑），在每 QALY 20000 英镑的阈值下，SC+UC 干预组具有成本效益的概率为 69%。结果对大多数敏感性分析都是稳健的，但对缺失数据假设（调查的 8 个方案中的 2 个）、SF-6D 和 ICECAP-A 生活质量结果敏感：症状门诊对于有持续性躯体症状的患者来说可能是一种具有潜在成本效益的治疗方法。

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引用次数: 0

CO9 Systematic Literature Review (SLR) of Disease Burden Related to First-Line (1L) Unresectable, Locally Advanced, or Metastatic Esophageal Squamous Cell Carcinoma (ESCC)

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-12-01 DOI: 10.1016/j.jval.2024.10.086

E Smyth , A Sibiga , AN Nehdi , A Krishna , K Kim , J Coaquira Castro , E Priedane , L Zhan

引用次数: 0