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Recommendations on the Use of Structured Expert Elicitation Protocols for Healthcare Decision Making: A Good Practices Report of an ISPOR Task Force 关于在医疗决策中使用结构化专家征询协议的建议：ISPOR 工作组的良好实践报告

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.027

Marta Soares PhD , Abigail Colson PhD , Laura Bojke PhD , Salah Ghabri PhD , Osvaldo Ulises Garay MSc , Jenna K. Felli PhD , Karen Lee MA , Elizabeth Molsen-David RN , Oswaldo Morales-Napoles PhD , Victoria A. Shaffer PhD , Maarten J. IJzerman PhD

Healthcare decision making, including regulatory and reimbursement decisions, is based on uncertain assessments of clinical and economic value. This arises from the evidence supporting those assessments being uncertain, incomplete, or even absent. Qualitative, structured expert elicitation (SEE) is a valuable tool for extracting expert knowledge about an uncertain quantity and formulating that knowledge as a probability distribution. This creates a useful input to decision modeling and support, particularly in areas with limited evidence, such as advanced therapy products, precision medicine, rare diagnoses, and other areas with high uncertainty.

Structured SEE protocols are used to improve the transparency, accuracy, and consistency of quantitative judgments from experts, limiting the effect of heuristics and biases. This task force report introduces 5 commonly used protocols for SEE (Sheffield elicitation framework; modified Delphi method; Cooke’s classical method; investigate, discuss, estimate, aggregate protocol; and the Medical Research Council reference protocol). It describes the common elements of SEE, discusses how these protocols differ in their implementation of those elements and illustrates the use of the protocols.

The report then reviews the relevant constraints on implementing SEE within the context of healthcare decision making and considers the strengths and weaknesses of these protocols in light of those considerations. Because this is an introductory report on an emerging topic, specific recommendations on practice are not made. However, there are broad recommendations based on the suitability of the different protocols in various decision contexts. The report concludes with recommendations for further research to better guide future practice.

医疗决策，包括监管和报销决策，都是基于对临床和经济价值的不确定评估。这是因为支持这些评估的证据不确定、不完整甚至不存在。定性、结构化的专家征询（SEE）是一种宝贵的工具，可用于提取有关不确定数量的专家知识，并将这些知识形成概率分布。结构化专家征询协议用于提高专家定量判断的透明度、准确性和一致性，限制启发式方法和偏见的影响。本特别工作组报告介绍了 5 种常用的 SEE 协议（谢菲尔德诱导框架；改良德尔菲法；库克经典法；调查、讨论、估计、汇总协议；以及医学研究委员会参考协议）。报告描述了SEE的共同要素，讨论了这些方案在实施这些要素时的不同之处，并说明了这些方案的使用方法。报告随后回顾了在医疗决策背景下实施SEE的相关限制因素，并根据这些因素考虑了这些方案的优缺点。由于这是一份关于新兴课题的介绍性报告，因此没有提出具体的实践建议。不过，报告根据不同方案在不同决策环境中的适用性提出了广泛的建议。报告最后提出了进一步研究的建议，以更好地指导未来的实践。

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引用次数: 0

EQ-5D-5L Population Scores in Mainland China: Results From a Nationally Representative Survey 2021 中国大陆 EQ-5D-5L 人口得分：2021 年全国代表性调查的结果。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.06.012

Objectives

There is a lack of monitoring changes in the population scores of the most recent version, EQ-5D-5L, in mainland China. This study aimed to address this knowledge gap by assessing the EQ-5D-5L scores in mainland China using a nationally representative sample.

Methods

Data were extracted from the 2021 Survey of Health Index of Chinese Families, which covered 31 provinces/autonomous regions/municipalities in mainland China. The survey used a multistage quota sampling strategy encompassing 120 prefecture-level cities. Quotas were allocated to each prefecture-level city in accordance with the 2020 China Population Census. This approach resulted in a final sample of 11 030 eligible questionnaires. The utility index (UI) and EuroQol Visual Analog Scale (EQ VAS) scores were reported for the entire sample (age-gender-urban/rural weighted) and by the characteristics of the study participants.

Results

The study participants had a weighted mean UI of 0.939 (SD 0.135) and EQ VAS score of 80.19 (SD 18.39). The most commonly reported problem was anxiety/depression (26.37%), whereas self-care was the least reported problem (6.18%). Those who were male, were younger, lived without chronic conditions and disabilities, had higher levels of education, earned higher monthly household income, and were covered by basic medical insurance for urban employees had higher scores in both the UI and EQ VAS.

Conclusion

This study revealed slightly lower UI scores despite a much higher drop in EQ VAS scores whereas China maintained minimum cases of COVID-19 in 2021 compared with the population norms recorded in 2019. Further studies are warranted to unveil the full impacts of COVID-19 outbreaks.

目的：中国大陆缺乏对最新版 EQ-5D-5L 人口评分变化的监测。本研究旨在利用具有全国代表性的样本，评估中国大陆的 EQ-5D-5L 评分，以填补这一知识空白：数据来自 2021 年中国家庭健康指数调查，该调查覆盖中国大陆 31 个省/自治区/直辖市。调查采用多阶段配额抽样策略，涵盖 120 个地级市。每个地级市的配额根据 2020 年中国人口普查结果进行分配。通过这种方法，最终获得了 11,030 份符合条件的问卷样本。报告了整个样本的效用指数和 EQ 视觉模拟量表（VAS）得分（年龄-性别-城乡加权）以及研究参与者的特征：研究参与者的加权平均效用指数为 0.939（SD=0.135），EQ VAS 得分为 80.19（SD=18.39）。最常报告的问题是焦虑/抑郁（26.37%），而自我护理是最少报告的问题（6.18%）。男性、年轻、无慢性病和残疾、受教育程度较高、家庭月收入较高、参加城镇职工基本医疗保险的人在效用指数和 EQ VAS 方面的得分都较高：本研究表明，与 2019 年记录的人口标准相比，尽管 EQ VAS 分数的下降幅度更大，但 2021 年中国 COVID-19 的病例数仍保持在最低水平。要揭示 COVID-19 爆发的全部影响，还需要进一步研究。

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引用次数: 0

Attribute Development in Health-Related Discrete Choice Experiments: A Systematic Review of Qualitative Methods and Techniques to Inform Quantitative Instruments 健康相关离散选择实验中的属性开发：定性方法和技术的系统回顾，为定量工具提供参考。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.05.014

Objectives

This review sought to identify the qualitative methods and techniques that researchers have used in the past decade to develop attributes and inform health-related discrete choice experiments (DCEs) surveys from a patient perspective.

Methods

The review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines for reporting systematic reviews. An adapted appraisal tool following guidelines for reporting qualitative research for quantitative instruments and criteria for attribute development in DCEs was applied for quality assessment and data extraction. A narrative approach was used to synthesize data. This examination included consideration of issues pertaining to sampling, data collection, data analysis, attribute list reduction, wording, methodological adaptations to capture patient preferences, and testing the pre-experimental design decisions of the DCE survey.

Results

Of 8505 articles identified for abstract screening, 680 were included for full-text screening, 36 of which met the inclusion criteria. Practices to improve methodological robustness included pre-data collection materials to inform instruments, data collection methods specific for decision-making scenarios, purposeful selection of data analysis methods to address the research question, and participants’ involvement in reducing the list of attributes. Examples of methodological adaptations for patients were noted.

Conclusions

DCEs have the potential to become a mixed-method approach in which the qualitative phase informs a reduced list of attributes for a survey, serves the predesign decisions of the experiment by testing trade-offs, overlapping, understandability, face, and content validity and provides explanations of the quantitative results. Establishing guidelines for using qualitative methods for DCE attribute development may help to broadly enhance the methodological robustness of DCEs.

目的：本综述旨在确定研究人员在过去十年中采用的定性方法和技术：本综述旨在确定研究人员在过去十年中采用的定性方法和技术，以便从患者的角度开发与健康相关的离散选择实验 (DCE) 调查的属性和信息：综述遵循 PRISMA 系统综述报告指南。根据定量工具定性研究报告指南和 DCEs 属性开发标准改编的评估工具被用于质量评估和数据提取。采用叙事方法对数据进行综合。审查包括考虑与抽样、数据收集、数据分析、属性列表缩减、措辞、捕捉患者偏好的方法调整以及测试 DCE 调查的前期实验设计决策有关的问题：在经过摘要筛选的 8,505 篇文章中，有 680 篇被纳入全文筛选，其中 36 篇符合纳入标准。提高方法稳健性的做法包括：为工具提供信息的前期数据收集材料、针对决策情景的特定数据收集方法、有目的地选择数据分析方法以解决研究问题，以及让参与者参与减少属性列表。此外，还提到了为患者调整方法的例子：DCE有可能成为一种混合方法，在这种方法中，定性阶段为减少调查的属性清单提供信息，通过测试权衡、重叠、可理解性、面效和内容效度为实验的前期设计决策服务，并为定量结果提供解释。为使用定性方法开发 DCE 属性制定指导原则可能有助于广泛提高 DCE 方法的稳健性。

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引用次数: 0

Development of a Prioritization Framework to Aid Healthcare Funding Decision Making in Health Technology Assessment in Australia: Application of Multicriteria Decision Analysis 在澳大利亚卫生技术评估 (HTA) 中制定优先次序框架以协助医疗保健资金决策：多标准决策分析的应用。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.003

Saval Khanal PhD , Son Nghiem PhD , Mel Miller PhD , Paul Scuffham PhD , Joshua Byrnes PhD

Objectives

This study develops a prioritization framework to aid healthcare funding decision making in health technology assessment (HTA) in Australia using a multiple criteria decision analysis (MCDA) approach.

Methods

MCDA frameworks for HTAs were reviewed through literature survey to identify the initial criteria and levels within each criterion. Key stakeholders and experts were consulted to confirm these criteria and levels. A conjoint analysis using 1000Minds was undertaken with policy makers from the Department of Health to establish ranking criteria and weighting scores. Monte Carlo simulations were used to examine the sensitivity of findings to factors affecting the ranking and weighting scores. The MCDA was then applied to 6 examples of chronic care models or technologies projects to demonstrate the performance of this approach.

Results

Five criteria (clinical efficacy/effectiveness, safety and tolerability, severity of the condition, quality/uncertainty, and direct impact on healthcare costs) were consistently ranked highest by healthcare decision makers. Among the criteria, patient-level health outcomes were considered the most important, followed by social and ethical values. The analyses were robust to inform the uncertainty in the parameter.

Conclusions

This study has developed an MCDA tool that effectively integrates key priorities for HTA reviews, reflecting the values and preferences of healthcare stakeholders in Australia. Although this tool aims to align the assessment process more closely with health benefits, it also highlights the importance of considering other criteria.

目的：本研究采用多标准决策分析（MCDA）方法，制定了一个优先次序框架，以帮助澳大利亚医疗卫生技术评估（HTA）中的医疗资金决策：本研究采用多标准决策分析（MCDA）方法，制定了一个优先顺序框架，以帮助澳大利亚医疗卫生技术评估（HTA）中的医疗资金决策：方法：通过文献调查回顾了用于 HTA 的 MCDA 框架，以确定初步标准和每项标准中的等级。咨询了主要利益相关者和专家，以确认这些标准和等级。与卫生部的政策制定者一起使用 1000Minds© 进行联合分析，以确定排序标准和加权分数。使用蒙特卡洛模拟来检查结果对影响排序和加权分数的因素的敏感性。然后将 MCDA 应用于六个慢性病护理模式或技术项目实例，以展示这种方法的性能：结果：医疗决策者始终将五项标准（临床疗效/有效性；安全性和耐受性；病情严重程度；质量/不确定性；对医疗成本的直接影响）排在首位。在这些标准中，患者层面的健康结果被认为是最重要的，其次是社会和伦理价值。分析结果对参数的不确定性很有说服力：本研究开发了一种 MCDA 工具，有效整合了 HTA 评审的关键优先事项，反映了澳大利亚医疗保健利益相关者的价值观和偏好。虽然该工具旨在使评估过程更贴近健康效益，但它也强调了考虑其他标准的重要性。

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引用次数: 0

First-Line Treatment With Atezolizumab Plus Bevacizumab and Chemotherapy for US Patients With Metastatic, Persistent, or Recurrent Cervical Cancer: A Cost-Effectiveness Analysis 美国转移性、顽固性或复发性宫颈癌患者接受阿特珠单抗加贝伐单抗和化疗的一线治疗：成本效益分析》。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.013

Jianying Lei MSc , Jiahao Zhang MSc , Caicong You MSc , Maobai Liu MSc , Na Li PhD

Objectives

The BETAcc clinical trial demonstrated that chemotherapy combined with bevacizumab plus atezolizumab (CBA) significantly prolonged progression-free survival and overall survival in patients with metastatic, persistent, or recurrent cervical cancer. However, to the best of our knowledge, the economic value of using this new therapy for this indication is currently unknown. Therefore, our study aimed to evaluate the cost-effectiveness of CBA for the first-line treatment of metastatic, persistent, or recurrent cervical cancer from the United States healthcare payers perspective.

Methods

A state-transition Markov model over a 10-year lifetime horizon was developed to compare the cost and effectiveness of CBA with that of chemotherapy plus bevacizumab (CB). The primary outcomes of our study included costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the results.

Results

CBA was associated with an additional 0.58 QALY at an extra cost of $172 495.90 compared with CB. The incremental cost-effectiveness ratio was $295 972.43/QALY, significantly higher than the willingness-to-pay threshold value of $150 000/QALY. One-way sensitivity analyses revealed that results were most sensitive to the progression-free disease utility, the unit cost of atezolizumab, and progressed disease utility. Probabilistic sensitivity analysis indicated that CBA achieved a 4.3% probability of cost-effectiveness at a $150 000/QALY threshold. To achieve cost-effectiveness, the unit price of atezolizumab must be reduced by approximately 56.6%.

Conclusions

CBA treatment is unlikely to be a cost-effective option compared with CB for patients with persistent, recurrent, or metastatic cervical cancer in the United States.

研究目的BETAcc 临床试验表明，化疗联合贝伐珠单抗加阿替珠单抗（CBA）可明显延长转移性、顽固性或复发性宫颈癌患者的无进展生存期（PFS）和总生存期（OS）。然而，据我们所知，将这种新疗法用于该适应症的经济价值目前尚不清楚。因此，我们的研究旨在从美国医疗支付方的角度评估 CBA 用于转移性、持续性或复发性宫颈癌一线治疗的成本效益：方法：我们建立了一个为期 10 年的状态转换马尔可夫模型，以比较 CBA 与化疗加贝伐单抗（CB）的成本和有效性。研究的主要结果包括成本、质量调整生命年（QALYs）和增量成本效益比（ICERs）。为了评估结果的稳健性，我们进行了单向敏感性分析和概率敏感性分析：与 CB 相比，CBA 额外增加了 0.58 QALY，额外成本为 172495.90 美元。ICER为295,972.43美元/QALY，明显高于支付意愿（WTP）阈值150,000美元/QALY。单向敏感性分析显示，研究结果对PFD效用、阿替佐珠单抗的单位成本和PD效用最为敏感。概率敏感性分析表明，在 150,000 美元/QALY 临界值下，CBA 获得成本效益的概率为 4.3%。要达到成本效益，阿特珠单抗的单价必须降低约56.6%：结论：在美国，与 CB 相比，CBA 治疗对持续性、复发性或转移性宫颈癌患者不太可能具有成本效益。

{"title":"First-Line Treatment With Atezolizumab Plus Bevacizumab and Chemotherapy for US Patients With Metastatic, Persistent, or Recurrent Cervical Cancer: A Cost-Effectiveness Analysis","authors":"Jianying Lei MSc , Jiahao Zhang MSc , Caicong You MSc , Maobai Liu MSc , Na Li PhD","doi":"10.1016/j.jval.2024.07.013","DOIUrl":"10.1016/j.jval.2024.07.013","url":null,"abstract":"<div><h3>Objectives</h3><div>The BETAcc clinical trial demonstrated that chemotherapy combined with bevacizumab plus atezolizumab (CBA) significantly prolonged progression-free survival and overall survival in patients with metastatic, persistent, or recurrent cervical cancer. However, to the best of our knowledge, the economic value of using this new therapy for this indication is currently unknown. Therefore, our study aimed to evaluate the cost-effectiveness of CBA for the first-line treatment of metastatic, persistent, or recurrent cervical cancer from the United States healthcare payers perspective.</div></div><div><h3>Methods</h3><div>A state-transition Markov model over a 10-year lifetime horizon was developed to compare the cost and effectiveness of CBA with that of chemotherapy plus bevacizumab (CB). The primary outcomes of our study included costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the results.</div></div><div><h3>Results</h3><div>CBA was associated with an additional 0.58 QALY at an extra cost of $172 495.90 compared with CB. The incremental cost-effectiveness ratio was $295 972.43/QALY, significantly higher than the willingness-to-pay threshold value of $150 000/QALY. One-way sensitivity analyses revealed that results were most sensitive to the progression-free disease utility, the unit cost of atezolizumab, and progressed disease utility. Probabilistic sensitivity analysis indicated that CBA achieved a 4.3% probability of cost-effectiveness at a $150 000/QALY threshold. To achieve cost-effectiveness, the unit price of atezolizumab must be reduced by approximately 56.6%.</div></div><div><h3>Conclusions</h3><div>CBA treatment is unlikely to be a cost-effective option compared with CB for patients with persistent, recurrent, or metastatic cervical cancer in the United States.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"27 11","pages":"Pages 1528-1534"},"PeriodicalIF":4.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141890237","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Clinical Outcome Assessments Are Never “Validated” 临床结果评估从未经过 "验证"。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.006

Christina K. Zigler PhD, MSEd , Theresa Coles PhD

引用次数: 0

The Cost-Effectiveness of Germline BReast CAncer Gene Testing in Metastatic Prostate Cancer Followed by Cascade Testing of First-Degree Relatives of Mutation Carriers 对转移性前列腺癌患者进行种系 BRCA 检测，然后对突变携带者的一级亲属进行连带检测的成本效益。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.06.019

Objectives

Patients with metastatic prostate cancer (mPCa) with BReast CAncer gene (BRCA) mutations benefit from targeted treatments (eg, olaparib). In addition, family members of affected patients have increased risk of hereditary cancers and benefit from early detection and prevention. International guidelines recommend genetic testing in mPCa; however, the value for money of testing patients with mPCa and cascade testing of blood-related family members has not been assessed. In this context, we evaluated the cost-effectiveness of germline BRCA testing in patients with mPCa followed by cascade testing of first-degree relatives (FDRs) of mutation carriers.

Methods

We conducted a cost-utility analysis of germline BRCA testing using 2 scenarios: (1) testing patients with mPCa only and (2) testing patients with mPCa and FDRs of those who test positive. A semi-Markov multi–health-state transition model was constructed using a lifetime time horizon. The analyses were performed from an Australian payer perspective. Decision uncertainty was characterized using probabilistic analyses.

Results

Compared with no testing, BRCA testing in mPCa was associated with an incremental cost of AU$3731 and a gain of 0.014 quality-adjusted life-years (QALYs), resulting in an incremental cost-effectiveness ratio of AU$265 942/QALY. Extending testing to FDRs of variant-positive patients resulted in an incremental cost-effectiveness ratio of AU$16 392/QALY. Probability of cost-effectiveness at a willingness-to-pay of AU$75 000/QALY was 0% in the standalone mPCa analysis and 100% in the cascade testing analysis.

Conclusion

BRCA testing when performed as a standalone strategy in patients with mPCa may not be cost-effective but demonstrates significant value for money after the inclusion of cascade testing of FDRs of mutation carriers.

目的：BRCA 基因突变的转移性前列腺癌（mPCa）患者可从靶向治疗（如奥拉帕尼）中获益。此外，受影响患者的家庭成员患遗传性癌症的风险也会增加，并从早期检测和预防中获益。国际指南建议对 mPCa 进行基因检测，但尚未评估对 mPCa 患者进行检测以及对血液相关家庭成员进行连带检测的性价比。在这种情况下，我们评估了对 mPCa 患者进行种系 BRCA 检测，然后对突变携带者的一级亲属（FDR）进行级联检测的成本效益：我们采用两种方案对种系 BRCA 检测进行了成本效益分析：1）仅对 mPCa 患者进行检测；2）对 mPCa 患者及检测呈阳性者的一级亲属 (FDR) 进行检测。采用终生时间跨度构建了一个半马尔可夫多健康状态转换模型。分析从澳大利亚支付方的角度进行。利用概率分析确定了决策不确定性的特征：与不进行检测相比，对 mPCa 进行 BRCA 检测的增量成本为 3,731 澳元，质量调整生命年 (QALY) 的收益为 0.014，因此增量成本效益比 (ICER) 为 265,942 澳元/QALY。如果将检测范围扩大到变异阳性患者的 FDRs，则 ICER 为 16,392 澳元/QALY。在独立 mPCa 分析中，在 75,000 澳元/QALY 的支付意愿下，成本效益概率为 0%，而在级联检测分析中，成本效益概率为 100%：结论：对 mPCa 患者单独进行 BRCA 检测可能不具成本效益，但在对突变携带者的 FDR 进行级联检测后，则显示出显著的性价比。

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引用次数: 0

Valuation of Multidimensional Health States With a Bolt-On: Is There a Shortcut? 用 "螺栓 "评估多维健康状态：有捷径吗？

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.06.018

Objectives

To use the EQ-5D questionnaire with bolt-on dimensions in economic evaluation studies, new value sets are needed. In this study, we explored the feasibility of a new approach called the scaling factor model, which estimates bolt-on value sets using estimated EQ-5D dimensional weights.

Methods

We designed a 2-arm study, inviting university students to value health states with and without bolt-on items using the composite time trade-off method. We selected 25 health states from an orthogonal array and added the 5 mildest EQ-5D states in the design. In arm 1, EQ-5D without self-care and standard EQ-5D states were valued, and in arm 2, standard EQ-5D states and EQ-5D with vision were valued. By arm, we compared the mean observed values of health states with and without bolt-on item. Next, by arm, we estimated value sets for the EQ-5D with bolt-on states using both standard model and scaling factor model. Model performances were compared in terms of prediction accuracy and correlation with likelihood-based mean values.

Results

Adding a five-level bolt-on to EQ-5D resulted in statistically lower values. This effect was consistent across 2 arms and bolt-on items. The scaling factor models outperformed the standard models in all statistics.

Conclusions

The scaling factor model offers a methodologically viable and low-cost option for producing value sets for EQ-5D supplemented with bolt-on items. Future studies should further test this method using other bolt-on items and more relevant study populations.

目的：为了在经济评估研究中使用带有附加维度的 EQ-5D 问卷，需要新的价值集。在本研究中，我们探索了一种名为缩放因子模型的新方法的可行性，该方法利用估算的 EQ-5D 维度权重估算附加值集：方法：我们设计了一项双臂研究，邀请大学生使用综合时间权衡（cTTO）法对有无栓塞项目的健康状态进行估值。我们从正交阵列中选取了 25 种健康状态，并在设计中加入了 5 种最轻的 EQ-5D 状态。在第一组中，我们评估了不含自我护理的 EQ-5D 状态和标准 EQ-5D 状态；在第二组中，我们评估了标准 EQ-5D 状态和含视力的 EQ-5D 状态。我们按治疗组比较了有和没有附加项目的健康状况的平均观察值。接下来，我们按研究组使用标准模型和缩放因子模型估算了EQ-5D（含栓塞项目）状态的值集。从预测准确性和与基于似然法的平均值的相关性方面比较了模型的性能：结果：在 EQ-5D 中添加第五级附加项目会导致统计值降低。这种效应在两臂和附加项目中是一致的。缩放因子模型在所有统计数据中均优于标准模型：缩放因子模型为 EQ-5D 值集的生成提供了一种方法上可行且成本低廉的选择，并辅以附加项目。未来的研究应使用其他附加项目和相关研究人群进一步测试这种方法。

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引用次数: 0

Key Issues of Economic Evaluations for Health Technology Assessment in China: A Nationwide Expert Survey 中国卫生技术评估经济评价的关键问题：全国专家调查。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.06.020

Objectives

Health technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations.

Methods

A nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants’ responses and demographics was examined using appropriate logistic models.

Results

A total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times gross domestic product (52.1% agreement); elevated thresholds for childhood diseases, rare diseases, end-of-life diseases, and first-in-class medicines (>78.0% agreement); a single pricing model for multi-indication medicines (60.4% agreement); consideration of multiple medicines as comparators (79.9% agreement); and avoiding the use of centrally procured medicines as comparators for medicines with a time to market of less than 3 years (71.0% agreement). Participants who are service provider had lower odds of selecting higher thresholds (odds ratio 0.26; P < .01) than responders from consultancy/contract research organization/industry.

Conclusions

Expert views indicate the need for substantial changes in China’s current HTA methods, highlighting the need for increased investment in HTA processes and expertise cultivation.

目的：卫生技术评估（HTA）在中国的药品价格谈判中越来越重要，但之前的评估显示，在关键的经济评估问题上存在全国性的差异：支付意愿（WTP）阈值、多适应症药品的定价模型和参照物选择原则。本研究旨在为未来的 HTA 评估收集专家对这些问题的意见：方法：在全国范围内进行匿名网络调查，调查对象包括学术界、HTA、咨询公司/合同研究组织 (CRO)/行业、服务提供商和支付方的专家。2023 年，利用便利抽样和滚雪球抽样法，通过微信发布了包含问卷网络链接的通用邀请函。采用描述性统计分析了问卷意见的一致率。使用适当的逻辑模型研究了参与者的回答与人口统计学之间的关系：结果：共收到来自 34 个城市的 303 份专家回复。专家的主要观点包括：建议的基本 WTP 临界值为 GDP 的 0.5 至 1.5 倍（52.1% 同意）；儿童疾病、罕见病、临终疾病和一流药品（FIC）的临界值提高（>78.0% 同意）；多适应症药品采用单一定价模式（60.4% 同意）；考虑将多种药品作为参照物（79.9% 同意）；上市时间在三年以下的药品避免使用集中采购药品作为参照物（71.0% 同意）。作为服务提供者的参与者选择较高阈值的几率较低（OR：0.26；PC 结论：专家观点表明，中国目前的 HTA 方法需要进行重大变革，并强调需要增加对 HTA 流程和专业知识培养的投资。

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引用次数: 0

When Are You Recovered? Defining Cutoff Points Using the Acute Respiratory Tract Infection Questionnaire 何时痊愈？使用急性呼吸道感染问卷确定分界点。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.002

Eskild Johansen MD , Volkert Siersma PhD , Malene P. Hansen MD , Rune M. Aabenhus MD

Objectives

Acute respiratory tract infections (ARTIs) are a heterogenous group of diseases. Often, it is difficult to obtain a precise diagnosis in general practice but also difficult to determine when the patient is recovered. The lack of a precise definition of recovery after ARTI complicates scientific research aiming to optimize diagnostics and compare treatments. The study aimed to define cutoff points to determine the end of an ARTI as a proxy for recovery in patients diagnosed with ARTI in general practice using a validated patient-reported outcome instrument; The ARTI Questionnaire (ARTIQ).

Methods

A total of 259 participants was divided in 2 groups—1 with ARTI and 1 without. Histograms and area under the curve were calculated for each of the 5 dimensions within the ARTIQ to evaluate the discriminative effect. For the most discriminative dimensions receiver operating comparison curves were performed to determine relevant cutoff points for having or not having ARTI symptoms and serve as a proxy for recovery in clinical research.

Results

The highest discriminative effect was found in 2 dimensions: “physical—upper airways” and “physical—lower airways.” When combining these dimensions, the area under the curve was 0.97. Sensitivity, specificity, and predictive values were calculated for selected cutoff points.

Conclusion

Cutoff points serving as proxy for recovery from ARTI using a patient-reported outcome were identified. The specific cutoff point for a certain research project must be selected considering the specific clinical situation of interest.

目的：急性呼吸道感染（ARTIs）是一类异质性疾病。通常情况下，在全科诊疗中很难获得准确诊断，也很难确定患者何时痊愈。由于缺乏对 ARTI 后康复的准确定义，使得旨在优化诊断和比较治疗方法的科学研究变得更加复杂。本研究旨在使用经过验证的患者报告结果（PRO）工具--急性呼吸道感染问卷（ARTIQ）--确定分界点，以确定 ARTI 的结束时间，作为在全科诊疗中被诊断为 ARTI 患者的康复时间：共有 259 名参与者被分为两组--一组患有 ARTI，另一组未患 ARTI。对 ARTIQ 的五个维度分别计算直方图和曲线下面积 (AUC)，以评估鉴别效果。对最具鉴别力的维度绘制了接收操作比较曲线（ROC），以确定有无 ARTI 症状的相关临界点，并在临床研究中作为康复的替代指标：结果：发现两个维度的判别效果最高：结果：在 "体征--上呼吸道 "和 "体征--下呼吸道 "这两个维度上发现了最高的分辨效果。将这两个维度结合起来，AUC 为 0.97。计算了选定截断点的敏感性、特异性和预测值：结论：利用 PRO 确定了作为 ARTI 康复替代指标的临界点。结论：利用 PRO 确定了作为 ARTI 康复替代指标的临界点。必须根据特定的临床情况为某个研究项目选择特定的临界点。

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