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Economic Evaluations of Interventions Addressing Inequalities in Cancer Care: A Systematic Review 针对癌症治疗中不平等现象的干预措施的经济评估:系统回顾。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.09.010
Bedasa Taye Merga MPH , Nikki McCaffrey PhD , Suzanne Robinson PhD , Ebisa Turi MPH , Anita Lal PhD

Objectives

Although substantial evidence exists on the costs and benefits of cancer care and screening programs for the general population, economic evidence of interventions addressing inequalities is less well known. This systematic review summarized economic evaluations of interventions addressing inequalities in cancer screening and care to inform decision makers on the value for money of such interventions.

Methods

Embase, MEDLINE, Cochrane Library, EconLit, and Scopus databases were searched for studies published from database inception to October 27, 2023. Studies were eligible for inclusion if they were economic evaluations of interventions to improve or address inequalities in cancer care among disadvantaged population groups. Study characteristics and cost-effectiveness results (US dollars 2023) were summarized. Study quality was assessed by 2 authors using the Drummond checklist.

Results

The searches yielded 2937 records, with 30 meeting the eligibility criteria for data extraction. In most of the studies (n = 27, 90%), interventions were considered cost-effective in addressing inequalities in cancer care and screening among disadvantaged populations. Notably, 60% of the studies were rated as high quality, 33.3% as good, and 6.7% as fair quality.

Conclusions

This systematic review identified cost-effective strategies addressing inequalities in cancer screening and care that have the potential to be replicated in other locations. The interventions were mainly focused on screening programs, and few addressed equity gaps around risk reduction and diagnostic and treatment outcomes. This underscores the need for targeted approaches to address inequalities in under-researched priority population groups along the cancer care continuum.
目的:尽管已有大量证据表明癌症护理和筛查项目对普通人群的成本和效益,但解决不平等问题的干预措施的经济证据却鲜为人知。本系统性综述总结了针对癌症筛查和护理中不平等现象的干预措施的经济评价,以告知决策者此类干预措施的资金价值:方法:在 Embase、Medline、Cochrane Library、EconLit 和 Scopus 数据库中检索了自数据库建立至 2023 年 10 月 27 日期间发表的研究。如果研究是对改善或解决弱势群体癌症护理不平等问题的干预措施进行经济评估,则符合纳入条件。对研究特点和成本效益结果(2023 美元)进行总结。研究质量由两位作者使用 Drummond 检查表进行评估:搜索共获得 2,937 条记录,其中 30 条符合数据提取的资格标准。在大多数研究(27 项,占 90%)中,干预措施被认为在解决弱势人群癌症治疗和筛查中的不平等方面具有成本效益。60%的研究被评为高质量,33.3%被评为良好,6.7%被评为一般质量:本系统综述确定了解决癌症筛查和护理不平等问题的具有成本效益的策略,这些策略有可能在其他地方推广。干预措施主要集中在筛查计划上,很少有干预措施能解决降低风险、诊断和治疗结果方面的公平差距。这突出表明,有必要采取有针对性的方法来解决癌症治疗过程中研究不足的重点人群的不平等问题。
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引用次数: 0
A Systematic Review and Quality Assessment of Cardiovascular Disease–Specific Health-Related Quality-of-Life Instruments: Part II Psychometric Properties 心血管疾病特定健康相关生活质量工具的系统回顾与质量评估》:第二部分 心理测量学特性。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.08.011
Xue Li PhD , Rui Li MSc , Meixuan Li MSc , Xu Hui MSc , Jing Li MSc , Liang Yao PhD , Harriette Van Spall MD, MPH , Kun Zhao PhD , Qiang Fu PhD , Feng Xie PhD

Objectives

Health-related quality-of-life instruments for cardiovascular diseases (CVDs) have been commonly used to measure important patient-reported outcomes in clinical trials and practices. This study aimed to systematically identify and evaluate the psychometric properties of CVD-specific health-related quality-of-life instruments.

Methods

We searched cumulative index to nursing and allied health literature, Embase, and PubMed from inception to January 20, 2022. Studies that reported psychometric properties of CVD-specific instruments were included. Two reviewers independently assessed the methodological quality using the Consensus-based Standards for the Selection of Health Measurement Instruments methods for evaluating measurement properties and quality of evidence. Seven psychometric properties, including structural validity, internal consistency, test-retest reliability, convergent validity, divergent validity, discriminative validity, and responsiveness, were evaluated.

Results

We identified 142 studies reporting psychometric properties of 40 instruments. Five (12.5%) instruments demonstrated measurement properties with sufficient or inconsistent ratings; 16 (40.0%) instruments did not report any responsiveness evidence. Of the 40 instruments, 15 (37.5%) instruments were rated sufficient with high quality of evidence on internal consistency; 4 (10.0%) on structural validity, convergent validity and divergent validity; and 3 (7.5%) on discriminative validity.

Conclusions

When measuring patient-reported outcomes in clinical trials or routine practice, it is important to choose instruments with established psychometric properties.
目的:心血管疾病(CVD)健康相关生活质量(HRQoL)工具通常用于测量临床试验和实践中患者报告的重要结果。本研究旨在系统识别和评估心血管疾病专用 HRQoL 工具的心理测量特性:我们检索了从开始到 2022 年 1 月 20 日的 CINAHL、Embase 和 PubMed。纳入了报告心血管疾病特定工具心理测量特性的研究。两位审稿人采用基于共识的健康测量工具选择标准对测量属性和证据质量的评估方法进行了独立评估。评估了七种心理测量特性,包括结构效度、内部一致性、重测可靠性、收敛效度、发散效度、区分效度和反应性:结果:我们确定了 142 项研究,报告了 40 种工具的心理测量特性。其中 5 项(12.5%)工具的测量属性被评为 "足够 "或 "不一致"。16(40.0%)项工具未报告任何反应性证据。在 40 种工具中,15 种(37.5%)工具的内部一致性被评为 "充分",证据质量较高,4 种(10.0%)工具的结构效度、收敛效度和发散效度被评为 "充分",3 种(7.5%)工具的区分效度被评为 "充分":在临床试验或常规实践中测量患者报告的结果时,选择具有成熟心理测量学特性的工具非常重要。
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引用次数: 0
An Application of the Checklist for Health Economic Quality Evaluations in a Systematic Review Setting 卫生经济质量评价清单(支票)在系统评价中的应用。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.10.3853
Stijntje W. Dijk MD, MSc , Skander Essafi MD , Myriam G.M. Hunink MD, PhD

Objective

Quality assessment tools serve an important role in evaluating economic evaluations. This article showcases the first application of the Checklist for Health Economic Quality Evaluations (CHEQUE) tool in a systematic review setting and offers descriptive reflections on its use.

Methods

We applied CHEQUE to 21 diverse economic evaluations in a systematic review of medical education. We visualized weighted CHEQUE scores, calculate correlations between methods and reporting sections, and provided all data and R code for reuse in future applications. Finally, we provided a detailed overview of our judgments and alternative considerations of the checklist items, and suggestions for further development.

Results

Scores ranged from 18% to 94% depending on the applied weighting method, with a positive correlation between the method and reporting quality. CHEQUE enables systematic and standardized assessment but may benefit from refinement in scoring clarity and burden reduction.

Conclusion

Our study provides insights for future guidance on applying and developing CHEQUE or similar tools in economic evaluation quality assessment.
目的:质量评价工具在评价经济评价中起着重要作用。本文展示了卫生经济质量评估清单(支票)工具在系统审查设置中的首次应用,并提供了对其使用的描述性反思。方法:采用检验方法对21项医学教育经济评价进行系统评价。我们可视化加权的支票分数,计算方法和报告部分之间的相关性,并提供所有数据和R代码,以便在未来的应用程序中重用。最后,我们提供了一个详细的概述,我们的判断和替代考虑的清单项目,并建议进一步发展。结果:根据所采用的加权方法,得分范围为18%至94%,方法与报告质量呈正相关。支票可以进行系统和标准化的评估,但可以从评分清晰度和减轻负担的改进中受益。结论:本研究为今后在经济评价质量评价中应用和开发支票或类似工具提供了指导。
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引用次数: 0
Reporting Uncertainty Around Health-State Values: A Standard Method and Worked Example 报告健康状态值的不确定性:一种标准方法和工作示例。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.010
Nancy J. Devlin PhD , Giselle Abangma MSc , Andrew Lloyd DPhil , David Parkin DPhil , Andrew Briggs DPhil

Objectives

Articles reporting value sets typically only report the standard errors (SEs) around each estimated coefficient in value set models. This is important information but does not help those building cost-effectiveness models, who need to know the uncertainty around the values of health states to conduct sensitivity analyses. This report’s aim is to demonstrate how SEs around health-related quality of life values can be calculated, using the example of the UK EQ-5D-3L value set.

Methods

We show how information from a model’s variance/covariance matrix can be used to estimate SEs for every health-state value, whether it is part of the modeling data set or not. Data from the Measurement and Valuation of Health study were used to replicate the original UK value set and the variance/covariance matrix and to produce SEs around the values for all 243 EQ-5D-3L states.

Results

The range of the SEs is small compared with the range of the health-state values but is conditional on a correct model specification and may be sensitive to alternative specifications.

Conclusions

Reporting these SEs should become routine practice in reporting value sets, to ensure that users are provided with information on parameter uncertainty. These SEs only capture one specific aspect of the sources of uncertainty around health-related quality of life values but represent a first step toward a more complete account of uncertainty in the preference weights used to estimate quality-adjusted life-years.
目的:报告值集的论文通常只报告值集模型中每个估计系数周围的标准误差(SEs)。这是重要的信息,但对那些建立成本效益模型的人没有帮助,因为他们需要知道健康状态值的不确定性,以便进行敏感性分析。本文的目的是利用英国EQ-5D-3L值集的例子,演示如何计算HRQoL值周围的se。方法:我们展示了如何使用来自模型方差/协方差矩阵的信息来估计每个健康状态值的se,无论它是否是建模数据集的一部分。来自健康测量和评估研究的数据被用来复制原始的UK值集和方差/协方差矩阵,并在所有243个EQ-5D-3L状态的值周围产生标准误差。结果:与健康状态值的范围相比,se的范围较小,但以正确的模型规格为条件,并且可能对替代规格敏感。结论:报告这些se应成为报告值集的常规做法,以确保向用户提供参数不确定性信息。这些se仅捕获HRQoL值不确定性来源的一个特定方面,但是代表了对用于估计QALYs的偏好权重的不确定性进行更完整说明的第一步。
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引用次数: 0
The Cost-Effectiveness of Frontline Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia: In Pursuit of Treatment-Free Remission and Dose Reduction 慢性髓系白血病患者一线TKI策略的成本-效果:追求无治疗缓解和剂量减少。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.12.005
Sanne J.J.P.M. Metsemakers MSc , Rosella P.M.G. Hermens PhD , Geneviève I.C.G. Ector PhD , Nicole M.A. Blijlevens PhD , Tim M. Govers PhD

Objectives

The management of chronic myeloid leukemia (CML) now includes dose reduction (DR) and treatment-free remission (TFR). Evaluating the cost-effectiveness of lifelong-prescribed expensive tyrosine kinase inhibitors (TKIs) for CML is crucial. Prior cost-effectiveness evaluations state that imatinib is the favorable frontline TKI. Some of these evaluations address TFR, but not DR, nor aging and second-generation (2G)-TKIs upcoming patent expirations. This study evaluates the cost-effectiveness of frontline TKIs for CML patients including these factors.

Methods

This Markov model evaluates the cost-effectiveness of frontline TKIs for newly diagnosed patients with CML using 17 health states. Transition probabilities, costs, and utilities were derived from literature data. Incremental cost-effectiveness ratios were calculated. Sensitivity analysis and model validation were conducted.

Results

Nilotinib is most effective (20.13 quality-adjusted life-years [QALYs]) and imatinib is least effective (17.25 QALYs) for the model including TFR and DR. Imatinib was favored over dasatinib (89.80%), nilotinib (62.70%), and bosutinib (78.40%), at a willingness-to-pay threshold of €80 000 per QALY. Without TFR and DR, fewer QALYs were generated. For patients at the age of 70 years, imatinib has a high probability of being more cost-effective than dasatinib, nilotinib, and bosutinib. With 50% 2GTKI cost reductions, nilotinib is considered more cost-effective compared with imatinib (98.40%), dasatinib (94.80%), and bosutinib (68.90%).

Conclusions

The findings indicate that 2GTKIs are more effective in generating QALYs, including for older (age >70 years) patients. Given the current TKI prices, imatinib remains cost-effective. Including DR and TFR in CML management generates more QALYs. Cost reductions from expected 2GTKIs patent expirations will greatly increase their cost-effectiveness. Results may inform 2GTKIs cost discussions after patent expiration, potentially broadening global availability. The findings also emphasize the importance of aiming for TFR and DR in CML management.
目的:慢性髓性白血病(CML)的治疗现在包括剂量减少(DR)和无治疗缓解(TFR)。评估终身处方昂贵的酪氨酸激酶抑制剂(TKIs)治疗CML的成本效益至关重要。先前的成本效益评估表明,伊马替尼是有利的一线TKI。其中一些评估涉及TFR,但不涉及DR,也不涉及老化和即将到期的第二代(2G) tki。本研究评估了包括这些因素在内的一线tki治疗CML患者的成本效益。方法:采用马尔可夫模型评估17种健康状态下一线tki治疗新诊断CML患者的成本-效果。转换概率、成本和效用来源于文献数据。计算增量成本-效果比。进行敏感性分析和模型验证。结果:在包括TFR和dr的模型中,尼洛替尼最有效(20.13 QALYs),伊马替尼最无效(17.25 QALYs),伊马替尼优于达沙替尼(89.80%)、尼洛替尼(62.70%)和博舒替尼(78.40%),WTP为80,000欧元/QALY。没有TFR和DR,产生的qaly较少。对于70岁的患者,与达沙替尼、尼洛替尼和博舒替尼相比,伊马替尼具有较高的成本效益。尼罗替尼的2GTKI成本降低了50%,与伊马替尼(98.40%)、达沙替尼(94.80%)和博舒替尼(68.90%)相比,尼罗替尼被认为具有成本效益。结论:研究结果表明,2GTKIs在产生QALYs方面更有效,包括老年(70岁)患者。考虑到目前TKI的价格,伊马替尼仍然具有成本效益。在cml管理中加入DR和TFR可以产生更多的qaly。从预计的2个gtki专利到期中减少的成本将大大提高其成本效益。研究结果可能会在专利到期后为2gtki的成本讨论提供信息,从而有可能扩大其在全球的可用性。研究结果还强调了在cml管理中以TFR和DR为目标的重要性。
{"title":"The Cost-Effectiveness of Frontline Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia: In Pursuit of Treatment-Free Remission and Dose Reduction","authors":"Sanne J.J.P.M. Metsemakers MSc ,&nbsp;Rosella P.M.G. Hermens PhD ,&nbsp;Geneviève I.C.G. Ector PhD ,&nbsp;Nicole M.A. Blijlevens PhD ,&nbsp;Tim M. Govers PhD","doi":"10.1016/j.jval.2024.12.005","DOIUrl":"10.1016/j.jval.2024.12.005","url":null,"abstract":"<div><h3>Objectives</h3><div>The management of chronic myeloid leukemia (CML) now includes dose reduction (DR) and treatment-free remission (TFR). Evaluating the cost-effectiveness of lifelong-prescribed expensive tyrosine kinase inhibitors (TKIs) for CML is crucial. Prior cost-effectiveness evaluations state that imatinib is the favorable frontline TKI. Some of these evaluations address TFR, but not DR, nor aging and second-generation (2G)-TKIs upcoming patent expirations. This study evaluates the cost-effectiveness of frontline TKIs for CML patients including these factors.</div></div><div><h3>Methods</h3><div>This Markov model evaluates the cost-effectiveness of frontline TKIs for newly diagnosed patients with CML using 17 health states. Transition probabilities, costs, and utilities were derived from literature data. Incremental cost-effectiveness ratios were calculated. Sensitivity analysis and model validation were conducted.</div></div><div><h3>Results</h3><div>Nilotinib is most effective (20.13 quality-adjusted life-years [QALYs]) and imatinib is least effective (17.25 QALYs) for the model including TFR and DR. Imatinib was favored over dasatinib (89.80%), nilotinib (62.70%), and bosutinib (78.40%), at a willingness-to-pay threshold of €80 000 per QALY. Without TFR and DR, fewer QALYs were generated. For patients at the age of 70 years, imatinib has a high probability of being more cost-effective than dasatinib, nilotinib, and bosutinib. With 50% 2GTKI cost reductions, nilotinib is considered more cost-effective compared with imatinib (98.40%), dasatinib (94.80%), and bosutinib (68.90%).</div></div><div><h3>Conclusions</h3><div>The findings indicate that 2GTKIs are more effective in generating QALYs, including for older (age &gt;70 years) patients. Given the current TKI prices, imatinib remains cost-effective. Including DR and TFR in CML management generates more QALYs. Cost reductions from expected 2GTKIs patent expirations will greatly increase their cost-effectiveness. Results may inform 2GTKIs cost discussions after patent expiration, potentially broadening global availability. The findings also emphasize the importance of aiming for TFR and DR in CML management.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 224-232"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898558","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
Slipping Away: Slippage in Hazard Ratios Over Datacuts and Its Impact on Immuno-oncology Combination Economic Evaluations 渐行渐远:数据中断导致的危害比滑坡及其对免疫肿瘤联合经济评估的影响。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.09.008
Dawn Lee MMath, MSc , Zain Ahmad , Caroline Farmer PhD , Maxwell S. Barnish PhD , Alan Lovell PhD , G.J. Melendez-Torres DPhil, MPH, RN

Objectives

This study examines the impact of slippage in hazard ratios (tending toward the null over subsequent datacuts) for overall survival for combination treatment with a PD-(L)-1 inhibitor and a tyrosine kinase inhibitor in advanced renal cell carcinoma.

Methods

Four trials’ Kaplan-Meier curves were digitized over several datacuts and fitted with standard parametric curves. Accuracy and consistency of early data projections were calculated versus observed restricted mean survival time and fitted lifetime survival from the longest follow-up datacut. The change in economically justifiable price (eJP) was calculated fitting the same curve to both arms, using an assumed average utility of 0.7 and willingness-to-pay threshold of £30 000 per quality-adjusted life-year. The eJP represents the lifetime justifiable price increment for the new treatment, including differences in drug-, administration-, and disease-related costs.

Results

Slippage in hazard ratios was observed in trials with longer follow-up, potentially influenced by subsequent PD-(L)-1 use after tyrosine kinase inhibitor monotherapy, early stoppage of PD-(L)-1, and development of resistance. Lognormal and log-logistic curves were more likely to overpredict the observed result; Gompertz and gamma underpredicted. Statistical measures of goodness of fit did not select the curves that resulted in the RMST closest to what was observed in the final data cut. Large differences in incremental mean life-years were observed between even the penultimate and final datacuts for most of the fitted curves, meaningfully affecting the eJP.

Conclusions

This work demonstrates the challenge in predicting treatment benefits with novel therapies using immature data. Incorporating information on the impact of subsequent treatment is likely to play a key role in improving predictions.
研究目的本研究探讨了晚期肾细胞癌(RCC)中PD-(L)-1抑制剂和酪氨酸激酶抑制剂(TKI)联合治疗总生存期危险比滑移(在随后的数据切分中趋向于空值)的影响:对四项试验的卡普兰-梅耶尔曲线进行数字化处理,并用标准参数曲线拟合。根据观察到的受限平均生存时间(RMST)和最长随访数据截面拟合的终生生存时间,计算早期数据预测的准确性和一致性。假设平均效用为 0.7,支付意愿阈值为每 QALY 30,000 英镑,通过拟合两臂的相同曲线,计算出经济合理价格(eJP)的变化。eJP 代表了新疗法的终生合理价格增量,包括药物、管理和疾病相关成本的差异:结果:在随访时间较长的试验中观察到了危险比的下滑,这可能是受TKI单药治疗后PD-(L)-1的后续使用、PD-(L)-1的早期停药以及耐药性的产生等因素的影响。对数正态曲线和对数-逻辑曲线更有可能过度预测观察到的结果;而贡珀茨曲线和伽马曲线则预测不足。拟合度的统计量不能可靠地预测 RMST。在大多数拟合曲线中,即使是倒数第二个数据截点和最后一个数据截点之间的平均寿命增量也存在很大差异,这对 eJP 产生了有意义的影响:这项研究表明,利用不成熟的数据预测新型疗法的治疗效果是一项挑战。纳入有关后续治疗影响的信息可能会在改进预测方面发挥关键作用。
{"title":"Slipping Away: Slippage in Hazard Ratios Over Datacuts and Its Impact on Immuno-oncology Combination Economic Evaluations","authors":"Dawn Lee MMath, MSc ,&nbsp;Zain Ahmad ,&nbsp;Caroline Farmer PhD ,&nbsp;Maxwell S. Barnish PhD ,&nbsp;Alan Lovell PhD ,&nbsp;G.J. Melendez-Torres DPhil, MPH, RN","doi":"10.1016/j.jval.2024.09.008","DOIUrl":"10.1016/j.jval.2024.09.008","url":null,"abstract":"<div><h3>Objectives</h3><div>This study examines the impact of slippage in hazard ratios (tending toward the null over subsequent datacuts) for overall survival for combination treatment with a PD-(L)-1 inhibitor and a tyrosine kinase inhibitor in advanced renal cell carcinoma.</div></div><div><h3>Methods</h3><div>Four trials’ Kaplan-Meier curves were digitized over several datacuts and fitted with standard parametric curves. Accuracy and consistency of early data projections were calculated versus observed restricted mean survival time and fitted lifetime survival from the longest follow-up datacut. The change in economically justifiable price (eJP) was calculated fitting the same curve to both arms, using an assumed average utility of 0.7 and willingness-to-pay threshold of £30 000 per quality-adjusted life-year. The eJP represents the lifetime justifiable price increment for the new treatment, including differences in drug-, administration-, and disease-related costs.</div></div><div><h3>Results</h3><div>Slippage in hazard ratios was observed in trials with longer follow-up, potentially influenced by subsequent PD-(L)-1 use after tyrosine kinase inhibitor monotherapy, early stoppage of PD-(L)-1, and development of resistance. Lognormal and log-logistic curves were more likely to overpredict the observed result; Gompertz and gamma underpredicted. Statistical measures of goodness of fit did not select the curves that resulted in the RMST closest to what was observed in the final data cut. Large differences in incremental mean life-years were observed between even the penultimate and final datacuts for most of the fitted curves, meaningfully affecting the eJP.</div></div><div><h3>Conclusions</h3><div>This work demonstrates the challenge in predicting treatment benefits with novel therapies using immature data. Incorporating information on the impact of subsequent treatment is likely to play a key role in improving predictions.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 260-268"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401462","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
The Influence of Recall and Timing of Assessment on the Estimation of Quality-Adjusted Life-Years When Health Fluctuates Recurrently 健康反复波动时,召回和评估时间对质量aly估计的影响。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.005
Sabina Sanghera PhD , Joanna Coast PhD , Axel Walther PhD , Tim J. Peters PhD

Objectives

When health fluctuates recurrently, estimating quality of life (QOL) is challenging, risking over-/underestimation due to measures’ recall periods and timing. To inform how/when to capture QOL, we compared responses using different recall periods and assessment timings.

Methods

For one 3-week chemotherapy cycle, cancer patients were randomly assigned to complete EQ-5D-5L or SF-12v2 (daily with a daily recall, weekly with a weekly recall, and at 3 weeks with a 3-week recall); a third group completed SF-12v2 daily with a 3-week recall. EQ-5D-5L and SF-6D utilities (anchored at 1 [full health] and 0 [dead]) were generated and repeated measures analysis of variance, t tests, and effect sizes were calculated to compare recall.

Results

A total of 503 patients consented; all 21 daily questionnaires were completed by 84 (50%), 67 (40%), and 72 (43%) in the groups. Both measures captured fluctuations in QOL suggesting differences are due to recall effects. Mean daily scores were greater than scores for the past week on days 7, 14, and 21 (P < .0001). Utility was underestimated (by 0.0782, 0.0374, and 0.0437) for EQ-5D-5L and (0.0387, 0.0266, and 0.0304) for SF-6D, with the EQ-5D-5L comparison on day 7 reaching a minimally important difference. The “past 3 weeks” generated the lowest scores (P < .0001), with utility underestimated by 0.0746 (EQ-5D-5L) and 0.0310 (SF-6D), heavily skewed by the first treatment week.

Conclusions

The current practice of using a single estimate at the beginning or end of a cycle with a daily (EQ-5D-5L) or longer (SF-12/SF-36) recall could bias cost-effectiveness estimates. QOL should be captured frequently with short recall when fluctuations are likely and less frequently with longer recall in stable periods.
目的:当健康状况反复波动时,估计生活质量是具有挑战性的,由于测量的回忆期和时间,有被高估/低估的风险。为了了解如何/何时捕捉生活质量,我们比较了使用不同回忆期和评估时间的反应。方法:在一个为期3周的化疗周期中,癌症患者被随机分配完成EQ-5D-5L或SF-12v2(每日召回,每日召回,每周召回,3周召回,3周召回);第三组每天完成SF-12v2,为期3周的召回。生成EQ-5D-5L和SF-6D效用(锚定在1(全健康)和0(死亡)),并计算重复测量方差分析、t检验和效应量,以比较召回率。结果:503例患者同意;21份日常问卷分别由84人(50%)、67人(40%)、72人(43%)完成。两种测量方法都捕捉到了生活质量的波动,表明差异是由于回忆效应造成的。平均每日评分大于过去一周在第7天、第14天和第21天的评分(p结论:目前在一个周期的开始或结束时使用单一评估的做法,每日(EQ-5D-5L)或更长(SF-12/SF-36)召回可能会使成本-效果评估产生偏差。在可能出现波动的情况下,应经常用短期回忆法记录生活质量,而在稳定时期,则较少使用较长回忆法记录生活质量。
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引用次数: 0
Generative Artificial Intelligence for Health Technology Assessment: Opportunities, Challenges, and Policy Considerations: An ISPOR Working Group Report 用于卫生技术评估的生成式人工智能:机遇、挑战和政策考虑--ISPOR 工作组报告。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.10.3846
Rachael L. Fleurence PhD , Jiang Bian PhD , Xiaoyan Wang PhD , Hua Xu PhD , Dalia Dawoud PhD , Mitchell Higashi PhD , Jagpreet Chhatwal PhD , ISPOR Working Group on Generative AI

Objectives

To provide an introduction to the uses of generative artificial intelligence (AI) and foundation models, including large language models, in the field of health technology assessment (HTA).

Methods

We reviewed applications of generative AI in 3 areas: systematic literature reviews, real-world evidence, and health economic modeling.

Results

(1) Literature reviews: generative AI has the potential to assist in automating aspects of systematic literature reviews by proposing search terms, screening abstracts, extracting data, and generating code for meta-analyses; (2) real-world evidence: generative AI can facilitate automating processes and analyze large collections of real-world data, including unstructured clinical notes and imaging; (3) health economic modeling: generative AI can aid in the development of health economic models, from conceptualization to validation. Limitations in the use of foundation models and large language models include challenges surrounding their scientific rigor and reliability, the potential for bias, implications for equity, as well as nontrivial concerns regarding adherence to regulatory and ethical standards, particularly in terms of data privacy and security. Additionally, we survey the current policy landscape and provide suggestions for HTA agencies on responsibly integrating generative AI into their workflows, emphasizing the importance of human oversight and the fast-evolving nature of these tools.

Conclusions

Although generative AI technology holds promise with respect to HTA applications, it is still undergoing rapid developments and improvements. Continued careful evaluation of their applications to HTA is required. Both developers and users of research incorporating these tools, should familiarize themselves with their current capabilities and limitations.
目的介绍生成式人工智能(AI)和基础模型(包括大型语言模型(LLM))在卫生技术评估(HTA)领域的应用:我们回顾了生成式人工智能在三个领域的应用:系统文献综述、现实世界证据(RWE)和卫生经济建模。结果:(1)文献综述:生成式人工智能有可能通过提出检索词、筛选摘要、提取数据和生成荟萃分析的代码,帮助实现系统性文献综述的自动化;(2)现实世界证据(RWE):生成式人工智能可以促进流程自动化,并分析包括非结构化临床笔记和成像在内的大量现实世界数据(RWD);(3)卫生经济建模:生成式人工智能可以帮助开发从概念化到验证的卫生经济模型。使用基础模型和 LLM 的局限性包括围绕其科学严谨性和可靠性的挑战、出现偏差的可能性、对公平的影响,以及对遵守监管和道德标准的非同小可的担忧,尤其是在数据隐私和安全方面。此外,我们还调查了当前的政策环境,并就如何负责任地将生成式人工智能整合到工作流程中为 HTA 机构提供了建议,同时强调了人工监督的重要性以及这些工具快速发展的特性:虽然生成式人工智能技术在 HTA 应用方面大有可为,但它仍在快速发展和改进之中。需要继续仔细评估其在 HTA 中的应用。使用这些工具进行研究的开发人员和用户都应熟悉其当前的能力和局限性。
{"title":"Generative Artificial Intelligence for Health Technology Assessment: Opportunities, Challenges, and Policy Considerations: An ISPOR Working Group Report","authors":"Rachael L. Fleurence PhD ,&nbsp;Jiang Bian PhD ,&nbsp;Xiaoyan Wang PhD ,&nbsp;Hua Xu PhD ,&nbsp;Dalia Dawoud PhD ,&nbsp;Mitchell Higashi PhD ,&nbsp;Jagpreet Chhatwal PhD ,&nbsp;ISPOR Working Group on Generative AI","doi":"10.1016/j.jval.2024.10.3846","DOIUrl":"10.1016/j.jval.2024.10.3846","url":null,"abstract":"<div><h3>Objectives</h3><div>To provide an introduction to the uses of generative artificial intelligence (AI) and foundation models, including large language models, in the field of health technology assessment (HTA).</div></div><div><h3>Methods</h3><div>We reviewed applications of generative AI in 3 areas: systematic literature reviews, real-world evidence, and health economic modeling.</div></div><div><h3>Results</h3><div>(1) Literature reviews: generative AI has the potential to assist in automating aspects of systematic literature reviews by proposing search terms, screening abstracts, extracting data, and generating code for meta-analyses; (2) real-world evidence: generative AI can facilitate automating processes and analyze large collections of real-world data, including unstructured clinical notes and imaging; (3) health economic modeling: generative AI can aid in the development of health economic models, from conceptualization to validation. Limitations in the use of foundation models and large language models include challenges surrounding their scientific rigor and reliability, the potential for bias, implications for equity, as well as nontrivial concerns regarding adherence to regulatory and ethical standards, particularly in terms of data privacy and security. Additionally, we survey the current policy landscape and provide suggestions for HTA agencies on responsibly integrating generative AI into their workflows, emphasizing the importance of human oversight and the fast-evolving nature of these tools.</div></div><div><h3>Conclusions</h3><div>Although generative AI technology holds promise with respect to HTA applications, it is still undergoing rapid developments and improvements. Continued careful evaluation of their applications to HTA is required. Both developers and users of research incorporating these tools, should familiarize themselves with their current capabilities and limitations.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 175-183"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628806","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
Exploring Social Preferences for Health and Well-Being Across the Digital Divide: A Qualitative Investigation Based on Tasks Taken From an Online Discrete Choice Experiment 跨越数字鸿沟,探索社会对健康和幸福的偏好。基于在线离散选择实验任务的定性调查。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.001
Becky Field PhD , Katherine E. Smith PhD , Clementine Hill O’Connor PhD , Nyantara Wickramasekera MSc , Aki Tsuchiya PhD

Objectives

Increasingly, discrete choice experiments (DCEs) are conducted online, with little consideration of the digitally excluded, who are unable to participate. Policy makers or others considering online research data need clarity about how views might differ across this “digital divide.” We took tasks from an existing online DCE designed to elicit social preferences for health and well-being outcomes. We aimed to explore (1) how telephone interview participants answered a series of choice tasks taken from an online DCE and (2) whether and how decision making for these tasks differed between digitally excluded and nonexcluded participants.

Methods

We conducted semistructured telephone interviews with members of the public (n = 27), recruited via an existing social research panel. Data were analyzed thematically to identify key approaches to decision making.

Results

Twelve participants were classed as “digitally excluded,” and 15 as “digitally nonexcluded.” Responses were similar between the 2 samples for most choice tasks. We identified 3 approaches used to reach decisions: (1) simplifying, (2) creating explanatory narratives, and (3) personalizing. Although these approaches were common across both samples, understanding the exercise seemed more challenging for the digitally excluded sample.

Conclusions

This novel study provides some assurance that the participants’ views over the choice tasks used are similar across the digital divide. The challenges we identified with understanding highlight the need to carefully examine the views held by the digitally excluded. If online data are to inform policy making, it is essential to explore the views of those who cannot participate in online DCEs.
目标:离散选择实验 (DCE) 越来越多地在网上进行,但却很少考虑到被数字技术排除在外、无法参与实验的人。政策制定者或其他考虑在线研究数据的人需要清楚地了解,在这种 "数字鸿沟 "中,人们的观点会有什么不同。我们从现有的在线 DCE 中选取了一些任务,该在线 DCE 旨在了解社会对健康和福利结果的偏好。我们的目的是探索:i) 电话访谈参与者如何回答取自在线 DCE 的一系列选择任务;ii) 数字排斥和非数字排斥参与者对这些任务的决策是否不同以及如何不同:方法:通过现有的社会研究小组对公众(n=27)进行半结构化电话访谈。对数据进行了主题分析,以确定决策的关键方法:结果:12 名参与者被归类为 "数字排斥",15 名参与者被归类为 "数字非排斥"。两个样本对大多数选择任务的回答相似。我们确定了做出决定的三种方法:(1) 简化;(2) 创建解释性叙述;(3) 个性化。虽然这些方法在两个样本中都很常见,但对于被数字排斥的样本来说,理解这项工作似乎更具挑战性:这项新颖的研究在一定程度上保证了参与者对所使用的选择任务的看法在数字鸿沟中是相似的。我们在理解方面发现的挑战突出表明,有必要谨慎研究被数字鸿沟排斥者所持有的观点。如果要让在线数据为政策制定提供信息,就必须探讨那些无法参与在线 DCE 的人的观点。
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引用次数: 0
Potential Biases in Post-Stroke Health Utility Estimates by Modified Rankin Scale Scores 修正Rankin量表评分在脑卒中后健康效用评估中的潜在偏差。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.07.028
Takashi Yoshioka MD, PhD
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引用次数: 0
期刊
Value in Health
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