Value in Health最新文献_第3页

Patient-Reported Outcome Measures in Clinical Trials: An Analysis of Trends From 2008 to 2023 临床试验中患者报告的结果测量：2008-2023年趋势分析

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-11-20 DOI: 10.1016/j.jval.2025.10.017

Maja Kuharic PhD , Justin Yu PharmD , Mrinmayee Joshi PhD , Jonathan L. Nazari PharmD , Fatih Zecic PharmD , David Cella PhD , A. Simon Pickard PhD

Objectives

To examine trends in patient-reported outcome (PRO) measure use in clinical trials (2008-2023) and analyze adoption patterns of common PRO measures across geographic regions, disease categories, and age groups.

Methods

We identified PRO measures from PROQOLID and health technology assessment recommendations, including generic instruments (EuroQol 5-Dimension [EQ-5D], SF-36/12/8/6D, Patient-Reported Outcomes Measurement Information System [PROMIS]), condition-specific measures (EORTC QLQ-C30/QLU-C10D, Functional Assessment of Chronic Illness Therapy), and pediatric instruments (Pediatric Quality of Life Inventory [PedsQL], EQ-5D-Y, PROMIS Pediatric, Child Health Questionnaire, Child Health Utility 9D). Using a custom Python algorithm (96% sensitivity, 97% specificity), we searched ClinicalTrials.gov for interventional studies incorporating PRO measures.

Results

Among 475 844 registered trials, PRO measure usage increased substantially. Adult trials incorporating PROs grew from 12.7% (n = 1783) in 2008 to 23.5% (n = 7808) in 2023; pediatric trials increased from 8.0% (n = 231) to 17.7% (n = 1118). EQ-5D surpassed SF measures as the most widely used generic instrument in 2017 (2023: EQ-5D n = 1023, SF n = 781), growing at a compound annual growth rate of 16.2%, whereas PROMIS demonstrated the most rapid expansion with a compound annual growth rate of 51.9%. PRO measures were most frequently implemented in trials studying pathological conditions, nervous system diseases, and neoplasms, with distinct regional preferences: EQ-5D predominated in Europe, whereas SF and PROMIS measures were more common in North America.

Conclusions

By 2023, nearly one-quarter of adult trials included PRO measures, with growing pediatric implementation. PRO integration continues expanding, driven by regulatory and health technology assessment requirements. EQ-5D’s emergence as the leading measure may reflect widespread health technology assessment endorsement. These findings inform PRO selection strategies and identify opportunities for greater pediatric implementation and regional adoption.

目的：研究临床试验中患者报告结果（PRO）测量方法的使用趋势（2008-2023年），并分析不同地理区域、疾病类别和年龄组中常见PRO测量方法的采用模式。方法：我们从PROQOLID和卫生技术评估建议中确定PRO指标，包括通用仪器（EQ-5D、SF-36/12/8/6D、PROMIS）、条件特异性仪器（EORTC QLQ-C30/ qu - c10d、FACIT）和儿科仪器（PedsQL、EQ-5D- y、PROMIS pediatric、CHQ、CHU9D）。使用自定义Python算法（96%灵敏度，97%特异性），我们在ClinicalTrials.gov网站上搜索包含PRO测量的介入性研究。结果：在475,844项注册试验中，PRO测量的使用显著增加。纳入pro的成人试验从2008年的12.7% （n= 1783）增加到2023年的23.5% (n= 7808)；儿科试验从8.0% （n=231）增加到17.7% （n= 1118）。EQ-5D在2017年（2023年：EQ-5D n=1,023, SF n=781）超过SF测量，成为使用最广泛的通用仪器，复合年增长率（CAGR）为16.2%，而PROMIS的增长速度最快，复合年增长率为51.9%。PRO测量最常用于研究病理状况、神经系统疾病和肿瘤的试验，具有明显的区域偏好：EQ-5D在欧洲占主导地位，而SF和PROMIS测量在北美更常见。结论：到2023年，近四分之一的成人试验包括PRO措施，儿科应用也越来越多。在法规和HTA要求的推动下，PRO集成继续扩展。EQ-5D作为主要措施的出现可能反映了HTA的广泛认可。这些发现为PRO选择策略提供了信息，并确定了更多儿科实施和区域采用的机会。

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引用次数: 0

Quantifying Accuracy and Cost in Public Preferences for Artificial Intelligence in Radiation Therapy 量化人工智能在放射治疗中的公众偏好的准确性和成本。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-12-17 DOI: 10.1016/j.jval.2025.10.019

Weihao Cheng BEng , Zekai Yu BEng

引用次数: 0

Economic Evaluation of Deprescribing in Older Adults: A Systematic Review 老年人开处方的经济评价：一项系统综述。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-20 DOI: 10.1016/j.jval.2025.10.001

Ying Zhang MS , Zhaoyan Chen MS , Xi Chen MS , Fangyuan Tian PhD

Objectives

This study aims to synthesize economic evaluation evidence on deprescribing in older adults across settings.

Methods

A comprehensive search was conducted across the PubMed, Embase, and Web of Science databases from inception through June 18, 2025, to identify studies evaluating the economic impact of deprescribing in older adults. Two independent reviewers selected relevant articles, extracted data, and assessed study quality according to the Consolidated Health Economic Evaluation Reporting Standards 2022.

Results

A total of 57 studies were included, covering multiple settings, such as communities, outpatient clinics, hospitals, geriatric care facilities, nursing homes, primary care, home, and online platforms. Medication review was the most frequently used deprescribing strategy, supplemented by education, pharmaceutical care, rounds, and pharmacist independent prescriber. Multiple deprescribing tools were used, including general and custom criteria. Physicians and pharmacists were the primary implementers of deprescribing interventions. Economic evaluation methods included cost, cost-utility, cost-benefit, cost-effectiveness, cost-consequence, and return-on-investment analysis, with various outcome indicators. Quality assessment revealed that the quality of the studies was good or very good, except for 3 studies that were of poor quality. Most studies (44 of 57) indicated that deprescribing led to cost-saving, medication cost reduction, or improved cost-effectiveness/benefit/utility.

Conclusions

The majority of studies support the economic benefit of deprescribing, although some report negative or inconclusive results. However, this review has some limitations in database retrieval, data abstraction, and risk of bias assessment, potentially affecting the findings. The contradiction in economic results stems from a variety of factors, which requires further optimization studies in the future.

目的：本研究的目的是综合不同环境下老年人处方的经济评价证据。方法：从2025年6月18日开始，对PubMed、Embase和Web of Science数据库进行了全面的搜索，以确定评估老年人开处方的经济影响的研究。两名独立审稿人选择相关文章，提取数据，并根据综合卫生经济评估报告标准2022评估研究质量。结果：纳入了57项研究，涵盖了多种环境，如社区、门诊诊所、医院、老年护理机构、养老院、初级保健、家庭和在线平台。药物回顾是最常用的处方解除策略，辅以教育、药学服务、查房和药剂师独立开处方者。使用了多种描述工具，包括通用标准和自定义标准。医生和药剂师是处方干预的主要实施者。经济评价方法包括成本分析、成本-效用分析、成本-效益分析、成本-效果分析、成本-后果分析和投资回报分析，并采用各种结果指标。质量评估显示，除了三个质量较差的研究外，研究的质量为好或非常好。大多数研究（57项中的44项）表明，开处方可以节省成本，降低药物成本，或提高成本效益/效益/效用。结论：大多数研究支持处方化的经济效益，尽管有些报告了负面或不确定的结果。然而，本综述在数据库检索、数据抽象和偏倚风险评估方面存在一定的局限性，可能影响研究结果。经济效果的矛盾源于多种因素，需要在未来进一步优化研究。

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引用次数: 0

Estimating the Willingness-to-Pay per Quality-Adjusted Life-Year to Aid Health Technology Assessment in India 估计每个质量调整生命年的支付意愿，以帮助印度的卫生技术评估。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-30 DOI: 10.1016/j.jval.2025.10.007

Yashika Chugh PhD , Gaurav Jyani PhD , Sitanshu Sekhar Kar MD , Binod Kumar Patro MD , Mayur Trivedi PhD , Sandra Albert PhD , Swati Raman PhD , Kavitha Rajsekar PhD , Shankar Prinja MD

Objectives

The study aimed to estimate the willingness to pay (WTP) for a quality-adjusted life-year (QALY) to help determine the threshold for cost-effectiveness in India. We also assessed the factors affecting WTP/QALY.

Methods

We used a multistage stratified random sampling strategy to select 5460 respondents across 6 Indian states. Participants were interviewed to gather household socioeconomic and demographic data, followed by assessments of health gains using time trade-off and WTP. Respondents were presented with 12 hypothetical health states to gauge health gains and their WTP to restore health. These values were combined to determine WTP per QALY using an aggregated approach. The health gains were discounted at 3%. Weighted estimates based on state, residence, gender, age, and education were computed. A mixed-effect regression model explored the relationship between socio-demographic variables and WTP/QALY.

Results

Based on 21 640 observations, we found that mean weighted WTP/QALY was estimated to be INR 2 12 307 (US$ 2535). Based on the different health states, the WTP/QALY ranged from INR 1 70 414 (US$ 2034) to INR 2 58 985 (US$ 3092). Age, gender, family size, education, marital status, occupation, presence of health insurance, consumption expenditure, and number of earning members in the household significantly impacted WTP per QALY.

Conclusions

To our knowledge, our study reports the first-ever estimate of WTP/QALY for the Indian population. The WTP/QALY ranges from 1 to 1.52 times the gross domestic product per capita. This could be considered as the cost-effectiveness threshold for health technology assessment in India.

目的：本研究旨在估计质量调整生命年（QALY）的支付意愿（WTP），以帮助确定印度的成本效益阈值。我们还评估了影响WTP/QALY的因素。方法：我们采用多阶段分层随机抽样策略，在印度6个邦选择5460名受访者。对参与者进行访谈，收集家庭社会经济和人口统计数据，然后使用时间权衡（TTO）和WTP评估健康收益。向应答者提供了12种假设的健康状态，以衡量健康收益和他们恢复健康的WTP。将这些值组合起来，使用聚合方法确定每个QALY的WTP。健康收益按3%折现。计算了基于州、居住地、性别、年龄和教育程度的加权估计。采用混合效应回归模型探讨社会人口学变量与WTP/QALY之间的关系。结果：基于21,640项观察，我们发现加权WTP/QALY的平均估计为2,12,307印度卢比（2,535美元）。根据不同的健康状况，WTP/QALY从170,414印度卢比（2,034美元）到2,58,985印度卢比（3,092美元）不等。年龄、性别、家庭规模、教育程度、婚姻状况、职业、是否有医疗保险、消费支出和家庭收入成员数量显著影响每个QALY的WTP。结论：我们的研究报告了印度人口WTP/QALY的首次估计。WTP/QALY为人均国内生产总值的1-1.52倍。这可被视为印度卫生技术评估的成本效益门槛。

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引用次数: 0

The Uneven Path of Psychological Distress: How Socioeconomic Status Shapes Distress Transitions in Australian Adults 心理困扰的不平坦路径：社会经济地位如何影响澳大利亚成年人的痛苦过渡。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-29 DOI: 10.1016/j.jval.2025.10.005

Muhammad Iftikhar ul Husnain MS , Mohammad Hajizadeh PhD , Hasnat Ahmad PhD , Rasheda Khanam PhD

Objectives

Psychological distress (PD) is a major public health concern linked to progressive disability. Estimating PD transition probabilities is essential for guiding targeted interventions and policies, particularly in the context of health economic models used for pharmaceutical reimbursement. We aimed to quantify how transition probabilities vary by socioeconomic status across 4 health states of PD (no PD, mild PD, moderate PD, and severe PD) among Australian adults with PD.

Methods

We obtained data on PD status and socioeconomic characteristics from the Household, Income and Labour Dynamics in Australia survey (2007-2021). We used a 4-state continuous-time Markov model to describe annual transitions between PD states. The model allowed tracking of both forward and backward transitions, enabling movement between any pair of states, including persistence of the same state. Socioeconomic status was assessed using education, employment, and income.

Results

Overall, 25 232 participants were identified for the study. The highest probabilities of worsening were for transitioning from mild PD to moderate PD (16.9%), whereas the highest probability of improvement was recorded for the transition from moderate to mild PD (32.3%). Higher recovery rates from severe PD to no PD were observed among individuals with higher education levels than in those with lower education (8.6% vs 5.8%), among the employed compared with the unemployed (7.0% vs 4.8%), and among those in the highest income bracket compared with those in the lowest (12.7% vs 5.7%).

Conclusions

The estimated transition probabilities can be used in health economic evaluations, designed to support reimbursement decision for interventions.

背景：心理困扰（PD）是与进行性残疾相关的主要公共卫生问题。估计PD过渡概率对于指导有针对性的干预措施和政策至关重要，特别是在用于药品报销的卫生经济模型的背景下。目的：我们旨在量化澳大利亚成年PD患者在四种PD健康状态（无PD、轻度PD、中度PD和重度PD）中过渡概率的社会经济状况变化。方法：我们从澳大利亚家庭、收入和劳动力动态调查（2007-2021）中获得PD状态和社会经济特征的数据。我们采用四状态连续时间马尔可夫模型来描述PD状态之间的年度转换。该模型允许跟踪向前和向后转换，支持在任何一对状态之间移动，包括相同状态的持久性。社会经济地位通过教育、就业和收入进行评估。结果：总共有25232名参与者参与了这项研究。从轻度PD过渡到中度PD的恶化概率最高（16.9%），而从中度PD过渡到轻度PD的改善概率最高（32.3%）。从严重PD到无PD的康复率在受教育程度较高的个体中高于受教育程度较低的个体（8.6%比5.8%），在就业人群中高于失业人群（7.0%比4.8%），在收入最高的人群中高于收入最低的人群（12.7%比5.7%）。结论：估计的转移概率可用于卫生经济评价，旨在为干预措施的报销决策提供支持。

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引用次数: 0

The Compounding Burden of Pregnancy Loss: A Longitudinal Analysis of Quality-of-Life Trajectories After Miscarriage 妊娠损失的复合负担：流产后生活质量轨迹的纵向分析。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-11-12 DOI: 10.1016/j.jval.2025.10.013

Corneliu Bolbocean PhD , Arri Coomarasamy MD , Leah Fitzsimmons PhD , Rosinder Kaur MBChB , Adam Devall PhD

Objectives

To characterize the trajectory of health-related quality of life, both overall utility and individual EuroQol 5-Dimension 5-Level (EQ-5D-5L) dimensions, over 12 months after miscarriage and assess whether this trajectory varies significantly for women with 2 or ≥3 miscarriages, compared with those with a single miscarriage within a new tiered Graded Model of Care.

Methods

Of the 203 women prospectively recruited into a cohort study at Birmingham Women’s and Children’s, the completion rate for the EQ-5D-5L questionnaire across 4 time points (baseline and follow-ups at 3, 6, and 12 months) was 71%. We used 2-way fixed-effects regression to estimate the effects of a subsequent miscarriage on the EQ-5D index and on optimal functioning outcomes in each health dimension.

Results

The mean baseline utility was 0.845; women with at least 3 miscarriages started lower (0.830). Relative to 1-loss trajectories, the recurrent miscarriage group showed additional decrements of −0.11 (95% CI −0.187 to −0.038) at 3 months and −0.12 (95% CI −0.198 to −0.036) at 6 months—exceeding the minimal clinically important difference. Deficits consistently arose from anxiety/depression. Women with 2 miscarriages exhibited a comparable mental health gap (≈30 percentage-point reduction in optimal anxiety/depression at 3 and 6 months). By 12 months, group differences were no longer significant.

Conclusions

The health-related quality-of-life burden of miscarriage compounds with successive miscarriages. Recurrent loss leaves women ≥0.12 utility points worse off for up to 6 months, and a second miscarriage triggers substantial mental health impairment. Stratified care pathways offering earlier, intensified psychological and clinical support for women with at least 2 miscarriages are warranted.

目的：描述流产后12个月内HRQoL的轨迹，包括总体效用和个体EQ-5D-5L维度，并评估两次或≥3次流产的女性与单次流产的女性相比，在新的分层分级护理模式下，HRQoL的轨迹是否有显著变化。方法：在伯明翰妇女和儿童医院前瞻性招募的203名妇女中，EQ-5D-5L问卷在四个时间点（基线和随访3,6和12个月）的完成率为71%。我们使用双向固定效应回归来估计随后流产对EQ-5D指数和每个健康维度的最佳功能结果的影响。结果：平均基线效用为0.845；至少有三次流产的妇女开始时较低（0.830）。相对于一次流产轨迹，复发性流产组在3个月时显示了-0.11 （95% CI: -0.187至-0.038）和6个月时-0.12 （95% CI: -0.198至-0.036）的额外下降，超过了最小的临床重要差异。缺陷总是由焦虑/抑郁引起的。两次流产的妇女表现出类似的心理健康差距（在3个月和6个月时，最佳焦虑/抑郁减少约30个百分点）。到12个月时，组间差异不再显著。结论：流产的HRQoL负担与连续流产有关。反复流产会使妇女在长达6个月的时间里的效用点数≥0.12，第二次流产会引发严重的精神健康损害。分层护理途径提供早期，加强心理和临床支持的妇女至少有两次流产是必要的。

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引用次数: 0

Comparative Effectiveness of Ixekizumab Versus Ustekinumab in Patients With Psoriasis: A Randomized Controlled Trial Replication Using Data From an Observational Study (Shanghai Psoriasis Effectiveness Evaluation Cohort) ixekizumab与ustekinumab在银屑病患者中的比较疗效：使用观察性研究（SPEECH）数据的RCT复制

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-12-03 DOI: 10.1016/j.jval.2025.11.009

Ning Yu MD, PhD , Lian Cui PhD, MD , Yuye Wang MD , Yu Wang MD , Xinyi Song MD , Qin Yang MD , Yuling Shi PhD, MD

Objectives

Randomized controlled trials provide high internal validity but often have limited generalizability. Benchmarking real-world data against randomized controlled trial findings helps evaluate external validity. The objectives were to assess whether the results of the IXORA-S trial comparing ixekizumab and ustekinumab could be replicated using real-world data from the Shanghai Psoriasis Effectiveness Evaluation Cohort (SPEECH) and to explore potential reasons for differences between trial and observational findings.

Methods

We conducted a prospective cohort study of adults with moderate-to-severe plaque psoriasis enrolled in SPEECH (2021-2023) and treated with ixekizumab or ustekinumab. Propensity-score methods and multiple imputation were applied to address confounding and missing data. Outcomes were benchmarked against IXORA-S using agreement metrics and sensitivity analyses, including population trimming. The primary endpoint was the proportion of patients achieving a ≥90% reduction in the Psoriasis Area and Severity Index (PASI 90) at week 12.

Results

Among 367 patients (ixekizumab n = 203; ustekinumab n = 164), ixekizumab achieved higher PASI 90 response rates at week 12 compared with ustekinumab. Effect estimates were consistent with IXORA-S in direction, magnitude, and statistical significance. Differences in baseline eligibility criteria and disease severity explained part of the efficacy-effectiveness gap.

Conclusions

The results of IXORA-S were successfully replicated using real-world data from SPEECH. This demonstrates that registry-based observational data can yield reliable estimates of treatment effects and may be used to investigate effectiveness questions not feasible to study in randomized trials, such as long-term outcomes and effects in underrepresented populations.

目的：随机对照试验（RCTs）具有较高的内部效度，但通常具有有限的推广能力。对照RCT结果对真实世界数据进行基准测试有助于评估外部有效性。为了评估IXORA-S试验比较ixekizumab和ustekinumab的结果是否可以使用来自上海银屑病有效性评估队列（SPEECH）的真实数据进行复制，并探讨试验结果和观察结果之间差异的潜在原因。方法：我们进行了一项前瞻性队列研究，纳入SPEECH（2021-2023）的中度至重度斑块性银屑病成人患者，并使用ixekizumab或ustekinumab治疗。采用倾向评分法和多重插值法来解决混淆和缺失数据。结果以IXORA-S为基准，采用一致性指标和敏感性分析，包括群体修剪。主要终点是第12周银屑病面积和严重程度指数（PASI 90）降低≥90%的患者比例。结果：在367例患者中（ixekizumab n = 203; ustekinumab n = 164）， ixekizumab在第12周获得了比ustekinumab更高的PASI 90缓解率。效应估计与IXORA-S在方向、幅度和统计显著性上一致。基线资格标准和疾病严重程度的差异部分解释了疗效差距。结论：IXORA-S的结果可以用SPEECH的真实数据成功复制。这表明，基于注册表的观察性数据可以产生可靠的治疗效果估计，并可用于调查在随机试验中不可行研究的有效性问题，例如在代表性不足的人群中的长期结果和效果。

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引用次数: 0

Psychometric Properties of Cognition Bolt-Ons for the EQ-5D-3L and EQ-5D-5L: A Systematic Review EQ-5D-3L和EQ-5D-5L认知锚点的心理测量特性：系统综述。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-02 DOI: 10.1016/j.jval.2025.09.3053

Fanni Rencz DSc , Stevanus Pangestu MBA , Brendan Mulhern PhD , Aureliano Paolo Finch PhD , Mathieu F. Janssen PhD

Objectives

Cognition is the most commonly used EQ-5D bolt-on, with many different versions varying by descriptors and response levels (3L vs 5L). We aimed to systematically review the psychometric properties of cognition bolt-ons for the EQ-5D-3L and EQ-5D-5L.

Methods

A systematic review was conducted in PubMed, Web of Science, and Google Scholar following PRISMA 2020 guidelines (PROSPERO:CRD42023445567). We assessed the bolt-ons’ performance both as individual items and when added to the EQ-5D. Each bolt-on version was rated as positive (+) or nonpositive (−) within each publication using a checklist, and scores were summed across publications to reflect overall performance.

Results

In total, 101 publications from 72 studies met the inclusion criteria, examining 15 3-level and 13 5-level bolt-ons. The most frequently reported psychometric properties were item-level ceiling (n = 75) and known-groups validity (n = 54). Fewer studies explored convergent or divergent validity (n = 8 for each), responsiveness (n = 3), patient-proxy agreement (n = 2), and test-retest reliability (n = 1). None reported on content validity. Five-level bolt-ons outperformed 3-level bolt-ons in terms of overall performance (3L: 55+/57−; 5L: 45+/28−). Supportive psychometric evidence varied by populations, eg, head/brain injury (3L: 11+/11−; 5L: 1+/3−) and dementia (3L: 9+/8−; 5L: 4+/4−). The most-tested bolt-ons were the Janssen 2013 (5L, cognition: 18+/15−) and Haagsma 2005 (3L, thinking ability: 8+/12−) versions, with fewer than 10 assessments for all other bolt-ons.

Conclusions

Despite several publications, the psychometric evidence remains insufficient to identify a preferred cognition descriptor. Future research should prioritize content validity testing to inform the selection of candidate items, with quantitative psychometric evaluation preferably conducted afterward.

目标：认知是最常用的EQ-5D附加功能，根据描述符和反应水平（3L vs. 5L），有许多不同的版本。本研究旨在系统回顾EQ-5D-3L和EQ-5D-5L认知栓接的心理测量特性。方法：根据PRISMA 2020指南（PROSPERO:CRD42023445567），在PubMed、Web of Science和b谷歌Scholar上进行系统评价。我们评估了螺栓的性能，既作为单独的项目，也添加到EQ-5D。在每个出版物中，使用检查表将每个附加版本评为积极（+）或非积极（-），并将所有出版物的分数相加以反映总体性能。结果：来自72项研究的101篇出版物符合纳入标准，检查了15个三级和13个五级螺栓。最常报告的心理测量属性是项目水平上限（n=75）和已知组效度（n=54）。较少的研究探讨了收敛效度或发散效度（各n=8）、反应性（n=3）、患者-代理协议（n=2）和测试-重测信度（n=1）。没有内容效度报告。五级螺栓的整体性能优于三级螺栓（3L: 55+/57-; 5L: 45+/28-）。支持性心理测量证据因人群而异，例如头部/脑损伤（3L: 11+/11-; 5L: 1+/3-）和痴呆（3L: 9+/8-; 5L: 4+/4-）。测试次数最多的是Janssen 2013 （5L，认知：18+/15-）和Haagsma 2005 （3L，思维能力：8+/12-）版本，其他所有版本的测试次数都少于10次。结论：尽管有一些出版物，心理测量证据仍然不足以确定首选的认知描述符。未来的研究应优先考虑内容效度测试，为候选项目的选择提供信息，之后最好进行定量心理测量评估。

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引用次数: 0

Genomic Testing in Australia: A Budget Impact Analysis Using Diffusion Modeling From a Healthcare System Perspective 基因组测试在澳大利亚：从医疗保健系统的角度使用扩散模型的预算影响分析。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-15 DOI: 10.1016/j.jval.2025.09.3067

Dylan A. Mordaunt MB, ChB, MPH , Zornitza Stark DM , Adam G. Elshaug PhD , Chris Schilling PhD

Objectives

Genomic testing can shorten the diagnostic odyssey for people with rare diseases, yet clinical uptake has lagged funding policy in Australia. Therefore, we evaluated the 10-year budget impact of alternative implementation strategies for publicly funded genomic testing using national claims data and diffusion modeling.

Methods

Monthly Medicare Benefits Schedule claims (1993–2025) were analyzed for chromosomal microarray analysis (CMA), Fragile X (FMR1) testing, and genomic tests across 7 rare-disease groups (syndromic and non-syndromic intellectual disability, neuromuscular, inherited cardiac, renal ciliopathies/tubulopathies, congenital hearing loss, mitochondrial). Logistic, Gompertz, and Bass diffusion functions and Seasonal Autoregressive Integrated Moving-Average models were fitted to uptake and used to forecast 2025 to 2034 volumes. Scenarios included status quo, broadened second-line eligibility, and first-line exome sequencing/genome sequencing (ES/GS) replacing CMA/FMR1 (60:40 ES:GS). Costs used were the 1 July 2024 Medicare Benefits Schedule fees; the results are in Australian (AUD) dollars.

Results

Observed genomic testing volumes were below diffusion-implied trajectories. The 10-year cumulative spending was as follows: status quo AUD 1.1 million; broadened second-line AUD 7.5 million (incremental +6.4 million vs status quo); and first-line ES/GS AUD 6.2 million (incremental +5.1 million). In 2028, it was status quo AUD 0.23 million, second-line AUD 1.21 million, and first-line AUD 0.97 million. ES/GS achieved lower cumulative spend than the broadened second-line despite higher per-test fees, reflecting substitution from CMA/FMR1 and efficient diagnostic pathways.

Conclusions

Indication-by-indication funding has yielded slower-than-expected uptake and likely under-budgeting. A first-line genomic testing pathway, aligned with CMA criteria, could better match clinical need while constraining spend versus expanding second-line eligibility. Harmonized eligibility and streamlined implementation would improve access and planning.

背景：基因组检测可以缩短罕见病患者的诊断过程，但临床应用滞后于澳大利亚的资助政策。我们使用国家索赔数据和扩散模型评估了公共资助基因组检测的替代实施策略的10年预算影响。方法：对每月医疗保险福利计划（MBS）索赔（1993-2025）进行染色体微阵列分析（CMA）、脆性X染色体（FMR1）检测和基因组检测，涵盖7个罕见疾病组（综合征和非综合征性智力残疾、神经肌肉、遗传性心脏、肾纤毛病/小管病、先天性听力损失、线粒体）。Logistic、Gompertz和Bass扩散函数和SARIMA拟合了吸收量，并用于预测2025-2034年的产量。场景包括：现状；扩大二线资格；一线ES/GS取代CMA/FMR1 （60:40 ES:GS）。成本使用2024年7月1日MBS时间表费用；结果以澳元表示。结果：观察到的基因组检测量低于扩散隐含轨迹。十年累计支出为：现状110万澳元；扩大二线750万澳元（比现状增加640万澳元）；一线ES/GS 620万澳元（增量+ 510万）。2028年，现状AUD 0.23m，二线AUD 121 m，一线AUD 0.97m。尽管每次检测费用较高，但ES/GS的累积支出低于扩大二线，这反映了CMA/FMR1的替代和有效的诊断途径。结论：各适应症的资金投入慢于预期，可能预算不足。与CMA标准一致的一线基因组检测途径可以更好地满足临床需求，同时限制支出，而不是扩大二线资格。统一的资格和精简的执行将改善获取和规划。

{"title":"Genomic Testing in Australia: A Budget Impact Analysis Using Diffusion Modeling From a Healthcare System Perspective","authors":"Dylan A. Mordaunt MB, ChB, MPH , Zornitza Stark DM , Adam G. Elshaug PhD , Chris Schilling PhD","doi":"10.1016/j.jval.2025.09.3067","DOIUrl":"10.1016/j.jval.2025.09.3067","url":null,"abstract":"<div><h3>Objectives</h3><div>Genomic testing can shorten the diagnostic odyssey for people with rare diseases, yet clinical uptake has lagged funding policy in Australia. Therefore, we evaluated the 10-year budget impact of alternative implementation strategies for publicly funded genomic testing using national claims data and diffusion modeling.</div></div><div><h3>Methods</h3><div>Monthly Medicare Benefits Schedule claims (1993–2025) were analyzed for chromosomal microarray analysis (CMA), Fragile X (<em>FMR1</em>) testing, and genomic tests across 7 rare-disease groups (syndromic and non-syndromic intellectual disability, neuromuscular, inherited cardiac, renal ciliopathies/tubulopathies, congenital hearing loss, mitochondrial). Logistic, Gompertz, and Bass diffusion functions and Seasonal Autoregressive Integrated Moving-Average models were fitted to uptake and used to forecast 2025 to 2034 volumes. Scenarios included status quo, broadened second-line eligibility, and first-line exome sequencing/genome sequencing (ES/GS) replacing CMA/<em>FMR1</em> (60:40 ES:GS). Costs used were the 1 July 2024 Medicare Benefits Schedule fees; the results are in Australian (AUD) dollars.</div></div><div><h3>Results</h3><div>Observed genomic testing volumes were below diffusion-implied trajectories. The 10-year cumulative spending was as follows: status quo AUD 1.1 million; broadened second-line AUD 7.5 million (incremental +6.4 million vs status quo); and first-line ES/GS AUD 6.2 million (incremental +5.1 million). In 2028, it was status quo AUD 0.23 million, second-line AUD 1.21 million, and first-line AUD 0.97 million. ES/GS achieved lower cumulative spend than the broadened second-line despite higher per-test fees, reflecting substitution from CMA/<em>FMR1</em> and efficient diagnostic pathways.</div></div><div><h3>Conclusions</h3><div>Indication-by-indication funding has yielded slower-than-expected uptake and likely under-budgeting. A first-line genomic testing pathway, aligned with CMA criteria, could better match clinical need while constraining spend versus expanding second-line eligibility. Harmonized eligibility and streamlined implementation would improve access and planning.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 3","pages":"Pages 375-382"},"PeriodicalIF":6.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145313765","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

Comparative Responsiveness of Preference-Based Health-Related Quality of Life, Social Care, and Well-Being Measures in the Context of Multiple Sclerosis 在多发性硬化症的背景下，基于偏好的健康相关生活质量、社会关怀和福利措施的比较响应性

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-03-01 Epub Date: 2025-10-14 DOI: 10.1016/j.jval.2025.09.3063

Elizabeth Goodwin PhD , Amy Heather MSc , Nia Morrish MSc , Jenny Freeman PhD , Kate Boddy MSc , Sarah Thomas PhD , Jeremy Chataway PhD , Rod Middleton PhD , Annie Hawton PhD

Objectives

To provide evidence on the responsiveness of social care and well-being preference-based measures (PBMs) compared with health-related quality of life PBMs in the context of multiple sclerosis (MS).

Methods

The ICEpop CAPability measure for Adults (ICECAP-A) and Adult Social Care Outcomes Toolkit (ASCOT) were completed online in September 2019, March 2020, September 2020, via the UK MS Register. Responses were linked to EQ-5D-3L and MS Impact Scale-8 Dimensions (MSIS-8D) values, and to MS Walking Scale-12, Hospital Anxiety and Depression Scale (HADS), and Fatigue Severity Scale scores. Responsiveness was assessed in relation to minimal important differences on MS Walking Scale-12, Hospital Anxiety and Depression Scale, and Fatigue Severity Scale between time points, using mean change scores, t tests, standardized effect sizes, standardized response means, and multivariable regression analyses.

Results

Data from 1742 people with MS were available for analysis. When using standardized values, MSIS-8D showed the greatest responsiveness and EQ-5D-3L the least. In contrast, when absolute utility values were used, EQ-5D-3L performed similarly to MSIS-8D and better than ICECAP-A and ASCOT. Standardized regression analyses indicated the MSIS-8Ds to be the most responsive, followed by the ASCOT, ICECAP-A, and EQ-5D-3L.

Conclusions

The ICECAP-A, ASCOT, and MSIS-8D were more responsive than the EQ-5D-3L in the context of MS when compared using standardized scores. The increased responsiveness of EQ-5D-3L when absolute values were used seems an artefact of the wide-ranging scale of this measure. This illustrates how the maximum potential range of values for a given PBM tariff could influence whether an intervention is found to be cost-effective.

目的：在多发性硬化症（MS）的背景下，与健康相关的生活质量PBMs相比，提供社会关怀和基于健康偏好的措施（PBMs）的响应性的证据。方法：通过英国MS Register于2019年9月、2020年3月和2020年9月在线完成成人icpop能力量表（ICECAP-A）和成人社会护理结果工具包（ASCOT）。反应与EQ-5D-3L和MS影响量表-8维度（MSIS-8D）值、MS步行量表-12 （msw -12）、医院焦虑和抑郁量表（HADS）和疲劳严重程度量表（FSS）得分相关联。使用平均变化评分、t检验、标准化效应量、标准化反应均值和多变量回归分析，评估与时间点之间MSWS-12、HADS和FSS的最小重要差异相关的响应性。结果：来自1742名多发性硬化症患者的数据可供分析。当使用标准值时，MSIS-8D表现出最大的响应性，EQ-5D-3L表现出最小的响应性。相比之下，当使用绝对效用值时，EQ-5D-3L的表现与MSIS-8D相似，优于ICECAP-A和ASCOT。标准化回归分析表明，msis - 8d反应最积极，其次是ASCOT、ICECAP-A和EQ-5D-3L。结论：在使用标准化评分进行比较时，ICECAP-A、ASCOT和MSIS-8D比EQ-5D-3L对MS的反应更敏感。当使用绝对值时，EQ-5D-3L的响应性增加似乎是该测量范围广泛的人工产物。这说明了给定PBM关税的最大潜在值范围如何影响干预措施是否具有成本效益。

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引用次数: 0