Value in Health最新文献_第4页

Developing Meaningful Score Differences for the Bayley-4 and Vineland-3 in Angelman Syndrome using a Delphi Panel. 利用德尔菲面板对Angelman综合征的Bayley-4和Vineland-3进行有意义的评分差异研究。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-05 DOI: 10.1016/j.jval.2025.11.021

Sonya Powers, Katherine N Anderson, Wen-Hann Tan, Julian Tillmann, Mark Daniel, Audrey Thum, Cristan Farmer, Susanne Clinch, Anne C Wheeler, Anjali Sadhwani

Objectives: To develop within-patient meaningful score differences (MSDs) on the Bayley Scales of Infant Development, Fourth Edition (Bayley-4), and the Vineland Adaptive Behavior Scales, Third Edition (Vineland-3), for individuals with Angelman syndrome (AS).

Methods: A Delphi method, involving a panel of 19 caregivers of individuals with AS, was used to establish MSDs for Bayley-4 and Vineland-3 Growth Scale Values. MSD was defined as the smallest change that would noticeably impact the daily functioning of an individual with AS or family quality of life in a way that was important to the individual with AS or their family. For each subscale of the Bayley-4 and Vineland-3, the panel was presented with 2 to 4 vignettes describing varying levels of baseline functioning and asked to select a MSD from a range of potential values. An iterative process involving three rounds of ratings and two rounds of discussion was used to build consensus. The median caregiver rating from round 3 was retained as the final recommended MSD value for each vignette.

Results: Final MSD ratings for the five subscales of Bayley-4 and 10 subscales of the Vineland-3 had an agreement rate of 70% or higher. MSD thresholds for each subscale were not single cut-offs, but rather reflected a range of MSD values dependent on level of baseline functioning.

Conclusions: The Delphi Panel method incorporates the caregiver perspective to provide preliminary estimates of what constitutes meaningful within-person change on the Bayley-4 and Vineland-3 in individuals with AS with various levels of baseline functioning.

目的：研究Angelman综合征（AS）患者在Bayley婴儿发育量表第四版（Bayley-4）和Vineland适应行为量表第三版（Vineland-3）上的患者内有意义评分差异（MSDs）。方法：采用德尔菲法，对19名AS患者的照顾者进行问卷调查，建立Bayley-4和Vineland-3生长量表值的MSDs。MSD被定义为能够显著影响as患者日常功能或家庭生活质量的最小变化，这种变化对as患者或其家庭非常重要。对于Bayley-4和Vineland-3的每个子量表，专家组都有2到4个描述基线功能不同水平的小图，并被要求从一系列潜在值中选择一个MSD。一个涉及三轮评级和两轮讨论的迭代过程被用来建立共识。第3轮的中位数护理人员评分保留为每个小插曲的最终推荐MSD值。结果：Bayley-4的5个子量表和Vineland-3的10个子量表的最终MSD评分的一致性率为70%或更高。每个子量表的MSD阈值不是单一的截止值，而是反映了依赖于基线功能水平的MSD值范围。结论：德尔菲小组方法结合了护理者的观点，对具有不同基线功能水平的AS患者的Bayley-4和Vineland-3的有意义的个人内部变化提供了初步估计。

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引用次数: 0

Pharmaceutical Pricing Evidence From a Healthcare System With Multiple Cost-per-Quality-Adjusted Life-Year Thresholds 具有多重质量成本阈值的医疗保健系统的药品定价证据。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.07.027

Anton P.H. Klockhoff MSc , Jonathan Siverskog PhD , Martin Henriksson PhD

Objectives

To study drug pricing in a reimbursement system accepting a higher cost per quality-adjusted life-year for drugs targeting conditions of higher severity. We investigate whether higher incremental cost-effectiveness ratios (ICERs) are observed for conditions of higher severity, how close to the highest acceptable cost-per-quality-adjusted life-year threshold drug prices are set, and whether the thresholds are applied as price ceilings. Furthermore, we explore factors other than severity that might affect pricing.

Methods

Disease severity and ICERs were extracted from publicly available reimbursement decisions made between 2017 and 2024 by the Swedish Dental and Pharmaceutical Benefits Agency. Linear regression assessed whether ICERs increase significantly with severity and other covariates and whether the same covariates explain deviations from the highest acceptable ICERs. We also assessed whether higher severity was associated with a higher likelihood of having confidential rebates in place.

Results

A total of 84 decisions reported ICERs and disease severity. ICERs increased significantly with severity (P < .01). The average ICER was 30.6% lower than the relevant threshold, and there was no evidence of pricing above the thresholds. Very high severity was associated with a 27.4% higher probability than moderate severity of the ICER including confidential rebates (P < .01). ICERs including rebates were associated with 19.1 percentage points smaller deviations from the threshold (P < .01).

Conclusions

Prices tend to be set below the reimbursement system’s thresholds and these appear to be ceilings, but decision makers should anticipate that drug companies will adjust prices in response to reimbursement policy, at least to some degree.

目的：研究在一个接受更高质量调整生命年（QALY）成本的报销系统中，针对更严重疾病的药物定价。我们调查了是否在更严重的情况下观察到更高的增量成本-效果比（ICERs），设定的药品价格与最高可接受的每质量成本阈值有多接近，以及阈值是否被用作价格上限。此外，我们探索了可能影响定价的其他因素。方法：从瑞典牙科和药品福利局（TLV） 2017年至2024年公开可获得的报销决定中提取疾病严重程度和ICERs。线性回归评估ICERs是否随严重程度和其他协变量显著增加，以及相同的协变量是否解释了与最高可接受ICERs的偏差。我们还评估了是否较高的严重性与较高的可能性有保密回扣到位。结果：84例患者报告了ICERs和疾病严重程度。结论：价格往往被设定在报销制度的阈值以下，这些阈值似乎是上限，但决策者应该预料到，制药公司至少会在某种程度上根据报销政策调整价格。

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引用次数: 0

Cost-Effectiveness of the 15 Drugs Selected for Initial Price Applicability Year 2027 by Centers for Medicare and Medicaid Services Drug Price Negotiation Program IPAY 2027 CMS药品价格谈判选择的15种药品成本效果分析

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.019

Feng Xie PhD , Ting Zhou PhD , Yue Ma MSc , Joshua T. Cohen PhD , Peter J. Neumann ScD

Objectives

To analyze published cost-effectiveness analyses (CEAs) for the 15 drugs selected for the Initial Price Applicability Year 2027 by the Centers for Medicare and Medicaid Services.

Methods

We identified CEAs for the 15 drugs from the Tufts Cost-Effectiveness Analysis Registry. For each drug, we included all base-case incremental cost-effectiveness ratios (ICERs) and calculated median and interquartile ranges for positive ICERs for each drug indication. We also performed the analyses on CEAs conducted in the United States, which used a lifetime horizon, or were industry sponsored. We present all costs in 2022 US dollars.

Results

A total of 20 drug-indications were included in the analysis. Most studies used a nonsocietal perspective and were industry sponsored. Median positive ICERs ranged from $1800 (linagliptin) to $640 000 (acalabrutinib) with 6 drug indications having a median ICER less than $50 000 and 10 more than $100 000. Among the 13 drug indications with US-based CEAs, 11 had a median ICER near or more than $100 000. Among CEAs with a lifetime horizon, 7 drug indications had a median ICER less than $50 000, and 5 more than $100 000. CEAs sponsored by industry had median ICERs more than $100 000 for 5 drug indications. The median ICERs differed considerably between the 2 covered indications for semaglutide, linaclotide, and rifaximin.

Conclusions

As Centers for Medicare and Medicaid Services continues to implement its drug price negotiation program, promoting greater transparency and consistency in how value is defined and applied to pricing decisions will be essential. CEA can serve as an important source of evidence, ensuring that negotiated prices more closely reflect the benefits drugs provide.

目的：分析美国医疗保险和医疗补助服务中心（CMS）为2027年初始价格适用年选择的15种药物已发表的成本-效果分析（CEAs）。方法：我们从塔夫茨成本-效果分析注册表中确定15种药物的cea。对于每种药物，我们纳入了所有基本病例增量成本效益比（ICERs），并计算了每种药物适应症ICERs阳性的中位数和四分位数范围。我们还对在美国进行的cea进行了分析，这些分析使用了生命周期，或者是由行业赞助的。我们以2022美元表示所有成本。结果：共纳入20个药物适应症。大多数研究采用非社会视角，由行业赞助。中位阳性ICERs从1800美元（利格列汀）到64万美元（阿卡拉布替尼）不等，有6种药物适应症的中位ICERs低于5万美元，10种高于10万美元。在美国CEAs的13种药物适应症中，11种的中位ICER接近或高于10万美元。在有生命周期的cea中，7个药物适应症的ICER中位数低于5万美元，5个高于10万美元。行业赞助的cea的5种药物适应症的ICERs中位数超过10万美元。西马鲁肽、利那洛肽和利福昔明这两种覆盖适应症的中位ICERs差异很大。结论：随着CMS继续实施其药品价格谈判计划，提高价值定义和定价决策的透明度和一致性将是至关重要的。CEA可以作为重要的证据来源，确保谈判价格更密切地反映药物提供的益处。

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引用次数: 0

Health Inequality Aversion in the United States 美国对健康不平等的厌恶。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.015

Julia F. Slejko PhD , Salome Ricci PharmD, MS , Susan dosReis PhD , Richard Cookson PhD , Stacey Kowal MSc

Objectives

Health inequality aversion parameters are used in distributional cost-effectiveness analysis, direct equity-based weighting to reflect societal preferences for improving total health (“efficiency”), and reducing health inequality between more and less socially advantaged groups (“equity”). We elicited a health inequality aversion parameter for the US population.

Methods

We adapted a benefit trade-off (BTO) instrument used in a UK study. Participants comprised the adult general public from June to December 2023. The online survey comprised (1) demographics and health views questions, (2) instructional videos, and (3) BTO exercise. The BTO asked participants to trade off quality-adjusted life expectancy from the better-off to worse-off quintiles of the US population, described by indicators of social vulnerability. Response patterns were classified into 15 ranks with corresponding inequality aversion parameters and implied equity weights.

Results

Among 1864 complete responses, inequality aversion was assessed for 1290 participants. The sample approximated US Census data for gender, race/ethnicity, and income. The median Atkinson parameter was 12.12, the corresponding equity weight was 6.7, and 88% were willing to trade off total health to reduce health inequality. Multivariable regression indicated no significant subgroup variation in trade-off responses by age or region; however, lower income groups and ethnic minority groups were slightly more averse to health inequality.

Conclusions

The inequality aversion statistics derived from this sample illustrate support for more robust and routine integration of equity concerns into healthcare decisions in the policy and health technology assessment arenas to advance distributional cost-effectiveness analysis in the United States.

目的：在分配成本效益分析（DCEA）和直接基于公平的加权中使用健康不平等厌恶参数，以反映社会对改善总体健康（“效率”）和减少更多和较少社会优势群体之间的健康不平等（“公平”）的偏好。我们为美国引入了健康不平等厌恶参数（IAP）。方法：我们采用了英国研究中使用的利益权衡（BTO）工具。参加者为成年市民，时间为2023年6月至12月。在线调查包括1)人口统计和健康观点问题，2)教学视频，3)BTO练习。BTO要求参与者根据社会脆弱性指标，在美国人口中按质量调整的预期寿命，从较富裕的五分之一到较贫穷的五分之一之间进行权衡。根据相应的iap和隐含权益权重，将反应模式分为15个等级。结果：在1864份完整的问卷中，1290名参与者对不平等厌恶程度进行了评估。样本在性别、种族/民族和收入方面近似于美国人口普查数据。Atkinson参数中位数为12.12；相应的权益权重为6.7；88%的人愿意牺牲整体健康来减少健康不平等。多变量回归表明，不同年龄或地区的权衡反应没有显著的亚组差异，但低收入群体和少数民族群体对健康不平等的厌恶程度略高。结论：从该样本中得出的不平等厌恶统计数据表明，支持在政策和卫生技术评估领域将公平问题更稳健和常规地整合到医疗保健决策中，以推进美国的DCEA。

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引用次数: 0

Unlocking Patient Preferences: The Potential of Stated-Preference Methods in Clinical Decision Making 解锁患者偏好：状态偏好方法在临床决策中的潜力。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.016

Semra Ozdemir PhD , Jorien Veldwijk PhD , Janine van Til PhD , Ilene L. Hollin PhD , Deborah A. Marshall PhD , Shelby D. Reed PhD

Stated-preference (SP) methods are gaining attention as tools to support patient-centered clinical decision making by quantifying individual preferences through structured tradeoffs. These methods may improve shared decision making by helping patients and clinicians better understand the relative importance of treatment attributes and the trade-offs patients are willing to make. However, integrating SP methods into clinical settings poses practical challenges, including concerns about complexity, patient burden, and relevance of hypothetical scenarios. Successful application will require thoughtful design, attention to accessibility, and collaboration across disciplines. Despite current limitations, SP methods hold promise for enhancing the quality and alignment of medical decisions with patient values.

状态偏好（SP）方法作为一种支持以患者为中心的临床决策的工具，通过结构化的权衡来量化个人偏好。这些方法可以帮助患者和临床医生更好地理解治疗属性的相对重要性以及患者愿意做出的权衡，从而改善共同决策。然而，将SP方法整合到临床环境中存在实际挑战，包括对复杂性、患者负担和假设情景相关性的担忧。成功的应用程序需要深思熟虑的设计、对可访问性的关注以及跨学科的协作。尽管目前存在局限性，但SP方法有望提高医疗决策的质量，并与患者的价值观保持一致。

引用次数: 0

Application of International Reference Pricing Rules to Forecast Pharmaceutical Launch Prices in 5 European Countries 国际参考定价规则在欧洲五国药品上市价格预测中的应用。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.003

Milena Izmirlieva MSc, MS

Objectives

To assess whether applying official International Reference Pricing (IRP) rules allows accurate prediction of pharmaceutical launch prices in Austria, Bulgaria, Croatia, The Netherlands, and North Macedonia.

Methods

Official pre-2019-reform IRP regulations were examined, with 1 Dutch rule further clarified via primary research. IRP rules were applied precisely to calculate the maximum price under IRP for all new chemical/molecular entities presentations first priced in each country in 2018 and customarily subject to IRP, using POLI pricing and reimbursement data for the referrer and the reference countries. Maximum allowed launch prices under IRP were calculated by using prices available for referencing 1 month before launch, the regulation-specified exchange rate and the definition of what constitutes an acceptable product for referencing. If an identical pack size/formulation was not available or multiple suitable products for referencing existed, the appropriate conversion rules were applied as stated in the IRP regulations. The actual first price was compared with the price calculated under IRP for each new chemical or molecular entity presentation, with several scenarios run to explain discrepancies.

Results

The mean absolute percentage error (MAPE) between the actual first price and the forecasted/IRP-based price across the sample was lowest for Bulgaria (3.04%), followed by The Netherlands (4.53%), Austria (4.91%), Croatia (9.63%), and North Macedonia (22.09%). North Macedonia’s high MAPE is because the country allows prices to exceed the IRP-based price by up to 20%. MAPE < 10% indicates outstanding model performance.

Conclusions

Precise IRP rules application can accurately predict launch prices in countries where IRP is binding and is the main price-setting method.

目的：评估应用官方国际参考定价（IRP）规则是否可以准确预测奥地利、保加利亚、克罗地亚、荷兰和北马其顿的药品上市价格。方法：审查2019年改革前的官方IRP法规，并通过初步研究进一步澄清了一项荷兰规则。IRP规则被精确地应用于计算IRP下的所有新化学/分子实体产品的最高价格，这些产品在2018年首次在每个国家定价，并且通常受IRP的约束，使用POLI定价和参考国家和参考国家的报销数据。IRP下允许的最高推出价格是根据推出前一个月可供参考的价格、规定的汇率和可供参考的可接受产品的定义来计算的。如果没有相同的包装尺寸/配方，或有多种合适的产品可供参考，则适用IRP条例所述的适当转换规则。实际的第一次价格与每次NCE展示的IRP下计算的价格进行了比较，并运行了几个场景来解释差异。结果：保加利亚的实际首次价格与基于irp的预测价格之间的平均绝对百分比误差（MAPE）最低（3.04%），其次是荷兰（4.53%）、奥地利（4.91%）、克罗地亚（9.63%）和北马其顿（22.09%）。北马其顿的高MAPE是因为该国允许价格超过irp基础价格的20%。结论：在有IRP约束的国家，精确应用IRP规则可以准确预测上市价格，是主要的定价方法。

{"title":"Application of International Reference Pricing Rules to Forecast Pharmaceutical Launch Prices in 5 European Countries","authors":"Milena Izmirlieva MSc, MS","doi":"10.1016/j.jval.2025.08.003","DOIUrl":"10.1016/j.jval.2025.08.003","url":null,"abstract":"<div><h3>Objectives</h3><div>To assess whether applying official International Reference Pricing (IRP) rules allows accurate prediction of pharmaceutical launch prices in Austria, Bulgaria, Croatia, The Netherlands, and North Macedonia.</div></div><div><h3>Methods</h3><div>Official pre-2019-reform IRP regulations were examined, with 1 Dutch rule further clarified via primary research. IRP rules were applied precisely to calculate the maximum price under IRP for all new chemical/molecular entities presentations first priced in each country in 2018 and customarily subject to IRP, using POLI pricing and reimbursement data for the referrer and the reference countries. Maximum allowed launch prices under IRP were calculated by using prices available for referencing 1 month before launch, the regulation-specified exchange rate and the definition of what constitutes an acceptable product for referencing. If an identical pack size/formulation was not available or multiple suitable products for referencing existed, the appropriate conversion rules were applied as stated in the IRP regulations. The actual first price was compared with the price calculated under IRP for each new chemical or molecular entity presentation, with several scenarios run to explain discrepancies.</div></div><div><h3>Results</h3><div>The mean absolute percentage error (MAPE) between the actual first price and the forecasted/IRP-based price across the sample was lowest for Bulgaria (3.04%), followed by The Netherlands (4.53%), Austria (4.91%), Croatia (9.63%), and North Macedonia (22.09%). North Macedonia’s high MAPE is because the country allows prices to exceed the IRP-based price by up to 20%. MAPE < 10% indicates outstanding model performance.</div></div><div><h3>Conclusions</h3><div>Precise IRP rules application can accurately predict launch prices in countries where IRP is binding and is the main price-setting method.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 1","pages":"Pages 73-81"},"PeriodicalIF":6.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144875442","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

The Cost-Effectiveness of Collagenase Injection Versus Limited Fasciectomy for Moderate Dupuytren’s Contracture: An Economic Evaluation of the Dupuytren’s Interventions Surgery Versus Collagenase Trial and a Decision Analytical Model 胶原酶注射与有限筋膜切除术治疗中度双膝挛缩的成本-效果：DISC试验的经济评估和决策分析模型。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.07.030

Qi Wu MSc , Catherine Arundel MSc , Charlie Welch MMath , Puvanendran Tharmanathan PhD , Nick Johnson PhD , Belen Corbacho MSc , Joseph J. Dias MD

Objectives

To compare the cost-effectiveness of collagenase injection (collagenase) and limited fasciectomy (LF) surgery in treating moderate Dupuytren’s contracture (DC) in the United Kingdom over different time horizons.

Methods

An incremental cost-effectiveness analysis was conducted alongside a multicenter, pragmatic, parallel randomized controlled trial (Dupuytren’s Interventions: Surgery versus Collagenase trial), to determine the short-term cost-effectiveness of collagenase compared with LF. A Markov decision analytic model was developed to assess long-term cost-effectiveness.

Results

Collagenase was associated with significantly lower cost and insignificantly lower quality-adjusted life-year (QALY) gain compared with LF at 1 year. The probability of collagenase being cost-effective was more than 99% at willingness-to-pay thresholds of £20 000 to £30 000 per QALY. At 2 years, collagenase was both significantly less costly and less effective compared with LF, and LF became cost-effective above a threshold of £25 488. There was a high level of uncertainty surrounding the 2-year results. Over a lifetime horizon, collagenase generated a cost saving of £2968 per patient but was associated with a mean QALY loss of −0.484. The probability of collagenase being cost-effective dropped to 22% and 16% at £20 000 to £30 000 per QALY, respectively.

Conclusions

Collagenase was less costly and less effective than LF in treating Dupuytren’s contracture. The cost-effectiveness of collagenase compared with LF was time dependent. Collagenase was highly cost-effective 1-year after treatment; however, the probability of collagenase being cost-effective declined over time. The Markov model suggested that LF is more cost-effective over a lifetime horizon. These findings emphasize the importance of longer follow-up when comparing surgical and nonsurgical interventions to fully capture overall costs and benefits.

目的：比较胶原酶注射（胶原酶）和有限筋膜切除术（LF）手术在不同时间范围内治疗中度Dupuytren's挛缩（DC）的成本-效果。方法：一项增量成本-效果分析与一项多中心、实用、平行随机对照试验（DISC）一起进行，以确定胶原酶与LF相比的短期成本-效果。建立了马尔可夫决策分析模型来评估长期成本效益。结果：与一年的胶原酶相比，胶原酶与较低的成本和较低的质量调整生命年（QALY）增益相关。在每个QALY的支付意愿阈值为20,000- 30,000英镑时，胶原酶具有成本效益的可能性超过99%。两年后，与LF相比，胶原酶的成本和效果都明显更低，超过25,488英镑的门槛后，LF变得具有成本效益。这两年的研究结果存在很大的不确定性。在患者的一生中，使用胶原酶可为每位患者节省2,968英镑的成本，但其QALY的平均损失为-0.484。在每个QALY花费2万至3万英镑时，胶原酶具有成本效益的概率分别降至22%和16%。结论：胶原酶治疗DC的成本较低，效果较低。与LF相比，胶原酶的成本-效果依赖于时间。治疗一年后，胶原酶具有很高的成本效益，但随着时间的推移，胶原酶具有成本效益的可能性下降。马尔可夫模型表明，在生命周期内，LF更具成本效益。这些发现强调了在比较手术和非手术干预措施时延长随访时间的重要性，以充分了解总体成本和收益。

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引用次数: 0

Predicting Healthcare Utilization Outcomes With Artificial Intelligence: A Large Scoping Review 用人工智能预测医疗保健利用结果：大范围回顾。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.007

Carlos Gallego-Moll MSc , Lucía A. Carrasco-Ribelles PhD , Marc Casajuana MSc , Laia Maynou PhD , Pablo Arocena PhD , Concepción Violán MD, PhD , Edurne Zabaleta-del-Olmo PhD

Objectives

To broadly map the research landscape to identify trends, gaps, and opportunities in data sets, methodologies, outcomes, and reporting standards for artificial intelligence (AI)-based healthcare utilization prediction.

Methods

We conducted a scoping review following the Joanna Briggs Institute methodology. We searched 3 major international databases (from inception to January 2025) for studies applying AI in predictive healthcare utilization. Extracted data were categorized into data sets characteristics, AI methods and performance metrics, predicted outcomes, and adherence to the Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) + AI reporting guidelines.

Results

Among 1116 records, 121 met inclusion criteria. Most were conducted in the United States (62%). No study incorporated all 6 relevant variable groups: demographic, socioeconomic, health status, perceived need, provider characteristics, and prior utilization. Only 7 studies included 5 of these groups. The main data sources were electronic health records (60%) and claims (28%). Ensemble models were the most frequently used (66.9%), whereas deep learning models were less common (16.5%). AI methods were primarily used to predict future events (90.1%), with hospitalizations (57.9%) and visits (33.1%) being the most predicted outcomes. Adherence to general reporting standards was moderate; however, compliance with AI-specific TRIPOD + AI items was limited.

Conclusions

Future research should broaden predicted outcomes to include process- and logistics-oriented events, extend applications beyond prediction—such as cohort selection and matching—and explore underused AI methods, including distance-based algorithms and deep neural networks. Strengthening adherence to TRIPOD-AI reporting guidelines is also essential to enhance the reliability and impact of AI in healthcare planning and economic evaluation.

目的：广泛地描绘研究前景，以确定基于人工智能的医疗保健利用预测的数据集、方法、结果和报告标准方面的趋势、差距和机会。方法：我们按照乔安娜布里格斯研究所的方法进行了范围审查。我们检索了三个主要的国际数据库（从成立到2025年1月），寻找将人工智能应用于预测性医疗保健利用的研究。提取的数据分为数据集特征、人工智能方法和性能指标、预测结果以及对TRIPOD+AI报告指南的遵守情况。结果：1116例病例中，符合纳入标准的121例。大多数是在美国进行的（62%）。没有一项研究纳入了所有六个相关变量组：人口统计学、社会经济、健康状况、感知需求、提供者特征和既往使用情况。只有7项研究包括了其中的5个群体。主要数据来源是电子健康记录（60%）和索赔（28%）。集成模型是最常用的（66.9%），而深度学习模型则不太常用（16.5%）。人工智能方法主要用于预测未来事件（90.1%），其中住院（57.9%）和就诊（33.1%）是预测最多的结果。对一般报告标准的遵守程度中等，但对AI特定TRIPOD+AI项目的遵守程度有限。结论：未来的研究应扩大预测结果，包括以过程和物流为导向的事件，将应用扩展到预测之外，如队列选择和匹配，并探索未充分利用的人工智能方法，包括基于距离的算法和深度神经网络。加强对TRIPOD-AI报告准则的遵守对于提高AI在医疗保健规划和经济评估中的可靠性和影响也至关重要。

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引用次数: 0

Adult Social Care Outcomes Toolkit and ICEpop Capability Measure in Decision Making: A Review of NICE Social Care and Public Health Guidelines ASCOT和ICECAP在决策中的作用：NICE社会关怀和公共卫生指南综述

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.021

Zhixin Zhang MSc , Tuba Saygın Avşar PhD , Sophie Cooper BSc , Jeremy Dietz MSc

Objectives

When the National Institute for Health and Care Excellence (NICE) assesses whether interventions in health and social care offer value for money, where possible, it considers health effects expressed in quality-adjusted life-years. NICE’s preferred measure of health-related quality of life is EQ-5D. For nonhealth effects, NICE cites Adult Social Care Outcomes Toolkit (ASCOT) and ICEpop Capability Measure (ICECAP) as possible outcomes. To date, to our knowledge, their use in NICE guidelines has not been reviewed. The objectives of this study were to (1) review how ASCOT and ICECAP have been used in NICE social care and public health guideliens and (2) contextualize the review via expert interviews.

Methods

NICE social care and public health guidelines published before August 26, 2025 were reviewed, and information on the use of ASCOT and ICECAP was extracted. Five experts were interviewed to contextualize the review findings.

Results

Of the eligible guidelines, ASCOT appeared as an outcome in 4% and ICECAP in 1%. Neither measure significantly affected committee’s decision making. Interview findings were grouped into 2 themes: (1) reasons behind the limited use of these measures (with 3 subthemes: conceptual, system-wide issues, and implementation challenges) and (2) ongoing developments and future opportunities.

Conclusions

ASCOT and ICECAP appeared infrequently in the NICE guidelines reviewed, and when used, their impact on committee decision making was limited—either because of trial-specific limitations or reliance on other forms of evidence. Experts suggested several barriers to the use of these measures, and although these barriers are not insurmountable, it is unclear whether such measures may appear more in future NICE social care and public health guidelines.

背景：当国家健康和护理卓越研究所（NICE）评估健康和社会护理干预措施是否物有所值时，在可能的情况下，它会考虑以质量调整生命年（QALYs）表示的健康影响。NICE对健康相关生活质量的首选测量是EQ-5D。对于非健康影响，NICE将ASCOT和ICECAP列为可能的结果。迄今为止，它们在NICE指南中的使用尚未被审查。目的：本研究的目的是1)回顾ASCOT和ICECAP在NICE社会护理和公共卫生指南中的应用情况，2)通过专家访谈将回顾置于背景中。方法：回顾2025年8月26日之前发布的NICE社会护理和公共卫生指南，提取有关ASCOT和ICECAP使用的信息。五位专家接受了采访，以了解审查结果的背景。结果：在符合条件的指南中，ASCOT作为结局出现的比例为4%，ICECAP为1%。这两项措施都没有显著影响委员会的决策。访谈结果分为两个主题：(1)这些措施有限使用背后的原因（有3个分主题：概念、全系统问题和实施挑战）；(2)持续的发展和未来的机会。结论：ASCOT和ICECAP在NICE审查的指南中很少出现，当使用时，它们对委员会决策的影响是有限的，要么是由于试验特定的限制，要么是依赖于其他形式的证据。专家们提出了使用这些措施的几个障碍，尽管这些障碍并非不可克服，但尚不清楚这些措施是否会更多地出现在未来的NICE社会护理和公共卫生指南中。

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引用次数: 0

Methodological Insights From Health Valuation Studies in Low- and Middle-Income Countries: A Scoping Review 中低收入国家健康评估研究的方法学见解：范围审查。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.014

Thomas Gadsden PhD , Janine Verstraete PhD , Audrey Moyo MPhil , Stanley Carries PhD , Nokwanda Sithole BA , Eugene Lee Davids PhD , Donela Besada PhD , Blake Angell PhD , Stephen Jan PhD , Darshini Govindasamy PhD

Objectives

Valuation studies generate utility values for health states using stated preference methods. These studies are complex and resource-intensive, posing implementation challenges in low- and middle-income countries (LMICs). This review aimed to examine how valuation studies in LMICs have navigated these challenges.

Methods

A scoping review was conducted, with database (MEDLINE, EMBASE, and CINAHL) and gray literature searches performed between April and June 2024. Inclusion was limited to valuation studies in LMICs using generic or disease-specific instruments and stated preference techniques with adult respondents and published in English. Results were narratively synthesized.

Results

A total of 36 studies from 22 LMICs were included. Studies were conducted across low (n = 2), lower-middle (n = 11), and upper middle-income countries (n = 9). Half were published since 2020. There were 33 studies that developed nationally representative value sets, 2 of which were based on patient preferences. Two pilot studies and 1 cancer-specific value set were also included. The EQ-5D-5L was used most (n = 16), followed by the EQ-5D-3L (n = 14), Short Form-6 Dimension (n = 4), Chinese medicine Quality of life-11 Dimensions (n = 1), and European Organization for Research and Treatment of Cancer-8 dimension (n = 1). Methodological adaptations included “lite” protocols, portable tools, and crosswalk methodology. Comprehension aids were reported in 11 studies; 5 included illiterate participants, and 7 were conducted in multiple languages.

Conclusions

Valuation studies are increasing rapidly in LMICs, and there is growing experimentation to reduce resource demands and enhance inclusivity. Although this is promising, the resource demands of valuation studies still limit their implementation in low-income settings. Therefore, these countries may still find it more cost-efficient to adapt value sets from neighboring countries rather than develop their own.

目的：评估研究使用既定偏好方法生成健康状态的效用值。这些研究复杂且资源密集，对低收入和中等收入国家的实施构成挑战。本综述旨在探讨中低收入国家的估值研究如何应对这些挑战。方法：采用数据库（MEDLINE、EMBASE和CINAHL）和灰色文献检索（2024年4 - 6月）进行范围综述。纳入的研究仅限于在低收入和中等收入国家进行的评估研究，这些研究使用通用或特定疾病的工具和成人受访者的陈述偏好技术，并以英语发表。对结果进行叙述性综合。结果：纳入了来自22个低收入国家的36项研究。研究在低收入国家（n=2）、中低收入国家（n=11）和中高收入国家（n=9）进行。其中一半是在2020年以后出版的。33项研究开发了具有全国代表性的价值集，其中两项基于患者的偏好。还包括两项试点研究和一项癌症特异性值集。使用最多的是EQ-5D-5L (n=16)，其次是EQ-5D-3L （n=14）、SF-6D （n=4）、CQ-11D （n=1）和EORTC-8D （n=1）。方法上的调整包括“精简”协议、便携式工具和人行横道方法。11项研究报告了理解辅助工具；其中5个是文盲，7个是用多种语言进行的。结论：估值研究在中低收入国家迅速增加，减少资源需求和增强包容性的实验也越来越多。虽然这是有希望的，但估价研究的资源需求仍然限制了它们在低收入环境中的执行。因此，各国可能仍会发现，采用邻国的价值体系比开发自己的价值体系更具成本效益。

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