首页 > 最新文献

Value in Health最新文献

英文 中文
A UK value set for the WAItE.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.002
Tomos Robinson, Sarah Hill, Yemi Oluboyede

Objectives: The Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE) is a weight-specific patient reported outcome measure for use in adolescence, consisting of seven domains, each with five response levels. The objective of this study was to generate a UK value set for the WAItE, enabling the calculation of utility values.

Methods: An online Discrete Choice Experiment (DCE) completed by an adult sample representative to the working population of the UK was used to estimate the preferences for the five levels of the seven domains. DCE data were analysed using Multinomial and Mixed Logit models. The latent values were then anchored onto the 0-1 death-full health Quality Adjusted Life Year (QALY) scale using two different anchoring techniques, the Time Trade Off (TTO) method and the DCE-Visual Analogue Scale (DCE-VAS) method.

Results: 1,004 adults from the UK were included in the final estimation sample for the DCE. From the latent estimates, the majority of the levels of the dimensions followed the monotonic nature of the WAItE, however some levels of the 'Tiredness', 'Walking' and 'Sports' related dimensions were not monotonic and combined to generate the final value set. The results from the TTO and DCE-VAS anchoring methods were similar, with values for the 'PITS state' (the worst health state possible from the WAItE) of 0.289 and 0.230.

Conclusions: This study has developed a value set for the WAItE based on the preferences of the UK population, enabling the use of the WAItE in cost utility analyses of interventions targeting obesity in adolescents.

{"title":"A UK value set for the WAItE.","authors":"Tomos Robinson, Sarah Hill, Yemi Oluboyede","doi":"10.1016/j.jval.2025.01.002","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.002","url":null,"abstract":"<p><strong>Objectives: </strong>The Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE) is a weight-specific patient reported outcome measure for use in adolescence, consisting of seven domains, each with five response levels. The objective of this study was to generate a UK value set for the WAItE, enabling the calculation of utility values.</p><p><strong>Methods: </strong>An online Discrete Choice Experiment (DCE) completed by an adult sample representative to the working population of the UK was used to estimate the preferences for the five levels of the seven domains. DCE data were analysed using Multinomial and Mixed Logit models. The latent values were then anchored onto the 0-1 death-full health Quality Adjusted Life Year (QALY) scale using two different anchoring techniques, the Time Trade Off (TTO) method and the DCE-Visual Analogue Scale (DCE-VAS) method.</p><p><strong>Results: </strong>1,004 adults from the UK were included in the final estimation sample for the DCE. From the latent estimates, the majority of the levels of the dimensions followed the monotonic nature of the WAItE, however some levels of the 'Tiredness', 'Walking' and 'Sports' related dimensions were not monotonic and combined to generate the final value set. The results from the TTO and DCE-VAS anchoring methods were similar, with values for the 'PITS state' (the worst health state possible from the WAItE) of 0.289 and 0.230.</p><p><strong>Conclusions: </strong>This study has developed a value set for the WAItE based on the preferences of the UK population, enabling the use of the WAItE in cost utility analyses of interventions targeting obesity in adolescents.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068296","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
A Value Set for the EQ-5D-5L for United Arab Emirates.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.003
Fatima Al Sayah, Bram Roudijk, Mohamed El Sadig, Asma Al Mannaei, Mohamad N Farghaly, Sara Dallal, Rima Kaddoura, Mirna Metni, Iffat Elbarazi, Samer A Kharroubi

Objectives To develop a value set for the EQ-5D-5L based on preferences of the general adult population of the United Arab Emirates (UAE). Methods The study followed the EuroQol EQ-5D-5L Valuation protocol and involved conducting interviewer-administered face-to-face or online interviews in Arabic or English, using the EuroQol Valuation Technology (EQ-VT) with a sample of 1005 adults representing the UAE general population. Sample recruitment involved a two-stage quota sampling strategy across the seven Emirates of the UAE, ensuring representation of nationals and expatriates. Various models using composite time-trade-off (cTTO) data only, discrete choice experiment (DCE) data only, and hybrid using both cTTO and DCE data were examined, along with various sensitivity analyses to examine the robustness of the models. Results Average age of respondents was 39 years (SD=10.8), 44.5% were female, and 11% were UAE nationals. The best performing model to generate the value set for the EQ-5D-5Lwas the hybrid Tobit model censored at -1.0, corrected for heteroskedasticity. Values ranged from - 0.654 for the worst health state (55555) to 1 for full health (11111) and 0.962 for 11211, with 15.3% of predicted values worse than dead. Mobility problems had the largest impact on health state preference values relative to other dimensions. Conclusion This value set will facilitate the application and use of the EQ-5D-5L instrument in the UAE population in generating local evidence on the cost-effectiveness of healthcare interventions, as well as to enhance other applications of EQ-5D in population health assessment and health systems.

{"title":"A Value Set for the EQ-5D-5L for United Arab Emirates.","authors":"Fatima Al Sayah, Bram Roudijk, Mohamed El Sadig, Asma Al Mannaei, Mohamad N Farghaly, Sara Dallal, Rima Kaddoura, Mirna Metni, Iffat Elbarazi, Samer A Kharroubi","doi":"10.1016/j.jval.2025.01.003","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.003","url":null,"abstract":"<p><p>Objectives To develop a value set for the EQ-5D-5L based on preferences of the general adult population of the United Arab Emirates (UAE). Methods The study followed the EuroQol EQ-5D-5L Valuation protocol and involved conducting interviewer-administered face-to-face or online interviews in Arabic or English, using the EuroQol Valuation Technology (EQ-VT) with a sample of 1005 adults representing the UAE general population. Sample recruitment involved a two-stage quota sampling strategy across the seven Emirates of the UAE, ensuring representation of nationals and expatriates. Various models using composite time-trade-off (cTTO) data only, discrete choice experiment (DCE) data only, and hybrid using both cTTO and DCE data were examined, along with various sensitivity analyses to examine the robustness of the models. Results Average age of respondents was 39 years (SD=10.8), 44.5% were female, and 11% were UAE nationals. The best performing model to generate the value set for the EQ-5D-5Lwas the hybrid Tobit model censored at -1.0, corrected for heteroskedasticity. Values ranged from - 0.654 for the worst health state (55555) to 1 for full health (11111) and 0.962 for 11211, with 15.3% of predicted values worse than dead. Mobility problems had the largest impact on health state preference values relative to other dimensions. Conclusion This value set will facilitate the application and use of the EQ-5D-5L instrument in the UAE population in generating local evidence on the cost-effectiveness of healthcare interventions, as well as to enhance other applications of EQ-5D in population health assessment and health systems.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068299","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
Social Return on Investment analysis: A Mixed Methods Approach to Assessing the Value of Adult Hospice Services.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.004
Nicole M Hughes, Jane Noyes, D Phil Trystan Pritchard, Carys Stringer

Objectives Hospice services offer invaluable support to individuals facing life-limiting illnesses, however, quantifying their positive impact presents a challenge. As the demand for palliative care rises due to complex illnesses and an aging population, hospices face the need to prove their value. With funding primarily reliant on charitable donations and limited statutory support, they must demonstrate their effectiveness to secure additional resources in a competitive landscape. Methods This study employed the Social Return on Investment (SROI) framework to evaluate the social value generated by four hospice sites offering inpatient and day therapy services across North Wales. Through a mixed-methods approach, quantitative and qualitative data were collected to explore stakeholder experiences, values, and outcomes, facilitating a thorough examination of the broader social impact of hospice care. Results The average input and output values for the inpatient unit were £602,100 and £1,667,861 respectively, thus returning a base case ratio of £2.77: £1. The day therapy unit had average input and output costs of £155,928 and £1,847,347 respectively, hence a base case ratio of £11.85: £1. Sensitivity analysis yielded estimates of between £2.20: £1 and £6.83: £1 for the inpatient unit and between £2:44: £1 and £19:51: £1 for the day therapy unit. CONCLUSION As healthcare providers globally confront challenges with resource constraints, adopting value-driven methodologies becomes crucial. Embracing such methodologies fosters a more comprehensive understanding of value, transcending traditional metrics to encompass social, environmental, and long-term sustainability considerations.

{"title":"Social Return on Investment analysis: A Mixed Methods Approach to Assessing the Value of Adult Hospice Services.","authors":"Nicole M Hughes, Jane Noyes, D Phil Trystan Pritchard, Carys Stringer","doi":"10.1016/j.jval.2025.01.004","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.004","url":null,"abstract":"<p><p>Objectives Hospice services offer invaluable support to individuals facing life-limiting illnesses, however, quantifying their positive impact presents a challenge. As the demand for palliative care rises due to complex illnesses and an aging population, hospices face the need to prove their value. With funding primarily reliant on charitable donations and limited statutory support, they must demonstrate their effectiveness to secure additional resources in a competitive landscape. Methods This study employed the Social Return on Investment (SROI) framework to evaluate the social value generated by four hospice sites offering inpatient and day therapy services across North Wales. Through a mixed-methods approach, quantitative and qualitative data were collected to explore stakeholder experiences, values, and outcomes, facilitating a thorough examination of the broader social impact of hospice care. Results The average input and output values for the inpatient unit were £602,100 and £1,667,861 respectively, thus returning a base case ratio of £2.77: £1. The day therapy unit had average input and output costs of £155,928 and £1,847,347 respectively, hence a base case ratio of £11.85: £1. Sensitivity analysis yielded estimates of between £2.20: £1 and £6.83: £1 for the inpatient unit and between £2:44: £1 and £19:51: £1 for the day therapy unit. CONCLUSION As healthcare providers globally confront challenges with resource constraints, adopting value-driven methodologies becomes crucial. Embracing such methodologies fosters a more comprehensive understanding of value, transcending traditional metrics to encompass social, environmental, and long-term sustainability considerations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068125","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
A Comparison of International Modelling Methods to Evaluate Health Economics of Colorectal Cancer Screening: A Systematic Review.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.007
Olivia Adair, Felicity Lamrock, James F O'Mahony, Mark Lawler, Ethna McFerran

Objectives: Cost-effectiveness analysis (CEA) is an accepted approach to evaluate cancer screening programmes. CEA estimates partially depend on modelling methods and assumptions used. Understanding common practice when modelling cancer relies on complete, accessible descriptions of prior work. This review's objective is to comprehensively examine published CEA modelling methods employed to evaluate Colorectal Cancer (CRC) screening from an aspiring modeller's perspective. It compares existing models highlighting the importance of precise modelling method descriptions, and essential factors when modelling CRC progression.

Methods: MEDLINE, EMBASE, Web of Science and Scopus electronic databases were used. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement and data items from previous systematic reviews formed a template to extract relevant data. Specific focus included model type, natural history, appropriate data sources and survival analysis.

Results: Seventy-eight studies, with 52 unique models found. Twelve previously published models were reported in 39 studies, with 39 newly developed models. CRC progression from the onset was commonly modelled, with only six models not including it as a model component.

Conclusions: Modelling methods needed to simulate CRC progression depend on the natural history structure and research requirements. For aspiring modellers, accompanying models with clear overviews and extensive modelling assumption descriptions is beneficial. Open-source modelling would also allow model replicability and result in appropriate decisions suggested for CRC screening programmes.

目的:成本效益分析(CEA)是评估癌症筛查计划的一种公认方法。成本效益分析的估计值部分取决于所使用的建模方法和假设。要了解癌症建模的常见做法,需要对先前的工作进行完整、易懂的描述。本综述旨在从一个有抱负的建模者的角度,全面考察已发表的用于评估结直肠癌(CRC)筛查的 CEA 建模方法。它对现有模型进行了比较,强调了精确建模方法描述的重要性,以及建立 CRC 进展模型时的基本要素:方法:使用 MEDLINE、EMBASE、Web of Science 和 Scopus 电子数据库。综合卫生经济评估报告标准(CHEERS)声明和以往系统综述中的数据项构成了提取相关数据的模板。具体重点包括模型类型、自然历史、适当的数据来源和生存分析:结果:共发现 78 项研究,52 个独特的模型。39 项研究报告了 12 个以前发表的模型,39 个新开发的模型。从发病开始的 CRC 进展模型很常见,只有 6 个模型没有将其作为模型的组成部分:结论:模拟 CRC 进展所需的建模方法取决于自然史结构和研究要求。对于有抱负的建模者来说,随模型提供清晰的概述和广泛的建模假设说明是有益的。开放源码建模还能使模型具有可复制性,并为 CRC 筛查计划提供适当的决策建议。
{"title":"A Comparison of International Modelling Methods to Evaluate Health Economics of Colorectal Cancer Screening: A Systematic Review.","authors":"Olivia Adair, Felicity Lamrock, James F O'Mahony, Mark Lawler, Ethna McFerran","doi":"10.1016/j.jval.2025.01.007","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.007","url":null,"abstract":"<p><strong>Objectives: </strong>Cost-effectiveness analysis (CEA) is an accepted approach to evaluate cancer screening programmes. CEA estimates partially depend on modelling methods and assumptions used. Understanding common practice when modelling cancer relies on complete, accessible descriptions of prior work. This review's objective is to comprehensively examine published CEA modelling methods employed to evaluate Colorectal Cancer (CRC) screening from an aspiring modeller's perspective. It compares existing models highlighting the importance of precise modelling method descriptions, and essential factors when modelling CRC progression.</p><p><strong>Methods: </strong>MEDLINE, EMBASE, Web of Science and Scopus electronic databases were used. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement and data items from previous systematic reviews formed a template to extract relevant data. Specific focus included model type, natural history, appropriate data sources and survival analysis.</p><p><strong>Results: </strong>Seventy-eight studies, with 52 unique models found. Twelve previously published models were reported in 39 studies, with 39 newly developed models. CRC progression from the onset was commonly modelled, with only six models not including it as a model component.</p><p><strong>Conclusions: </strong>Modelling methods needed to simulate CRC progression depend on the natural history structure and research requirements. For aspiring modellers, accompanying models with clear overviews and extensive modelling assumption descriptions is beneficial. Open-source modelling would also allow model replicability and result in appropriate decisions suggested for CRC screening programmes.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068292","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
Changes in Out-of-Pocket Drug Expenditures Among Medicare Beneficiaries with Dementia Under the Inflation Reduction Act: A Simulation Study.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.008
Yingying Zhu, Natalia Olchanski, Karen M Freund, Joshua T Cohen, Peter J Neumann, Pei-Jung Lin

Objectives: We investigated how the Inflation Reduction Act (IRA) Medicare Part D benefit redesign may reduce out-of-pocket (OOP) drug expenditures for Medicare beneficiaries with dementia. Methods Design Utilizing data from the Health and Retirement Study (HRS) linked with Medicare claims, we simulated post-redesign OOP drug spending by applying the 2025 prescription drug cost-sharing rules to each beneficiary's pre-redesign Part D medication utilization data for 2016, adjusting for inflation. Participants Our study population comprised HRS respondents aged 65 and older in 2016, enrolled in Medicare fee-for-service, with at least one Part D drug claim in 2016, and diagnosed with dementia between 2000 and 2016 (n=1,677). Main Measures We compared pre- and post-redesign annual OOP drug expenditures stratified by: 1) low-income subsidy (LIS) eligibility status; 2) household income among non-LIS beneficiaries; 3) comorbidity count, and 4) cognitive impairment severity.

Results: Post-redesign, we project average annual OOP drug expenditures among LIS beneficiaries with dementia to decrease from $56 to $25 - representing a $31, or 55% reduction. In contrast, among non-LIS beneficiaries, average OOP drug expenditures is projected to decrease from $772 to $576, a $196, or 25% reduction. We project the reduction in OOP drug expenditures to be greater among beneficiaries with 6-8 comorbidities (45%) than among beneficiaries with fewer comorbidities (21-26%).

Conclusions: The IRA Medicare Part D benefit redesign will reduce OOP drug spending for beneficiaries with dementia, resulting in potentially marked savings not only for low-income beneficiaries and beneficiaries with a high comorbidity burden, but also for higher-income beneficiaries.

{"title":"Changes in Out-of-Pocket Drug Expenditures Among Medicare Beneficiaries with Dementia Under the Inflation Reduction Act: A Simulation Study.","authors":"Yingying Zhu, Natalia Olchanski, Karen M Freund, Joshua T Cohen, Peter J Neumann, Pei-Jung Lin","doi":"10.1016/j.jval.2025.01.008","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.008","url":null,"abstract":"<p><strong>Objectives: </strong>We investigated how the Inflation Reduction Act (IRA) Medicare Part D benefit redesign may reduce out-of-pocket (OOP) drug expenditures for Medicare beneficiaries with dementia. Methods Design Utilizing data from the Health and Retirement Study (HRS) linked with Medicare claims, we simulated post-redesign OOP drug spending by applying the 2025 prescription drug cost-sharing rules to each beneficiary's pre-redesign Part D medication utilization data for 2016, adjusting for inflation. Participants Our study population comprised HRS respondents aged 65 and older in 2016, enrolled in Medicare fee-for-service, with at least one Part D drug claim in 2016, and diagnosed with dementia between 2000 and 2016 (n=1,677). Main Measures We compared pre- and post-redesign annual OOP drug expenditures stratified by: 1) low-income subsidy (LIS) eligibility status; 2) household income among non-LIS beneficiaries; 3) comorbidity count, and 4) cognitive impairment severity.</p><p><strong>Results: </strong>Post-redesign, we project average annual OOP drug expenditures among LIS beneficiaries with dementia to decrease from $56 to $25 - representing a $31, or 55% reduction. In contrast, among non-LIS beneficiaries, average OOP drug expenditures is projected to decrease from $772 to $576, a $196, or 25% reduction. We project the reduction in OOP drug expenditures to be greater among beneficiaries with 6-8 comorbidities (45%) than among beneficiaries with fewer comorbidities (21-26%).</p><p><strong>Conclusions: </strong>The IRA Medicare Part D benefit redesign will reduce OOP drug spending for beneficiaries with dementia, resulting in potentially marked savings not only for low-income beneficiaries and beneficiaries with a high comorbidity burden, but also for higher-income beneficiaries.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066655","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
Rare Disease Policy in High-Income Countries: An Overview of Achievements, Challenges, and Solutions.
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2024.12.009
Arianna Gentilini, Emilie Neez, Durhane Wong-Rieger

Objectives: To provide an overview of policy initiatives in high-income countries aimed at supporting the development and accessibility of treatments for rare diseases.

Methods: We examine how legislative, research, and pricing policies in high-income countries address barriers that have historically hindered innovation and access to rare disease treatments. By analysing examples from the EU, UK, US, Canada, Japan, and Australia, the article identifies ongoing initiatives, outlines current challenges, and explores proposed solutions to foster a sustainable, innovative, and accessible rare disease treatment ecosystem.

Results: The review highlights policies like legislative incentives in the EU, US, and Japan for orphan drug development, public-private partnerships to boost innovation, and patient registries to support research and clinical trials. Despite these efforts, major challenges persist, including high therapy costs, limited access to innovation for ultra-rare diseases, and diagnostic delays, with significant disparities across regions.

Conclusions: Overcoming these challenges will require sustainable pricing and reimbursement frameworks, alongside stronger collaboration between stakeholders, particularly for ultra-rare diseases. Advanced technologies like AI hold promise for improving diagnostic accuracy and data collection, supported by enhanced coding systems and registries to facilitate more robust research.

{"title":"Rare Disease Policy in High-Income Countries: An Overview of Achievements, Challenges, and Solutions.","authors":"Arianna Gentilini, Emilie Neez, Durhane Wong-Rieger","doi":"10.1016/j.jval.2024.12.009","DOIUrl":"https://doi.org/10.1016/j.jval.2024.12.009","url":null,"abstract":"<p><strong>Objectives: </strong>To provide an overview of policy initiatives in high-income countries aimed at supporting the development and accessibility of treatments for rare diseases.</p><p><strong>Methods: </strong>We examine how legislative, research, and pricing policies in high-income countries address barriers that have historically hindered innovation and access to rare disease treatments. By analysing examples from the EU, UK, US, Canada, Japan, and Australia, the article identifies ongoing initiatives, outlines current challenges, and explores proposed solutions to foster a sustainable, innovative, and accessible rare disease treatment ecosystem.</p><p><strong>Results: </strong>The review highlights policies like legislative incentives in the EU, US, and Japan for orphan drug development, public-private partnerships to boost innovation, and patient registries to support research and clinical trials. Despite these efforts, major challenges persist, including high therapy costs, limited access to innovation for ultra-rare diseases, and diagnostic delays, with significant disparities across regions.</p><p><strong>Conclusions: </strong>Overcoming these challenges will require sustainable pricing and reimbursement frameworks, alongside stronger collaboration between stakeholders, particularly for ultra-rare diseases. Advanced technologies like AI hold promise for improving diagnostic accuracy and data collection, supported by enhanced coding systems and registries to facilitate more robust research.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068112","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
Developing an Australian Value Set for the Recovering Quality of Life-Utility Index Instrument Using Discrete Choice Experiment With Duration. 使用持续时间的离散选择实验为恢复生活质量-效用指数(ReQoL-UI)工具开发澳大利亚值集。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-17 DOI: 10.1016/j.jval.2024.12.008
Thao Thai, Lidia Engel, Jemimah Ride, Brendan Mulhern, Richard Norman, Cathrine Mihalopoulos

Objectives: The Recovering Quality of Life-Utility Index (ReQoL-UI) instrument was designed to measure the quality-of-life outcomes for people older than 16 years with mental health problems. We aimed to elicit societal preferences for the ReQoL-UI health states to facilitate better decision making in Australia.

Methods: A discrete choice experiment with duration was embedded in a self-completed online survey and administered to a representative sample (n = 1019) of the Australian adult population aged 18 years and older stratified by age, sex, and geographic location. A partial subset design discrete choice experiment was used with 3 fixed attributes and 5 varying attributes, containing 240 choice tasks that were divided into 20 blocks so that each respondent was assigned a block of 12 choice tasks. The value set was modeled using the conditional logit model with utility decrements directly anchored on the 0 to 1 dead-full health scale. Preference heterogeneity was tested using a mixed logit model.

Results: The final value set reflects the monotonic nature of the ReQoL-UI descriptive systems where the best health state defined by the descriptive system has a value of 1 and the worst state has a value of -0.585. The most important dimension was physical health problems, whereas the least important attribute was self-perception. Sensitivity and preference heterogeneity analyses revealed the stability of the value set.

Conclusions: The value set, which reflects the preferences of the Australian population, facilitates the calculation of an index for quality-adjusted life-years in mental health intervention cost-utility analyses.

目的:设计康复生活质量-效用指数(ReQoL-UI)测量16岁以上有心理健康问题人群的生活质量结果。我们的目标是引出社会对ReQoL-UI健康状态的偏好,以促进澳大利亚更好的决策。方法:将离散选择实验(DCE)嵌入一份自我完成的在线调查中,并对具有代表性的样本(n=1019)进行管理,这些样本年龄在18岁及以上,按年龄、性别和地理位置分层。采用部分子集设计DCE,其中包含3个固定属性和5个变化属性,包含240个选择任务,这些选择任务被划分为20个块,因此每个被调查者被分配到12个选择任务的块。该值集是使用条件logit模型建模的,其效用递减直接锚定在0到1的完全死亡生命值范围内。采用混合logit模型检验偏好异质性。结果:最终值集反映了ReQoL-UI描述系统的单调性,其中描述系统定义的最佳健康状态值为1,最差状态值为-0.585。最重要的维度是身体健康问题,而最不重要的属性是自我认知。敏感性分析和偏好异质性分析显示了该值集的稳定性。结论:该数值集反映了澳大利亚人口的偏好,便于在心理健康干预成本-效用分析中计算质量调整生命年指数。
{"title":"Developing an Australian Value Set for the Recovering Quality of Life-Utility Index Instrument Using Discrete Choice Experiment With Duration.","authors":"Thao Thai, Lidia Engel, Jemimah Ride, Brendan Mulhern, Richard Norman, Cathrine Mihalopoulos","doi":"10.1016/j.jval.2024.12.008","DOIUrl":"10.1016/j.jval.2024.12.008","url":null,"abstract":"<p><strong>Objectives: </strong>The Recovering Quality of Life-Utility Index (ReQoL-UI) instrument was designed to measure the quality-of-life outcomes for people older than 16 years with mental health problems. We aimed to elicit societal preferences for the ReQoL-UI health states to facilitate better decision making in Australia.</p><p><strong>Methods: </strong>A discrete choice experiment with duration was embedded in a self-completed online survey and administered to a representative sample (n = 1019) of the Australian adult population aged 18 years and older stratified by age, sex, and geographic location. A partial subset design discrete choice experiment was used with 3 fixed attributes and 5 varying attributes, containing 240 choice tasks that were divided into 20 blocks so that each respondent was assigned a block of 12 choice tasks. The value set was modeled using the conditional logit model with utility decrements directly anchored on the 0 to 1 dead-full health scale. Preference heterogeneity was tested using a mixed logit model.</p><p><strong>Results: </strong>The final value set reflects the monotonic nature of the ReQoL-UI descriptive systems where the best health state defined by the descriptive system has a value of 1 and the worst state has a value of -0.585. The most important dimension was physical health problems, whereas the least important attribute was self-perception. Sensitivity and preference heterogeneity analyses revealed the stability of the value set.</p><p><strong>Conclusions: </strong>The value set, which reflects the preferences of the Australian population, facilitates the calculation of an index for quality-adjusted life-years in mental health intervention cost-utility analyses.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143012526","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
Advancing Mental Health Economics: Insights from the Themed Section. 推进心理健康经济学:来自主题部分的见解。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-17 DOI: 10.1016/j.jval.2024.12.007
Rachael L Fleurence, Jagpreet Chhatwal
{"title":"Advancing Mental Health Economics: Insights from the Themed Section.","authors":"Rachael L Fleurence, Jagpreet Chhatwal","doi":"10.1016/j.jval.2024.12.007","DOIUrl":"https://doi.org/10.1016/j.jval.2024.12.007","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143012523","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
Unpaid Caring and Health-Related Quality of Life: Longitudinal Analysis of Understanding Society (the UK Household Longitudinal Survey) 无偿照料和与取暖相关的生活质量:对 "理解社会"(英国家庭纵向调查)的纵向分析。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.08.004
Becky M. Pennington MSc, Mónica Hernández Alava PhD, Mark Strong PhD

Objectives

Decision models for economic evaluation are increasingly including health-related quality of life (HRQoL) for informal/unpaid carers, but these estimates often come from poor quality data and typically rely on cross-sectional analysis. We aimed to identify within-person effects using longitudinal analysis of 13 waves of Understanding Society (the UK Household Longitudinal Survey).

Methods

We analyzed data for coresident carer and care-recipient dyads, where the carer reported “looking after or giving special help to” the care recipient in any of the 13 waves. We used fixed-effects models to study the effects of caring for the care recipient (the “caregiving” effect) using volume of care (hours per week) and continuous duration of caregiving (years) and caring about the care recipient (the “family” effect) using the care recipient’s HRQoL on the carer’s HRQoL. HRQoL was measured using the Short Form 6 Dimension, calculated from the Short Form 12.

Results

We found consistent evidence for the family effect: improving care recipient’s HRQoL by 0.1 would improve carer’s HRQoL by approximately 0.012. We also consistently found evidence of a small but statistically significant decrement to carer’s HRQoL for each additional year of caring. These findings were robust to scenario analyses. Evidence for the relationship between volume of care and carer’s HRQoL was less clear.

Conclusions

We propose that our estimates can be used to populate economic models to predict changes in carers’ HRQoL over time and allow disutilities to be estimated separately for the family and caregiving effect.
目的:用于经济评估的决策模型越来越多地包括非正式/无偿照护者的健康相关生活质量(HRQoL),但这些估算通常来自质量较差的数据,而且通常依赖于横截面分析。我们的目标是通过对 13 次 "了解社会"(英国家庭纵向调查)的纵向分析来确定人内效应:我们分析了同住照护者和受照护者二元组的数据,其中照护者在 13 次调查中的任何一次中都报告了 "照护或给予特别帮助 "受照护者的情况。我们使用固定效应模型来研究照顾受照顾者("照顾 "效应)对照顾者的影响(使用照顾量(每周小时)和连续照顾时间(年));以及照顾受照顾者("家庭 "效应)对照顾者的影响(使用受照顾者的 HRQoL)。HRQoL 采用简表 6 维(SF-6D)进行测量,由 SF-12 计算得出:我们发现了家庭效应的一致证据:护理对象的 HRQoL 提高 0.1 将使护理者的 HRQoL 提高约 0.012。我们还一致发现,照顾者每多照顾一年,其 HRQoL 就会略有下降,但在统计学上具有显著意义。这些发现在情景分析中都是可靠的。护理量与照护者 HRQoL 之间关系的证据不太明确:我们建议将我们的估算结果用于经济模型,以预测照护者的 HRQoL 随时间的变化,并分别估算家庭效应和照护效应的损失。
{"title":"Unpaid Caring and Health-Related Quality of Life: Longitudinal Analysis of Understanding Society (the UK Household Longitudinal Survey)","authors":"Becky M. Pennington MSc,&nbsp;Mónica Hernández Alava PhD,&nbsp;Mark Strong PhD","doi":"10.1016/j.jval.2024.08.004","DOIUrl":"10.1016/j.jval.2024.08.004","url":null,"abstract":"<div><h3>Objectives</h3><div>Decision models for economic evaluation are increasingly including health-related quality of life (HRQoL) for informal/unpaid carers, but these estimates often come from poor quality data and typically rely on cross-sectional analysis. We aimed to identify within-person effects using longitudinal analysis of 13 waves of Understanding Society (the UK Household Longitudinal Survey).</div></div><div><h3>Methods</h3><div>We analyzed data for coresident carer and care-recipient dyads, where the carer reported “looking after or giving special help to” the care recipient in any of the 13 waves. We used fixed-effects models to study the effects of caring for the care recipient (the “caregiving” effect) using volume of care (hours per week) and continuous duration of caregiving (years) and caring about the care recipient (the “family” effect) using the care recipient’s HRQoL on the carer’s HRQoL. HRQoL was measured using the Short Form 6 Dimension, calculated from the Short Form 12.</div></div><div><h3>Results</h3><div>We found consistent evidence for the family effect: improving care recipient’s HRQoL by 0.1 would improve carer’s HRQoL by approximately 0.012. We also consistently found evidence of a small but statistically significant decrement to carer’s HRQoL for each additional year of caring. These findings were robust to scenario analyses. Evidence for the relationship between volume of care and carer’s HRQoL was less clear.</div></div><div><h3>Conclusions</h3><div>We propose that our estimates can be used to populate economic models to predict changes in carers’ HRQoL over time and allow disutilities to be estimated separately for the family and caregiving effect.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 138-147"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354728","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
Most Common Symptomatic Adverse Reactions of Cancer Treatments From US Drug Labels (2015-2021) to Inform Selection of Patient-Reported Outcomes 美国药品标签中癌症治疗最常见的症状性不良反应(2015-2021 年),为选择患者报告结果提供依据。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2025-01-01 DOI: 10.1016/j.jval.2024.09.009
Erica G. Horodniceanu MPH , Tejaswi Datla , Meena N. Murugappan PharmD, MPH, PhD , Bindu Kanapuru MD , Laleh Amiri-Kordestani MD , Erin Larkins MD , Paul Kluetz MD , Vishal Bhatnagar MD

Objectives

Incorporating patient-reported outcomes (PROs) to assess symptomatic adverse events (AEs) in cancer clinical trials (CTs) is important to characterize treatment tolerability. Cancer therapies approved over the past decade have expanded the types of expected toxicities. To inform future symptomatic AE PRO item selection, we identified the most common symptomatic adverse reactions from recently approved products.

Methods

We reviewed approvals from 2015 to 2021 for lung, breast, and hematologic cancer indications. Using United States Prescribing Information safety data, we recorded symptomatic adverse reactions reported in ≥20% of patients in the experimental arm of CTs supporting approvals. We calculated the proportion of arms reporting each symptomatic adverse reaction.

Results

In total, 130 experimental arms were included (lung = 30, breast = 10, hematologic = 90). For all cancer types, fatigue and diarrhea were reported in >50% of the arms. Nausea was reported in ≥50% of the arms for all except lung. Vomiting, decreased appetite, and alopecia, were reported in ≥50% of breast cancer arms. Rash, musculoskeletal pain, and cough were reported in >50% of leukemia/lymphoma arms. Cough was common (50%) in multiple myeloma arms.

Conclusions

Heterogeneity in symptomatic adverse reactions across CTs supports the use of item libraries when building a PRO strategy to assess tolerability. Fatigue, diarrhea, and nausea were the most frequent symptomatic adverse reactions reported in contemporary cancer CTs and could provide a starting point when selecting PRO symptomatic AE items. Additional symptomatic AE PRO items should be selected based on the mechanism of action, early clinical data, published literature, and patient and clinician input.
目的:在癌症临床试验(CTs)中纳入患者报告结果(PROs)来评估症状性不良事件(AEs)对于描述治疗耐受性非常重要。过去十年中批准的癌症疗法扩大了预期毒性的类型。为了给未来的症状性 AE PRO 项目选择提供信息,我们确定了最近批准的产品中最常见的症状性不良反应:我们回顾了 2015-2021 年肺癌、乳腺癌和血液肿瘤适应症的批准情况。利用美国处方信息安全性数据,我们记录了支持批准的 CT 实验臂中≥20% 的患者报告的症状性不良反应。我们计算了报告每种症状性不良反应的试验组比例:结果:共纳入 130 个实验臂(肺癌=30 个,乳腺癌=10 个,血液肿瘤=90 个)。在所有癌症类型中,超过 50% 的实验组报告了疲劳和腹泻。除肺癌外,≥50% 的实验组报告有恶心症状。≥50%的乳腺癌治疗组出现呕吐、食欲下降和脱发。在白血病/淋巴瘤治疗组中,报告皮疹、肌肉骨骼疼痛和咳嗽的比例>50%。在多发性骨髓瘤治疗组中,咳嗽很常见(50%):结论:不同临床试验中症状性不良反应的异质性支持在建立PRO策略以评估耐受性时使用项目库。疲劳、腹泻和恶心是当代癌症 CT 中最常见的症状性不良反应,可作为选择 PRO 症状性 AE 项目的起点。应根据作用机制、早期临床数据、已发表文献以及患者和临床医生的意见选择其他症状性 AE PRO 项目。
{"title":"Most Common Symptomatic Adverse Reactions of Cancer Treatments From US Drug Labels (2015-2021) to Inform Selection of Patient-Reported Outcomes","authors":"Erica G. Horodniceanu MPH ,&nbsp;Tejaswi Datla ,&nbsp;Meena N. Murugappan PharmD, MPH, PhD ,&nbsp;Bindu Kanapuru MD ,&nbsp;Laleh Amiri-Kordestani MD ,&nbsp;Erin Larkins MD ,&nbsp;Paul Kluetz MD ,&nbsp;Vishal Bhatnagar MD","doi":"10.1016/j.jval.2024.09.009","DOIUrl":"10.1016/j.jval.2024.09.009","url":null,"abstract":"<div><h3>Objectives</h3><div>Incorporating patient-reported outcomes (PROs) to assess symptomatic adverse events (AEs) in cancer clinical trials (CTs) is important to characterize treatment tolerability. Cancer therapies approved over the past decade have expanded the types of expected toxicities. To inform future symptomatic AE PRO item selection, we identified the most common symptomatic adverse reactions from recently approved products.</div></div><div><h3>Methods</h3><div>We reviewed approvals from 2015 to 2021 for lung, breast, and hematologic cancer indications. Using United States Prescribing Information safety data, we recorded symptomatic adverse reactions reported in ≥20% of patients in the experimental arm of CTs supporting approvals. We calculated the proportion of arms reporting each symptomatic adverse reaction.</div></div><div><h3>Results</h3><div>In total, 130 experimental arms were included (lung = 30, breast = 10, hematologic = 90). For all cancer types, fatigue and diarrhea were reported in &gt;50% of the arms. Nausea was reported in ≥50% of the arms for all except lung. Vomiting, decreased appetite, and alopecia, were reported in ≥50% of breast cancer arms. Rash, musculoskeletal pain, and cough were reported in &gt;50% of leukemia/lymphoma arms. Cough was common (50%) in multiple myeloma arms.</div></div><div><h3>Conclusions</h3><div>Heterogeneity in symptomatic adverse reactions across CTs supports the use of item libraries when building a PRO strategy to assess tolerability. Fatigue, diarrhea, and nausea were the most frequent symptomatic adverse reactions reported in contemporary cancer CTs and could provide a starting point when selecting PRO symptomatic AE items. Additional symptomatic AE PRO items should be selected based on the mechanism of action, early clinical data, published literature, and patient and clinician input.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 108-115"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401461","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
期刊
Value in Health
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1