Value in Health最新文献

{"title":"Social Decision-Making Analysis: A General Approach to Inform Decisions on Resources in the Public Sector","authors":"Francesco Longo PhD ,&nbsp;Karl Claxton PhD ,&nbsp;Susan Griffin PhD ,&nbsp;Anne Mason MA ,&nbsp;Simon Walker MSc ,&nbsp;Helen Weatherly MSc","doi":"10.1016/j.jval.2024.01.015","DOIUrl":"10.1016/j.jval.2024.01.015","url":null,"abstract":"<div><h3>Objectives</h3><p>Public expenditure aims to achieve social objectives by improving a range of socially valuable attributes of benefit (arguments in a social welfare function). Public expenditure is typically allocated to public sector budgets, where budget holders are tasked with meeting a subset of social objectives.</p></div><div><h3>Methods</h3><p>Decision makers require an evidence-based assessment of whether a proposed investment is likely to be worthwhile given existing levels of public expenditure. However, others also require some assessment of whether the overall level and allocation of public expenditure are appropriate. This article proposes a more general theoretical framework for economic evaluation that addresses both these questions.</p></div><div><h3>Results</h3><p>Using a stylized example of the economic evaluation of a new intervention in a simplified UK context, we show that this more general framework can support decisions beyond the approval or rejection of single projects. It shows that broader considerations about the level and allocation of public expenditure are possible and necessary when evaluating specific investments, which requires evidence of the range of benefits offered by marginal changes in different types of public expenditure and normative choices of how the attributes of benefit gained and forgone are valued.</p></div><div><h3>Conclusions</h3><p>The proposed framework shows how to assess the value of a proposed investment and whether and how the overall level of public expenditure and its allocation across public sector budgets might be changed. It highlights that cost-benefit analysis and cost-effectiveness analysis can be viewed as special cases of this framework, identifying the weakness with each.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524000457/pdfft?md5=203bc6ea4aab845d9d5ffd010891c1e5&pid=1-s2.0-S1098301524000457-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139668645","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}

{"title":"Construct Validity, Reliability, and Responsiveness of the 10-Item Well-Being Instrument for Use in Economic Evaluation Studies","authors":"Judith A.M. Bom PhD ,&nbsp;Daphne C. Voormolen PhD ,&nbsp;Werner B.F. Brouwer PhD ,&nbsp;Esther W. de Bekker-Grob PhD ,&nbsp;Job van Exel PhD","doi":"10.1016/j.jval.2024.02.014","DOIUrl":"10.1016/j.jval.2024.02.014","url":null,"abstract":"<div><h3>Objectives</h3><p>Economic evaluations of interventions in health and social care require outcome measures that capture their full benefits, including those beyond health. This study aimed to assess construct validity, test-retest reliability, and responsiveness of the newly developed 10-item Well-being instrument (WiX).</p></div><div><h3>Methods</h3><p>Data were gathered via an online survey in a representative sample of the adult general population in The Netherlands (N = 1045). Construct validity was assessed by inspecting convergent, structural, and discriminant validity, following the COnsensus-based Standards for the selection of health status Measurement INstruments methodology. Regression analyses of the WiX and its items on other validated measures of well-being were performed to assess the convergent validity of the instrument and the relevance of its items. Dimensionality of the WiX was assessed using exploratory factor analysis. To assess discriminant validity, several hypotheses in terms of well-being differences were assessed. Finally, a second survey was sent out 2 weeks after the initial survey (n = 563; 53.9% response rate) to assess the test-retest reliability and responsiveness of the WiX.</p></div><div><h3>Results</h3><p>The WiX showed to be correlated with alternative well-being measures as expected and able to sufficiently differentiate between relevant subgroups in the population. Moreover, the dimensionality analysis indicated that the WiX captures a broad array of elements relevant to well-being, including physical and mental health. The test-retest reliability was good, with an intraclass correlation coefficient of 0.82.</p></div><div><h3>Conclusions</h3><p>The results regarding the WiX are favorable and indicate that this new instrument may be a promising alternative for existing measures of well-being for evaluating interventions in health and social care.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S109830152400086X/pdfft?md5=0f68ebc6e489acc861e710826a896c35&pid=1-s2.0-S109830152400086X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140047249","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}

{"title":"From Health to Well-Being: Toward a Monetary Valuation of a Well-Being-Adjusted Life-Year","authors":"Carolin Brinkmann MSc ,&nbsp;Tom Stargardt PhD ,&nbsp;Werner B.F. Brouwer PhD","doi":"10.1016/j.jval.2024.02.015","DOIUrl":"10.1016/j.jval.2024.02.015","url":null,"abstract":"<div><h3>Objectives</h3><p>Economic evaluations using broader measures to capture benefits beyond improved health can inform policy making, but only if the monetary value of gains measured using these instruments is understood. This study explored contingent valuation as a method to estimate the monetary value of a well-being-adjusted life-year (WALY) as measured by ICEpop Capability Measure for Adults (ICECAP-A).</p></div><div><h3>Methods</h3><p>In a large online survey of representative samples from 7 European countries, participants valued a change in the ICECAP-A from their current health state to a randomly assigned hypothetical state. Participants were instructed that an unspecified treatment could avoid a loss or produce a gain in well-being and were asked for their willingness to pay (WTP) for this treatment. WTP per WALY was calculated using an aggregated approach that used ICECAP-A tariffs from the United Kingdom.</p></div><div><h3>Results</h3><p>We analyzed a sample of 7428 observations, focusing on avoided losses (n = 6002) because the results for gains were not theoretically valid. Different cutoff points for a marginal change were explored. Depending on the definition of a marginal change, WTP per WALY averaged between €13 323.28 and €61 375.63 for avoided losses between [0, 0.5] and [0, 0.1], respectively, for 1 month. Mean WTP per WALY varied across the countries as follows: Denmark (€17 867.93-€88 634.14), France (€10 278.35-€45 581.28), Germany (€12 119.39-€54 566.56), Italy (€11 753.69-€52 161.25), The Netherlands (€14 612.88-€58 951.74), Spain (€11 904.12-€57 909.17), and United Kingdom (€13 133.75-€68 455.85).</p></div><div><h3>Conclusion</h3><p>Despite the inherent limitations of our study, it offers valuable insights into methods for eliciting the WTP for changes in capability well-being as measured with ICECAP-A.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524000871/pdfft?md5=efd8324fdaa5184dbfd957dd3928a94a&pid=1-s2.0-S1098301524000871-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140047160","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}

{"title":"A Head-to-Head Comparison of EQ-HWB and EQ-5D-5L in Patients, Carers, and General Public in China","authors":"Chen Long PhD ,&nbsp;Zhuxin Mao PhD ,&nbsp;Zhihao Yang PhD","doi":"10.1016/j.jval.2024.02.012","DOIUrl":"10.1016/j.jval.2024.02.012","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to understand the psychometric properties of EQ Health and Wellbeing (EQ-HWB) and to examine its relationship with EQ-5D-5L in a sample covering patients, carers, and general public.</p></div><div><h3>Methods</h3><p>A cross-sectional study was conducted in Guizhou Province, China. The acceptability, convergent validity (using Spearman correlation coefficients), internal structure (using exploratory factor analysis), and known-group validity of EQ-HWB, EQ-HWB-Short (EQ-HWB-S), and EQ-5D-5L were reported and compared.</p></div><div><h3>Results</h3><p>A total of 323 participants completed the survey, including 106 patients, 101 carers, and 116 individuals from the general public. Approximately 7.4% of participants had at least 1 missing response. In the EQ-HWB and EQ-5D-5L items related to activities, there were more level 1 responses. The correlations between EQ-HWB and EQ-5D-5L items ranged from low to high, confirming the convergent validity of similar aspects between the 2 instruments. Notably, EQ-HWB measures 2 additional factors compared with EQ-5D-5L or EQ-HWB-S, both of which share 3 common factors. When the patient group was included, EQ-5D-5L had the largest effect size, but it failed to differentiate between the groups of general public and carers. Both EQ-HWB and EQ-HWB-S demonstrated better known-group validity results when carers were included.</p></div><div><h3>Conclusions</h3><p>EQ-HWB measures a broader quality of life construct that goes beyond health measured by EQ-5D-5L. By encompassing a broader scope, the impact of healthcare interventions may become diluted, given that other factors can influence well-being outcomes as significantly as health conditions do.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140047527","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}

{"title":"The Ceiling Effects of EQ-5D-3L and 5L in General Population Health Surveys: A Systematic Review and Meta-Analysis","authors":"Ling Jie Cheng MPH, RN ,&nbsp;Tianxin Pan PhD ,&nbsp;Le Ann Chen BSc ,&nbsp;Jing Ying Cheng BSN (Hons), RN ,&nbsp;Brendan Mulhern PhD ,&nbsp;Nancy Devlin PhD ,&nbsp;Nan Luo PhD","doi":"10.1016/j.jval.2024.02.018","DOIUrl":"10.1016/j.jval.2024.02.018","url":null,"abstract":"<div><h3>Objectives</h3><p>This review aims to examine the ceiling effects of EQ-5D-3L (3L) and EQ-5D-5L (5L) in general adult populations and identify the factors influencing these effects.</p></div><div><h3>Methods</h3><p>We searched 8 databases for observational studies published in English from inception to 24 July 2023. Ceiling effects were calculated by dividing the number of participants reporting full health at dimension or profile level by the total sample size. Subgroup analysis and meta-regression using the <em>metafor</em> package in R software were performed.</p></div><div><h3>Results</h3><p>We identified 94 studies from 70 articles, including 4 543 647 adults across 37 countries. The global pooled proportion of individuals reporting full health (“11111”) was 56% (95% CI 51%-62%) for 3L and 49% (95% CI 44%-54%) for 5L. The self-care dimension showed the highest ceiling effects (3L: 97%; 5L: 94%), whereas pain/discomfort had the lowest (3L: 69%; 5L: 60%). The ceiling effects in East/South-East Asia were higher than in Europe by 25% (95% CI 18%-32%) in 3L and 9% (95% CI −2%-20%) in 5L. Adjusting for mean age and proportion of males, significant regional differences persisted in the overall profile level of 3L, in all 3L dimensions (except for self-care), and 5L dimensions (except for pain/discomfort and anxiety/depression).</p></div><div><h3>Conclusions</h3><p>This review highlights significant ceiling effects in the EQ-5D, especially in Asian populations. The 5L version exhibited fewer ceiling effects than the 3L, indicating its superiority for general population surveys. Further research is crucial to understand the disparities in self-reported health outcomes between Asians and other populations.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140102482","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}

{"title":"Cost-Effectiveness of 2 Models of Pessary Care for Pelvic Organ Prolapse: Findings From the TOPSY Randomized Controlled Trial","authors":"Sarkis Manoukian PhD ,&nbsp;Helen Mason PhD ,&nbsp;Suzanne Hagen PhD ,&nbsp;Rohna Kearney MD ,&nbsp;Kirsteen Goodman PhD ,&nbsp;Catherine Best PhD ,&nbsp;Andrew Elders MSc ,&nbsp;Lynn Melone BSc (Hons) ,&nbsp;Lucy Dwyer MSc ,&nbsp;Melanie Dembinsky PhD ,&nbsp;Aethele Khunda ,&nbsp;Karen Lesley Guerrero ,&nbsp;Doreen McClurg PhD ,&nbsp;John Norrie MSc ,&nbsp;Ranee Thakar MD ,&nbsp;Carol Bugge PhD","doi":"10.1016/j.jval.2024.03.004","DOIUrl":"10.1016/j.jval.2024.03.004","url":null,"abstract":"<div><h3>Objectives</h3><p>Pelvic organ prolapse is the descent of one or more reproductive organs from their normal position, causing associated negative symptoms. One conservative treatment option is pessary management. This study aimed to to investigate the cost-effectiveness of pessary self-management (SM) when compared with clinic-based care (CBC). A decision analytic model was developed to extend the economic evaluation.</p></div><div><h3>Methods</h3><p>A randomized controlled trial with health economic evaluation. The SM group received a 30-minute SM teaching session, information leaflet, 2-week follow-up call, and a local helpline number. The CBC group received routine outpatient pessary appointments, determined by usual practice. The primary outcome for the cost-effectiveness analysis was incremental cost per quality-adjusted life year (QALY), 18 months post-randomization. Uncertainty was handled using nonparametric bootstrap analysis. In addition, a simple decision analytic model was developed using the trial data to extend the analysis over a 5-year period.</p></div><div><h3>Results</h3><p>There was no significant difference in the mean number of QALYs gained between SM and CBC (1.241 vs 1.221), but mean cost was lower for SM (£578 vs £728). The incremental net benefit estimated at a willingness to pay of £20 000 per QALY gained was £564, with an 80.8% probability of cost-effectiveness. The modeling results were consistent with the trial analysis: the incremental net benefit was estimated as £4221, and the probability of SM being cost-effective at 5 years was 69.7%.</p></div><div><h3>Conclusions</h3><p>Results suggest that pessary SM is likely to be cost-effective. The decision analytic model suggests that this result is likely to persist over longer durations.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524001189/pdfft?md5=9e9db88ba5d1f9fc10b5902dc82e3d20&pid=1-s2.0-S1098301524001189-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140140809","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}

{"title":"Anti–Vascular Endothelial Growth Factor Drugs Compared With Panretinal Photocoagulation for the Treatment of Proliferative Diabetic Retinopathy: A Cost-Effectiveness Analysis","authors":"Matthew Walton MSc ,&nbsp;Laura Bojke PhD ,&nbsp;Mark Simmonds PhD ,&nbsp;Ruth Walker MSc ,&nbsp;Alexis Llewellyn MSc ,&nbsp;Helen Fulbright PhD ,&nbsp;Sofia Dias PhD ,&nbsp;Lesley A. Stewart PhD ,&nbsp;Tom Rush ,&nbsp;David H. Steel MD ,&nbsp;John G. Lawrenson PhD ,&nbsp;Tunde Peto PhD ,&nbsp;Robert Hodgson PhD","doi":"10.1016/j.jval.2024.03.007","DOIUrl":"10.1016/j.jval.2024.03.007","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to evaluate the cost-effectiveness of anti–vascular endothelial growth factor drugs (anti-VEGFs) compared with panretinal photocoagulation (PRP) for treating proliferative diabetic retinopathy (PDR) in the United Kingdom.</p></div><div><h3>Methods</h3><p>A discrete event simulation model was developed, informed by individual participant data meta-analysis. The model captures treatment effects on best corrected visual acuity in both eyes, and the occurrence of diabetic macular edema and vitreous hemorrhage. The model also estimates the value of undertaking further research to resolve decision uncertainty.</p></div><div><h3>Results</h3><p>Anti-VEGFs are unlikely to generate clinically meaningful benefits over PRP. The model predicted anti-VEGFs be more costly and similarly effective as PRP, generating 0.029 fewer quality-adjusted life-years at an additional cost of £3688, with a net health benefit of −0.214 at a £20 000 willingness-to-pay threshold. Scenario analysis results suggest that only under very select conditions may anti-VEGFs offer potential for cost-effective treatment of PDR. The consequences of loss to follow-up were an important driver of model outcomes.</p></div><div><h3>Conclusions</h3><p>Anti-VEGFs are unlikely to be a cost-effective treatment for early PDR compared with PRP. Anti-VEGFs are generally associated with higher costs and similar health outcomes across various scenarios. Although anti-VEGFs were associated with lower diabetic macular edema rates, the number of cases avoided is insufficient to offset the additional treatment costs. Key uncertainties relate to the long-term comparative effectiveness of anti-VEGFs, particularly considering the real-world rates and consequences of treatment nonadherence. Further research on long-term visual acuity and rates of vision-threatening complications may be beneficial in resolving uncertainties.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524001220/pdfft?md5=fd49e3e2681da560c1b1aedc6ef3eb07&pid=1-s2.0-S1098301524001220-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140319340","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}

{"title":"Understanding the Complex Interactions Between Interventions, Well-Being, and Resource Allocation","authors":"Aleksandra Torbica PhD ,&nbsp;Brendan Mulhern PhD, MRes ,&nbsp;Richard Norman PhD","doi":"10.1016/j.jval.2024.05.012","DOIUrl":"10.1016/j.jval.2024.05.012","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141200150","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}

{"title":"The Measurement Properties of the EQ-HWB and the EQ-HWB-S in Italian Population: A Comparative Study With EQ-5D-5L","authors":"Maja Kuharić MPharm, MSc, PhD ,&nbsp;A. Simon Pickard PhD ,&nbsp;Clara Mukuria PhD ,&nbsp;Aureliano Paolo Finch PhD","doi":"10.1016/j.jval.2024.03.002","DOIUrl":"10.1016/j.jval.2024.03.002","url":null,"abstract":"<div><h3>Objectives</h3><p>The EQ Health and Well-being, EQ-HWB (25-item) and the EQ-HWB-S (9-item), are new generic measures of health and well-being. The purpose of this study was to examine the measurement properties of the EQ-HWB and EQ-HWB-S measures in relation to the EQ-5D-5L among the Italian general population.</p></div><div><h3>Methods</h3><p>A cross-sectional survey was conducted from October 2020 to February 2021, followed by secondary analysis of collected data from Italian adults. This analysis included response pattern distributions, correlation strength, and known-group comparison. Known-group comparison was assessed using effect sizes (ES) across health conditions, caregiver status, and social care usage. The EQ-HWB-S index-based score was based on the UK pilot value set, whereas the Italian value set was used for scoring the EQ-5D-5L index.</p></div><div><h3>Results</h3><p>Out of the 1182 participants, 461 reported having a chronic condition, 185 identified as caregivers, and 42 were social care users. EQ-HWB items (7.5%) showed fewer ceiling effects than EQ-5D-5L items (34.7%). Strong correlations (r_s &gt; 0.5) were found between overlapping EQ-HWB and EQ-5D-5L items. EQ-HWB-S and EQ-5D-5L index scores demonstrated similar discrimination based on symptomatic chronic conditions (ES d = 0.68 vs d = 0.71), but EQ-HWB-S had slightly higher ES for social care users (ES d = 0.84 vs d = 0.74).</p></div><div><h3>Conclusions</h3><p>Initial evidence supports the validity of EQ-HWB/EQ-HWB-S because outcome measures in the Italian population. EQ-HWB-S performed comparably to EQ-5D-5L among patients and was better in differentiating social care users. A slight decrease in discriminative properties for caregivers was observed when transitioning from EQ-HWB to EQ-HWB-S.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524001165/pdfft?md5=56297fd9e8b0eed8a8de2eb484ecf7a8&pid=1-s2.0-S1098301524001165-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140137242","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}

{"title":"Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making","authors":"Cindy L. Gauvreau PhD ,&nbsp;Leighton Schreyer BSc ,&nbsp;Paul J. Gibson MD ,&nbsp;Alicia Koo PharmD ,&nbsp;Wendy J. Ungar PhD ,&nbsp;Dean Regier PhD ,&nbsp;Kelvin Chan MD, PhD ,&nbsp;Robin Hayeems PhD ,&nbsp;Jennifer Gibson PhD ,&nbsp;Antonia Palmer MSc ,&nbsp;Stuart Peacock DPhil ,&nbsp;Avram E. Denburg MD, PhD","doi":"10.1016/j.jval.2024.03.012","DOIUrl":"10.1016/j.jval.2024.03.012","url":null,"abstract":"<div><h3>Objectives</h3><p>A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach.</p></div><div><h3>Methods</h3><p>We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis.</p></div><div><h3>Results</h3><p>The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework’s key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores.</p></div><div><h3>Conclusions</h3><p>We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140319341","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}