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APPRAISE: A Tool for Appraising Potential for Bias in Real-World Evidence Studies on Medication Effectiveness or Safety. 评价:在药物有效性或安全性的真实证据研究中评估潜在偏倚的工具。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-06 DOI: 10.1016/j.jval.2025.07.024
Katsiaryna Bykov, Ashley Jaksa, Jennifer L Lund, Jessica M Franklin, Cynthia J Girman, Madlen Gazarian, Hongbo Yuan, Stephen Duffield, Seamus Kent, Elisabetta Patorno
{"title":"APPRAISE: A Tool for Appraising Potential for Bias in Real-World Evidence Studies on Medication Effectiveness or Safety.","authors":"Katsiaryna Bykov, Ashley Jaksa, Jennifer L Lund, Jessica M Franklin, Cynthia J Girman, Madlen Gazarian, Hongbo Yuan, Stephen Duffield, Seamus Kent, Elisabetta Patorno","doi":"10.1016/j.jval.2025.07.024","DOIUrl":"10.1016/j.jval.2025.07.024","url":null,"abstract":"<p><strong>Objectives: </strong>Real-world evidence (RWE) plays an increasingly important role in health technology assessment (HTA), as well as regulatory and clinical decision making. RWE studies, however, are subject to multiple sources of bias, which are often not easy to identify, impeding credibility and inclusion of RWE in decision making. A comprehensive, fit-for-purpose, and easy-to-use bias assessment tool would help streamline RWE evaluation, enabling efficient utilization of RWE for decision making globally.</p><p><strong>Methods: </strong>A working group of the International Society for Pharmacoepidemiology collaborated with HTA experts to develop a tool that could guide bias assessment in observational studies on the comparative safety and effectiveness of medications, building upon existing methodological tools, best practice guidelines, and checklists for the analysis of real-world data. The tool was further tested and refined in collaboration with HTA agencies.</p><p><strong>Results: </strong>APPRAISE (APpraisal of Potential for Bias in ReAl-World EvIdence StudiEs) covers key domains through which bias might be introduced into an RWE study: inappropriate study design and analysis, exposure and outcome misclassification, and confounding. Each domain contains a series of questions. Responses to questions auto-populate a summary of the potential for bias within each domain and of the actions to take to avoid, mitigate, or explore the impact of bias.</p><p><strong>Conclusions: </strong>APPRAISE is a tool to guide bias assessment in observational studies on medication comparative effectiveness or safety. Although the tool was designed for HTA, it will be useful for many other users of RWE and will help guide optimized RWE generation.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12392756/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144800398","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
Incorporating Informal Caregiving Time Into Cost-Effectiveness Analysis for Adults Aged 50 and Older: Estimating From Health-Related Quality of Life and Functional Status. 将非正式护理时间纳入50岁及以上成年人的成本-效果分析:从健康相关生活质量和功能状态估计
IF 6 2区 医学
Value in Health Pub Date : 2025-08-06 DOI: 10.1016/j.jval.2025.07.026
Boshen Jiao, Xinran Lu, Darius N Lakdawalla, William V Padula, Fangli Geng
{"title":"Incorporating Informal Caregiving Time Into Cost-Effectiveness Analysis for Adults Aged 50 and Older: Estimating From Health-Related Quality of Life and Functional Status.","authors":"Boshen Jiao, Xinran Lu, Darius N Lakdawalla, William V Padula, Fangli Geng","doi":"10.1016/j.jval.2025.07.026","DOIUrl":"10.1016/j.jval.2025.07.026","url":null,"abstract":"<p><strong>Objectives: </strong>Informal caregiving, often provided by family and friends, is essential for supporting individuals with functional impairments. However, these caregiving costs are frequently excluded from cost-effectiveness analyses (CEAs), potentially biasing the evaluation of medical interventions dependent on informal caregiving. Although guidelines recommend including these costs in CEA from a societal perspective, implementation has been inconsistent because of limited data. To address this gap, we developed US-based models estimating informal caregiving time by linking it to health-related quality of life (HrQoL) of care recipients.</p><p><strong>Methods: </strong>Using data from the 2010 to 2018 waves of the Health and Retirement Study, a nationally representative survey of Americans aged 50+, we focused on individuals with limitations in activities of daily living (ADLs) or instrumental ADLs (IADLs). We performed regression analyses to estimate the association between informal caregiving time and HrQoL, developing separate models with and without ADL/IADL predictors. Additionally, we created models with both preference-based health utility scores from the Health Utilities Index Mark 3 and visual analog scale scores as the HrQoL measures.</p><p><strong>Results: </strong>The models demonstrated strong predictive accuracy based on cross-validation. Findings from the models using HrQoL as the primary predictor indicate that lower Health Utilities Index or visual analog scale scores are associated with increased caregiving time. The models incorporating the number of ADL and IADL limitations as additional predictors further highlight the growing demand for informal caregiving as functional impairments worsen.</p><p><strong>Conclusions: </strong>These models provide practical tools to facilitate the inclusion of informal caregiving costs in US-based CEAs.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144804883","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
"Adrift From the World": Exploring the Lived Experiences of Individuals Affected by an Inherited Optic Neuropathy in the United Kingdom-A Qualitative Study. “漂离世界”:探索受英国遗传性视神经病变影响的个体的生活经历-一项定性研究。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-06 DOI: 10.1016/j.jval.2025.07.023
Benson S Chen, Chloe Seikus, James Ferguson, Valerija Tadić, Mike Horton, Patrick Yu-Wai-Man, Stephanie Archer
{"title":"\"Adrift From the World\": Exploring the Lived Experiences of Individuals Affected by an Inherited Optic Neuropathy in the United Kingdom-A Qualitative Study.","authors":"Benson S Chen, Chloe Seikus, James Ferguson, Valerija Tadić, Mike Horton, Patrick Yu-Wai-Man, Stephanie Archer","doi":"10.1016/j.jval.2025.07.023","DOIUrl":"10.1016/j.jval.2025.07.023","url":null,"abstract":"<p><strong>Objectives: </strong>Little is understood about the lived experiences of individuals affected by inherited optic neuropathies (IONs) in the United Kingdom. The aim of this study was to understand how autosomal dominant optic atrophy (DOA) and Leber hereditary optic neuropathy, the 2 more commonly encountered IONs, impact affected individuals and the factors contributing to their vision-related quality of life (VRQoL).</p><p><strong>Methods: </strong>Semistructured qualitative interviews were conducted with 20 individuals with a genetic diagnosis of DOA (10 participants) or Leber hereditary optic neuropathy (10 participants) and affected by vision loss. Eligible participants were purposively sampled to achieve variation in participant age, sex, duration of visual impairment, and location in the United Kingdom. Using inductive thematic analysis, a range of themes and subthemes were developed.</p><p><strong>Results: </strong>Participants' experiences could be broadly summarized across 4 overarching themes: (1) IONs affected all aspects of life, most notably psychosocial and emotional well-being; (2) participants learned to cope by adapting and adjusting to visual impairment, often on their own, with little external support or resources; (3) participants' identities as visually impaired people were determined by how they viewed themselves and others' reactions to their disability; and (4) good VRQoL was defined as having independence with the support of others.</p><p><strong>Conclusions: </strong>Visual impairment due to an ION threatens the independence of affected individuals, leading to psychosocial losses and reduced emotional well-being. Despite the challenges they face, people living with an ION describe a \"relatively\" good VRQoL, often because of the positive impact of social support, enabling them to lead fulfilling lives.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144800397","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
Combining simulation model-based outcomes with county-level data for geographic health equity impact evaluations of new interventions. 将基于模拟模型的结果与县级数据相结合,用于新干预措施的地理卫生公平影响评估。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-06 DOI: 10.1016/j.jval.2025.07.025
Jeroen P Jansen, Michael P Douglas, Kathryn A Phillips
{"title":"Combining simulation model-based outcomes with county-level data for geographic health equity impact evaluations of new interventions.","authors":"Jeroen P Jansen, Michael P Douglas, Kathryn A Phillips","doi":"10.1016/j.jval.2025.07.025","DOIUrl":"https://doi.org/10.1016/j.jval.2025.07.025","url":null,"abstract":"<p><strong>Objective: </strong>Geographic health disparities persist across the United States, with substantial variations in health outcomes between regions. Evaluating how emerging health technologies might affect these disparities is crucial for developing equitable health policies. This paper introduces an approach for geographic health equity impact evaluation by combining predicted outcomes by equity-relevant subgroup from a simulation model with US county-level data on subgroup proportions.</p><p><strong>Methods: </strong>The approach involves the following steps: 1) Create a dataset with county-level information on equity-relevant factors and lifetime risk of the target indication; 2) Estimate QALYs and costs with and without the intervention for different combinations of equity-relevant factors with the simulation model; 3) Calculate expected and incremental QALYs in target population, incremental net health benefits per 100,000 general population, and quality adjusted life expectancy at birth (QALEs) without and with LB for each county based on its distribution of equity-relevant factors and step 2 estimates; and 4) Quantify inequality in QALYs and QALEs between counties with and without the technology and the corresponding health equity impact.</p><p><strong>Results: </strong>We illustrate the approach using liquid biopsy for first-line treatment in non-small cell lung cancer. Future applications should incorporate more detailed information on the equity-relevant groups by county.</p><p><strong>Conclusion: </strong>Combining simulation model outcomes with county-level data on equity-relevant subgroups provides a novel approach for health equity impact evaluations of new interventions. It facilitates examining how introducing a new health technology can impact geographic disparities in health, and can help identify areas that may benefit most from a new intervention.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144804932","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 Head-to-Head Comparison of the Psychometric Properties of the EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D in Children Aged 8 to 18 Years With Eczema. EQ-5D-Y-3L、EQ-5D-Y-5L和CHU9D对8-18岁湿疹患儿心理测量特性的正面比较
IF 6 2区 医学
Value in Health Pub Date : 2025-08-06 DOI: 10.1016/j.jval.2025.07.021
Rachel Lee-Yin Tan, Jian Yi Soh, Elizabeth Huiwen Tham, Anne Eng Neo Goh, Mark Jean Aan Koh, Nisha Suyien Chandran, Madeline Sheun Ling Ho, Lucinda Siyun Tan, Michael Herdman, Nan Luo
{"title":"A Head-to-Head Comparison of the Psychometric Properties of the EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D in Children Aged 8 to 18 Years With Eczema.","authors":"Rachel Lee-Yin Tan, Jian Yi Soh, Elizabeth Huiwen Tham, Anne Eng Neo Goh, Mark Jean Aan Koh, Nisha Suyien Chandran, Madeline Sheun Ling Ho, Lucinda Siyun Tan, Michael Herdman, Nan Luo","doi":"10.1016/j.jval.2025.07.021","DOIUrl":"10.1016/j.jval.2025.07.021","url":null,"abstract":"<p><strong>Objectives: </strong>This study evaluated and compared the psychometric properties (ceiling effects, construct validity, and responsiveness) of EQ-5D-Y-3L (Y-3L), EQ-5D-Y-5L (Y-5L), and child health utility 9D (CHU9D) in pediatric patients with eczema.</p><p><strong>Methods: </strong>Patients completed the Y-3L, Y-5L, CHU9D, and the Child Dermatology Life Quality Index and were followed up during their next visit. Clinicians assessed patients' skin status using the Validated Investigator Global Assessment scale for Atopic Dermatitis. Ceiling effects of the 3 instruments were compared. Convergent validity was assessed using correlation with the Child Dermatology Life Quality Index; known-group validity was evaluated using F-statistics; and responsiveness was assessed using standardized effect sizes (SESs) among patients reporting improved health.</p><p><strong>Results: </strong>A total of 164 patients (mean age: 12.2 years; female: 47.6%) participated in the study. Of those, 67 patients (mean age:11.9; female: 53.8%) completed the follow-up survey. The ceiling effects of Y-3L, Y-5L, and CHU9D were 34.8%, 28.7%, and 9.8%, respectively. All known-group hypotheses, defined by the Validated Investigator Global Assessment scale for Atopic Dermatitis and scratching frequency, were met, with Y-5L showing the strongest performance in discriminating between patients based on symptom severity. Convergent validity was met in 70.0%, 70.0%, and 83.3% of hypotheses tested for Y-3L, Y-5L, and CHU9D, respectively. CHU9D (SES = 0.53) was relatively more responsive to change than Y-3L (SES= 0.18) or Y-5L (SES = 0.43).</p><p><strong>Conclusions: </strong>Y-3L and Y-5L were more sensitive to clinical outcome differences, whereas CHU9D appeared to be more responsive to self-rated health improvement. Further research using clinical measures and more diverse eczema patient samples is needed to confirm these findings and support evidence-based instrument selection.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144800396","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
Do We Always Need a New Preference Study? A Scoping Review of Promising Research Areas for Meta-Analyses and Benefit Transfers of Patient Preference Studies 我们是否总是需要新的偏好研究?荟萃分析和患者偏好研究的利益转移的前景研究领域的范围综述。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-05 DOI: 10.1016/j.jval.2025.06.004
Michael Bui MSc , Catharina G.M. Groothuis-Oudshoorn PhD , A. Cecilia Jimenez-Moreno PhD , Byron Jones PhD , Conny Berlin Dipl-Math , Janine A. van Til PhD
{"title":"Do We Always Need a New Preference Study? A Scoping Review of Promising Research Areas for Meta-Analyses and Benefit Transfers of Patient Preference Studies","authors":"Michael Bui MSc ,&nbsp;Catharina G.M. Groothuis-Oudshoorn PhD ,&nbsp;A. Cecilia Jimenez-Moreno PhD ,&nbsp;Byron Jones PhD ,&nbsp;Conny Berlin Dipl-Math ,&nbsp;Janine A. van Til PhD","doi":"10.1016/j.jval.2025.06.004","DOIUrl":"10.1016/j.jval.2025.06.004","url":null,"abstract":"<div><h3>Objectives</h3><div>Although patient preference (PP) studies are costly, time intensive, and burdensome on patients, their findings are rarely used beyond the purpose of the original study. If PP study findings could be transferred to other contexts through meta-regression (benefit transfers), resources could be better utilized. We conducted a scoping review to assess the readiness of the current PP study landscape for evidence synthesis and benefit transfers.</div></div><div><h3>Methods</h3><div>Quantitative PP studies examining risks and benefits of treatments were identified through a systematic search on PubMed, Scopus, and Web of Science. Based on benefit transfer guidelines from environmental economics, prospects for transferring PP study findings were judged based on the number of studies across indications, consistency in elicitation methods, consistency in treatment attributes, and consistency in computed preference parameters.</div></div><div><h3>Results</h3><div>In total, 777 studies were included. Of these, 580 were discrete choice experiments (DCEs). Geographically, most studies were conducted in the United States (<em>N</em> = 271), multicountry designs (<em>N</em> = 105), Germany (<em>N</em> = 61), the United Kingdom (<em>N</em> = 59), and The Netherlands (<em>N</em> = 54). Indication wise, most research was concentrated in type 2 diabetes (T2D) (46 DCEs, 7 non-DCEs), psoriasis (24 DCEs, 8 non-DCEs), and multiple sclerosis (21 DCEs, 7 non-DCEs).</div></div><div><h3>Conclusions</h3><div>The landscape of PP studies is dispersed across various indications and therapeutic focus areas, which generally limits interstudy comparisons. However, numerous DCEs on T2D exhibited a high consistency in computed preference parameters and a moderately high degree of overlap in studied attributes (hypoglycemia, glycemic control, weight change, and out-of-pocket costs). Hence, benefit transfers seem feasible in T2D.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 9","pages":"Pages 1426-1443"},"PeriodicalIF":6.0,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144785398","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
Genotype-Specific Tricyclic Antidepressant Dosing in Patients With Major Depressive Disorder: A Trial-Based Economic Evaluation. 重度抑郁症患者基因型特异性三环抗抑郁药剂量:一项基于试验的经济评估。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-05 DOI: 10.1016/j.jval.2025.07.018
Sophie E Ter Hark, Wietske Kievit, Gerjon Hannink, Cornelis F Vos, Jan Spijker, Annemarie van der Meij, Anne J Grotenhuis, Raluca Mihaescu, Rogier Donders, Rob E Aarnoutse, Arnt F A Schellekens, Marieke J H Coenen, Joost G E Janzing
{"title":"Genotype-Specific Tricyclic Antidepressant Dosing in Patients With Major Depressive Disorder: A Trial-Based Economic Evaluation.","authors":"Sophie E Ter Hark, Wietske Kievit, Gerjon Hannink, Cornelis F Vos, Jan Spijker, Annemarie van der Meij, Anne J Grotenhuis, Raluca Mihaescu, Rogier Donders, Rob E Aarnoutse, Arnt F A Schellekens, Marieke J H Coenen, Joost G E Janzing","doi":"10.1016/j.jval.2025.07.018","DOIUrl":"10.1016/j.jval.2025.07.018","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate the cost-effectiveness of preemptive CYP2D6 and CYP2C19 genotype-informed tricyclic antidepressant dosing from a societal and a healthcare perspective.</p><p><strong>Methods: </strong>A trial-based cost-effectiveness analysis was conducted with data from the Pharmacogenetics for Individualized Tricyclic Antidepressant dosing study. This multicenter randomized controlled trial (n = 111) compared pharmacogenetic-informed treatment (PIT) with treatment as usual. Quality-adjusted life-years (QALY) and costs were measured at 13 and 26 weeks. Prices were based on or indexed to 2022 tariffs. Single imputation nested in the bootstrap percentile method (using 5000 bootstrap replications) was performed to address missing data and to estimate uncertainty around cost-effectiveness outcomes. Incremental net monetary benefit (iNMB) was calculated based on a willingness to pay (WTP) of €50 000/QALY.</p><p><strong>Results: </strong>Our data showed a marginal difference of -0.0125 QALYs (95% confidence interval [CI] -0.0404 to 0.0149, week 13) and 0.0012 QALYs (95% CI -0.0491 to 0.0574, week 26) for PIT versus treatment as usual. From the healthcare perspective, a cost saving of €148 (week 13) and €521 (26 week) was found for PIT. The societal perspective showed increased costs of €1300 (week 13) and €1704 (26 weeks) for PIT. The mean iNMB from a healthcare perspective was positive at 26 weeks, the other iNMBs (13 weeks and societal perspective) were negative.</p><p><strong>Conclusions: </strong>We observed marginal differences of QALYs in both the healthcare and societal perspective with cost savings from the healthcare perspective and additional cost from the societal perspective. These mixed results warrant more long-term observational studies to determine whether preemptive genotyping in tricyclic antidepressant dosing will be cost-effective.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144795650","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
Toward a Common Ground for Defining Digital Health Interventions, Mapping Digital Health Frameworks to PICOTS-ComTeC: An ISPOR Special Interest Group Report. 迈向定义数字健康干预措施的共同基础,将数字健康框架映射到picot - comtec: ISPOR特别兴趣小组报告。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-05 DOI: 10.1016/j.jval.2025.07.022
Annette Champion, Anita Burrell, Anke-Peggy Holtorf, Rossella Di Bidino, Jagadeswara Rao Earla, Artem T Boltyenkov, Masami Tabata-Kelly, Carl Asche, Brian Seal, Zsombor Zrubka
{"title":"Toward a Common Ground for Defining Digital Health Interventions, Mapping Digital Health Frameworks to PICOTS-ComTeC: An ISPOR Special Interest Group Report.","authors":"Annette Champion, Anita Burrell, Anke-Peggy Holtorf, Rossella Di Bidino, Jagadeswara Rao Earla, Artem T Boltyenkov, Masami Tabata-Kelly, Carl Asche, Brian Seal, Zsombor Zrubka","doi":"10.1016/j.jval.2025.07.022","DOIUrl":"10.1016/j.jval.2025.07.022","url":null,"abstract":"<p><strong>Objectives: </strong>Digital health interventions (DHIs) should be defined in a comparable, structured manner to facilitate research informing clinical and financial decisions. The Population, Intervention, Comparator, Outcomes, Timing, Setting, Communication, Technology, Context (PICOTS-ComTeC) framework was developed to define patient-facing DHIs for health economics and outcomes research. Our objective was to compare PICOTS-ComTeC with established DHI frameworks and guidelines to determine the degree of overlap, additional value of PICOTS-ComTeC, and how the frameworks might be used together.</p><p><strong>Methods: </strong>An expert group selected comparator frameworks. Reviewer pairs extracted information and mapped DHI definitions to 9 domains and 32 subcategories of PICOTS-ComTeC. A third reviewer checked for consistency across frameworks and missing data.</p><p><strong>Results: </strong>A diverse group of 16 frameworks (9 international, regarding DHI classification, quality, labeling, and reporting; 6 national DHI health technology assessment and payer; 1 international health economic reporting) were compared with PICOTS-ComTeC. Across all frameworks, 81% (116/144) of PICOTS-ComTeC domains matched (range 44%-100%). The mean number of domain matches for a framework was 7.3. Comparator frameworks matched 48% (247/512) of PICOTS-ComTeC subcategories (range 16%-81%).</p><p><strong>Conclusions: </strong>The degree to which PICOTS-ComTeC is congruous with items in diverse DHI frameworks suggests that PICOTS-ComTeC represents a common ground for defining patient-facing DHIs for research, reporting, and assessment purposes, thereby improving patient care by accelerating adoption of effective DHIs. PICOTS-ComTeC contains items not uniformly present in comparator frameworks. PICOTS-ComTeC can be used to define patient-facing DHIs by adding missing PICOTS-ComTeC items to comparator frameworks or using information from comparator frameworks to describe PICOTS-ComTeC items.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144800399","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
EQ-5D-5L in multiple sclerosis: Estimated utility values, minimal (clinically) important changes, and direct and indirect influences over time. EQ-5D-5L在多发性硬化症中的作用:估计效用值,最小(临床)重要变化,以及随时间的直接和间接影响。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-04 DOI: 10.1016/j.jval.2025.07.019
Carolyn A Young, Roger J Mills, Alan Tennant
{"title":"EQ-5D-5L in multiple sclerosis: Estimated utility values, minimal (clinically) important changes, and direct and indirect influences over time.","authors":"Carolyn A Young, Roger J Mills, Alan Tennant","doi":"10.1016/j.jval.2025.07.019","DOIUrl":"https://doi.org/10.1016/j.jval.2025.07.019","url":null,"abstract":"<p><strong>Objectives: </strong>In multiple sclerosis (MS), studies reporting mean EQ-5D-5L health utility values ranged from 0.31-0.82 and a 2024 systematic review found no published anchor-based estimates for EQ-5D-5L Minimal Important Change (MIC). We derived age-sex specific utility reference values and MIC of the EQ-5D-5L in a large UK sample with MS.</p><p><strong>Methods: </strong>The Trajectories of Outcomes in Neurological Conditions-MS study calculated EQ-5D-5L age-sex and disability-subtype specific utility estimates. Multiple regression identified possible predictors of health status for inclusion into a Structural Equation Model (SEM). Calculation of MIC used an anchor-based method.</p><p><strong>Results: </strong>Among 5509 participants, female (73.7%), aged 17-87, mean EQ-5D-5L utility was 0.682 (95% CI: 0.675-0.688; range -0.28-1.0). There was an age gradient and difference between male and female utilities across each age group (age: F 34.3 (df 11, 497): p<0.001; sex: t 5.794; (df 5507): p<0.001). In SEM, higher self-efficacy improved EQ-5D-5L while cognitive problems, progressive disease, fatigue, bladder problems and stigma decreased EQ-5D-5L. In the longitudinal sample comprising 2066 pwMS, mean utility value at follow-up was 0.673 (95% CI: 0.662-0.683), MIC 0.146 (95% CI: 0.137-0.154). MIC varied significantly by MS subtype and for those reporting a relapse within the past year.</p><p><strong>Conclusions: </strong>Health utility values for MS are consistently lower than those found in the English population, and are influenced by factors amenable to clinical intervention. Anchor-based MIC varied by subtype and relapse history, showing that when assessing the significance of changing health status from the perspective of those with MS, a uniform MIC should not be employed.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144795649","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 Functional Assessment of Cancer Therapy Eight Dimension (FACT-8D), a Multi-Attribute Utility Instrument Derived From the Cancer-Specific FACT-General (FACT-G) Quality of Life Questionnaire: Development and Australian Value Set 癌症治疗八维度功能评估 (FACT-8D),一种从癌症专用 FACT-G 生活质量问卷衍生而来的多属性实用性工具:开发和澳大利亚价值集。
IF 6 2区 医学
Value in Health Pub Date : 2025-08-01 DOI: 10.1016/j.jval.2024.06.001
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引用次数: 0
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