Value in Health最新文献

{"title":"Developing an Australian Value Set for the Recovering Quality of Life-Utility Index (ReQoL-UI) 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":"https://doi.org/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 over 16 years of age with mental health problems. We aim 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 (DCE) with duration was embedded in a self-complete online survey and administered to a representative sample (n=1019) of the Australian adult population aged 18 years and more, stratified for age, sex and geographic location. A partial subset design DCE was used with 3 fixed attributes and 5 varying attributes containing 240 choice tasks that were blocked into 20 blocks, so that each respondent was assigned a block of 12 choice tasks. The value set was modelled using the conditional logit model with utility decrements directly anchored on the 0 to 1 dead-full health scale. Preference heterogeneity was tested using the 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 while the least important attribute was self-perception. Sensitivity analysis and analysis of preference heterogeneity show the stability of the value set.</p><p><strong>Conclusion: </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 interventions 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}

{"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}

{"title":"Unpaid Caring and Health-Related Quality of Life: Longitudinal Analysis of Understanding Society (the UK Household Longitudinal Survey).","authors":"Becky M Pennington, Mónica Hernández Alava, Mark Strong","doi":"10.1016/j.jval.2024.08.004","DOIUrl":"10.1016/j.jval.2024.08.004","url":null,"abstract":"<p><strong>Objectives: </strong>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).</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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, Tejaswi Datla, Meena N Murugappan, Bindu Kanapuru, Laleh Amiri-Kordestani, Erin Larkins, Paul Kluetz, Vishal Bhatnagar","doi":"10.1016/j.jval.2024.09.009","DOIUrl":"10.1016/j.jval.2024.09.009","url":null,"abstract":"<p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","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}

{"title":"Primer on Health Equity Research in Health Economics and Outcomes Research: An ISPOR Special Interest Group Report.","authors":"Matthew J S Griffiths, Richard Cookson, Anton L V Avanceña, Manuel A Espinoza, Caroline M Jacobsen, Jesse Sussell, Stacey Kowal","doi":"10.1016/j.jval.2024.09.012","DOIUrl":"https://doi.org/10.1016/j.jval.2024.09.012","url":null,"abstract":"<p><strong>Objectives: </strong>Disparities in health and healthcare between more and less socially advantaged groups are pervasive, multidimensional, and far-reaching. The material and social conditions in which people are born, grow, work, live, and age are systematically associated with their health and with the volume, quality, and outcomes of care received by the vast majority of the general population, as well as by specific marginalized populations. The field of health economics and outcomes research (HEOR) has an important role in supporting health equity goals. This publication aimed to act as a \"primer\" for conducting health equity research within the field of HEOR, establishing foundational understanding of key concepts.</p><p><strong>Methods: </strong>The ISPOR Special Interest Group on Health Equity Research was established in 2021 to advance equity-informative methods and data to better enable researchers to empirically investigate-and ultimately reduce-unfair social differences in health. This publication was developed by the ISPOR Special Interest Group leadership team with input from the group membership.</p><p><strong>Results: </strong>The resultant publication provides an overview of health equity research methods and data considerations as they relate to HEOR-relevant topics including clinical trials, real-world evidence and economic evaluation. Reflecting the current body of research on health equity in HEOR, particular focus is given to the latter. It also brings together a list of core reference material to support future learning.</p><p><strong>Conclusions: </strong>This report provides the HEOR community with a tailored \"state of play\" overview of health equity, to support development of foundational understanding and inspire increased engagement.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"16-24"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955752","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":"Proposal for a General Outcome-Based Value Attribution Framework for Combination Therapies.","authors":"Lotte Steuten, Mickael Lothgren, Andrew Bruce, Marco Campioni, Adrian Towse","doi":"10.1016/j.jval.2024.07.019","DOIUrl":"10.1016/j.jval.2024.07.019","url":null,"abstract":"<p><strong>Objectives: </strong>Valuing and pricing the components of combination therapies can be difficult because of competition law issues, difficulty implementing different prices for the same product in alternative uses, and attributing value to each component of the combination. We propose a value attribution solution that allows all combination components to be priced according to their relative value in the combination.</p><p><strong>Methods: </strong>We developed a value attribution solution that is universal, symmetrical, and neutral to each combination constituent, regardless of whether it is the backbone or the add-on, and complete, meaning that it will always attribute the full value of the combination between the component parts. Moreover, it can be applied to any number of components in the combination (eg, triplets or quadruplets). We compared this solution with 2 other existing approaches.</p><p><strong>Results: </strong>The results of the proposed value attribution solution sit between those of the 2 other value attribution approaches as it combines elements of each. As the degree of additivity moves further away from one in either direction, then our general approach ratios also move, reflecting the impact of the incremental value.</p><p><strong>Conclusions: </strong>The proposed value attribution solution for combination therapies differs from 2 existing approaches by being universally applicable and allowing for symmetry when neutral to the constituent components of the combination. To optimally contribute to policy debate and practice, various requirements for its implementation need to be well understood, including how to overcome (1) partial information, (2) whether its assumptions can be relaxed, and (3) implementation issues.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":"81-87"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914159","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":"Will the Institute for Clinical and Economic Review's Shared Savings Approach Decrease Value-Based Prices Most for the Most Severe Diseases?","authors":"Jason Shafrin, Shanshan Wang, Khounish Sharma, Kathryn Spurrier, Robert J Nordyke","doi":"10.1016/j.jval.2024.09.002","DOIUrl":"10.1016/j.jval.2024.09.002","url":null,"abstract":"<p><strong>Objectives: </strong>To identify the types of disease most likely to be affected by the Institute for Clinical and Economic Review's (ICER) shared savings assumptions.</p><p><strong>Methods: </strong>For diseases with treatments that were Food and Drug Administration approved between 2019 and 2023, annual direct and indirect economic burden and characteristics of each disease were extracted from peer-reviewed literature. ICER's shared savings methodology was applied 2 ways: 50/50 shared savings and $150 000 cost-offset cap. The primary outcome was the difference in eligible cost savings provided by a hypothetical disease cure under ICER's 2 shared savings methods. Characteristics of diseases most impacted by these 2 methods were evaluated descriptively.</p><p><strong>Results: </strong>Food and Drug Administration approved 260 therapies for 89 unique diseases between 2019 and 2023. Shared savings reduced value of a hypothetical cure for hemophilia A most (50/50 method: -$367 670 per year; cap method: -$585 340 per year), followed by acute hepatic porphyria (50/50 method: -$333 948; cap method: -$517 896) and paroxysmal nocturnal hemoglobinuria (50/50 method: -$291 997; cap method: -$433 993). Compared with diseases with annual burdens <$150 000, those ≥$150 000 had earlier disease onset by 22.0 years (age 12.3 vs 34.3), lower life expectancy by 10.6 years (55.8 vs 66.4 years), and lower disease prevalence (4.7 vs 1981.5 per 100 000). Shared savings' impact on health-benefit price benchmarks was projected to be larger for diseases with shorter life expectancy (ρ = -0.319; p =.005), worse quality of life (ρ = -0.263; P =.020), and lower prevalence (ρ = -0.418; P < .001).</p><p><strong>Conclusions: </strong>ICER's shared savings assumptions would most likely have the largest negative impact on health-benefit price benchmarks for rare, severe, and pediatric diseases.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":"25-30"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142366714","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":"An Attribution of Value Framework for Combination Treatments.","authors":"Andrew H Briggs, Alexis Doyle-Connolly, John Schneider, Tanja Podkonjak, Helen Taylor, Emma Roffe, Eric Low, Sarah Davis, Martin Kaiser, Anthony J Hatswell, Neil Rabin","doi":"10.1016/j.jval.2024.08.012","DOIUrl":"10.1016/j.jval.2024.08.012","url":null,"abstract":"<p><strong>Objectives: </strong>The use of cost-effectiveness methods to support policy decisions has become well established, but difficulties can arise when evaluating a new treatment that is indicated to be used in combination with an established backbone treatment. If the latter has been priced close to the decision maker's willingness-to-pay threshold, this may mean that there is no headroom for the new treatment to demonstrate value, at any price, even if the combination is clinically effective. Without a mechanism for attributing value to component treatments within a combination therapy, the health system risks generating negative funding decisions for combinations of proven clinical benefit to patients. The aim of this work was to define a value attribution methodology, which could be used to allocate value between the components of any combination treatment.</p><p><strong>Methods: </strong>The framework is grounded in the standard decision rules of cost-effectiveness analysis and provides solutions according to key features of the problem: perfect/imperfect information about component treatment monotherapy effects and balanced/unbalanced market power between their manufacturers.</p><p><strong>Results: </strong>The share of incremental value varies depending on whether there is perfect/imperfect information and balance/imbalance of market power, with some scenarios requiring the manufacturers to negotiate a share of the incremental value within a range defined by the framework.</p><p><strong>Conclusions: </strong>It is possible to define a framework that is independent of price and focuses on benefits expressed as quality-adjusted life-year gains (and/or quality-adjusted life-year equivalents for cost savings), a standard metric used by many health technology assessment agencies to evaluate novel treatments.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":"72-80"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142558801","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":"Cancer Drug Wastage and Mitigation Methods: A Systematic Review.","authors":"Krishna Sabareesh Rajangom, F Safa Erenay, Qi-Ming He, Rachel Figueiredo, Kelvin K W Chan, Matthew C Cheung, Lauren F Charbonneau, Susan E Horton, Avram Denburg","doi":"10.1016/j.jval.2024.08.006","DOIUrl":"10.1016/j.jval.2024.08.006","url":null,"abstract":"<p><strong>Objectives: </strong>To systematically review published evidence on cancer drug wastage and the effectiveness of mitigation methods.</p><p><strong>Methods: </strong>Search keywords for Scopus, PubMed, and EMBASE were developed using the Pearl Growing technique. Relevant articles were identified in a 2-step process: first, based on titles/abstracts, then on full article reviews. Among the identified English peer-reviewed articles, those considering adults ≥18 years and relevant cancer drug wastage outcomes were included. Key concepts and measures for drug wastage and its mitigation were tabulated. Trends in publication numbers were analyzed using Mann-Kendall tests. Costs were converted first to 2024 local currencies using country-wise consumer price indexes and then to 2024 USD using exchange rates.</p><p><strong>Results: </strong>Among 6298 unique articles, 94 met the inclusion criteria. Seventy-four (79%) of these were published since 2015, highlighting increasing attention to cancer drug wastage. Twenty-three articles (24%) explicitly reported drug wastage amounts, whereas 52 articles (55%) considered the mitigation methods. Most articles focused on high-income countries (n = 67), single-hospital settings (n = 45), and retrospective study designs (n = 55). Wastage mitigation techniques included vial sharing (n = 21), dose rounding (n = 17), closed-system transfer device (n = 9), centralized drug preparation (n = 7), and vial size optimization (n = 7). A trend toward higher median wastage cost was evident in US settings ($135.35/patient-month) compared with other countries ($37.71/patient-month), whereas mitigation methods across countries were not statistically significant.</p><p><strong>Conclusions: </strong>High cancer drug costs highlight the importance of minimizing drug wastage to reduce healthcare expenditure. Our review demonstrates that wastage varies by healthcare setting and mitigation technique. Future studies would benefit from reporting standards for cancer drug wastage that include reporting wastage (both in mg and cost, preferably in terms of purchase power parity), as well as cohort size, considered vial sizes, considered dosages, and used mitigation methods separately for each drug. This approach would account for variability in cancer drug wastage and help identify optimal mitigation practices tailored to the health system context.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":"148-160"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354769","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":"Dysmenorrhea-Related Impact on Functioning Scale: Development and Measurement Properties for Cisgender Women and Transgender Men.","authors":"Guilherme T Arruda, Maria Eduarda C B da Silva, Barbara I da Silva, Patricia Driusso, Mariana A Avila","doi":"10.1016/j.jval.2024.08.007","DOIUrl":"10.1016/j.jval.2024.08.007","url":null,"abstract":"<p><strong>Objectives: </strong>To develop the Dysmenorrhea-related Impact on Functioning Scale (DIFS) to assess the impact of dysmenorrhea on functioning in cisgender women and transgender men and to evaluate its measurement properties.</p><p><strong>Methods: </strong>Mixed and online design study conducted with adolescents and adult cisgender women and transgender men with dysmenorrhea. We developed the DIFS based on the International Classification of Functioning, Disability, and Health. Content validity was assessed with experts and people with dysmenorrhea. Item Response Theory developed the DIFS total score. Structural validity was assessed by exploratory and confirmatory factor analysis and internal consistency by Cronbach's α and McDonald's Ω. Construct validity and test-retest reliability were assessed by correlation between DIFS and World Health Organization Disability Assessment Schedule and intraclass correlation coefficient, respectively. Measurement error was also assessed.</p><p><strong>Results: </strong>A total of 3335 people participated in the study. The DIFS is a 15-item instrument divided into \"Bodily Functions\" and \"Daily Activities and Social Participation\" sections and \"Functioning\" as a general factor. Internal consistency (α and Ω > 0.7) and test-retest reliability (intraclass correlation coefficient > 0.9) were adequate. No systematic error was found. Correlation was positive and strong between World Health Organization Disability Assessment Schedule and \"Functioning\" (r = 0.62, P ≤ .05). For the DIFS total score, higher scores indicate a greater impact of dysmenorrhea on functioning, and 44 points is the cutoff point for classifying the person with a significant impact of dysmenorrhea on functioning.</p><p><strong>Conclusions: </strong>DIFS showed excellent measurement properties for assessing the impact of dysmenorrhea on functioning for cisgender women and transgender men.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":"99-107"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354771","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}