PharmacoEconomics最新文献

{"title":"Making Composite Time Trade-Off Sensitive for Worse-than-Dead Health States.","authors":"Michał Jakubczyk, Bram Roudijk, Stefan A Lipman, Peep Stalmeier","doi":"10.1007/s40273-025-01471-6","DOIUrl":"10.1007/s40273-025-01471-6","url":null,"abstract":"<p><strong>Objective: </strong>The utilities elicited with the composite time trade-off (cTTO) method for health states worse-than-dead (WTD) often correlate poorly with other severity measures, indicating a poor sensitivity of cTTO. We aimed to explore modifications to cTTO to better understand this phenomenon and identify potential improvements.</p><p><strong>Methods: </strong>A total of 480 respondents completed an online TTO interview, each valuing 12 EQ-5D-5L health states. The participants were randomized into four arms, A-D. Arm A followed the standard cTTO, serving as a reference. In arm B, we removed the sorting question comparing immediate death versus 10 years in a valued state. Arm C allowed for utility values <math><mrow><mo><</mo> <mo>-</mo> <mn>1</mn></mrow> </math> by reducing the time in the valued state in the lead-time TTO (LT-TTO) part of cTTO. In arm D, we randomly selected the starting negative utility in LT-TTO. Utility value distributions, correlations between utilities and level sum score (LSS), and inconsistencies between Pareto-ordered states were analyzed.</p><p><strong>Results: </strong>Arm A replicated the lack of significant correlation between LSS and the negative utility observed in previous work. Of the experimental arms, only arm B exhibited a significant negative correlation. Compared with arm A, arm B produced a higher proportion of WTD states ( <math><mrow><mn>46.5</mn> <mo>%</mo></mrow> </math> versus  <math><mrow><mn>26.3</mn> <mo>%</mo></mrow> </math> ), less negative utility for WTD states on average ( <math><mrow><mo>-</mo> <mn>0.571</mn></mrow> </math> versus  <math><mrow><mo>-</mo> <mn>0.752</mn></mrow> </math> ), and a lower mean censored utility for 55555 ( <math><mrow><mo>-</mo> <mn>0.486</mn></mrow> </math> versus  <math><mrow><mo>-</mo> <mn>0.406</mn></mrow> </math> ).</p><p><strong>Conclusions: </strong>The observed lack of correlation between LSS and utility for WTD states appears linked to the use of comparison with immediate death in the sorting question. LT-TTO is capable of eliciting utility values in a way that is sensitive to severity. Modifying the initial questions in cTTO to identify whether health states are BTD or WTD should be considered.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"665-675"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12081533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143502145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Review of Heterogeneity in Comparative Economic Analysis, with Specific Considerations for the Decentralized US Setting and Patient-Centered Care.","authors":"Michael S Willis, Andreas Nilsson, Cheryl A Neslusan","doi":"10.1007/s40273-025-01478-z","DOIUrl":"10.1007/s40273-025-01478-z","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Patient-centered care emphasizes individual preferences, but insurer coverage decisions-based on population-level evidence-may restrict treatment options for patients who differ from the average. This highlights the importance of considering heterogeneity, which refers to differences in health and cost outcomes that are systematically linked to variations in factors like patient characteristics, insurer policies, and provider practices. Failing to account for heterogeneity in economic evaluations can lead to suboptimal decisions, inferior outcomes, and inefficiency. This study aimed to assess the tools and methods for addressing heterogeneity in economic evaluations, examine the extent to which, and how, heterogeneity has been addressed in US cost-utility studies, and provide insights and recommendations to promote more fuller consideration of heterogeneity in US economic evaluations. We reviewed and adapted a seminal taxonomy of heterogeneity to the US setting, highlighting key drivers like patient preferences and insurance design. Methods for addressing heterogeneity in economic evaluations were also reviewed and summarized. We used data from the Tufts Medical Center Cost-Effectiveness Analysis Registry to assess empirical practices in US cost-utility applications, specifically the frequency, types, and impact of a subgroup analysis, and whether rationales for including or excluding subgroups were provided. The revised taxonomy highlights key drivers of heterogeneity in the diverse and decentralized US healthcare ecosystem, such as the diversity of patient preferences and in non-patient factors like access to healthcare providers and insurance coverage. Methods to explore, confirm, and incorporate heterogeneity into a comparative economic analysis exist, but are often challenged by data availability. In addition to the trade-off between potential efficiency gains and increasing uncertainty in comparative value estimates, ethical implications of stratified decisions were highlighted in the literature. We found that a subgroup analysis was rare, and primarily performed for clinical factors like age and disease severity. Only 2 of the 85 studies published between 2015 and 2022 with subgroup-level results were found to consider non-patient factors, and none considered preferences. One-third of studies reported incremental cost-effectiveness ratios differing by more than 50% from the unstratified estimate. No studies provided a rationale for omitting a subgroup analysis, and only two motivated inclusion of a subgroup analysis, limiting our ability to assess the appropriateness of these decisions. Despite well-documented methods to address heterogeneity, its application is limited in US cost-utility studies, especially regarding patient preferences and non-patient factors. As these factors often drive real-world health outcomes and costs in the USA, proper consideration of, and reporting on, heterogeneity is essential to avoid erroneous market ","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"601-616"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12081492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586635","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Monetary Value of a Statistical Life in the Context of Atherosclerotic Cardiovascular Disease.","authors":"Jorge-Eduardo Martínez-Pérez, Fernando-Ignacio Sánchez-Martínez, José-María Abellán-Perpiñán, Domingo Pascual-Figal","doi":"10.1007/s40273-025-01482-3","DOIUrl":"10.1007/s40273-025-01482-3","url":null,"abstract":"<p><strong>Aim: </strong>This study aims to estimate the value of a statistical life (VSL) in the context of atherosclerotic cardiovascular disease (ASCVD) in Spain using a contingent valuation/standard gamble (CV/SG) chained approach.</p><p><strong>Methods: </strong>The study employed a two-stage preference elicitation method that combined contingent valuation and a modified standard gamble technique. Specifically, willingness-to-pay and willingness-to-accept values were obtained for two health states depicting hypothetical outcomes following cardiovascular events. Subsequently, relative utility losses for the health states were derived using a modified standard gamble framing two risky choices. Chaining these elicited values allowed for VSL calculation without requiring direct valuation of small mortality risk reductions. The study was conducted through in-person interviews with a representative sample of 412 Spanish adults selected by stratified quotas.</p><p><strong>Results: </strong>The estimated VSL range is from 1.59 to 2.06 million euros. Minor differences emerge between VSL figures on the basis of each of the two health states. These VSL estimates for ASCVD are congruent with the recent update of the official VSL estimated for Spain in the context of road traffic accidents, though the upper limit of the range is slightly higher (almost 9%).</p><p><strong>Conclusions: </strong>VSL estimates align with existing ranges in other European countries, particularly in the context of road safety, where a significant portion of existing studies is concentrated. Comparisons with other contexts, involving cardiovascular diseases, also lend support to the estimates presented here.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"677-689"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12081548/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743551","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"What are the Revealed and Stated Population Preferences for Environmental Sustainability in Healthcare? A Scoping Review.","authors":"Charlotte Desterbecq, Mark Harrison, Sandy Tubeuf","doi":"10.1007/s40273-025-01479-y","DOIUrl":"10.1007/s40273-025-01479-y","url":null,"abstract":"<p><strong>Objective: </strong>Collective changes in healthcare practices are required to ensure real environmental gains. As patient-centred care is increasingly considered to enhance the ability of health systems to meet the expectations of the population, it is crucial for policymakers and health professionals to account for the preferences of the wider public regarding environmentally friendly healthcare. This article synthesises and appraises evidence from empirical studies to understand how people value environmental concerns when making decisions within medical-related or pharmaceutical sectors.</p><p><strong>Methods: </strong>We conducted electronic searches of the PubMed, Scopus, and Embase literature databases. Studies were eligible if they conducted a quantitative experiment to understand participants' preferences regarding sustainability and green initiatives in the medical sector or for pharmaceuticals.</p><p><strong>Results: </strong>Of the 1138 documents identified, 32 studies were deemed eligible. More than 60% were published since 2020. Different methods were used to elicit the revealed and/or stated preferences of participants. In most studies, respondents valued the environment positively and were willing to change their behaviour or practices to support sustainability. However, concerns such as disease severity or clinical effectiveness of medicines or medical interventions were often prioritised over environmental considerations. The wide heterogeneity in study participants emphasises the need to involve all stakeholders to achieve the transition to a greener and sustainable healthcare system.</p><p><strong>Conclusion: </strong>The identified studies used various methods but were consistent in finding broad support for environmental considerations within the healthcare sector.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"617-627"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Nirsevimab for Preventing Respiratory Syncytial Virus-Related Lower Respiratory Tract Disease in Dutch Infants: An Analysis Including All-Infant Protection.","authors":"Florian Zeevat, Simon van der Pol, Alexia Kieffer, Maarten J Postma, Cornelis Boersma","doi":"10.1007/s40273-025-01469-0","DOIUrl":"10.1007/s40273-025-01469-0","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to assess the cost effectiveness of nirsevimab, a recently authorized monoclonal antibody (mAb) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV), in comparison with the standard practice involving palivizumab for high-risk infants during their first RSV season in the Netherlands.</p><p><strong>Methods: </strong>A static cost-effectiveness model was populated for the Netherlands to evaluate different immunization strategies for nirsevimab over a single RSV season from a societal perspective. The model considered the most recently published RSV incidence data (average incidence from 2006 to2018), costs (adjusted to the 2023 price year), and associated health effects. Extensive scenario analyses were conducted to explore various strategies, and sensitivity analysis was performed to assess the model's robustness.</p><p><strong>Results: </strong>In the base-case scenario, all-infant protection-a strategy of in-season with catch-up immunization for all infants-nirsevimab has the potential to prevent numerous RSV-related cases, including 2333 hospitalizations and 150 intensive-care admissions, in the overall population compared with the standard of care. Nirsevimab appears to be cost effective under this strategy with an economically justifiable acquisition price for nirsevimab of €220 at a willingness-to-pay threshold of €50,000 per quality-adjusted life-year. Sensitivity analyses indicate a 52% probability that nirsevimab is cost effective at this threshold. Comparison of different vaccination strategies revealed that the all-infant protection approach was the one that prevented the higher number of cases.</p><p><strong>Conclusions: </strong>This study indicates that universal infant immunization with nirsevimab has the potential to be cost effective and significantly reduces the burden of RSV among Dutch infants. These findings underscore the importance of implementing effective protective measures against RSV-LRTD, reducing the pressure on the healthcare system during the RSV season.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"569-582"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011648/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acceptance of Evidence Transfer Within German Early Benefit Assessment of New Drugs for Pediatric and Adolescents Target Populations.","authors":"Georgia Pick, Dirk Müller, Charalabos-Markos Dintsios","doi":"10.1007/s40273-024-01467-8","DOIUrl":"10.1007/s40273-024-01467-8","url":null,"abstract":"<p><strong>Background and objective: </strong>In Germany, all new drugs undergo an early benefit assessment (EBA) by the decision-making body (G-BA). Due to limited access to clinical data in pediatric healthcare since 2017, evidence transfer has allowed for data from adult studies to be used in the EBA of pediatric drugs. This study examines the acceptance of evidence transfer, aiming to understand its correlation with granted added benefit.</p><p><strong>Methods: </strong>By searching the G-BA database, relevant EBAs were identified. In addition to descriptive statistics, agreement statistics regarding binary and ordinal extent of added benefit and binary logistic regression with and without intercept were performed to investigate acceptance of evidence transfer, juxtaposing it with manufacturers' claims, and to evaluate the impact of identified factors on evidence transfer.</p><p><strong>Results: </strong>In 14 of 36 identified EBAs, the evidence transfer was accepted by the G-BA. They referred to four therapeutic areas, received a non-quantifiable added benefit and were subject to a pediatric investigation program. Non-quantifiable added benefit implies an added value in itself which can range from minor to major added benefit and is considering the genuine uncertainty mainly induced in theese EBAs due to evidence transfer, which is not allowing a quantification of an added benefit. The binary agreement between manufacturers' claims and G-BA's appraisals was less than by chance [kappa - 0.054 (- 0.158 to 0.050)] whereas the ordinal agreement became fair [kappa 0.333 (0.261-0.406)]. Congruence of the mechanism of action, alignment of disease pattern, transferability of efficacy and safety, and same comparator were fundamental for evidence transfer. Additionally, supportive evidence, therapeutic breakthroughs, and small-scale approval enhanced the acceptance of evidence transfer. The regression models yielded similar results showing different model fit and explained variance.</p><p><strong>Conclusions: </strong>Evidence transfer hinges upon fulfilling various minimum criteria and additional supportive evidence. Availability of study data from adult or older patients and the pediatric group under evaluation is crucial.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"521-553"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011912/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009453","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model.","authors":"Antal Zemplenyi, Jim Leonard, Garth C Wright, Michael J DiStefano, Kavita Nair, Kelly E Anderson, R Brett McQueen","doi":"10.1007/s40273-025-01474-3","DOIUrl":"10.1007/s40273-025-01474-3","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.</p><p><strong>Methods: </strong>Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.</p><p><strong>Results: </strong>The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.</p><p><strong>Conclusions: </strong>The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"583-594"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011968/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468544","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction: De Novo Cost‑Effectiveness Model Framework for Nonalcoholic Steatohepatitis-Modeling Approach and Validation.","authors":"Peter Gal, Gyorgyi Feldmajer, Margarida Augusto, Ray Gani, Emma Hook, Ash Bullement, Zoe Philips, Inger Smith","doi":"10.1007/s40273-025-01475-2","DOIUrl":"10.1007/s40273-025-01475-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"595-596"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EQ-5D-5L or EQ-HWB-S: Which is the Better Instrument for Capturing Spillover Effects in Parental Carers of Children with COVID-19?","authors":"Wenjing Zhou, Bo Ding, Jan Busschbach, Michael Herdman, Zhihao Yang, Yanming Lu","doi":"10.1007/s40273-025-01473-4","DOIUrl":"10.1007/s40273-025-01473-4","url":null,"abstract":"<p><strong>Background and objectives: </strong>'Caregiver health spillovers' refer to the broader impacts of an individual's illness and interventions on informal caregivers' health and well-being. This study focuses on the spillover effects experienced by parental carers of children with coronavirus disease 2019 (COVID-19), aiming to compare the psychometric properties of the EQ-5D-5L and the experimental EQ Health and Wellbeing Short version (EQ-HWB-S) in capturing these effects.</p><p><strong>Methods: </strong>A longitudinal study was conducted with 861 parental carers of children aged 0-18 years with COVID-19 and 231 parents of healthy children as the control group. The EQ-5D-5L and EQ-HWB-S were used to assess parental health and well-being. Analyses included known-groups validity (multivariable regression), test-retest reliability (Gwet's AC1, intraclass correlation coefficient) and responsiveness to health improvement (Glass' Δ effect size).</p><p><strong>Results: </strong>Parents of infected children reported more problems than those of healthy controls. The EQ-HWB-S better discriminated between sub-groups defined by the child's COVID-19 presence, caring time and work impact. Test-retest reliability was fair to good for EQ-HWB-S dimensions (Gwet's AC1: 0.33-0.79), moderate to good for EQ-5D-5L (Gwet's AC1: 0.40-0.76), and good for index scores and EQ VAS (intraclass correlation coefficient: 0.70-0.77). Parental health and well-being improved as children recovered, with the EQ-5D-5L showing slightly higher responsiveness (effect size: 0.77-0.87) than EQ-HWB-S (effect size: 0.62-0.74).</p><p><strong>Conclusions: </strong>Both EQ-HWB-S and EQ-5D-5L are valid, reliable and responsive for measuring parental spillover effects related to a child's COVID-19 infection. EQ-HWB-S outperformed in distinguishing social and emotional impacts of caregiving, while EQ-5D-5L better captured physical health improvements. The choice between tools may depend on study objectives.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"555-567"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011911/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Taxonomy for Assessing Whether HRQoL Value Sets Are Obsolete.","authors":"Richard Norman, Bram Roudijk, Marcel Jonker, Elly Stolk, Saskia Knies, Raoh-Fang Pwu, Ciaran O'Neill, Kirsten Howard, Nancy Devlin","doi":"10.1007/s40273-025-01476-1","DOIUrl":"10.1007/s40273-025-01476-1","url":null,"abstract":"<p><p>Providing health-related quality of life (HRQoL) value sets to enable estimation of quality adjusted life years (QALYs) is important in facilitating economic evaluation and in supporting reliable decision-making about healthcare. However, as the field matures, many value sets across a range of HRQoL instruments are now old, based on potentially outdated valuation methodologies and preference data from samples that no longer represent the contemporary population. Having a clear strategy for identification and mitigation of obsolescence is important to ensure policy makers retain confidence in their country-specific value sets. In this Current Opinion, we develop a taxonomy of value set obsolescence. We then explore how the different types of obsolescence might be identified and how methodologists might work with local policymakers to address obsolescence and therefore ensure HRQoL instruments remain relevant for use. The taxonomy of obsolescence consists of four main areas: (1) the value set no longer aligns with current normative health technology assessment (HTA) requirements; (2) the methods used to generate it are no longer considered robust or adequately close to best practice; (3) the population composition has moved too far from the characteristics of the sample in which the original value set was derived; and (4) even after controlling for population differences, preferences are likely to have changed since the original data collection. Through identification of the type of obsolescence that applies in a particular setting, we then suggest a range of possible solutions to each, ranging from recommending particular sensitivity analyses, through reweighting of existing data to better account for population differences, to collecting new data for an updated value set. Obsolescence of existing value sets is driven by more than just time since data collection is often a matter of judgment rather than based on a clear definition. The taxonomy presented here provides a tool for assessing whether value sets are obsolete and what the appropriate response to this obsolescence should be. Working closely with local policymakers and involving discussions regarding the ongoing appropriateness of existing value sets should form an important part of future activities. This should include the consideration of updating value sets in contemporary populations using current best-practice methods. However, the benefits of updating value sets have to be balanced against the cost of doing so, including the challenges faced by policymakers when new values sets require a transition to new local decision-making processes.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"473-481"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011929/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}