PharmacoEconomics最新文献

{"title":"The Influence of Perspective on the Valuation of the EQ-5D-Y-3L: A Comparison Using the OPUF Tool and a Discrete Choice Experiment.","authors":"Jake Hitch, Gayathri Kumar, Paul Schneider, Nancy Devlin, Koonal Shah, David Mott","doi":"10.1007/s40273-025-01545-5","DOIUrl":"https://doi.org/10.1007/s40273-025-01545-5","url":null,"abstract":"<p><strong>Background: </strong>The choice of perspective in valuation tasks is likely to affect the scale of EQ-5D-Y-3L value sets, but less is known about how it affects the relative importance of different dimensions.</p><p><strong>Objectives: </strong>The aim of this study was to examine how preferences for EQ-5D-Y-3L health states differ according to different perspectives utilising two methods: the Online elicitation of Personal Utility Functions (OPUF) tool and a discrete choice experiment (DCE).</p><p><strong>Methods: </strong>An online survey was designed containing the OPUF tool and a DCE. Adult respondents from the United Kingdom were randomised to one of five different perspective arms: (1) 4-year-old child, (2) 10-year-old child, (3) a child of unspecified age, (4) another adult, and (5) own health. The resulting OPUF value sets (social utility functions), and relative importance scores for the five dimensions from both methods, were compared across perspectives.</p><p><strong>Results: </strong>Results differed by perspective in both valuation tasks. In both tasks, 'looking after myself' was less important and 'pain or discomfort' was more important in the child perspectives than in the adult perspectives. Furthermore, the scale of the value sets produced by the OPUF tool differed by perspective, with the value of the worst health state being significantly lower in the adult perspectives than in the child perspectives.</p><p><strong>Conclusion: </strong>Our results suggest that the valuation of the EQ-5D-Y-3L is affected by the perspective that adult respondents are asked to take. Researchers should be aware of the potential impact and ensure that relevant stakeholders understand this when designing valuation studies.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145233169","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":"Challenges in Modelling the Cost Effectiveness of Pharmacotherapies for Obesity.","authors":"Becky Pennington, Ewen Cummins, Albany Chandler, James Fotheringham","doi":"10.1007/s40273-025-01520-0","DOIUrl":"10.1007/s40273-025-01520-0","url":null,"abstract":"<p><p>The cost effectiveness of pharmacotherapies for obesity (such as semaglutide, tirzepatide, liraglutide, and newer agents) is increasingly being appraised by health technology assessment (HTA) bodies. Modelling is required to extrapolate weight change observed over relatively short clinical trial durations to long-term weight loss and associated cardio-metabolic outcomes and costs. Extrapolation is a common issue in HTA, but there is a unique challenge for anti-obesity drugs because of the number of interacting uncertainties. This is a particular concern given the substantial eligible population sizes and associated high financial decision risk of providing lifetime treatment. We describe four key challenges in modelling pharmacotherapies for obesity: (1) modelling long-term body mass index (BMI) trajectories with and without obesity pharmacotherapy, (2) modelling time on treatment, (3) using risk equations to link changes in BMI to clinical outcomes, and (4) modelling clinical outcomes not (solely) related to BMI changes. We discuss each of these challenges and the impact they have had in global HTA appraisals for pharmacotherapies. We speculate how these challenges relating to short-term clinical trials could be overcome to more robustly predict long-term outcomes and the role that observational data may play. As clinical trial and real-world evidence for technologies for obesity evolves, analysts and decision-makers need to determine which evidence sources are most appropriate and how they should be combined.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1171-1178"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450117/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732655","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":"Challenges and Criteria for Single-Arm Trials Leading to an Added Benefit in German Health Technology Assessments.","authors":"Jörg Tomeczkowski, Tanja Heidbrede, Birte Eichinger, Ulrike Osowski, Friedhelm Leverkus, Sarah Schmitter, Charalabos-Markos Dintsios","doi":"10.1007/s40273-025-01524-w","DOIUrl":"10.1007/s40273-025-01524-w","url":null,"abstract":"<p><strong>Background: </strong>Randomized controlled trials are the standard for health technology assessment, but when they are infeasible or unethical, single-arm trials (SATs) are submitted.</p><p><strong>Objectives: </strong>This study examined when SATs were accepted for added benefit by the Institute for Quality and Efficiency in Health Care (IQWiG) and/or the Federal Joint Committee (G-BA) in Germany.</p><p><strong>Methods: </strong>We identified health technology assessments via the AMNOG-Monitor database through December 2024, with additional details from G-BA documents. We compared the SATs and other evidence for added benefit decisions (granted/not granted), stratified by orphan drug status, special marketing authorization, approved indication (chronic hepatitis C/others), and population (adults/children). Added benefit claims by manufacturers, IQWiG recommendations, and G-BA appraisals were compared.</p><p><strong>Results: </strong>Among 1738 G-BA decisions, 85.8% (1491/1738) of the subpopulations were fully assessed by IQWiG, with 13.5% (202/1491) based on SATs. Among the 247 orphan drugs assessed by the G-BA, 37.7% (93/247) were SAT-based. Overall, SAT-based assessments demonstrated an added benefit in 12.2% (36/295) of cases. This included 13.4% (27/202) of full assessments and 9.7% (9/93) of orphan drug assessments. IQWiG accepted only 18.5% (5/27) of the SATs endorsed by the G-BA. Statistical tests revealed significant differences between manufacturers' claims, IQWiG recommendations, and G-BA appraisals. SATs were most frequently accepted for chronic hepatitis C treatments (mostly with non-standard marketing authorization) and paediatric indications. The G-BA cited reasons such as dramatic effects, rare diseases, a lack of alternatives, or fewer side effects, although justifications were often unclear.</p><p><strong>Conclusion: </strong>Acceptance rates for SATs remain low, and criteria for added benefit are not always explicitly defined. To enable benefit assessments when randomised controlled trials are infeasible or unethical, clear and binding criteria developed in collaboration with the G-BA are essential.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1223-1233"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732646","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":"Integrating Environmental Impact in Health Technology Assessment: An Exploratory Study.","authors":"Coline Ducrot, Julien Péron, Matthieu Delaye, David Pérol, Isabelle Durand-Zaleski, Max Piffoux","doi":"10.1007/s40273-025-01521-z","DOIUrl":"10.1007/s40273-025-01521-z","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To what extent a care pathway, due to its associated pollution, may be more detrimental to future health than beneficial to contemporary patients is still an open question. We present a methodological framework to integrate pollutant-induced future health damages in health technology assessment (HTA) metrics like quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) for a better evaluation of the cost effectiveness of care pathways.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We used the ReCiPe model to estimate the future detrimental health impact (in disability-adjusted life years [DALY]) of pollutants from the US healthcare system, showing the major impact of GHG emissions compared with other pollutants. An adapted version of the ReCiPe model was used to convert GHG emissions from care pathways into future DALY&lt;sub&gt;GHG&lt;/sub&gt;, QALY&lt;sub&gt;GHG&lt;/sub&gt;, and life years (LY&lt;sub&gt;GHG&lt;/sub&gt;), as well as the associated confidence intervals. For a given care pathway, future health damages were compared with patient benefits (e.g., QALY&lt;sub&gt;GHG&lt;/sub&gt;/QALY&lt;sub&gt;patient&lt;/sub&gt;). Damages may also be integrated in the ICER&lt;sub&gt;GHG&lt;/sub&gt; by subtracting future health losses from patient health benefits. Case applications are provided.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Future damages to health emerging from pollutants emitted by the US healthcare system were estimated at 7,363,000 DALYs per year. Focusing on GHG emissions to estimate pollutant impact is reasonable, as they represent &gt;90% of future damages. We provide estimates to convert GHG emissions into future health damages in DALY, QALY, or LY (and associated uncertainty), taking into account future impacts over different time horizons (20, 100, or 500-1000 years) and using different discount rates for future health impact (0 or 3%). We recommend estimating future damages using an egalitarian perspective (with a 0% discount rate) to maintain intergenerational equity. The QALY&lt;sub&gt;GHG&lt;/sub&gt;/QALY&lt;sub&gt;patient&lt;/sub&gt; ratio allows weighting future detrimental effects of care pathways against their benefits. For health economic evaluations, we recommend integrating GHG emissions into the ICER, preferably in its denominator (QALY, DALY, LY). When focusing on specific care pathways, health gains may be substantially limited by future GHG-related detrimental impacts, especially for chronic treatments in low-risk populations. Some care pathways, like influenza vaccination, improve patient health while mitigating GHG. Accounting for GHG emissions may substantially favor or penalize one strategy over another in terms of ICER. Confidence intervals of the results were wide due to large uncertainties regarding long-term predictions.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;HTA should consider care pathways' impact on future health to better assess the impact and cost effectiveness of health technologies. Under the hypothesis of intergenerational equity, GHG accounting has a substantial ","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1205-1222"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450120/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144668150","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":"Cost Comparisons in NICE Technology Appraisals: An External Assessment Group Perspective.","authors":"Marten J Poley, Nigel Armstrong, Huiqin Yang, Mubarak Patel, Lisa Stirk, Maiwenn J Al, Isaac Corro Ramos","doi":"10.1007/s40273-025-01522-y","DOIUrl":"10.1007/s40273-025-01522-y","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1165-1169"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144691117","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":"The Burden of Informal Caregiving for Adults with Musculoskeletal Conditions: A Systematic Review.","authors":"Nanati Legese Alemu, Neha Das, Jennifer J Watts, Suzanne Robinson, Gang Chen, Lan Gao","doi":"10.1007/s40273-025-01523-x","DOIUrl":"10.1007/s40273-025-01523-x","url":null,"abstract":"<p><strong>Background and objective: </strong>Informal caregivers play a critical role in supporting individuals with musculoskeletal conditions. This systematic review aimed to evaluate the psychological and economic burdens associated with caregiving for musculoskeletal conditions.</p><p><strong>Methods: </strong>We conducted a systematic review in accordance with PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) guidelines, searching MEDLINE, Embase, CINAHL, EconLIT, and APA PsycINFO for studies published between 2000 and April 2025. Studies were eligible if they examined the psychological and economic burden of informal caregiving for adults with musculoskeletal conditions. Screening and data extraction were conducted using EndNote 21 and Covidence. Risk of bias was assessed using the CASP checklist for psychological burden studies and the EVERS criteria for economic burden studies. Data were synthesized narratively. An exploratory meta-analysis of informal care hours was conducted using a subset of studies with sufficient statistical data.</p><p><strong>Results: </strong>A total of 41 studies were included, with 24 reporting psychological burdens, 16 economic burdens, and one for both. Caregiving burden included emotional, social, financial, and time-related impacts, impacting the caregivers' quality of life. Higher anxiety and depression were correlated with a greater caregiver burden. Informal care costs varied by musculoskeletal condition type, location, severity, intensity, and valuation method. Reported informal care hours showed substantial variation across studies. The overall risk of bias across included studies was low.</p><p><strong>Conclusions: </strong>This systematic review highlights the considerable psychological, economic, and time-related burdens faced by informal caregivers of individuals with musculoskeletal conditions. Caregivers face high stress, physical strain, and opportunity costs. The lack of standardized assessments hinders accurate burden quantification, economic evaluation, and policy responses. Future efforts should focus on adopting consistent measurement instruments and valuation methods, alongside implementing structured policies, financial support, and psychological interventions to better support the caregivers.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1179-1204"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450227/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144668151","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":"Can We Make Health Economic Decision Models as Simple as Possible, But Not Simpler? Introducing SMART tool.","authors":"Teebah Abu-Zahra, Sabine E Grimm, Mirre Scholte, Manuela Joore","doi":"10.1007/s40273-025-01515-x","DOIUrl":"10.1007/s40273-025-01515-x","url":null,"abstract":"<p><strong>Background: </strong>Developing health economic decision-analytic models requires making modelling choices to simplify reality while addressing the decision context. Finding the right balance between a decision-analytic model's simplicity and its adequacy is important but can be challenging.</p><p><strong>Objective: </strong>We aimed to develop a tool that supports the systematic reporting and justification of modelling choices in a decision-analytic model, ensuring it is adequate and only as complex as necessary for addressing the decision context.</p><p><strong>Methods: </strong>We identified decision-analytic model features from the key literature and our expertise. For each feature, we defined both simple and complex modelling choices that could be selected, and the consequences of simplifying a feature contrary to requirements of the decision context. Next, we designed the tool and assessed its clarity and completeness through interviews and expert workshops. To ensure consistency of use, we developed a glossary sheet and applied the tool in an illustrative case: a decision-analytic model on a repurposed drug for treatment-resistant hypertension.</p><p><strong>Results: </strong>We conducted five interviews and two workshops with 18 decision-analytic model experts. The developed SMART (Systematic Model adequacy Assessment and Reporting Tool) consists of a framework of 28 model features, allowing users to select modelling choices per feature, then assessing the consequences of their choices for validity and transparency. SMART also includes a glossary sheet. The treatment resistant hypertension case example is provided separately.</p><p><strong>Conclusions: </strong>SMART supports decision-analytic model development and assessment, by promoting clear reporting and justification of modelling choices, and highlighting their consequences for model validity and transparency. Thoughtful and well-justified modelling choices can help optimise the use of resources and time for model development, while ensuring the model is adequate to support decision making.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1235-1250"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450218/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732645","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 Cost-Effective Price of Lecanemab for Patients with Early Alzheimer's Disease in Sweden.","authors":"Xin Xia, Sandar Aye, Oskar Frisell, Emil Aho, Ron Handels, Yunfei Li, Anders Wimo, Bengt Winblad, Maria Eriksdotter, Tobias Borgh Skillbäck, Silke Kern, Henrik Zetterberg, Linus Jönsson","doi":"10.1007/s40273-025-01527-7","DOIUrl":"10.1007/s40273-025-01527-7","url":null,"abstract":"<p><strong>Introduction: </strong>We sought to estimate the cost-effective price for lecanemab for treating early Alzheimer's disease in Sweden from the perspective of formal care payers.</p><p><strong>Methods: </strong>We developed a Markov model with states defined by disease severity and care setting. The model was populated by integrated clinical and economic data from Swedish registers. We included patients with biomarker-confirmed Alzheimer's disease and fitted survival models for transitions between model states. Costs in 2023 Swedish kronor (SEK), life-years (LYs), and quality-adjusted LYs (QALYs) over a 10-year time horizon were estimated for standard of care and for lecanemab in addition to standard of care, assuming a maximum treatment duration of 3 years with lecanemab and no treatment effect after treatment stops. We also explored the impact of different assumptions regarding treatment efficacy and duration.</p><p><strong>Results: </strong>Treatment with lecanemab over 3 years resulted in 0.13 LYs gained, 0.17 QALYs gained, and a net cost increase of 87,146 SEK (€1 = 11.5 SEK, $US1 = 10.6 SEK) due to administration and monitoring, before considering the cost of drug. The cost-effective price of lecanemab at a willingness-to-pay level of 1 million SEK per QALY was 33,886 SEK per year of treatment. The health gain, net costs, and cost-effective price of lecanemab varied significantly by treatment duration, potential residual effects, and patient characteristics.</p><p><strong>Conclusions: </strong>The future price of lecanemab in European countries is unknown. However, treatment with lecanemab is unlikely to be cost effective in Sweden at the levels of current list prices in the USA.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1251-1266"},"PeriodicalIF":4.6,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450216/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144743921","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 Economic Burden of Inflammatory Arthritis: A Systematic Review.","authors":"Xiaoyu Zhang, Jiaru Liu, Zhengwei Wang, James Galloway, Sam Norton, Sumeet Singla, Huajie Jin","doi":"10.1007/s40273-025-01534-8","DOIUrl":"https://doi.org/10.1007/s40273-025-01534-8","url":null,"abstract":"<p><strong>Background and objective: </strong>Inflammatory arthritis is a common condition treated in rheumatology clinics, contributing significantly to healthcare costs and societal burden. Understanding the economic impact of inflammatory arthritis requires a comprehensive analysis through cost-of-illness studies. This systematic review aims to gather up-to-date cost-of-illness data on inflammatory arthritis from various countries, identify the primary cost drivers, describe shifts in cost components and appraise the quality of cost-of-illness study reporting in this field.</p><p><strong>Methods: </strong>An electronic search was performed across four databases, including MEDLINE, Embase, the Cochrane Database of Systematic Reviews and the Health Management Information Consortium, to identify cost-of-illness studies on inflammatory arthritis published over the past two decades. The primary outcome was the annual cost per patient with inflammatory arthritis, categorised by cost components. All costs were standardised to 2024 US dollar values. The quality of the included studies was evaluated using the Larg and Moss checklist and the modified Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist.</p><p><strong>Results: </strong>From an initial 12,264 publications, 82 studies were included in this review, covering axial spondyloarthritis (n = 49), psoriatic arthritis (n = 30), reactive arthritis (n = 2), rheumatoid arthritis (n = 13; 2019 onwards) and seronegative/seropositive rheumatoid arthritis (n = 8). Annual total societal costs varied considerably across inflammatory arthritis subtypes and countries. Medication expenditures consistently emerged as the primary direct healthcare cost driver, while productivity losses due to morbidity constituted the major component of indirect costs. Carer productivity loss represented a substantial proportion of indirect costs (up to 60.9%), yet was infrequently reported. Over time, we observed an increasing proportion of medication-related costs and a decreasing proportion of productivity losses for axial spondyloarthritis, alongside a reduction in inpatient care costs for psoriatic arthritis. These evolving cost distributions mirror patterns previously reported in rheumatoid arthritis. Methodological gaps were evident, with most studies lacking sensitivity analyses and comprehensive cost perspectives.</p><p><strong>Conclusions: </strong>A substantial economic impact of inflammatory arthritis across different regions and subtypes was identified. This review emphasises the importance of including comprehensive cost components to fully assess the economic burden of inflammatory arthritis and provides methodological recommendations for future studies.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145176612","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":"Scoping Review of Productivity-Adjusted Life Years (PALYs): Methods, Applications and Policy Implications.","authors":"Zanfina Ademi, Dina Abushanab, Maria J Alfonso Arvez, Clara Marquina, Karl Vivoda, Janne Martikainen, Piia Lavikainen, Melanie Lloyd, Danny Liew","doi":"10.1007/s40273-025-01544-6","DOIUrl":"https://doi.org/10.1007/s40273-025-01544-6","url":null,"abstract":"<p><strong>Objective: </strong>To understand the application of productivity-adjusted life years (PALYs) as an outcome measure across various disease contexts.</p><p><strong>Methods: </strong>We conducted a scoping review of studies published between 2018 and April 2025 that utilised PALYs to illustrate their potential applications and identify methodological approaches that have been applied. Using a citation-based search, we selected studies that applied PALYs to quantify societal health burdens in specific diseases or contexts. Extracted data included health conditions, country, timeframe, model type, outcomes, productivity index components, gross domestic product and sensitivity analysis. Findings were summarised through narrative synthesis.</p><p><strong>Results: </strong>A total of 41 studies conducted between 2018 and 2025 were reviewed, including chronic diseases such as diabetes and cardiovascular diseases, as well as environmental factors. Conditions such as breast cancer, leukaemia, kidney disease, mental health, knee osteoarthritis, epilepsy and sleep apnoea had the lowest productivity indices. Most of these studies originated from high-income countries (n = 27), followed by upper-middle-income (n = 10), and lower-middle-income (n = 4) settings. Life table models were the most common methodological approach adopted (n = 26), followed by dynamic models (n = 10). Studies focused on disease prevention (n = 21) outnumbered those addressing disease management (n = 18). Most studies accounted for both absenteeism and presenteeism (n = 30). Estimates of productivity loss per person using gross domestic product ranged from US$1137 to AU$217,983 annually.</p><p><strong>Conclusions: </strong>PALYs have been utilised in diverse diseases and contexts, highlighting their utility in measuring societal health impacts. However, adding unpaid and informal work makes burden estimates more accurate. The increasing emphasis on prevention indicates a strategic change in health policy and economic assessment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150448","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}