PharmacoEconomics最新文献

{"title":"A Framework for Reliable, Transparent, and Reproducible Population-Adjusted Indirect Comparisons.","authors":"K Jack Ishak, Conor Chandler, Fei Fei Liu, Sven Klijn","doi":"10.1007/s40273-025-01503-1","DOIUrl":"10.1007/s40273-025-01503-1","url":null,"abstract":"<p><p>Population-adjusted indirect comparison (PAIC) methods aim to address some of the potential shortcomings of conventional approaches to indirect treatment comparisons by adjusting for imbalances in effect modifiers or prognostic factors and allowing for unanchored indirect treatment comparisons from disconnected networks of evidence. Health technology assessment bodies have published guidance and best practice recommendations for PAICs. However, recently published reviews of published PAICs have highlighted notable variability in implementation and a lack of transparency in the decision-making process in analyses and reporting; this hinders the interpretation and reproducibility of analyses, which, in turn, could affect reimbursement decision-making. We propose a systematic framework to address these challenges by describing considerations on six key elements of analyses: (1) definition of the comparison of interest (e.g., in terms of an estimand), (2) selection of the PAIC method, (3) selection of adjustment variables, (4) application of adjustment method, (5) risk-of-bias assessment, and (6) comprehensive reporting.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"691-710"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015053","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":"A Hemophilia Joint Health Score-Based Model for the Economic Evaluation of Hemophilia A Prophylaxis Interventions.","authors":"Sam Hirniak, Andrea N Edginton, Alfonso Iorio, William W L Wong","doi":"10.1007/s40273-025-01484-1","DOIUrl":"10.1007/s40273-025-01484-1","url":null,"abstract":"<p><strong>Background and objective: </strong>Hemophilia A is a costly, lifelong illness with multiple prophylaxis options. Previously, these options were assessed using a Peterson score-based model to simulate joint damage over time. This study built a model for the economic evaluation of hemophilia A with less socioeconomic selection bias utilizing the hemophilia joint health score (HJHS).</p><p><strong>Methods: </strong>A mechanistically defined HJHS-based state-transition microsimulation model was implemented for the cost-utility analysis conducted over a lifetime horizon from a Canadian provincial Ministry of Health perspective, with a 1.5% discount rate on (costs and outcomes), to compare the following interventions: standard half-life (SHL), extended half-life (EHL), emicizumab, and efanesocotog alfa (EA). The health states are HJHS levels, waiting for surgery, postoperative time, and death. Individuals experience bleeds, joint bleeds (increasing the HJHS), and surgery in each health state. Disutilities include injections and postoperative time. Model validation included face validity, internal validity, comparison analysis, external validity, and predictive validity. Probabilistic analysis, pricing threshold analysis, and one-way scenario analyses were completed.</p><p><strong>Results: </strong>EA showed lower levels of hospitalizations and surgeries and an improved joint damage experience in the simulation. However, EA was not cost-effective against emicizumab, which continued to be the most cost-effective intervention. Pricing threshold analysis indicated that a price decrease would be required for EA to dominate SHL (50% decrement) and emicizumab (55% decrement).</p><p><strong>Conclusions: </strong>This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"765-778"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753897","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 Psychometric Performance of Generic Preference-Based Measures in Informal Carers: A Systematic Review of Validation Studies.","authors":"Jan Faller, Valeriia Sokolova, Yared Belete Belay, Gang Chen, Cathrine Mihalopoulos, Brendan Mulhern, Lidia Engel","doi":"10.1007/s40273-025-01509-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01509-9","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objective: &lt;/strong&gt;A growing number of health technology assessment agencies recommend inclusion of informal carer outcomes in health economic evaluations. While generic preference-based measures (GPBMs) are favoured, the evidence regarding their performance in measuring the health-related quality of life of informal carers has not been synthesised. The aim of this systematic review was to synthesise the psychometric evidence of GPBMs in informal carers.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, a literature search (indexed through October 2024) was conducted in CINAHL, PsycInfo, Embase and MEDLINE databases, supplemented with forward and backward citation searches. Publications were included that reported the psychometric performance of GPBMs in informal carers, regardless of care recipients' condition. Narrative synthesis was used to summarise the evidence. Quality of studies was evaluated using the COSMIN risk of bias checklist. International Prospective Register of Systematic Reviews (PROSPERO) registration is CRD42023434651.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Twenty-one studies (published between 2001 and 2024) were identified, with nine evaluating multiple GPBMs (head-to-head comparisons). The EQ-5D 3-level (EQ-5D-3L) [n = 9] and EQ-5D 5-level (EQ-5D-5L) [n = 7] were the most frequently evaluated, followed by the Short-form 6-Dimension version 1 (SF-6Dv1) [n = 4], EuroQol Health and Wellbeing Short Form (EQ-HWB-9) [n = 4], Health Utilities Index (HUI) marks 2/3 (n = 3), Health-related Quality of Life Instrument with 8 Items (HINT-8) [n = 1] and Quality of Well Being Self-Administered (QWB-SA) [n = 1]. Studies were conducted in the USA (n = 6), UK (n = 4), China (n = 4), Australia (n = 3), Italy (n = 1), Iran (n = 1) and South Korea (n = 1), including a multi-country study (UK, Germany and France) study (n = 1). Care recipient conditions included carers of unspecified conditions, adults using long-term care, Alzheimer's disease or dementia, autism, cancer, leukaemia, craniofacial malformations, meningitis and multiple sclerosis. The EQ-5D-3L and EQ-5D-5L had evidence of ceiling effects at the index level. The EQ-5D-3L, EQ-5D-5L and EQ-HWB-9 demonstrated at least 'good' (intraclass correlation coefficient &gt; 0.60) test-retest reliability. Known-group validity evidence was available for the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3 and SF-6Dv1 where each GPBM was able to discriminate over 60% of the groups (known or exploratory). Convergent validity studies reported that the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3, SF-6Dv1 and QWB-SA had moderate correlations with at least one care-specific preference-based measure (Adult Social Care Outcomes Toolkit for Carers [ASCOT-Carer], Care-Related Quality of Life [CarerQol] and Carer Experience Scale [CES]). Responsiveness was evaluated for the EQ-5D-5L, EQ-HWB-9 and SF-6Dv1 where mixed evidence was repor","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529178","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":"Key Considerations for Assessing Real-World Comparative Effectiveness in the Context of the Drug Price Negotiation Program: A Case Study of Pembrolizumab.","authors":"Antal T Zemplenyi, Nai-Chia Chen, Kelly E Anderson, Blythe Adamson, Michael J DiStefano, Kavita V Nair, Robert B McQueen","doi":"10.1007/s40273-025-01514-y","DOIUrl":"https://doi.org/10.1007/s40273-025-01514-y","url":null,"abstract":"<p><strong>Background and objective: </strong>The Centers for Medicare and Medicaid Services increasingly rely on real-world evidence to inform drug price negotiations under the Inflation Reduction Act. This study aims to evaluate methodological decisions that impact real-world comparative effectiveness outcomes using a case example of first-line pembrolizumab versus therapeutic alternatives in advanced non-small cell lung cancer among the Medicare population.</p><p><strong>Methods: </strong>This study used a deidentified, electronic health record-derived, advanced non-small cell lung cancer dataset (2011-23) to analyze Medicare-eligible stage IV patients in three indications: (1) non-squamous, epidermal growth factor receptor, and anaplastic lymphoma kinase negative; (2) squamous; and (3) epidermal growth factor receptor and anaplastic lymphoma kinase negative with programmed death ligand-1 expression ≥1%. Indications (1)-(2) involved pembrolizumab combinations, while (3) referred to pembrolizumab monotherapy. Comparators included common non-platinum-based chemotherapy regimens. Propensity score-based inverse probability weighting was applied. The primary outcomes were real-world progression-free survival and overall survival. Scenario analyses examined the influence of time period selection, programmed death ligand-1 inclusion, therapeutic alternatives, and treatment switching on comparative effectiveness estimates.</p><p><strong>Results: </strong>In the non-squamous cohort (1), overall survival benefits of pembrolizumab therapies compared to alternatives varied from a non-significant difference to an improvement of 2.7 months (95% confidence interval 1.2, 4.8), depending on analytical choices. In the squamous cohort (2), pembrolizumab combinations consistently demonstrated overall survival benefits, which ranged from 1.4 months (95% confidence interval 0.1, 3.0) to up to 3.6 months (95% confidence interval 0.1, 5.9). However, for pembrolizumab monotherapy (3), overall survival differences were statistically non-significant. Scenario analyses indicated substantial variability in outcomes based on methodological choices.</p><p><strong>Conclusions: </strong>This study underscores the importance of transparent reporting and scenario analyses in real-world evidence to support Centers for Medicare & Medicaid Services decision making during drug price negotiations. Findings highlight the need for rigorous methodological standards to ensure the external validity of real-world evidence and its alignment with clinical practice.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144476223","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":"Innovation Headroom for a Highly Accurate PD-L1 Companion Diagnostic in Non-small Cell Lung Cancer.","authors":"Toluwase Akinsoji, Nick Dragojlovic, Cécile Darviot, Michel Meunier, Mark Harrison, Larry D Lynd","doi":"10.1007/s40273-025-01512-0","DOIUrl":"10.1007/s40273-025-01512-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Companion diagnostics (CDx) are critical to precision medicine. Developing and commercializing new CDx faces regulatory and economic challenges. This study aims to illustrate the utility of an early health technology assessment in quantifying the unmet clinical need and commercial opportunity created by the limited accuracy of existing programmed cell death ligand 1 CDx.</p><p><strong>Methods: </strong>The study uses an early health technology assessment and market sizing to assess the potential value of a novel programmed cell death ligand 1 CDx for non-small cell lung cancer (NSCLC). Decision tree-based cost-effectiveness models were used to evaluate clinical and economic outcomes for improved programmed cell death ligand 1 testing in atezolizumab-treated patients with stage II-IIIA and metastatic NSCLC from a US payer perspective in 2023 US Dollars. Three strategies were examined: standard care, new CDx for cytology specimens only, and new CDx for all patients. Commercial opportunities from the perspectives of diagnostics and pharmaceutical manufacturers were assessed using headroom and threshold analyses.</p><p><strong>Results: </strong>Headroom analyses indicated that a new CDx is not cost effective for metastatic NSCLC but holds significant value for stage II-IIIA NSCLC. Assuming perfect sensitivity and specificity, the incremental cost-effectiveness ratio for the new CDx in stage II-IIIA NSCLC was $57,650/quality-adjusted life-year and $54,950/quality-adjusted life-year for cytology specimens only and all patients, respectively. A threshold analysis showed that at a $500 price point, the new CDx is cost effective at sensitivity levels of 0.9 for all patients and 0.8 for cytology only. The total addressable US market for the CDx manufacturer was estimated at $2.6 million per year with a $500/test kit price.</p><p><strong>Conclusions: </strong>A novel, highly accurate CDx for stage II-IIIA NSCLC could provide significant value to patients, payers, and manufacturers.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333728","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":"An Updated Systematic Literature Review of the Economic Costs of Loneliness and Social Isolation and the Cost Effectiveness of Interventions.","authors":"Lidia Engel, Muhammad Fikru Rizal, Sharon Clifford, Jan Faller, Michelle H Lim, Long Khanh-Dao Le, Mary Lou Chatterton, Cathrine Mihalopoulos","doi":"10.1007/s40273-025-01516-w","DOIUrl":"https://doi.org/10.1007/s40273-025-01516-w","url":null,"abstract":"<p><strong>Purpose: </strong>There has been growing interest in understanding the economic impacts of loneliness and social isolation. This study updates a previous review on the economic costs of loneliness and social isolation and the cost effectiveness of related interventions.</p><p><strong>Methods: </strong>We conducted a systematic search in the MEDLINE, PsycInfo, CINAHL, and Embase databases from 2018 to 13 August 2024, supplemented by a search of the grey literature. Studies included cost-of-illness studies, economic evaluations, and social return on investment (SROI) analyses published in the English language. All studies were evaluated for quality and summarised using a narrative approach. Costs reported were converted into US$, year 2024 values.</p><p><strong>Results: </strong>In total, 15 studies were included: six cost-of-illness studies, four economic evaluations, and five SROI studies. Cost-of-illness studies primarily examined healthcare and productivity costs. All but one study reported excess costs linked to loneliness and social isolation, ranging from US$2 billion to US$25.2 billion per annum. Among four economic evaluations, three were model-based cost-utility or cost-effectiveness analyses (targeting older adults and the general population), and one was trial based (focusing on low-income individuals with health issues). One study found an intervention cost effective, whereas cost-effectiveness probabilities in others ranged from 54% to 68%. One study concluded that an intervention to reduce severe loneliness in older adults was cost effective but unlikely to be cost saving. All SROI studies reported positive returns, with SROI ratios ranging from US$2.28 to US$13.72.</p><p><strong>Conclusion: </strong>This review highlights additional evidence on the economic burden of loneliness and social isolation. Future research should explore broader cost impacts beyond healthcare and expand cost-effectiveness studies to younger populations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310273","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":"Inequalities in Quality-Adjusted Life Expectancy in Australia by Educational Attainment.","authors":"Sheridan E Rodda, Melanie Lloyd, Jennifer Welsh, Jedidiah Morton, Rosemary Korda, Zanfina Ademi","doi":"10.1007/s40273-025-01517-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01517-9","url":null,"abstract":"<p><strong>Introduction: </strong>Summary measures such as quality-adjusted life expectancy (QALE) are increasingly used to monitor health inequalities. Socioeconomic inequalities in health are well documented in Australia, including inequalities by education. However, estimates for QALE by level of education are lacking for Australia. We aimed to provide QALE stratified by age and sex across levels of educational attainment for the Australian population aged 25 years and above.</p><p><strong>Methods: </strong>We categorized educational attainment as low (completed year 11 or below), intermediate (completed year 12 and/or other non-tertiary or vocational qualification) or high (completed a bachelor's degree or above). Mean Short-Form Six-Dimension health utility was estimated for sex- and education-specific subgroups from the Household, Income and Labour Dynamics in Australia survey (2022). We constructed life tables using age-sex-education-specific mortality rates for 2019 obtained from linked 2016 Census and Death Registrations data. Health utility was incorporated into the life tables to derive age- and sex-specific QALE across education levels.</p><p><strong>Results: </strong>At age 25 years, males with high education had 7.3 years greater life expectancy than those with low education (61.0 versus 53.7 years undiscounted) and larger QALE (39.9 versus 28.8 years undiscounted), a gap of 11.1 years (39% relative difference). Females aged 25 years with a high level of education experienced 3.9 years greater life expectancy (LE; 63.1 versus 59.2 years, undiscounted) and an additional 7.6 years of QALE (36.9 versus 29.3 years, undiscounted), compared with those with low education, a 26% relative difference in QALE.</p><p><strong>Conclusions: </strong>Significant disparities in QALE by educational attainment exist in Australia. These findings can inform policies aimed at reducing health inequity by guiding resource allocation and supporting future equity-informative economic evaluations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310274","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":"Bayesian Meta-Analysis: A Practical Introduction.","authors":"Christopher S Hollenbeak","doi":"10.1007/s40273-025-01510-2","DOIUrl":"https://doi.org/10.1007/s40273-025-01510-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144249118","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":"Using AI in the Economic Evaluation of AI-Based Health Technologies.","authors":"Salah Ghabri","doi":"10.1007/s40273-025-01496-x","DOIUrl":"10.1007/s40273-025-01496-x","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"597-600"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144041256","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":"Market and Non-Market Productivity Losses Associated with Invasive Meningococcal Disease in the USA.","authors":"Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Mei Grace, Shah Alam Khan, Elise Kuylen, Shahina Begum, Frederik Verelst, Zeki Kocaata","doi":"10.1007/s40273-025-01477-0","DOIUrl":"10.1007/s40273-025-01477-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Invasive meningococcal disease (IMD) is an uncommon but serious disease associated with a risk of death and severe long-term sequelae, impacting both patients and their caregivers. Productivity losses due to IMD have not previously been comprehensively evaluated in the USA. This study evaluated both market and non-market productivity losses to better estimate the economic burden of IMD in the USA.</p><p><strong>Methods: </strong>An economic model estimated lifetime market (labour) and non-market (unpaid household, caring and voluntary services) productivity losses due to acute IMD, premature death due to IMD, reduced life expectancy in IMD survivors and IMD-related sequelae for patients 16 years of age or older and their caregivers based on IMD cases diagnosed in the USA in 2021 (due to data availability). Time use data were used to characterise IMD-incurred disruptions as market or non-market productivity losses. Time lost during the acute phase (assumed equal for patients and caregivers) was estimated based on hospital length-of-stay data. Time lost due to premature death from acute IMD or reduced remaining life expectancy (only calculated for patients) was estimated by subtracting the age at IMD acquisition or life expectancy of IMD survivors from average life expectancy. Time lost due to IMD-related sequelae was estimated based on sequelae event rates. Time lost was multiplied by earnings per hour (derived from median salary) to estimate productivity losses. Assumptions about sequelae impact on productivity were derived from the literature and expert clinical opinion. Scenario and sensitivity analyses assessed the impact of different inputs and assumptions on the results. Costs were inflated to 2023 US dollars.</p><p><strong>Results: </strong>Lifetime productivity losses for IMD cases diagnosed in the USA in 2021 (N = 121) totalled $87.4 million ($722,458 per case) for patients 16 years of age and older and their caregivers, with market and non-market losses accounting for approximately 72% and 28%, respectively. Premature death, reduced life expectancy and long-term sequelae were responsible for the majority of total productivity losses for patients and caregivers ($87.1 million); the acute phase accounted for $314,850. Results were most sensitive to the ratio of total benefits, median salary, case-fatality rates and specific sequelae included.</p><p><strong>Conclusions: </strong>Despite being an uncommon disease, the high mortality rate and severe long-term consequences of IMD result in a substantial economic impact. Comprehensive market and non-market productivity losses for both patients and caregivers should be considered when evaluating and communicating the true burden of IMD.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"651-664"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12081490/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616743","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}