PharmacoEconomics最新文献

{"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}

{"title":"Economic Evaluation of Interventions to Reduce Antimicrobial Resistance: A Systematic Literature Review of Methods.","authors":"Kristina Aluzaite, Marta O Soares, Catherine Hewitt, Julie Robotham, Chris Painter, Beth Woods","doi":"10.1007/s40273-024-01468-7","DOIUrl":"10.1007/s40273-024-01468-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Economic evaluation of antimicrobial resistance (AMR) interventions is complicated by the multisectoral, inter-temporal and international aspects of the problem, further hindered by a lack of available data and theoretical understanding of the emergence and transmission of AMR. Despite the substantial global focus on the problem, there is a lack of comprehensive economic evaluation literature on AMR policies. The goal of this work is to review the available literature on the economic evaluation of AMR interventions focusing on methods used to quantify the effects on AMR and the associated health consequences and costs.</p><p><strong>Methods: </strong>The studies included in the review were identified by a previous study by Painter et al. that included all full economic evaluations of AMR policies in the peer-reviewed and grey literature published between 2000 and 2020. The current review extracted additional information to (1) summarise the types and the key features of the AMR intervention economic evaluation literature available; (2) systemise the types of intervention effects on AMR quantified and describe these across the dimensions of AMR burden: time, space, wider pathogen pool and different sectors (One Health framework); and (3) categorise the methods used to derive these outcomes and how were these linked to health consequences and costs.</p><p><strong>Results: </strong>Thirty-one studies were included within this review, of which 18 evaluated interventions that aimed to reduce infection rates and 11 evaluated interventions that aimed to optimise antimicrobial use. Almost all were conducted with a high-income and/or upper-middle income country perspective and focused on human health. Thirteen of 31 studies were cost-utility analyses. Fifteen of 31 and 7/31 studies estimated the AMR effects through decision tree and/or Markov models and transmission models, respectively. Transmission models and linkage of AMR outcomes to quality-adjusted life-years and costs were more common in evaluations of interventions aimed at reducing infection rates. Most of the included studies restricted the scope of evaluation to a short time horizon and a narrow geographical scope and did not consider the wider impact on other pathogens and other settings, potentially resulting in an incomplete capture of the effects of interventions.</p><p><strong>Conclusions: </strong>This review found limited available literature that mainly focused on high-income countries and infection prevention/reduction strategies. Most evaluations used a narrow study scope, which might have prevented the full capture of the costs and outcomes associated with interventions. Finally, despite the known complexities associated with quantifying AMR effects, and the corresponding methodological challenges, the implications of these choices were rarely discussed explicitly.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"631-646"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567844","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":"Artificial Intelligence as a New Research Ally? Performing AI-Assisted Systematic Literature Reviews in Health Economics.","authors":"Sietse van Mossel, Martijn Johan Oude-Wolcherink, Rafael Emilio de Feria Cardet, Lioe-Fee de Geus-Oei, Dennis Vriens, Hendrik Koffijberg, Sopany Saing","doi":"10.1007/s40273-025-01481-4","DOIUrl":"10.1007/s40273-025-01481-4","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"647-650"},"PeriodicalIF":4.4,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12081476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019590","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":"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":"Health Care Resource Utilization and Costs Associated with US Medicaid Sobriety Restrictions on Direct-Acting Antivirals for Hepatitis C Virus: A Retrospective Claims Database Analysis.","authors":"Michelle T Martin, Krithika Rajagopalan, Dilip Makhija, Fatema Turkistani, Caroline Burk, Marvin Rock, Alice Hsiao, Nancy Reau","doi":"10.1007/s40273-025-01487-y","DOIUrl":"https://doi.org/10.1007/s40273-025-01487-y","url":null,"abstract":"<p><strong>Background and aims: </strong>Many state Medicaid programs implemented sobriety restrictions that delay timely initiation of direct-acting antivirals (DAAs) for patients with hepatitis C virus (HCV) infections. This claims database study examined the economic impact of sobriety restrictions on DAAs among Medicaid-insured patients with HCV.</p><p><strong>Methods: </strong>A retrospective database analysis of the Anlitiks All Payor Claims data (APCD) during the period January 1, 2020 to June 30, 2022 was conducted. Continuously enrolled adult (aged 18-64 years) Medicaid-insured patients with HCV who initiated DAAs (i.e., index date) during the period January 1, 2021 to December 31, 2021 with ≥ 12 months pre-index and ≥ 6 months post-index follow-up were categorized into two cohorts (states with sobriety restriction [SR] and states with no sobriety restriction [NSR]) based on the sobriety restriction status in the state of residence on the index date. Measures analyzed were the proportion of patients with one or more all-cause medical health care resource utilization (HCRU) (inpatient hospitalization [IP], emergency department [ED], outpatient [OP], professional office [PV], and other [OV] visits) and mean per-patient medical, pharmacy, and overall costs. HCRU and cost differences were compared using adjusted multivariable logistic and gamma-log link regression models, respectively.</p><p><strong>Results: </strong>Patients in the SR (n = 2,295) versus NSR (n = 4,623) cohort had a higher mean age (45 ± 12.02 vs. 43 ± 11.51 years), fewer males (50.28% vs. 58.1%), and they had lower substance use rates (44.10% vs. 59.68%), all significant at p < 0.05. The SR vs. NSR cohort had higher rates of patients with all-cause HCRU by type (IP 22.0% vs.18.1%; ED 42.3% vs. 37.4; OP 62.5% vs. 55.4%; PV 76.4% vs. 69.1%; other visits 47.4% vs. 46.5%). The SR vs. NSR cohort had a significantly higher adjusted odds ratio (95% confidence interval) for IP (2.09; 1.59-2.73) and OP (1.52; 1.28-1.82). Similarly, the SR versus NSR cohort had a significantly higher all-cause adjusted least squares mean cost per patient for IP ($42,616 vs. $15,063), ED ($982 vs. $420), OP ($715 vs. $349), PV ($840 vs. $621), medical ($11,845 vs. $3,850), pharmacy ($53,453 vs. $38,298), and overall ($63,935 vs. $41,524).</p><p><strong>Conclusion: </strong>Patients who initiated DAAs with SR versus NSR had 2 times and 1.5 times greater likelihood of IP and OP visits, respectively. Similarly, the SR versus NSR cohort had 3 times greater medical costs. Restricting DAA access among patients with HCV increases HCRU and cost burden, potentially impeding World Health Organization (WHO) 2030 HCV global elimination goals.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144161024","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":"Global Assessment of Health Utilities Associated with Pneumococcal Disease in Children-Targeted Literature Reviews.","authors":"Min Huang, Jipan Xie, Hela Romdhani, Yan Song, Sun Lee, Daisy Liu, Elamin Elbasha, Salini Mohanty, Donna Rowen, Matthew S Kelly","doi":"10.1007/s40273-025-01504-0","DOIUrl":"https://doi.org/10.1007/s40273-025-01504-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Pneumococcal disease can significantly impact the quality of life (QoL) of children. Health utilities are used to measure the disease burden and calculate quality-adjusted life year (QALY) estimates. These estimates provide critical inputs in economic evaluations of pneumococcal vaccines in children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This study aimed to synthesize utility values used in cost-utility analyses (CUAs) of pediatric pneumococcal vaccines and to summarize published utility studies on pneumococcal disease and post-meningitis sequelae (PMS) in children on a global scale.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Two targeted literature reviews were conducted to identify CUAs of pediatric pneumococcal vaccines and original studies on health utilities of pneumococcal disease and PMS. Both literature reviews identified relevant studies using published reviews, supplemented by de novo searches conducted in MEDLINE in June 2024 to cover periods not included in those reviews. References from published literature reviews on QoL of pneumococcal disease and CUAs were screened to identify additional original utility studies. Health utility values applied in the CUAs were summarized and the source studies for these utilities were reviewed. For original utility studies, methods and utility estimates were summarized for each condition.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The study identified 45 CUAs of pediatric pneumococcal vaccines in North America and Europe published from 2004 to 2024, and 21 original utility studies on pneumococcal disease or PMS in children published globally from 1994 to 2017. QALY decrement was the most common utility input in CUAs. Most CUAs referenced an earlier CUA for utility inputs, which were often sourced from one or two original utility studies for each health state. Most source studies were published more than two decades ago; some common source studies were conducted in adults. Utility estimates from original studies showed considerable variability, with ranges of -0.330 to 0.6882 for meningitis, -0.331 to 0.93 for non-meningitis invasive pneumococcal disease (IPD), -0.054 to 0.71 for inpatient pneumonia, 0.412-0.82 for outpatient pneumonia, 0.389-0.97 for acute otitis media (AOM)/simple AOM, 0.434-0.540 for recurrent AOM, -0.33 to 0.89 for neurological deficits, and 0.217-0.97 for hearing loss. Variability in methods, including in the surveyed population, utility elicitation method, and use of different country-specific preference weights, substantially impacted utility values. Overall, the methods were not suitable for temporary health states. Additionally, many studies used instruments that have not been validated in children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Original utility studies demonstrated that pneumococcal disease and PMS are associated with impaired QoL in children; however, there was considerable variability in utility estimates across studies, reflecting the inherent methodologica","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128234","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}