PharmacoEconomics最新文献

{"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":"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":"1047-1063"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370830/pdf/","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":"OA","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":"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":"1109-1122"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370557/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Are Economic Evaluations of Task Shifting Too Narrow in Focus? A Rapid Review.","authors":"Peter Murphy, Susan Griffin, Helen Fulbright, Simon Walker","doi":"10.1007/s40273-025-01507-x","DOIUrl":"10.1007/s40273-025-01507-x","url":null,"abstract":"<p><strong>Background and objectives: </strong>Task shifting between different cadres of health worker has been proposed as an approach to address workforce shortages. Whether such reallocation is a useful strategy for a health system depends on the potential costs and consequences. Too narrow a focus has implications for population health as resources could be incorrectly directed towards inefficient activities owing to important costs and/or benefits being omitted from the evaluation. We aim to identify the key issues when evaluating the value for money of task shifting and review the applied literature to determine whether it is fit for purpose.</p><p><strong>Methods: </strong>We developed an a priori logic model of task shifting and searched five databases (MEDLINE, Embase, EconLit, Social Sciences Citation Index and CEA Registry) for economic evaluations of task shifting published between 2014 and 2024. We performed forwards and backwards citation searching. We considered the scope of the evaluations with respect to the ability to capture key costs and outcomes of task shifting from the logic model. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist.</p><p><strong>Results: </strong>The rapid review identified 26 studies for inclusion covering 16 countries. Studies evaluated task shifting to community health workers and lay health workers as well as from doctors to radiographers, non-physician clinicians and nurse-midwives. The studies included health costs and outcomes but few included changes in the capacity of the workforce to undertake tasks, access, waiting times, productivity, burden on other staff, patient satisfaction, patient productivity and health equity concerns. There was a predominance for cost-effectiveness analysis to be used to assess the value for money of task shifting but the literature did include a cost-benefit analysis, a cost-consequence analysis and an extended cost-effectiveness analysis.</p><p><strong>Conclusions: </strong>The majority of studies identified a range of costs and consequences that may only be appropriate for resource allocation under the strong assumption that all longer term costs and consequences would be unaffected by the task shift.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1083-1108"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370793/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128211","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":"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":"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":"1123-1133"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370855/pdf/","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":"OA","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":"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":"1147-1160"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370795/pdf/","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":"OA","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":"10.1007/s40273-025-01509-9","url":null,"abstract":"<p><strong>Background and objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 > 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":"1065-1082"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370823/pdf/","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":"OA","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":"10.1007/s40273-025-01510-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"999-1000"},"PeriodicalIF":4.6,"publicationDate":"2025-09-01","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":"Developing an EQ-5D-5L Value Set for Singapore.","authors":"Nan Luo, Annushiah Vasan Thakumar, Ling Jie Cheng, Zhihao Yang, Kim Rand, Yin Bun Cheung, Julian Thumboo","doi":"10.1007/s40273-025-01519-7","DOIUrl":"https://doi.org/10.1007/s40273-025-01519-7","url":null,"abstract":"<p><strong>Objectives: </strong>The EQ-5D-5L is a multi-attribute utility instrument recommended by many health technology assessment agencies. This study aimed to develop an EQ-5D-5L value set for Singapore.</p><p><strong>Methods: </strong>A 'lite' version of the EuroQol Research Foundation's EQ-5D-5L valuation protocol, which was designed to value a total of 91 health states using a composite time trade-off (cTTO) method, was followed. Five hundred members of the general public in Singapore were quota-sampled and invited to a personal interview face-to-face or via Zoom. All participants completed 20 cTTO tasks administered using the EuroQol Valuation Technology (EQ-VT) program. Cross-validation analysis was performed to identify the best-performing model for estimating the values of all the 3,125 EQ-5D-5L health states.</p><p><strong>Results: </strong>A 20-parameter main-effects model with two two-way interaction terms outperformed other models in the cross-validation analysis. The value set estimated using this model ranges from - 0.851 (for state 55555) to 1.000 (for state 11111), with pain/discomfort and anxiety/depression dimensions associated with the greatest disutility.</p><p><strong>Conclusions: </strong>We developed an EQ-5D-5L value set based on the health preferences of Singaporeans. We recommend EQ-5D-5L users in Singapore to use this value set and encourage a more systematic and dedicated methodological effort to understand interaction effects and potential non-linearities in the valuation of multi-attribute health descriptive systems.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964307","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-Utility Analysis of Treatment Sequences for Moderate-to-Severe Crohn's Disease.","authors":"Matthijs M Versteegh, Simone A Huygens, Inigo Bermejo, Sabine Grimm, Marieke Pierik, Tessa Römkens, Fiona van Schaik, Peter Wahab","doi":"10.1007/s40273-025-01531-x","DOIUrl":"10.1007/s40273-025-01531-x","url":null,"abstract":"<p><strong>Objectives: </strong>The clinical pathway for patients with moderate-to-severe Crohn's disease (CD) typically includes sequential pharmacologic treatment as well as surgery, but positioning of different therapies within these sequences remains challenging. Cost-utility analysis rarely captures these sequences and does not incorporate registry data on long-term effectiveness. In this study, we aim to overcome these limitations.</p><p><strong>Methods: </strong>We developed an individual state transition model with four health states (active disease, remission, and remission due to surgery and death), five sequential treatment lines, and surgery. Efficacy data from network meta-analyses (NMA) for biologic naive and biologic exposed patients were combined with Dutch registry data to forecast long-term benefit, calculate costs, and estimate utilities. Analyses had a Dutch societal perspective with a lifetime time horizon. Costs were reported in 2023 euros and discounted with 3%. Effects were reported in quality-adjusted life years (QALYs) and discounted with 1.5%. The cost-per-QALY threshold was €20,000. Deterministic analyses for the base case, three scenarios (including recently published trials or price declines for ustekinumab), and one-way sensitivity analysis were run with 30,000 patients. The probabilistic sensitivity analysis was conducted by sampling 1000 patients in 1000 model runs.</p><p><strong>Results: </strong>When opting for step-up sequences, the most cost-effective sequence (out of 156 sequences) starts with either azathiopurine/6-mp or methotrexate and is followed by combination therapy (infliximab + azathioprine) when patients discontinue their first line owing to disease activity or discontinuation. The most cost-effective top-down sequence (out of 72) starts with combination therapy (infliximab + azathioprine). After two lines of treatment, differences in cost-effectiveness between biologics become smaller. To be equally cost-effective as anti-tumor necrosis factor (TNF) combination therapy, a price decline for ustekinumab (biosimilars) of 81% is required or 50% to become the preferred option after combination therapy. Validation against external data suggested good predictive capabilities of the model.</p><p><strong>Conclusions: </strong>Integrating NMA and registry data improves the quality of cost-effectiveness models for treatment sequences in CD. This open-source model can be easily updated for future therapies and holds the potential to become a standard model for use in clinical guideline development and the economic evaluation of new drugs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964318","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":"Eliciting and Anchoring Health State Preferences Using Discrete Choice Experiments Among Adults, Adolescents, and Children.","authors":"Shitong Xie, Tianxin Pan, Juan Manuel Ramos-Goni, Brendan Mulhern, Zhihao Yang, Richard Norman, Nancy Devlin, Feng Xie","doi":"10.1007/s40273-025-01530-y","DOIUrl":"10.1007/s40273-025-01530-y","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to compare EQ-5D-Y-5L health state preferences among children, adolescents, and adults in Canada using a discrete choice experiment (DCE), and to explore the feasibility of a rescaling latent DCE using anchoring tasks collected from adolescents.</p><p><strong>Methods: </strong>An online survey was conducted to elicit preferences for EQ-5D-Y-5L health states from children (aged 12-15 years), adolescents (aged 16-17 years), and adults (aged ≥ 18 years). All respondents completed 12 latent DCE tasks. Adults and adolescents were randomly assigned to three additional anchoring tasks using a DCE with duration or with dead. The tasks were framed from the perspective of a 10-year-old child for adults and their own perspective for children and adolescents. Respondents provided feedback on the difficulty of latent DCE tasks. Mixed logit models were used to analyze latent DCE data. Anchored DCE models using duration/dead tasks were estimated and compared between adults and adolescents.</p><p><strong>Results: </strong>Overall, 546 children, 508 adolescents, and 908 adults were included in the analyses. A higher proportion of children indicated it easy to complete DCE tasks compared with adolescents and adults. Monotonicity of coefficients were observed in latent DCE models among adults but not among children and adolescents. Anchored DCE modeling performed better in adults than in adolescents regarding monotonicity and statistical significance of coefficients, and the DCE with duration performed slightly better than the DCE with dead.</p><p><strong>Conclusions: </strong>There were differences in health state preferences elicited using DCEs between children/adolescents and adults. Anchoring tasks appeared feasible for adolescents, with a DCE with duration performing slightly better than a DCE with dead.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144874460","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}