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Hospital Burden and Care Pathways of ANCA-Associated Vasculitis in Italy: A Focus on Clinical, Economic, and Geographic Dimensions. 意大利anca相关血管炎的医院负担和护理途径:临床、经济和地理维度的重点。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-21 DOI: 10.1007/s41669-026-00689-0
Helen Banks, Giovanni Fattore, Claudio Jommi, Marco Soro, Federico Alberici, Luca Quartuccio
{"title":"Hospital Burden and Care Pathways of ANCA-Associated Vasculitis in Italy: A Focus on Clinical, Economic, and Geographic Dimensions.","authors":"Helen Banks, Giovanni Fattore, Claudio Jommi, Marco Soro, Federico Alberici, Luca Quartuccio","doi":"10.1007/s41669-026-00689-0","DOIUrl":"https://doi.org/10.1007/s41669-026-00689-0","url":null,"abstract":"<p><strong>Background: </strong>Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs) are rare autoimmune diseases characterized by heterogeneous clinical presentation and substantial morbidity. Evidence suggests high healthcare utilization and economic burden of ANCA-associated vasculitis (AAV), particularly owing to hospitalization as well as geographic variation in the organization of specialized care. However, national-level analyses examining outcomes, resource use, and geographic differences in care pathways remain limited.</p><p><strong>Objective: </strong>The study aimed to identify hospitalized patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) using administrative data, characterize their hospital burden and geographic care pathways in Italy, and investigate determinants of hospital costs and in-hospital mortality.</p><p><strong>Methods: </strong>Nationwide Italian hospital discharge records (2010-2019) were used to identify patients with GPA and MPA using International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM)-based algorithms. Hospital resource use, costs, in-hospital mortality, inter-regional mobility, and continuity of specialized care were estimated. Determinants of hospital costs were analyzed using generalized linear models (gamma distribution and log link), and in-hospital mortality was analyzed using the Cox proportional hazards model.</p><p><strong>Results: </strong>We identified 3763 patients with GPA and 1206 patients with MPA, with a mean of 6.9 and 8.6 admissions per patient, respectively, over 10 years. In-hospital mortality was 20.3% (GPA) and 21.4% (MPA), with over 70% of deaths occurring within 2 years of the first observed key diagnosis in hospital records. Mean cumulative observed hospital costs during 2010-2019 were substantial (€28,275 for GPA and €36,462 for MPA per patient), with around 60% of costs concentrated in the year of the first observed key diagnosis and adjacent years. Marked regional variation in care pathways was observed, with patients residing in southern regions frequently receiving AAV-related care in northern hospitals. Lower hospital loyalty (seeking AAV-related care in numerous hospitals) was more commonly observed in several southern regions. In regression analyses, infections were the strongest predictors of both higher costs and in-hospital mortality, while lower hospital loyalty was associated with substantially higher costs but not in-hospital mortality.</p><p><strong>Conclusions: </strong>Our analysis showed that hospitalized patients with AAV in Italy experienced substantial inpatient burden, frequent admissions, and high in-hospital mortality. Infections and renal involvement were strongly associated with both costs and in-hospital mortality. The observed regional differences in mobility patterns and continuity of specialized care suggest important geographic variation in AAV care pathways within","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149228566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Health State Valuation in Nigeria: An Insight into Discrete Choice Experiment-Based Preferences for Children and Adolescents from the Lenses of Adult Respondents. 尼日利亚的健康状况评估:从成人受访者的角度对儿童和青少年离散选择实验偏好的洞察。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-13 DOI: 10.1007/s41669-026-00690-7
Abdulmuminu Isah, Iheomimichineke Ojiakor, Chinelo Obi, Chinelo Ikeanyi, Chibueze Anosike, Ezinwanne Jane Ugochukwu, Stacey Orangi, Edwine Barasa, Felix Masiye, Obinna Onwujekwe
{"title":"Health State Valuation in Nigeria: An Insight into Discrete Choice Experiment-Based Preferences for Children and Adolescents from the Lenses of Adult Respondents.","authors":"Abdulmuminu Isah, Iheomimichineke Ojiakor, Chinelo Obi, Chinelo Ikeanyi, Chibueze Anosike, Ezinwanne Jane Ugochukwu, Stacey Orangi, Edwine Barasa, Felix Masiye, Obinna Onwujekwe","doi":"10.1007/s41669-026-00690-7","DOIUrl":"https://doi.org/10.1007/s41669-026-00690-7","url":null,"abstract":"<p><strong>Background: </strong>Developing local value sets for child and adolescent health is critical for economic evaluation in Nigeria. Discrete choice experiments (DCEs) can elicit these preferences, yet data on choice behaviour from Nigerian populations are lacking.</p><p><strong>Objectives: </strong>The aim of this study was to analyse DCE-based preferences for EQ-5D-Y-3L health states among Nigerian adult proxies, examining dimension-level drivers of choice, prevalence and correlates of preference reversals, and overall data quality.</p><p><strong>Methods: </strong>A DCE was administered to 2699 adults across six Nigerian states, generating 40,485 choice task observations. Paired health states were constructed using an efficient design. The EuroQol Portable Valuation Technology (EQ-PVT) recorded choices and identified logical reversals. Analyses included descriptive statistics, mixed-effects logistic regression, and comparative tests examining differences between reversed and normal tasks.</p><p><strong>Results: </strong>Reversals, which reflect respondent-level software orientation rather than task-level choice behaviour, occurred in 50.32% of tasks; severity, extreme-problem status, and demographics did not differ between EQ-PVT-reversed and normal tasks, confirming this measure as a data-entry artifact rather than evidence of decision strategy. A level-sum-score-based measure of substantive preference inconsistency, distinct from the EQ-PVT reversal, identified inconsistency in 10.7% of non-tied pairs. Pain/discomfort (β = - 1.544) and anxiety/depression (β = - 1.328) were the strongest predictors of choosing the better health state (p < 0.001).</p><p><strong>Conclusions: </strong>This large-scale DCE provides foundational evidence for a Nigerian EQ-5D-Y-3L value set, confirming pain/discomfort and anxiety/depression as primary drivers of proxy preferences for child health. The EQ-PVT reversal rate reflects a balanced software-orientation assignment rather than flawed decision making; the true behavioural inconsistency rate (10.7% of non-tied pairs) was low and supports the feasibility of generating locally relevant preference data for health technology assessment in Nigeria.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Conceptualising Health Economic Models Using a Graph Retrieval-Augmented, Agentic AI Framework with Human-in-the-Loop Validation: Proof-of-Concept Study. 概念化健康经济模型使用图形检索增强,人工智能框架与人在循环验证:概念验证研究。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-12 DOI: 10.1007/s41669-026-00687-2
Tushar Srivastava, Hanan Irfan, Shilpi Swami
{"title":"Conceptualising Health Economic Models Using a Graph Retrieval-Augmented, Agentic AI Framework with Human-in-the-Loop Validation: Proof-of-Concept Study.","authors":"Tushar Srivastava, Hanan Irfan, Shilpi Swami","doi":"10.1007/s41669-026-00687-2","DOIUrl":"https://doi.org/10.1007/s41669-026-00687-2","url":null,"abstract":"<p><strong>Objective: </strong>Health economic models (HEMs) are crucial for assessing the cost-effectiveness of healthcare interventions, informing policy decisions and guiding reimbursement strategies. This study examines the feasibility of employing generative artificial intelligence (Gen-AI) for the conceptualisation of HEMs. Two proof-of-concept studies were conducted, implementing a first-of-its-kind agentic Gen-AI-augmented approach for model conceptualisation.</p><p><strong>Methods: </strong>An agentic Gen-AI framework was developed, incorporating the chain-of-thought (CoT) reasoning algorithm through advanced prompt engineering, compounded with self-consistency, executed in an external Python programming environment. Within this framework, an agentic execution layer (the 'agent') autonomously orchestrated a multi-step retrieval-reasoning-generation task. Additionally, a graph-based retrieval-augmented generation (RAG) technique was implemented by linking the large language model (LLM) to an external database curated with user-selected data specific to HEM. Two case studies were explored: breast cancer, representing a data-rich disease area, and transthyretin amyloid polyneuropathy (ATTR-PN), exemplifying a data-sparse rare disease.</p><p><strong>Results: </strong>The agentic Gen-AI framework successfully generated two key outputs that were subsequently validated against published literature and expert review from health economic (HE) modelers and clinicians: (1) a disease concept diagram illustrating the progression of the disease and its associated health states, and (2) a model structure diagram depicting patient transitions between various health states.</p><p><strong>Conclusions: </strong>The findings suggest that Gen-AI-augmented methodologies can enhance multiple aspects of HEM conceptualisation, including problem definition, disease and treatment effect pathway development, model structure recommendations, and drafting of model specification plans. To the best of the authors' knowledge, this is the first study to integrate cutting-edge Gen-AI techniques such as human-in-the-loop (HITL), chain of thought-self-consistency (CoT-SC), and graph-based RAG for HEM conceptualisation or broader health economics and outcomes research (HEOR) applications.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Economic and Humanistic Burden of Phenylketonuria Among Patients and Their Caregivers: A Systematic Literature Review. 苯丙酮尿症患者及其护理人员的经济和人文负担:系统文献综述。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-10 DOI: 10.1007/s41669-026-00683-6
Judit Józwiak-Hagymásy, Tamás Zelei, Shailja Vaghela, Dóra Mezei, Hédi Varga, Márk Hámori, Kristóf Gyöngyösi, Vanja Sikirica, Sue Perera
{"title":"The Economic and Humanistic Burden of Phenylketonuria Among Patients and Their Caregivers: A Systematic Literature Review.","authors":"Judit Józwiak-Hagymásy, Tamás Zelei, Shailja Vaghela, Dóra Mezei, Hédi Varga, Márk Hámori, Kristóf Gyöngyösi, Vanja Sikirica, Sue Perera","doi":"10.1007/s41669-026-00683-6","DOIUrl":"https://doi.org/10.1007/s41669-026-00683-6","url":null,"abstract":"<p><strong>Background/objectives: </strong>Phenylketonuria (PKU) is a rare autosomal recessive metabolic disorder resulting from a deficiency in phenylalanine hydroxylase (PAH) enzyme activity. This systematic literature review (SLR) aims to synthesize the available evidence on the economic and humanistic burden of PKU on patients and their caregivers.</p><p><strong>Methods: </strong>A comprehensive SLR was conducted using PubMed, Embase, Cochrane Library, International Prospective Register of Systematic Reviews (PROSPERO), EconLit, and the Cost-Effectiveness Analysis (CEA) Registry, from database inception to June 2026. Eligible studies were published in English, included individuals diagnosed with PAH deficiency (including PKU) and/or their caregivers, and reported original data on either the economic or humanistic burden of the condition.</p><p><strong>Results: </strong>Of 2131 records identified, 1330 were screened after deduplication, and 313 underwent full-text review. A total of 130 studies were included, with 44 reporting on economic burden and 87 covering humanistic burden-related outcomes. The economic burden of dietary management was substantial, with mean per patient per year (PPPY) costs ranging from US$1634 to US$35,946, and an estimated 233-359 h PPPY dedicated to diet-related activities. Humanistic burden was also significant; the long-term metabolic control significantly influenced multiple domains of health-related quality of life (HRQoL). In a study involving adults with PKU (n = 88), dietary protein restriction was found to be negatively associated with HRQoL (Rp = 0.265, p < 0.05), and similarly, anxiety was also negatively correlated with blood phenylalanine (Phe) levels (Rs = 0.186-0.209, p < 0.05).</p><p><strong>Conclusions: </strong>Findings underscore the substantial financial, time, and psychological burden of PKU, particularly in relation to dietary management.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149172118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Budget Impact Model of a Novel cfRNA Test-and-Treat Strategy for Preeclampsia in Advanced Maternal Age Pregnancies Compared with Current Guidelines. 与现行指南相比,一种新型cfRNA检测和治疗策略对高龄产妇子痫前期的预算影响模型
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-05 DOI: 10.1007/s41669-026-00682-7
April Zambelli-Weiner, Brian Nathanson, Susan Hahn
{"title":"Budget Impact Model of a Novel cfRNA Test-and-Treat Strategy for Preeclampsia in Advanced Maternal Age Pregnancies Compared with Current Guidelines.","authors":"April Zambelli-Weiner, Brian Nathanson, Susan Hahn","doi":"10.1007/s41669-026-00682-7","DOIUrl":"https://doi.org/10.1007/s41669-026-00682-7","url":null,"abstract":"<p><strong>Background: </strong>Preeclampsia (PE) affects 8% of United States (US) pregnancies, driving substantial maternal and neonatal morbidity, costs, and mortality. Current United States Preventive Services Task Force (USPSTF) risk-based stratification has limited specificity, which may reduce the ability to effectively target preventive interventions and optimize PE prevention.</p><p><strong>Objectives: </strong>We evaluated the budget impact of adopting a cell-free RNA (cfRNA) PE blood test compared with USPSTF screening in advanced maternal age (AMA; maternal age ≥ 35 years at delivery) pregnancies without pre-existing high-risk conditions. The evaluated intervention was a cfRNA-guided test-and-treat strategy in which high-risk pregnancies received targeted preventive management including low-dose aspirin.</p><p><strong>Methods: </strong>An observational claims analysis and deterministic budget impact model was developed from the US commercial payer perspective to simulate a 4.5-million-member health plan with 10,000 eligible AMA pregnancies annually. Costs were assessed at year 1 and year 5 timepoints at the maternal-infant dyad level, capturing both maternal and neonatal expenditures. Clinical performance inputs were derived from published data; cost inputs were drawn from commercial claims. The model compared USPSTF screening with cfRNA testing and targeted use of prophylactic low-dose aspirin. Economic endpoints included maternal and neonatal costs, budget impact, and return on investment (ROI).</p><p><strong>Results: </strong>In the base case, the cfRNA test-and-treat strategy was projected to generate first-year net health plan savings (in 2024 US dollars) of $36.6 million per 10,000 eligible AMA pregnancies tested. On a total allowed-payment basis, projected first-year net budget savings after testing and intervention costs were $37.4 million, gross clinical-event savings (before testing and intervention costs) were $56.1 million, and ROI was 3.00:1. Projected annual net savings increased to $56.1 million using net plan payments and $57.2 million using total allowed payments in year 5.</p><p><strong>Conclusions: </strong>Cell-free RNA-based screening combined with targeted preventive interventions and changes in preventive management was projected to reduce severe PE cases and associated maternal and neonatal healthcare costs, resulting in substantial payer savings and favorable ROI in this modeled analysis. Results depend on assumptions regarding clinical recommendations, adherence, treatment effects, gestational-age shifts, test cost, and neonatal payments.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Healthcare Utilization and Direct Medical Costs of Hepatocellular Carcinoma Treatment - A German Claims Data Study of 2778 Patients. 肝细胞癌治疗的医疗保健利用和直接医疗费用——2778例德国索赔数据研究
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-02 DOI: 10.1007/s41669-026-00685-4
Selina Becht, Sami Orabi, Simone Schrodi, Najib Ben Khaled, Michael von Bergwelt-Baildon, Peter Buggisch, Wolf-Peter Hofmann, Ursula Marschall, Julia Mayerle, Alexander Philipp, Jörg Trojan, Tobias Weiglein, Larissa Schwarzkopf, Enrico N De Toni, Karin Berger-Thürmel
{"title":"Healthcare Utilization and Direct Medical Costs of Hepatocellular Carcinoma Treatment - A German Claims Data Study of 2778 Patients.","authors":"Selina Becht, Sami Orabi, Simone Schrodi, Najib Ben Khaled, Michael von Bergwelt-Baildon, Peter Buggisch, Wolf-Peter Hofmann, Ursula Marschall, Julia Mayerle, Alexander Philipp, Jörg Trojan, Tobias Weiglein, Larissa Schwarzkopf, Enrico N De Toni, Karin Berger-Thürmel","doi":"10.1007/s41669-026-00685-4","DOIUrl":"https://doi.org/10.1007/s41669-026-00685-4","url":null,"abstract":"<p><strong>Background/objective: </strong>Hepatocellular carcinoma (HCC) is a cancer with rising incidence and economic burden. However, evidence on healthcare resource utilization (HCRU) and associated costs is scarce in Europe. This study aims to determine HCRU and costs of HCC from a German third-party payer's perspective.</p><p><strong>Methods: </strong>This longitudinal claims study (BARMER statutory health insurance) included patients with HCC (ICD-10-GM C22.0; 2016-2020). HCRU and costs within 2 years after diagnosis were analyzed descriptively and reported per patient (PP) and per patient per month (PPPM).</p><p><strong>Results: </strong>The cohort included 2778 patients. Mean age was 71.9 years (SD ±9.7); 69% were male. Total cost within 2 years after diagnosis was approximately €105 million, with mean cost PP of €37,970 (SD ±€43,840), primarily due to inpatient stays (67%). Median costs PP were highest for patients receiving liver transplantation (€126,943; interquartile range [IQR]: €104,340-€167,205) and lowest for patients without any tumor-specific therapy (€10,132; IQR: €5875-€19,663). Considering the observation period, costs PPPM were lowest for liver resection (€2434; IQR: €1282-€5425) and thermal ablation (€2349; IQR: €1233-€4316), whereas costs PPPM were highest for patients without tumor-specific HCC treatment (€5984; IQR: €2764-€10,908). Similar patterns were observed in the HCRU assessment.</p><p><strong>Conclusions: </strong>In this German claims data analysis, PP costs and HCRU in patients with HCC were higher among those who underwent liver transplantation and lower among those without tumor-specific treatment. However, after adjustment for follow-up duration, the high costs PP among patients receiving curative-intent treatments were significantly relativized. These findings support structured HCC surveillance to potentially improve clinical and economic outcomes.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness Analysis of Ribociclib plus an Aromatase Inhibitor (AI) Versus AI Alone in the Adjuvant Treatment of Stage II and III Pre- and Postmenopausal Women with HR+/HER2- Early Breast Cancer (EBC): A Canadian Payer Perspective. Ribociclib联合芳香酶抑制剂(AI)与单独使用AI辅助治疗II期和III期绝经前和绝经后HR+/HER2-早期乳腺癌(EBC)的成本-效果分析:加拿大支付款人的观点
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-02 DOI: 10.1007/s41669-026-00674-7
Dina Soliman, Christine Brezden-Masley, Sandeep Sehdev, Khalid El Ouagari, Natalia Sabater Anaya, Daniel Stellato, Talitha Vincken
{"title":"Cost-Effectiveness Analysis of Ribociclib plus an Aromatase Inhibitor (AI) Versus AI Alone in the Adjuvant Treatment of Stage II and III Pre- and Postmenopausal Women with HR+/HER2- Early Breast Cancer (EBC): A Canadian Payer Perspective.","authors":"Dina Soliman, Christine Brezden-Masley, Sandeep Sehdev, Khalid El Ouagari, Natalia Sabater Anaya, Daniel Stellato, Talitha Vincken","doi":"10.1007/s41669-026-00674-7","DOIUrl":"https://doi.org/10.1007/s41669-026-00674-7","url":null,"abstract":"<p><strong>Background: </strong>Cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitors have transformed the treatment of hormone receptor-positive (HR+) human epidermal growth factor receptor 2-negative (HER2-) breast cancer. Ribociclib plus an aromatase inhibitor (AI) was approved in Canada as adjuvant treatment for high-risk stage II-III early breast cancer (EBC) based on the phase 3 NATALEE trial, demonstrating improved invasive disease-free survival (iDFS) versus AI treatment alone. This study evaluated the cost-effectiveness of ribociclib plus AI versus AI alone and tamoxifen from a Canadian public payer perspective.</p><p><strong>Methods: </strong>A non-homogeneous, semi-Markov cohort model with six health states (iDFS, second primary malignancy, non-metastatic recurrence, remission, distant recurrence [DR], and death) was developed, using Ontario-based costs (Canadian dollars) and a lifetime horizon. Transition probabilities from iDFS were derived from NATALEE trial data; other transitions were informed by published sources. DR was stratified by endocrine therapy (ET) sensitivity based on recurrence timing (≤ 12 months post-ET: resistant; > 12 months: sensitive), with outcomes and costs informed by prior health technology assessment evidence in metastatic disease. Results were reported as incremental cost-effectiveness ratios (ICERs) per quality-adjusted life-year (QALY) gained.</p><p><strong>Results: </strong>Ribociclib plus AI yielded a probabilistic ICER of $45,097/QALY versus AI alone; tamoxifen was dominated by AI alone. Sensitivity analyses showed results were most sensitive to iDFS hazard rates and ribociclib drug costs. In the probabilistic analysis, ribociclib was preferred in 58.5% of simulations at a $50,000 willingness-to-pay threshold.</p><p><strong>Conclusion: </strong>Ribociclib plus AI is cost-effective for HR+/HER2- EBC with an ICER of $45,097, below the $50,000 willingness-to-pay threshold versus AI or tamoxifen alone.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Generalized Pustular Psoriasis: A Cost-of-Illness and Health-Related Quality of Life Study in Italy. 全身性脓疱性牛皮癣:意大利疾病成本和健康相关生活质量研究
IF 2.4
PharmacoEconomics Open Pub Date : 2026-10-02 DOI: 10.1007/s41669-026-00684-5
Ludovica Borsoi, Patrizio Armeni, Annalisa M Scopinaro, Paolo Gisondi, Francesco Costa
{"title":"Generalized Pustular Psoriasis: A Cost-of-Illness and Health-Related Quality of Life Study in Italy.","authors":"Ludovica Borsoi, Patrizio Armeni, Annalisa M Scopinaro, Paolo Gisondi, Francesco Costa","doi":"10.1007/s41669-026-00684-5","DOIUrl":"https://doi.org/10.1007/s41669-026-00684-5","url":null,"abstract":"<p><strong>Objective: </strong>Generalized pustular psoriasis (GPP) is a life-threatening rare disease, with an estimated prevalence of 1-3 cases per million individuals in Italy. Despite its clinical severity, evidence on the economic impact of GPP remains limited. This study aimed to estimate the annual societal economic burden of GPP in Italy and assess the health-related quality of life (HRQoL) of patients with GPP.</p><p><strong>Methods: </strong>A prevalence-based, probabilistic cost-of-illness model was developed using a societal perspective. Data were drawn from primary and secondary sources, including published and grey literature, expert opinion, and a socio-economic questionnaire administered to Italian patients with GPP. Patient-reported HRQoL was assessed using the Psoriasis Disability Index (PDI). Parameter uncertainty was addressed through both deterministic and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>Our study estimated that the annual economic burden per GPP patient in Italy ranged from €24,914 to €25,270 (total societal cost: €2.94-€2.98 million/year), substantially exceeding the costs associated with plaque psoriasis. While the majority of costs were attributable to the consumption of healthcare resources, more than one-third of the total burden was due to indirect costs. PDI data revealed a high level of perceived disability among patients with GPP, particularly in relation to daily activities and interpersonal relationships.</p><p><strong>Conclusions: </strong>Our analysis indicates that GPP imposes a considerable societal economic burden in Italy and markedly affects patients' quality of life. The substantial direct and indirect costs of GPP, which far exceed those of plaque psoriasis, underscore the importance of recognizing GPP as a distinct clinical entity rather than a subtype of plaque psoriasis. This distinction could support the development of more targeted management strategies and health policies for GPP (as a rare disease), including disease-specific treatment approvals and reimbursement.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Health Outcomes, Healthcare Resource Use, and Economic Burden of Invasive Meningococcal Disease in the United States Among Children Aged <11 Years. 美国11岁以下儿童侵袭性脑膜炎球菌病的健康结局、医疗资源使用和经济负担
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-21 DOI: 10.1007/s41669-026-00676-5
Oscar Herrera-Restrepo, Elizabeth Packnett, Megan Richards, Elise Kuylen, Tosin O Olaiya, Thatiana Pinto, Lindsay Landgrave, Ryan Ross, Andrew G Allmon
{"title":"Health Outcomes, Healthcare Resource Use, and Economic Burden of Invasive Meningococcal Disease in the United States Among Children Aged <11 Years.","authors":"Oscar Herrera-Restrepo, Elizabeth Packnett, Megan Richards, Elise Kuylen, Tosin O Olaiya, Thatiana Pinto, Lindsay Landgrave, Ryan Ross, Andrew G Allmon","doi":"10.1007/s41669-026-00676-5","DOIUrl":"https://doi.org/10.1007/s41669-026-00676-5","url":null,"abstract":"<p><strong>Background/objective: </strong>Although uncommon, invasive meningococcal disease (IMD) is a serious and life-threatening illness. In the United States (US), incidence is highest among infants aged <1 year, but research characterizing pediatric IMD burden is limited. This study aimed to evaluate health outcomes and economic burden (healthcare resource utilization [HCRU] and costs [associated with health outcomes and HCRU]) in US individuals aged <11 years with IMD and without IMD, and their caregivers, from the patient and payer (commercial and Medicaid insurance) perspective.</p><p><strong>Methods: </strong>Retrospective claims analysis utilizing real-world data (1/1/2005-12/31/2022) from the MarketScan<sup>®</sup> Commercial and Multi-State Medicaid Databases compared health outcomes, HCRU (e.g., inpatient and outpatient admissions, surgical procedures, pharmacy claims), and costs (associated with health outcomes and HCRU) in commercially and Medicaid-insured infants (aged <1 year), toddlers (aged 1-4 years), and children (aged 5-10 years) with IMD versus those without IMD (matched 1:5), and their caregivers. Length of follow-up was variable, divided into acute/post-acute phases for individuals with IMD.</p><p><strong>Results: </strong>Infants, toddlers, and children with IMD versus without IMD generally experienced more negative health outcomes and HCRU, and higher associated costs. Common health outcomes among infants with IMD included neurological, physical, and neurodevelopmental/mental health disorders. In the commercially insured cohort, infants with IMD had more inpatient admissions, specialist office visits, and surgical procedures than those without IMD. In the Medicaid-insured cohort, HCRU was higher for infants with IMD versus without IMD. Costs associated with health outcomes and HCRU were higher for infants with IMD versus those without IMD, regardless of insurance cohort. Health outcomes, HCRU, and HCRU-associated costs did not differ for caregivers to individuals with IMD versus without IMD.</p><p><strong>Conclusion: </strong>In this retrospective claims analysis from a dual US patient and payer perspective, infants, toddlers, and children with IMD experienced substantial negative health outcomes and economic burden, regardless of insurance status. Preventive interventions in childhood may lessen the impact of IMD on individuals, families, and healthcare systems.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017445","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Economic Burden of Inappropriate Antibiotic Prescribing Attributable to Dengue in Singapore. 新加坡因登革热导致的不适当抗生素处方的经济负担。
IF 2.4
PharmacoEconomics Open Pub Date : 2026-09-15 DOI: 10.1007/s41669-026-00680-9
Peter Yong Sheng Ong, Hwee Lin Wee, Tun-Linn Thein, Yee-Sin Leo, David Chien Boon Lye, Jue Tao Lim, Annelies Wilder-Smith, Yi Wang, Borame L Dickens
{"title":"Economic Burden of Inappropriate Antibiotic Prescribing Attributable to Dengue in Singapore.","authors":"Peter Yong Sheng Ong, Hwee Lin Wee, Tun-Linn Thein, Yee-Sin Leo, David Chien Boon Lye, Jue Tao Lim, Annelies Wilder-Smith, Yi Wang, Borame L Dickens","doi":"10.1007/s41669-026-00680-9","DOIUrl":"https://doi.org/10.1007/s41669-026-00680-9","url":null,"abstract":"<p><strong>Background: </strong>Dengue has a clinical presentation similar to that of bacterial infections, creating diagnostic uncertainty that may lead to inappropriate antibiotic prescribing.</p><p><strong>Objective: </strong>To estimate the magnitude of inappropriate antibiotic prescribing attributable to dengue in Singapore and assess the associated economic burden from healthcare system and societal perspectives over the period 2015-2024.</p><p><strong>Methods: </strong>A decision tree model was developed to determine costs associated with 142,525 laboratory-confirmed dengue cases reported in Singapore during 2015-2024. National surveillance data and prescribing rates informed model inputs. Costs were estimated from healthcare system and societal perspectives and expressed in 2025 Singapore dollars (SGD). Deterministic and probabilistic sensitivity analyses were conducted.</p><p><strong>Results: </strong>The model estimated 7055 antibiotic prescriptions, of which 6866 were inappropriate; 79.6% of inappropriate prescriptions occurred in ambulatory care. The cumulative burden associated with the 2015-2024 caseload was SGD 14.32 million from the healthcare system perspective and SGD 16.13 million from the societal perspective. Prolonged hospitalisation was the largest cost component followed by adverse drug events due to antibiotic use. In the probabilistic analysis, the mean societal burden was SGD 16.16 million, with a 95% uncertainty interval of SGD 2.78-49.60 million.</p><p><strong>Conclusions: </strong>In this analysis, inappropriate antibiotic prescribing attributable to dengue was associated with an estimated cumulative economic burden of SGD 14.32 million from the healthcare system perspective and SGD 16.13 million from the societal perspective in Singapore during 2015-2024, expressed in 2025 values. Direct medical costs accounted for most of the burden, and the findings support further evaluation of approaches to reduce unnecessary prescribing.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148977661","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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