Franciso Álvarez García, Federico Martinón-Torres, Valentí Pineda, Alejandra López-Ibáñez de Aldecoa, Paulina Gálvez, An Ta, Johnna Perdrizet
{"title":"Cost-effectiveness analysis of 20-valent anti-pneumococcal vaccination in the Spanish pediatric population.","authors":"Franciso Álvarez García, Federico Martinón-Torres, Valentí Pineda, Alejandra López-Ibáñez de Aldecoa, Paulina Gálvez, An Ta, Johnna Perdrizet","doi":"10.1080/14737167.2025.2521445","DOIUrl":"10.1080/14737167.2025.2521445","url":null,"abstract":"<p><strong>Objectives: </strong>We evaluated the cost-effectiveness of implementing different pneumococcal conjugate vaccines (PCV) - 20-valent (PCV20; 3 + 1), 13-valent (PCV13; 2 + 1), and 15-valent (PCV15; 2 + 1) - into the Spanish pediatric national immunization program (NIP) for pneumococcal disease prevention.</p><p><strong>Methods: </strong>A Markov model adopting a Spanish National Healthcare System perspective and annual cycles estimated the health and cost impact of PCV20, PCV13, and PCV15 over 10 years among children. Epidemiological, cost, and utility inputs were derived from published literature and official databases; vaccine efficacy inputs were based on PCV13 clinical effectiveness and 7-valent PCV efficacy and impact studies. Sensitivity analyses evaluated model robustness.</p><p><strong>Results: </strong>PCV20 implementation was predicted to reduce the pneumococcal disease burden, preventing > 1,000,000 pneumococcal disease cases and > 150 deaths, versus both comparators. The adoption of PCV20 was estimated to result in cost-savings of approximately €1 billion versus PCV13 and PCV15. PCV20 demonstrated dominance over both alternatives, with 100% of 1,000 probabilistic sensitivity analysis iterations indicating PCV20 dominance.</p><p><strong>Conclusion: </strong>Incorporating PCV20 3 + 1 into the Spanish pediatric NIP was predicted to be more effective at a lower cost than PCV13 2 + 1 and PCV15 2 + 1 due to its broader serotype coverage and enhanced protection against pneumococcal disease.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1101-1113"},"PeriodicalIF":1.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144332726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Fernando Martínez Valle, Lucía Galán Dávila, Juan Buades Reinés, Juan Gonzalez-Moreno, Inés Losada López, Teresa Sevilla, Francisco Muñoz Beamud, José Eulalio Bárcena Llona, Manuel Romero Acebal, Patricia Tarilonte, Francesca Setaro, Javier Rejas-Gutierrez, Alejandra López-Ibáñez de Aldecoa, Carmen Peral
{"title":"Economic and healthcare resource utilization for hereditary transthyretin amyloidosis with polyneuropathy in Spain: real-world insights from early-stage patients and mutation carriers.","authors":"Fernando Martínez Valle, Lucía Galán Dávila, Juan Buades Reinés, Juan Gonzalez-Moreno, Inés Losada López, Teresa Sevilla, Francisco Muñoz Beamud, José Eulalio Bárcena Llona, Manuel Romero Acebal, Patricia Tarilonte, Francesca Setaro, Javier Rejas-Gutierrez, Alejandra López-Ibáñez de Aldecoa, Carmen Peral","doi":"10.1080/14737167.2025.2527269","DOIUrl":"10.1080/14737167.2025.2527269","url":null,"abstract":"<p><strong>Objective: </strong>Variant transthyretin (ATTRv) amyloidosis is a rare genetic multisystem disease inducing progressive morbidity. Evidence about its economic burden is scarce. This study estimated annual use and costs of healthcare-resource-utilization (HRU) and indirect costs for ATTRv mutation carriers and patients with polyneuropathy from a societal perspective in Spain.</p><p><strong>Methods: </strong>A cross-sectional, retrospective non-interventional multicenter study was conducted in 2018-2020 to characterize asymptomatic carriers of mutations in the TTR gen (AC) and Coutinho stage 1 transthyretin amyloidosis polyneuropathy patients (PA). HRU, associated costs, and indirect wages were assessed during 12 months prior to enrollment.</p><p><strong>Results: </strong>One-hundred-five participants were analyzed: 86 AC and 19 PA. Average healthcare cost-per-person-per-year was significantly higher in PA: €2,323.6 vs. €953.3 (<i>p</i> < 0.001), due to higher utilization of medical specialty visits, hospitalizations, and nerve conduction studies. Indirect costs were statistically not different (€380.1 for AC and €781.3 for PA, <i>p</i> = 0.581). Days worked with fatigue/pain were significantly higher in PA: 53.9 vs 5.5 (<i>p</i> = 0.034). Total cost was significantly higher in PA than AC (€3,105.0 vs €1,333.4; <i>p</i> < 0.001).</p><p><strong>Conclusions: </strong>Both AC and early-stage ATTRv-PN patients showed a meaningful economic burden to the National Health System and society. We observed an annual incremental economic burden of €1,771.5 in PA versus AC.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1115-1123"},"PeriodicalIF":1.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559644","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-effectiveness of 13-valent pneumococcal polysaccharide conjugate vaccine in Indian adults aged ≥50 years.","authors":"Namrata Kulkarni, Ahuva Averin, Santosh Taur, Liping Huang, Dhwani Hariharan, Mark Atwood, Neha Gupta","doi":"10.1080/14737167.2025.2508255","DOIUrl":"10.1080/14737167.2025.2508255","url":null,"abstract":"<p><strong>Objectives: </strong>Lacking national policy for adult pneumococcal vaccination, local Indian guidelines recommend 13-valent pneumococcal polysaccharide conjugate vaccine (PCV13) followed by 23-valent pneumococcal polysaccharide vaccine (PPV23). However, no vaccination ('NV') or PPV23 alone are most common in clinical practice. We evaluated cost-effectiveness of PCV13 versus NV and, alternatively, PPV23 in India.</p><p><strong>Methods: </strong>Cohort model with Markov-type process projected lifetime cases, deaths, and costs associated with invasive pneumococcal disease and all-cause non-bacteremic pneumonia (discounting = 5%/year). Cost per quality-adjusted life year (QALY) gained was assessed among at-risk/high-risk adults aged 50-59 years and all aged 60-99 years (<i>N</i> = 209.5 M) from private/patient and government/payer perspectives, which differed on medical costs, vaccination costs, and uptake rates.</p><p><strong>Results: </strong>From private/patient perspective, PCV13 versus NV yielded ₹480,908/QALY (ΔCosts=₹27.2B;ΔQALYs = 56,560), whereas PCV13 was dominant versus PPV23 (ΔCosts=-₹359.1 M; ΔQALYs = 53,861).From government/payer perspective, corresponding ratios were ₹610,178/QALY (ΔCosts=₹84.4B;ΔQALYs = 138,382), and ₹456,048/QALY (ΔCosts=₹60.2B; ΔQALYs = 131,933). In probabilistic sensitivity analyses, cost-effectiveness was <₹600,000/QALY in 72.9%-99.7% of replications (<i>N</i> = 1000/analysis), depending on comparison/perspective.</p><p><strong>Conclusions: </strong>PCV13 versus NV among at-risk/high-risk adults aged 50-59 years and all aged ≥ 60 years would be cost-effective from both perspectives considering willingness-to-pay equaling approximately 3× gross domestic product/capita. Furthermore, PCV13 versus PPV23 would be cost-effective and cost saving from government/payer and private/patient perspectives, respectively.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1087-1099"},"PeriodicalIF":1.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144215318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Understanding the upper bound of survival for conducting and critically appraising economic evaluations of low-risk cancers.","authors":"Xuanqian Xie, Ishita Joshi, Myra Wang, Chunmei Li","doi":"10.1080/14737167.2025.2522326","DOIUrl":"10.1080/14737167.2025.2522326","url":null,"abstract":"<p><strong>Background: </strong>In most developed countries, overall survival rates for low-risk cancers (e.g. localized prostate and thyroid cancer) are comparable to those of the general population. The general population's survival rate may serve as an upper bound for survival in people with these cancers.</p><p><strong>Methods: </strong>By applying this concept, we demonstrated limitations of using Markov models for low-risk cancers and proposed an alternative modeling approach.</p><p><strong>Results: </strong>Markov disease progression models typically depict a gradual progression from early to advanced cancer stages and an increasing risk of cancer-specific mortality over time. However, data showed that the risk of death from cancer was often the greatest within the first few years following diagnosis. We therefore proposed an alternative modeling approach. This method involves calculating the average quality-adjusted life-years (QALYs) from the integrals of the survival curve, multiplied by the corresponding health utility curve. Alternatively, QALYs may be estimated by averaging survival and utility within each time interval and summing these estimates across intervals. We also applied these concepts to the critical appraisal of published economic evaluations.</p><p><strong>Conclusions: </strong>Understanding the upper bound of survival for low-risk cancers enables health economists to more accurately conduct cost-effectiveness analyses and assess the credibility of published economic evaluations. [Figure: see text].</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1011-1020"},"PeriodicalIF":1.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ilaria Ruotolo, Andrea Carenza, Giovanni Sellitto, Rachele Simeon, Francescaroberta Panuccio, Andrea Marini Padovani, Emanuele Amadio, Alessandro Ugolini, Anna Berardi, Giovanni Galeoto
{"title":"Exploring the psychometric properties of the fatigue severity scale: results from a systematic review and reliability meta-analysis.","authors":"Ilaria Ruotolo, Andrea Carenza, Giovanni Sellitto, Rachele Simeon, Francescaroberta Panuccio, Andrea Marini Padovani, Emanuele Amadio, Alessandro Ugolini, Anna Berardi, Giovanni Galeoto","doi":"10.1080/14737167.2025.2511128","DOIUrl":"10.1080/14737167.2025.2511128","url":null,"abstract":"<p><strong>Introduction: </strong>Fatigue is a multifactorial phenomenon that affects individuals across several conditions. Fatigue Severity Scale (FSS) is a widely used questionnaire to measure perceived fatigue. This systematic review aims to identify the psychometric properties of FSS across different populations where it has been validated.</p><p><strong>Methods: </strong>The study selection process was conducted from April 2024 to January 2025. The databases used to identify included studies were MEDLINE (via PubMed), SCOPUS, Web of Science, and CINAHL (via EBSCO). The risk of bias was evaluated using the COSMIN checklist. For the meta-analysis, studies reporting the internal consistency of the scale were analyzed.</p><p><strong>Results: </strong>At the end of the selection process, 59 articles were included in the review; the sample sizes ranged from 20 to 2017 participants, and the mean age spanned from 21,86 to 68,1 years.</p><p><strong>Conclusion: </strong>The FSS is a valid and reliable instrument for fatigue assessment. It would be desirable to investigate heterogeneity; it would be beneficial to investigate responsiveness and to explore the settings in which it is used. Another advise would be to calculate cutoff in healthy subjects to better understand fatigue symptoms and the differences in perception between populations.</p><p><strong>Registration: </strong>PROSPERO (CRD42025635211).</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1021-1036"},"PeriodicalIF":1.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132322","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-effectiveness of durvalumab with or without olaparib as a first-line treatment for dMMR type of advanced/recurrent endometrial cancer.","authors":"Jerusha Daggolu, Javeria Khalid, Hua Chen, Moosa Tatar","doi":"10.1080/14737167.2025.2550426","DOIUrl":"https://doi.org/10.1080/14737167.2025.2550426","url":null,"abstract":"<p><strong>Background: </strong>In the phase III DUO-E trial, durvalumab with or without olaparib significantly increased survival outcomes compared to chemotherapy alone among patients with endometrial cancer. The aim of this study was to assess the cost-effectiveness of durvalumab in patients with advanced/recurrent dMMR endometrial cancer.</p><p><strong>Research design and methods: </strong>A Markov model was used to compare the cost-effectiveness of durvalumab with or without olaparib compared to chemotherapy alone in the treatment of advanced/recurrent dMMR endometrial cancer, using the data from phase III DUO-E trial. The outcomes evaluated were total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICER). One-way sensitivity and probabilistic sensitivity analyses were performed to account for uncertainty.</p><p><strong>Results: </strong>The base case analysis found that durvalumab and chemotherapy had an ICER of $239,884/QALY, whereas durvalumab with olaparib and chemotherapy had an ICER of $259,651/QALY compared to chemotherapy alone. The model was most sensitive to the cost of durvalumab. Probabilistic sensitivity analysis revealed that durvalumab had a 0% likelihood of being considered cost effective at a WTP threshold of $150,000 per QALY.</p><p><strong>Conclusions: </strong>Durvalumab was not cost-effective as a first-line treatment of patients with advanced/recurrent dMMR subtype of endometrial cancer. Reducing the cost of durvalumab might improve economic outcomes.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-8"},"PeriodicalIF":1.5,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948228","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marzieh Nosrati, Hossein Vatankhah, Mahdi Asfia, Mohammad Hossein Sharifnia, Farhad Shahi, Mandana Hasanzad, Akbar Abdollahi Asl, Mohammad Abdollahi, Shekoufeh Nikfar
{"title":"Proposed method for economic evaluation based on basket trials: a case study of pembrolizumab.","authors":"Marzieh Nosrati, Hossein Vatankhah, Mahdi Asfia, Mohammad Hossein Sharifnia, Farhad Shahi, Mandana Hasanzad, Akbar Abdollahi Asl, Mohammad Abdollahi, Shekoufeh Nikfar","doi":"10.1080/14737167.2025.2526065","DOIUrl":"10.1080/14737167.2025.2526065","url":null,"abstract":"<p><strong>Background: </strong>Precision medicine's emphasis on genetic profiles rather than diseases has reshaped oncology trials, prioritizing biomarker-specific approaches named basket trials. They involve heterogeneous populations, necessitating new economic evaluation methods, as traditional approaches fail to capture this diversity. This study introduced a methodology for economic evaluations based on basket trials, exemplified through pembrolizumab's cost-effectiveness analysis using Keynote-158 data as a case study and compared the results to disease-specific methods.</p><p><strong>Research design and methods: </strong>A regression-based approach was developed to aggregate costs and QALYs and calculate transition probabilities for economic evaluations in populations sharing a biomarker and various cancers. The proposed method involved five steps: designing a general model, selecting comparator arms, comparing Kaplan-Meier curves and conducting network analysis to evaluate treatment responses, calculating disease-specific coefficients to aggregate QALYs, and aggregating costs using weighted averages.</p><p><strong>Result: </strong>All the steps have been followed for conducting cost-effectiveness of Pembrolizumab in patients with dMMR/MSI-H biomarker. The results revealed significant differences in ICERs calculated by the proposed method versus disease-specific evaluations.</p><p><strong>Conclusion: </strong>This study introduces a biomarker-focused model for estimating ICERs across diverse diseases, enabling informed decision-making in healthcare resource allocation. Further research is recommended to broaden the model's applicability and address additional population heterogeneity factors.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-10"},"PeriodicalIF":1.5,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Preference research is underutilized in health prevention.","authors":"Brett Hauber, Mickael Hiligsmann","doi":"10.1080/14737167.2025.2548279","DOIUrl":"10.1080/14737167.2025.2548279","url":null,"abstract":"<p><strong>Introduction: </strong>Preference research is crucial in shaping effective patient-centered preventive healthcare strategies. Preventive measures such as vaccinations, screenings, and proactive treatment of asymptomatic high-risk conditions aim to reduce future health risks, often in healthy individuals. Understanding patient preferences in these contexts is vital to ensure that interventions are clinically effective, acceptable, and actionable. Failing to consider patient preferences when developing preventive strategies may result in low uptake and adherence to prevention programs, ultimately limiting their impact on patient and population health.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"1-6"},"PeriodicalIF":1.5,"publicationDate":"2025-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144845067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}