PharmacoEconomics最新文献

{"title":"Anchoring the Hemophilia Joint Health Score for Conventional Value Assessment in the Care of People Living with Severe Hemophilia A.","authors":"Satoko Ito, Manraj Sra, George Goshua","doi":"10.1007/s40273-025-01508-w","DOIUrl":"10.1007/s40273-025-01508-w","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"761-764"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167127/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132696","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":"Exploring the Economic Burden of Pulmonary Arterial Hypertension and Its Relation to Disease Severity and Treatment Escalation: A Systematic Literature Review.","authors":"Gautam Ramani, Vishal Bali, Heather Black, Danny Bond, Ina Zile, Ashley C Humphries, Dominik Lautsch","doi":"10.1007/s40273-025-01492-1","DOIUrl":"10.1007/s40273-025-01492-1","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary arterial hypertension (PAH) is a highly progressive disease characterized by luminal narrowing of the pulmonary arteries, leading to progressive dyspnoea and restricted functional capacity, which can ultimately result in right ventricular failure and death. Treatment goals include improving functional class and walk distance, recovering right ventricular function, halting disease progression, and improving survival. PAH carries a high mortality rate, and treatment escalation is a common feature of disease management. Due to the substantial impact of PAH, a high economic burden has been observed. A systematic literature review (SLR) was carried out to assess the contemporary economic burden of PAH, including the impact of disease severity and treatment escalation.</p><p><strong>Methods: </strong>An electronic database search was conducted and supplemented with a hand search of health technology assessments and conference materials. Studies were included from 2012 to 2024, with no restrictions on geographical location. The inclusion criteria specified that adult patients with PAH (≥ 18 years) and only English language studies were captured.</p><p><strong>Results: </strong>The review included 148 studies and evaluations, 110 of which were observational studies, 14 were economic evaluations, and 24 were health technology assessments. The studies identified reported on several healthcare resource utilization (HCRU) outcomes including hospitalization, PAH-related hospitalization, inpatient visits, emergency department (ED) visits, intensive care unit (ICU) visits, and outpatient visits. Cost data were also reported, including total costs and costs for each of the above-mentioned types of HCRU, as well as specific costs such as pharmacy and drug costs. The results provide an overview of the high economic burden caused by PAH, indicating that the economic burden increases with increasing severity; reported mean monthly costs were as high as US $14,614 (cost converted to USD 2024) for the highest severity group. These data also demonstrated the impact of PAH-specific therapies in reducing HCRU, with efficacious treatment shifting management from an inpatient to outpatient setting (i.e., reduced inpatient admissions and length of stay). Further, while treatment escalation resulted in increased pharmacy costs, this was offset by a reduction in HCRU, including hospitalizations and ED visits. Timely diagnosis was also associated with reduced economic burden, as patients with a longer delay prior to diagnosis reported a higher mean number of monthly hospitalizations, ICU stays, and ED visits. Functional limitation is a common feature of PAH disease progression and can severely impact a patient's ability to work. This SLR identified few studies that investigated such outcomes as well as broader indirect costs, such as out-of-pocket costs and productivity loss.</p><p><strong>Discussion: </strong>This study highlights the","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"741-760"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167309/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034609","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 Framework for Reliable, Transparent, and Reproducible Population-Adjusted Indirect Comparisons.","authors":"K Jack Ishak, Conor Chandler, Fei Fei Liu, Sven Klijn","doi":"10.1007/s40273-025-01503-1","DOIUrl":"10.1007/s40273-025-01503-1","url":null,"abstract":"<p><p>Population-adjusted indirect comparison (PAIC) methods aim to address some of the potential shortcomings of conventional approaches to indirect treatment comparisons by adjusting for imbalances in effect modifiers or prognostic factors and allowing for unanchored indirect treatment comparisons from disconnected networks of evidence. Health technology assessment bodies have published guidance and best practice recommendations for PAICs. However, recently published reviews of published PAICs have highlighted notable variability in implementation and a lack of transparency in the decision-making process in analyses and reporting; this hinders the interpretation and reproducibility of analyses, which, in turn, could affect reimbursement decision-making. We propose a systematic framework to address these challenges by describing considerations on six key elements of analyses: (1) definition of the comparison of interest (e.g., in terms of an estimand), (2) selection of the PAIC method, (3) selection of adjustment variables, (4) application of adjustment method, (5) risk-of-bias assessment, and (6) comprehensive reporting.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"691-710"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015053","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Adjuvant Alectinib Versus Chemotherapy for Patients with Resectable, ALK-positive Non-small Cell Lung Cancer in Canada.","authors":"Nick Jovanoski, Sarah Vaselenak, Andrew Hogan, Jasmine Turki, Quincy Chu","doi":"10.1007/s40273-025-01488-x","DOIUrl":"10.1007/s40273-025-01488-x","url":null,"abstract":"<p><strong>Background: </strong>For patients with resected non-small cell lung cancer (NSCLC), the risk of disease recurrence and progression is associated with a substantial humanistic, clinical, and economic burden. In the phase III ALINA trial (NCT03456076), adjuvant alectinib significantly improved disease-free survival (DFS) compared with chemotherapy in patients with resected ALK-positive NSCLC.</p><p><strong>Objective: </strong>The aim of this study was to assess the cost-effectiveness of adjuvant alectinib versus chemotherapy for patients with resected ALK-positive NSCLC in Canada.</p><p><strong>Methods: </strong>A cost-utility model comprising eight health states was developed to estimate lifetime patient outcomes and costs of patients with resected, ALK-positive NSCLC treated with adjuvant alectinib versus platinum-based chemotherapy from a societal perspective. Patterns of disease recurrence and progression were based on ALINA and other trial data; model assumptions were consistent with existing models and validated through consultation with expert Canadian clinicians. Cost-effectiveness was assessed in terms of estimated effect on life-years, quality-adjusted life-years (QALYs), and healthcare costs. In addition, scenario and probabilistic analyses were performed to explore model uncertainty. An annual discount rate of 1.5% was applied to both costs and outcomes (evaluated for 2023).</p><p><strong>Results: </strong>Compared with chemotherapy, alectinib was associated with greater total life-years (19.2 versus 13.1 years) and QALYs (15.0 versus 10.1). Alectinib was dominant over platinum-based chemotherapy as it yielded a lower lifetime cost (CA $480,967.00) versus chemotherapy (CA $592,959.00). Scenario analyses showed model robustness and consistent dominance in cost-effectiveness. Probabilistic analyses results were similar to those from the base case and scenario analyses; alectinib was dominant over chemotherapy in 93.6% of simulations of incremental costs versus incremental QALYs, and remained under a willingness-to-pay threshold of CA$50,000.00 per QALY gained in 99.7% of simulations.</p><p><strong>Conclusions: </strong>Our analysis suggests that adjuvant alectinib is dominant (i.e., more effective and less costly) to platinum-based chemotherapy in Canadian patients with resected ALK-positive NSCLC. Together with the DFS benefit seen in ALINA, this analysis supports adjuvant alectinib as an important new treatment strategy.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"805-817"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167269/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143992189","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":"Cost-Effectiveness Analysis Methods Used in Evaluations of Treatment for Cystic Fibrosis: A Scoping Review.","authors":"Dominique Seo, David C Young, Eberechukwu Onukwugha, T Joseph Mattingly","doi":"10.1007/s40273-025-01497-w","DOIUrl":"10.1007/s40273-025-01497-w","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis (CF) is a rare genetic condition requiring extensive medical care, which has a significant impact on people with CF. Advances in treatment have extended life expectancy, yet there remains a significant economic burden to manage CF. Cost-effectiveness analysis (CEA) is crucial for evaluating the economic value of treatments and screening for CF. This scoping review seeks to highlight the best practices and gaps in the current evidence base, contributing to robust and comparable CEAs in CF research.</p><p><strong>Methods: </strong>A scoping review was conducted using PubMed and Embase. Studies were included if they featured a CEA focused on CF treatment. Data extraction covered study characteristics, model inputs, and modeling assumptions. A qualitative synthesis was conducted to assess the inclusion of considerations for both healthcare and societal impacts.</p><p><strong>Results: </strong>In total, 11 studies were included. Of these, six focused on evaluations of supportive therapies for CF and five focused on evaluation of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Heterogeneity in comparators and drug costing methods complicated cross-study comparisons. A qualitative review revealed differences in the types of costs and outcomes considered. Studies captured long-term disease progression, health-related quality-of-life effects, and direct medical costs.</p><p><strong>Conclusions: </strong>This review highlights the complexity of CEAs for CF treatment and underscores the need for standardized methodologies and comprehensive evaluations, including broader economic impacts, to support more robust analyses and better-informed decision-making in CF treatment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"711-721"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144049586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Hemophilia Joint Health Score-Based Model for the Economic Evaluation of Hemophilia A Prophylaxis Interventions.","authors":"Sam Hirniak, Andrea N Edginton, Alfonso Iorio, William W L Wong","doi":"10.1007/s40273-025-01484-1","DOIUrl":"10.1007/s40273-025-01484-1","url":null,"abstract":"<p><strong>Background and objective: </strong>Hemophilia A is a costly, lifelong illness with multiple prophylaxis options. Previously, these options were assessed using a Peterson score-based model to simulate joint damage over time. This study built a model for the economic evaluation of hemophilia A with less socioeconomic selection bias utilizing the hemophilia joint health score (HJHS).</p><p><strong>Methods: </strong>A mechanistically defined HJHS-based state-transition microsimulation model was implemented for the cost-utility analysis conducted over a lifetime horizon from a Canadian provincial Ministry of Health perspective, with a 1.5% discount rate on (costs and outcomes), to compare the following interventions: standard half-life (SHL), extended half-life (EHL), emicizumab, and efanesocotog alfa (EA). The health states are HJHS levels, waiting for surgery, postoperative time, and death. Individuals experience bleeds, joint bleeds (increasing the HJHS), and surgery in each health state. Disutilities include injections and postoperative time. Model validation included face validity, internal validity, comparison analysis, external validity, and predictive validity. Probabilistic analysis, pricing threshold analysis, and one-way scenario analyses were completed.</p><p><strong>Results: </strong>EA showed lower levels of hospitalizations and surgeries and an improved joint damage experience in the simulation. However, EA was not cost-effective against emicizumab, which continued to be the most cost-effective intervention. Pricing threshold analysis indicated that a price decrease would be required for EA to dominate SHL (50% decrement) and emicizumab (55% decrement).</p><p><strong>Conclusions: </strong>This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"765-778"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"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}