PharmacoEconomics最新文献

{"title":"Defining Biological and Clinical Plausibility: The DICSA Framework for Protocolized Assessment in Survival Extrapolations Across Therapeutic Areas.","authors":"Bart Heeg, Dawn Lee, Jane Adam, Maarten Postma, Mario Ouwens","doi":"10.1007/s40273-025-01485-0","DOIUrl":"10.1007/s40273-025-01485-0","url":null,"abstract":"<p><strong>Background: </strong>Numerous health technology assessment guidance documents emphasize the importance of biological/clinical plausibility of modeled lifetime incremental survival without clearly defining it.</p><p><strong>Objectives: </strong>This paper defines biologically and clinically plausible lifetime survival extrapolations and proposes a framework to systematically assess this by comparing survival expectations estimated premodeling, with the final modeled survival extrapolations. This framework is embedded in a survival extrapolation protocol template, which ensures that both the expectations and extrapolations are based on unified, comprehensive evidence.</p><p><strong>Methods: </strong>A targeted review was conducted of 29 guidance documents from National Institute for Health and Care Excellence, Pharmaceutical Benefits Advisory Committee, Haute Autorité de Santé, Canada's Drug Agency, and European joint clinical assessment, focusing on survival analysis, evidence synthesis, cost-effectiveness modeling methods, and use of observational data.</p><p><strong>Results: </strong>Survival extrapolations are biologically/clinically plausible when \"predicted survival estimates that fall within the range considered plausible a-priori, obtained using a-priori justified methodology.\" These a priori expectations should utilize the totality of evidence available and take into account local target setting (i.e., survival-influencing aspects such as patient population, treatment pathway, and country). Pre-protocolized biologically/clinically plausible survival extrapolation was operationalized in a five-step DICSA approach: (1) Describe the target setting as defined by all relevant treatment and disease aspects that influence survival; (2) collect Information from relevant sources; (3) Compare survival-influencing aspects across information sources; (4) Set pre-protocolized survival expectations and plausible ranges; and (5) Assess how trial-based extrapolations align with the set expectations by comparing modeled survival extrapolations to the range of values a priori considered to be plausible.</p><p><strong>Conclusion: </strong>The definition of plausibility of survival extrapolations, the operationalization of its assessment, and the corresponding extrapolation protocol template can contribute to the transparent development of biologically/clinically plausible survival extrapolations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"793-803"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167276/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743541","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":"Reply to Comment on \"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria\".","authors":"Daniel Gallacher","doi":"10.1007/s40273-025-01499-8","DOIUrl":"10.1007/s40273-025-01499-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"839-840"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119445","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":"Comment on \"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria\".","authors":"Benjamin Farrar, Daniel Gladwell, Matthew Woods","doi":"10.1007/s40273-025-01500-4","DOIUrl":"10.1007/s40273-025-01500-4","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"835-837"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119512","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":"How do Health State Values Differ When Respondents Consider Adults Versus Children Living in Those States? A Systematic Review.","authors":"Ashwini De Silva, Alexander van Heusden, Zhongyu Lang, Nancy Devlin, Richard Norman, Kim Dalziel, Tessa Peasgood, Tianxin Pan","doi":"10.1007/s40273-025-01493-0","DOIUrl":"10.1007/s40273-025-01493-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This systematic review examines how different perspectives influence the valuation of child health-related quality of life (HRQoL). Specifically, it explores differences in values when health states are assessed by children, adolescents, or adults (or some combination of these), from the perspective of the first person (self) or the third person (other), and whether specifying (or not) the age of the person living the described health state affects the valuations. Recent studies suggest discrepancies for descriptively similar health states potentially owing to differences in respondents' willingness to trade length-of-life for quality-of-life for children, though findings are inconsistent. This review aims to assess: (1) differences in peoples' willingness to trade, (2) differences between the relative importance of dimensions, and (3) factors influencing these differences.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This systematic review follows PRISMA guidelines. A search in Ovid MEDLINE, Ovid Embase, and EconLit up to November 2024 was undertaken. We included studies where different perspectives and different valuation instruments were considered. We extracted information on study characteristics, instruments, valuation methods, perspective, study design, analytical methods, sample characteristics, differences in values by respondents, and perspective. A multi-level meta-regression assessed the impact of factors affecting the mean differences between perspectives.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;In total, 24 studies were included, which were from 2004 to 2024. Studies used a range of preference elicitation methods and nearly half (38%) used mixed valuation methods. Most studies (71%) used the EQ- 5D-Y- 3L instrument. Overall, 54% of studies compared adults valuing health states for themselves, or other adult versus adults valuing for other children or themselves as children. The multi-level meta-regression found that the severity of the health state and the valuation method has a significant impact on the mean differences between child and adult values for child health states. In most of the studies when adults are respondents, pain or discomfort was considered as the most important dimension. When adolescent respondents value health states the results are mixed. Qualitative studies identified respondents' difficulty imagining a child in ill health and becoming emotional while thinking about child poor health and early death as potential reasons behind differences in child values versus adult values.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;The evidence suggests that differences in mean values arise when different perspectives are used in valuing severe child health states by adults. These differences are influenced by factors such as health state severity and valuation method. While the review identified the key factors influencing the differences in mean values, an uncertainty remains regarding the optimal choice of preferenc","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"723-740"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167273/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986868","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":"Do the Age of Children and Parental Status Matter in Valuing the Child Health Utility 9D (CHU9D)?","authors":"Xiuqin Xiong, Li Huang, Natalie Carvalho, Kim Dalziel, Nancy Devlin","doi":"10.1007/s40273-025-01494-z","DOIUrl":"10.1007/s40273-025-01494-z","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to test whether preferences for children's health states differ (a) when considering those aged 2-4 years compared with older children and (b) by parental status; we also aim to provide a value set that can be used for 2-4 years old.</p><p><strong>Methods: </strong>Health states were described using the Child Health Utility 9D (CHU9D). A discrete choice experiment (DCE) survey was administered between September 2023 and March 2024 to a representative sample of the Australian general adult population which included a 20% quota of parents of 0-18-year-old children. Participants were randomly allocated to two study arms considering the health of a 2-4-year-old or a 10-year-old child. A conditional logit model was used to obtain the latent values from the choice responses. The differences in latent values between the two ages and between parental status were analyzed using relative attribute importance (RAI), poolability test, and pooled model with interaction terms. Visual Analogue Scale (VAS) responses were used to anchor the latent values onto a 0-1 utility scale.</p><p><strong>Results: </strong>In all, 3112 participants were included. Results suggested that the preferences between the two age perspectives were similar, with only 1 out of 36 interaction terms being significant. Preferences of parents of children aged 0-18 years differed from those who were not, as indicated by three significant interaction terms and failure in poolability testing, having smaller disutility for severe health states in the Pain, Tired, and Joining in Activities dimensions.</p><p><strong>Conclusion: </strong>In the valuation of CHU9D health states, asking respondents to consider a 2-4-year-old compared with a 10-year-old did not influence adults' preferences; however, the preferences of respondents who were parents of 0-18-year-olds at that time differed from those who were not. Two CHU9D value sets are reported for children 2 years and older, one derived from the general adult population and the other from parents.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"819-833"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144009590","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 Potential Health Economic Value of Adding Magnetomotive Ultrasound to Current Diagnostic Methods for Detecting Lymph Node Metastases in Rectal Cancer.","authors":"Emelie Andersson, Ulrika Axelsson, Carl-Fredrik Rönnow, Henrik Thorlacius, Linda Persson, Adam Fridhammar","doi":"10.1007/s40273-025-01490-3","DOIUrl":"10.1007/s40273-025-01490-3","url":null,"abstract":"<p><strong>Background: </strong>Local resection of early rectal cancer (RC) is a desirable treatment option compared with surgery, offering reduced morbidity, mortality, health care costs and avoidance of stoma. However, local resection is restricted to cases without suspicion of lymph node metastases (LNM). Current methods to diagnose LNM and risk estimations based on histopathology cannot reliably identify patients eligible for local resection. The NanoEcho diagnostic system is based on a novel method for lymph node staging in RC. The aim of this study was to perform a health economic analysis at an early stage of clinical development to estimate the potential value of adding NanoEcho diagnostics to current diagnostic methods in RC.</p><p><strong>Methods: </strong>A Markov model for RC diagnosis was developed where the costs and health outcomes, including quality-adjusted life years (QALYs), for adding the NanoEcho diagnostics to current diagnostic methods were compared with current diagnostic methods alone. The diagnostic performance of the NanoEcho diagnostic system is still unknown and the base-case analysis was performed at an assumed 85% sensitivity and 85% specificity. Two testing strategies corresponding to two alternative ways of implementing the diagnostic test in clinic were evaluated: (1) examine all patients diagnosed with RC and (2) examine only patients diagnosed with clinical stages T1 and T2.</p><p><strong>Results: </strong>Adding the NanoEcho diagnostic system resulted in a gain of 0.032 life years and 0.124 QALYs per patient in the target population compared with current diagnostic methods alone. At a cost-neutral level, the estimated justifiable price of NanoEcho diagnostics was SEK 6995 in the first testing strategy and SEK 50,658 in the second testing strategy. The justifiable price of the NanoEcho diagnostics at a willingness to pay of 500,000 SEK/QALY was SEK 10,654 in the first testing strategy and SEK 65,132 in the second testing strategy.</p><p><strong>Conclusion: </strong>The results indicate that adding NanoEcho diagnostics to standard of care can potentially reduce healthcare costs and increase quality of life in RC patients, assuming a sensitivity and specificity of 85%.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"779-791"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167262/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973431","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":"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":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;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":"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}