Applied Health Economics and Health Policy最新文献

{"title":"Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations.","authors":"Adéla Bártová, Jiří Samek, Vera Pinheiro, Barbora Říhová, João Vasco Santos","doi":"10.1007/s40258-025-01008-0","DOIUrl":"https://doi.org/10.1007/s40258-025-01008-0","url":null,"abstract":"<p><strong>Objectives: </strong>Due to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.</p><p><strong>Methods: </strong>HTA organisations across the 27 EU Member States and the UK were identified via INAHTA, EUnetHTA, and ISPOR reference listings. Publicly available documents were screened, and 29 relevant national guidelines were selected. A structured document analysis was performed using a predefined coding framework. Key terms were systematically searched, and content was categorised into thematic domains.</p><p><strong>Results: </strong>Among the 29 guidelines, 16 included references to rare disease populations, and 12 to paediatric populations. For paediatric populations, most references focused on quality-of-life measurement and proxy assessments. Adaptations for rare diseases recognised flexible cost-effectiveness thresholds (e.g. adjusted incremental cost-effectiveness ratios [ICERs] or gross domestic product [GDP]-based modifiers), tailored economic modelling, and acceptance of alternative data sources. However, significant variability was observed across countries, with no consistent pattern.</p><p><strong>Conclusion: </strong>While several HTA bodies have introduced adjustments for paediatric and rare disease populations, guidance often remains limited and heterogeneous. The findings indicate that HTA guidelines and recommendations require further collaboration to properly define and account for the specific needs of these patients. Methodological shortcomings are mainly due to the nature of these diseases, where the limited data available are primarily from clinical practice and often lack comparative effectiveness evidence.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145298227","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":"Opportunities for CMS to Further Specify and Broaden Its Approach to Assessing Unmet Medical Need in the Context of the Medicare Drug Price Negotiation Program.","authors":"Michael J DiStefano, R Brett McQueen, Hanke Zheng, Harry Gyimah Gyamfi, David Ameyaw, Jonathan D Campbell, Antal Zemplenyi","doi":"10.1007/s40258-025-01009-z","DOIUrl":"https://doi.org/10.1007/s40258-025-01009-z","url":null,"abstract":"<p><p>The United States Inflation Reduction Act of 2022 established the Medicare Drug Price Negotiation Program, directing the Centers for Medicare and Medicaid Services (CMS) to establish \"maximum fair prices\" for select drugs. In arriving at maximum fair prices, CMS is required to consider several criteria, including the extent to which a selected drug addresses unmet medical need (UMN). Through a targeted literature review of 48 original research studies, we identified and categorized 40 potential elements of UMN as they relate to pharmaceuticals, as well as treatment-related barriers to addressing this UMN, from the patient, caregiver, and societal perspectives. We synthesized these elements of UMN into seven domains: (1) traditional clinical effectiveness, (2) impacts on patient, caregiver, or family quality of life, (3) economic burden on the patient, caregiver, or family, (4) economic burden on society, (5) societal perspective elements, (6) elements of treatment administration, and (7) availability of other treatments. Comparing these elements with the United States Food and Drug Administration guidance regarding UMN reveals potential key gaps in the current CMS approach to assessing UMN in the Medicare Drug Price Negotiation Program, particularly regarding domains 2 through 6, including quality of life, economic burdens, societal elements, and treatment administration.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145273343","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":"The Role of Price Variation in Economic Analyses for Cancer Screenings: A Rapid Review.","authors":"Austin J Triana, Mason N Alford-Holloway","doi":"10.1007/s40258-025-01007-1","DOIUrl":"https://doi.org/10.1007/s40258-025-01007-1","url":null,"abstract":"<p><strong>Background: </strong>Health care spending continues to rise, and recent policies have made prices more visible.</p><p><strong>Purpose: </strong>To assess how cost effectiveness analyses obtain price information for common cancer screenings and account for price variation.</p><p><strong>Methods: </strong>A systematic search of PUBMED was conducted, extracting studies from 2021 to 2024 in English and evaluating health care services in the USA. Cost-effectiveness analyses were included for four common cancer screenings: prostate cancer, breast cancer, colon cancer, and lung cancer. A single investigator extracted data and assessed quality, reviewed by a second investigator.</p><p><strong>Results: </strong>A total of 16 articles met inclusion criteria. Nearly all (94%) cited the Medicare Fee Schedule as the data source for pricing information. About half (44%) of analyses included a degree of price variation. Only three articles (19%) performed a cost-effectiveness analysis with a wide degree of price variation that accurately reflected the true degree of price variation observed in empirical data.</p><p><strong>Limitations: </strong>The sample size of included studies was modest, and generalizability is limited beyond these four common cancer screenings.</p><p><strong>Conclusions: </strong>Cost-effectiveness analyses in the USA need to reflect the wide price variation that exists in health care, and publicly available price transparency data should guide future work.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145237579","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":"Burden of Disease in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS): A Scoping Review.","authors":"Patricia Vester, Stefanos Boudouroglou-Walter, Jonas Schreyögg, Chantal Wieting, Christine Blome","doi":"10.1007/s40258-025-01006-2","DOIUrl":"https://doi.org/10.1007/s40258-025-01006-2","url":null,"abstract":"<p><strong>Objective: </strong>Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) is a serious chronic and complex multi-system disease characterised by symptoms such as post-exertional malaise, fatigue, cognitive impairment and pain. Diagnosis is based on international consensus criteria, and no curative treatment is available. In the USA, its prevalence is estimated at 0.42% among adults, with women affected three times as often as men. Prevalence is expected to increase due to the COVID-19 pandemic. In addition to its severe symptoms, ME/CFS has a substantial economic impact. This scoping review aimed to systematically examine the global health, social and economic burden of ME/CFS.</p><p><strong>Methods: </strong>We conducted a systematic literature search following the Preferred Reporting Items for Systematic Reviews and Meta-Analysis extension for Scoping Reviews (PRISMA-ScR) guidelines in six databases and supplemented it with a citation search. We assessed study quality using a modified version of the Mixed Methods Appraisal Tool.</p><p><strong>Results: </strong>We included 20 studies that assessed costs (n = 16), disability-adjusted life years (DALYs) (n = 3), employment rates (n = 1), and school attendance (n = 1) as indicators of disease burden. Reported costs per patient ranged from USD 2,916 to USD 119,611, with indirect costs accounting for the largest proportion. DALYs reported for the USA ranged from 0.714 million in 2016 to 5.77 million in 2022.</p><p><strong>Conclusion: </strong>ME/CFS imposes a substantial health, social and economic burden of disease. Discrepancies in estimates are probably due to differences in study samples, methodologies, cost components, and healthcare systems. Because ME/CFS is assumed to be underdiagnosed, its true burden may be even higher.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145123972","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":"Aligning Indigenous and Western Concepts of Health Resource Decision Making in a Western Canadian First Nations Context.","authors":"Aidan Neill, Stephanie Montesanti, Lea Bill, Barbara S E Verstraeten, Rhonda C Bell, Richard T Oster, Arto Ohinmaa, Mike Paulden","doi":"10.1007/s40258-025-01004-4","DOIUrl":"https://doi.org/10.1007/s40258-025-01004-4","url":null,"abstract":"<p><strong>Background: </strong>Western health economic evaluation tools often fail to reflect the relational, collective, and holistic perspectives that underpin Indigenous concepts of health. These limitations pose challenges when applying Western measures in Indigenous contexts. The individualistic foundation of the Western definition of health and the values that inform it are significantly different from the community-based values typically found in Canadian Indigenous communities. For health economics to effectively support Indigenous health decision making, a values-based approach should initially be undertaken to identify conceptual commonalities with Western perspectives.</p><p><strong>Aims: </strong>This study aimed to develop a conceptual framework that identifies shared elements between Western and First Nations understandings of health resource decision making, with the goal of supporting the creation of culturally meaningful health outcome measures.</p><p><strong>Methods: </strong>Through a Health Economics Technical Advisory Group (HE-TAG) in Alberta, Canada, co-led by Indigenous and non-Indigenous researchers, we conducted a conceptual exploration of health resource decision making. Fourteen HE-TAG sessions held between July 2021 and June 2023 were transcribed and analyzed using a hybrid approach-combining Q methodology, thematic analysis (Braun & Clarke), and Walker and Avant's concept analysis.</p><p><strong>Data: </strong>Transcripts from 14 HE-TAG sessions provide the qualitative data upon which analysis was conducted. Sessions were held online using virtual meeting technology, and recordings were transcribed and analyzed.</p><p><strong>Results: </strong>Indigenous and Western conceptual frameworks allow for a common understanding of health resourcing. Indigenous community and culture and Western economic evaluation and social determinants of health were the four main observed themes, each of which contained two subthemes which differentiated between concepts of 'health.' Five concepts were found to resonate between Indigenous and Western themes when exploring health resource thinking: values, holism, time, resources, and context. Concepts and themes were mapped to illustrate common approaches to understanding health resource decision making.</p><p><strong>Conclusions: </strong>This Indigenous-informed research aligns concepts of resource decision making by showing the thematic backgrounds of First Nations and Western thinking, which are linked by the common concepts of values, holism, time, resources, and context. Centering future community engagement on these shared concepts while grounding them in community-generated health value sets can advance the development of novel, culturally relevant health outcome measures.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145102462","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":"Willingness to Pay per QALY: A Systematic Review of Demand-Side Valuations with a Focus on Age and Disease Severity.","authors":"Marios Athanasios Loupas, Kostas Athanasakis, Dimitris Zavras","doi":"10.1007/s40258-025-01005-3","DOIUrl":"https://doi.org/10.1007/s40258-025-01005-3","url":null,"abstract":"<p><strong>Background: </strong>Willingness-to-pay (WTP) studies offer a demand-side perspective on the monetary value of health gains, typically expressed as WTP per quality-adjusted life year (WTP/Q). These estimates can complement supply-side cost-effectiveness thresholds (CETs) and inform whether healthcare budgets align with public preferences. However, existing thresholds often overlook heterogeneity by condition or population characteristics.</p><p><strong>Objective: </strong>This study sought to systematically review literature on WTP/Q estimates derived from stated preference methods by (i) mapping the broader landscape of demand-side valuations and (ii) synthesizing how WTP/Q varies by both respondent and scenario-assigned age and disease severity in general-health contexts.</p><p><strong>Methods: </strong>A systematic review was conducted across Medline, Embase, and EconLit, following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Eligible studies estimated the monetary value of a quality-adjusted life year (QALY) from a societal, demand-side perspective. We first conducted a broad narrative mapping of all included studies to describe methodological characteristics and contextual diversity. We then focused on a subset of studies using general-health scenarios to explore how willingness to pay per QALY varied by age and severity. A structured four-axis framework was applied to distinguish between respondent characteristics and scenario-assigned attributes. Data were classified and tabulated. A formal quality assessment was conducted across the full set of studies using the National Institutes of Health (NIH) tool.</p><p><strong>Results: </strong>A total of 67 studies met inclusion criteria. Most originated from high-income countries and used contingent valuation or discrete choice experiments. Among general-health studies, WTP/Q decreased with respondents' actual age in 49% of studies and increased with respondents' own health severity in 79%. In studies assessing scenario-assigned attributes, WTP/Q increased with hypothetical disease severity in 91% of studies and decreased with scenario-assigned age in 83%. Substantial heterogeneity was observed in elicitation methods, framing, and utility measurement.</p><p><strong>Conclusions: </strong>The findings support the need for condition- and population-specific WTP thresholds, as valuations appear to vary depending on the nature of the health gain and the characteristics of the beneficiary-whether real or hypothetical.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145074406","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":"A Systematic Review of Economic Evaluations on Interventions Targeting Insomnia or Hypersomnia.","authors":"Phuong Hong Le, Long Khanh-Dao Le, Dai Quy Le, Shantha M W Rajaratnam, Cathrine Mihalopoulos","doi":"10.1007/s40258-025-00997-2","DOIUrl":"https://doi.org/10.1007/s40258-025-00997-2","url":null,"abstract":"<p><strong>Objective: </strong>Insomnia and hypersomnia are sleep conditions associated with significant costs to the healthcare system and society. This study aimed to review the cost-effectiveness evidence of interventions for insomnia and hypersomnia, including psychotherapy, pharmacotherapy, and complementary and alternative medicine (CAM), across the age spectrum.</p><p><strong>Methods: </strong>A systematic search (from inception to 18th February 2025) was conducted in electronic databases (Medline, PsycINFO, CINAHL, Econlit and Embase) and Health Technology Assessment websites. Full economic evaluations and return-on-investment analyses were included if they focused on treatments targeting insomnia or hypersomnia in people aged ≥12 years. The Drummond checklist was used to evaluate the quality of eligible studies. Narrative synthesis was applied to extract study characteristics and economic evaluation outcomes.</p><p><strong>Results: </strong>Twenty-eight studies met the pre-defined criteria, including 26 for adults and older adults with insomnia, two for adolescents with insomnia, and no studies were found for hypersomnia treatment. Cognitive behavioural therapy for insomnia (CBT-I) and pharmacotherapy were likely to be cost-effective interventions for insomnia compared to inactive controls. Digital CBT-I was found to generate healthcare and societal cost savings when compared to face-to-face CBT-I or pharmacotherapy. The cost-effectiveness of CAM interventions is under-researched and remains unclear.</p><p><strong>Conclusion: </strong>Among insomnia interventions, CBT-I has the strongest cost-effectiveness credentials. Future studies should focus on hypersomnia, adolescent insomnia, and comorbid insomnia and related conditions.</p><p><strong>Prospero registration number: </strong>CRD42022343067.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145068911","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":"Correction: Therapy, Pills and Unmet Needs for Financial Reasons: Socioeconomic Inequalities and Inequities in Access to Mental Health Care in Spain 2014–2020","authors":"Rosa M. Urbanos‑Garrido,&nbsp;Laura Agúndez","doi":"10.1007/s40258-025-01003-5","DOIUrl":"10.1007/s40258-025-01003-5","url":null,"abstract":"","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 6","pages":"1131 - 1132"},"PeriodicalIF":3.3,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-025-01003-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063438","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Mixed-Methods Assessment of India's Health Technology Assessment Ecosystem.","authors":"Yashika Chugh, Josyula K Lakshmi, Pankaj Bahuguna, Navneet Kaur, Stephen Jan, Shankar Prinja","doi":"10.1007/s40258-025-00995-4","DOIUrl":"https://doi.org/10.1007/s40258-025-00995-4","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to evaluate the technical quality of health technology assessment (HTA) studies conducted in India. Second, we aim to identify process-related challenges across the life cycle of an HTA from commissioning to policy translation.</p><p><strong>Methods: </strong>A mixed-methods approach was employed to assess HTA studies conducted between 2018 and 2023 conducted by ten regional resource centers. The quantitative assessment involved reviewing 26 HTA reports using the Indian HTA Quality Appraisal Checklist. The qualitative component included semi-structured interviews with staff from six regional resource centers and the Health Technology Assessment in India secretariat to explore the processes of topic selection, study conduct, stakeholder engagement, and evidence to policy translation. Quantitative data were analyzed through scoring and categorization into quality ratings, while qualitative data were analyzed thematically using the framework method.</p><p><strong>Results: </strong>In the quantitative assessment, 14% (n = 3) of studies were found to be of excellent quality, 50% (n = 11) were deemed to be of good quality, 32% (n = 7) were of average quality, and only one (4%) of poor quality. The qualitative findings highlighted limited adherence to guidelines, challenges in framing the topic, and gaps in technical expertise for advanced analyses. Additionally, a high staff turnover, the need for better stakeholder consultations, and strategies to disseminate the evidence were also highlighted.</p><p><strong>Conclusions: </strong>These findings emphasize the need for improvements in adherence to guidelines, transparency in topic selection, and alignment of HTA findings with policy needs. Investments in training, advanced methodology guidance, and systematic communication between researchers and policy makers are crucial to enhancing HTA's impact in India.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005818","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":"A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders.","authors":"Diaz M Prameyllawati, Hester F Lingsma, Marjon H Cnossen, Renske M T Ten Ham","doi":"10.1007/s40258-025-00996-3","DOIUrl":"https://doi.org/10.1007/s40258-025-00996-3","url":null,"abstract":"<p><strong>Objective: </strong>The aim of this review is to identify and assess modelling approaches in published model-based economic evaluations of treatments for individuals with inherited bleeding disorders.</p><p><strong>Methods: </strong>A literature search was performed on seven electronic databases, from database inception until 30 May, 2024. Inclusion criteria were cost-effectiveness or cost-utility analyses using decision-analytic models. The approaches from included models were identified and assessed, and these approaches were compared across bleeding disorders and treatments.</p><p><strong>Results: </strong>This review included a total of 47 decision-analytic models. The identified models primarily evaluated treatments for severe haemophilia A and B. For haemophilia without inhibitors, factor concentrates were the most evaluated intervention (n = 21, 68%), followed by gene therapies (n = 6, 19%) and emicizumab (n = 4, 13%). For haemophilia with inhibitors, assessed interventions included emicizumab (n = 8, 50%), immune tolerance induction with factor concentrates (n = 5, 31%) and bypassing agents (n = 3, 19%). Markov models were often used as a model type (n = 27, 57%), followed by decision trees (n = 9, 19%), Markov decision trees and decision process (n = 5, 11%) and individual-level models (n = 5, 11%). Regardless of the model type, most authors used a lifetime horizon, a 1-year cycle length, and bleeding events-particularly joint bleeds-as key health states of the models.</p><p><strong>Conclusions: </strong>As the reviewed decision-analytic models mainly assessed treatments for severe haemophilia, the identified common approaches may only be generalisable to evaluating these treatments. Further research is required to evaluate their relevance for evaluating treatments of milder forms of haemophilia or other inherited bleeding disorders.</p><p><strong>Systematic review protocol registration: </strong>PROSPERO registration number CRD42023416560.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144939987","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}