Applied Health Economics and Health Policy最新文献

{"title":"Funding Multi-indication Medicines: A Scoping Review of Health Technology Assessment and Reimbursement Frameworks","authors":"Isaiah Luc,&nbsp;Mah Laka,&nbsp;Drew Carter,&nbsp;Tracy Merlin","doi":"10.1007/s40258-025-01020-4","DOIUrl":"10.1007/s40258-025-01020-4","url":null,"abstract":"<div><h3>Background</h3><p>Medicines with multiple indications pose efficiency problems for health technology assessment (HTA). Evaluating indications separately may result in years of duplicative evaluation work and ultimately delay patient access. The price of these medicines is also difficult to determine because the value may vary between the different indications.</p><h3>Objectives</h3><p>A scoping review was conducted to identify HTA and reimbursement frameworks for evaluating and pricing multi-indication medicines for reimbursement decisions and to explore whether there are elements in these frameworks that would have utility if implemented in jurisdictions with well-established HTA systems.</p><h3>Methods</h3><p>PubMed, Embase and Scopus were systematically searched and a targeted search of HTA agency websites was conducted in September 2025. Documents were included in the review if they discussed HTA and reimbursement frameworks concerning multi-indication medicines in high-income countries.</p><h3>Results</h3><p>Sixteen frameworks were identified, describing four main approaches: policy (3), pricing (6), HTA evaluation methods (4) and decision-making (3). Policy approaches included implementing conditional listing processes and streamlining the evaluation of multi-indication medicines. Pricing frameworks including indication-based pricing frameworks and volume-based agreements were reported most frequently.</p><h3>Conclusions</h3><p>While pricing frameworks are commonly used in high-income countries, they do not help reduce duplication of effort in the evaluation and appraisal of these medicines. Policy change allowing for the streamlined incremental evaluation of each new indication may be helpful, although the methodological characteristics of a streamlined HTA have yet to be defined. Subscription-based approaches to purchasing medicines represent another option. Stakeholder engagement is needed to determine the merit and feasibility of different approaches.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"293 - 307"},"PeriodicalIF":3.3,"publicationDate":"2025-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12924833/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145707003","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":"Improving Consumer Engagement in Health Technology Assessment and Decision Making: Lessons from ‘Values in Science’","authors":"Isabella Carnovale,&nbsp;Kevin Elliott,&nbsp;Ian Kerridge,&nbsp;Wendy Lipworth","doi":"10.1007/s40258-025-01018-y","DOIUrl":"10.1007/s40258-025-01018-y","url":null,"abstract":"<div><p>Health technology assessments (HTA) and related decision making by regulators, payers and clinical policymakers (henceforth HTA-DM) are not simply technical processes, but normative ones, shaped by economic, moral, and scientific values. Since these value-laden decisions affect consumers, it is broadly accepted that their values need to be taken into consideration in HTA-DM and that they should be actively engaged in some aspects of these processes. This paper begins by examining current approaches to consumer engagement in HTA-DM. We then identify key challenges inherent in meaningful consumer participation, including procedural obstacles, representation issues, and difficulties in values interpretation. After briefly reviewing existing responses to these challenges, we demonstrate how insights from the philosophy of science—particularly scholarship on values in scientific inquiry—can provide new insights into the challenges of integrating consumers’ values into HTA-DM and offer some strategies for more targeted and effective consumer involvement.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"247 - 260"},"PeriodicalIF":3.3,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145676491","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":"EQ-5D-5L Values and Determinants for a Vietnamese Population","authors":"Nguyen Thanh Nam,&nbsp;Ngo Thi Tam,&nbsp;Nguyen Thi Lan Huong,&nbsp;Nguyen Tat Cuong,&nbsp;Pham Van Dem,&nbsp;Nguyen Hoang Thanh","doi":"10.1007/s40258-025-01019-x","DOIUrl":"10.1007/s40258-025-01019-x","url":null,"abstract":"<div><h3>Objective</h3><p>The aim of this study was to update EQ-5D-5L data on health-related quality of life (HRQoL) in the Vietnamese population using the EQ-5D-5L instrument and examine associated sociodemographic and health-related factors.</p><h3>Method</h3><p>A cross-sectional online survey was conducted between April and May 2025 among 3550 Vietnamese adults aged 18 years and older across three geographic regions. Participants were recruited through convenience and snowball sampling using online platforms. HRQoL was assessed using the EQ-5D-5L index (derived from the Vietnamese value set) and the EQ-VAS. Descriptive statistics, <i>t</i>-tests, ANOVA, and multivariable Tobit regressions were used.</p><h3>Results</h3><p>The mean EQ-5D-5L index score was 0.896 (SD 0.14) and the mean EQ-VAS score was 86.7 (SD 11.8). Significant differences were observed across age groups, employment status, education levels, and chronic disease presence (<i>p</i> &lt; 0.001). Older age, unemployment, low education, and multiple morbidities were independently associated with lower HRQoL scores.</p><h3>Conclusion</h3><p>The findings provide updated data for HRQoL in Vietnam, confirming the impact of age, socioeconomic status, and health conditions. These results are useful for healthcare planning, health economic evaluations, and prioritizing public health interventions in Vietnam.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"407 - 419"},"PeriodicalIF":3.3,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145686835","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 Cost–Utility Analysis of Surgical Site Infection Prevention: Broadening the Scope","authors":"Agi McFarland,&nbsp;Sarkis Manoukian,&nbsp;Helen Mason,&nbsp;Jacqui Reilly","doi":"10.1007/s40258-025-01015-1","DOIUrl":"10.1007/s40258-025-01015-1","url":null,"abstract":"<div><h3>Background</h3><p>Surgical site infections (SSI) present a significant burden in terms of excess length of stay, distress, disability and death. SSI risk and the associated economic burden may be reduced through programmes of infection prevention and control (IPC) although evidence of their cost-effectiveness is limited. Patient-level data from the Evaluation of Cost of Nosocomial Infection (ECONI) study provided opportunity for analysis.</p><h3>Aim</h3><p>The aim of this study is to explore the cost-effectiveness of enhanced SSI prevention in terms of costs and quality of life for adult surgical patients in the UK National Health Service (NHS).</p><h3>Methods</h3><p>A Monte Carlo microsimulation model was built to evaluate the cost-effectiveness [cost per quality-adjusted life years (QALY)] of SSI prevention in three surgery types (coronary artery bypass graft, hip arthroplasty and caesarean section) by comparing an enhanced programme of SSI IPC to current clinical sequelae over 1 month and 1 year in the NHS. Uncertainty was explored through probabilistic sensitivity analysis, scenario analysis and the use of alternative utility valuation sets.</p><h3>Results</h3><p>In most surgeries and time points, enhanced IPC was associated with lower costs and higher QALY gains than current SSI IPC measures. The results were sensitive to utility valuation methods used. Scenario analyses identified factors relating to SSI rate, IPC programme efficacy and cost resulted in strategy dominance changes over all three surgery types.</p><h3>Conclusions</h3><p>Enhanced programmes of IPC for SSIs may deliver improved health outcomes at a lower cost; however, this is not consistent across all surgery types and timepoints and is sensitive to various factors.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"421 - 435"},"PeriodicalIF":3.3,"publicationDate":"2025-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145676531","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":"Preferences for Digital Mental Health Services in Urban China: A Discrete Choice Experiment","authors":"Defang Xiang,&nbsp;Yuling Qian,&nbsp;Di Liang","doi":"10.1007/s40258-025-01014-2","DOIUrl":"10.1007/s40258-025-01014-2","url":null,"abstract":"<div><h3>Background</h3><p>Despite the high prevalence of mental disorders in China, most patients do not utilize mental health services. Digital mental health services offer a potential solution, but their full potential remains untapped. Understanding consumer preferences is key to tailoring these services and boosting acceptance and adoption.</p><h3>Objective</h3><p>This study aims to elicit preferences for digital mental health services in urban China.</p><h3>Methods</h3><p>A discrete choice experiment was conducted with 867 participants, including both current and potential consumers of mental health services, recruited from urban areas in China. Under the guidance of the Theory of Planned Behavior, attributes relevant to digital mental health services were identified through a multi-stage process including a literature review and in-depth semi-structured interviews with 30 consumers and potential consumers of mental health services, and five professionals from Internet-based healthcare and online psychological counseling fields. Six attributes were identified: (1) professional engagement; (2) intervention type; (3) endorser/recommender; (4) mode of content delivery; (5) monthly costs; and (6) privacy policy. Recruited via a market research company, participants completed a discrete choice experiment survey with 11 option sets online. Participants’ choices were analyzed with a mixed logit model and further stratified by experience with mental health services, sex, education, income, age, and risk for mental health conditions. The relative importance score was calculated to interpret the results of the discrete choice experiment. Additionally, the willingness-to-pay approach was used to estimate the monetary value of each attribute level.</p><h3>Results</h3><p>The most important influence on the choice of digital mental health services was the level of professional engagement (<i>β</i> = 2.470, <i>β</i> = 1.011), followed by the customized and adaptive intervention (<i>β</i> = 0.508) and data usage solely used for iterative product upgrades (<i>β</i> = 0.492). The relative importance score also revealed the most important attributes for consumers were professional engagement, intervention type, and privacy policy. Considering the professional engagement attribute, consumers were willing to pay an additional ¥3916.5 per month for services provided by mental health professionals throughout. Preferences diverged based on participant characteristics including experience with mental health services, sex, and education.</p><h3>Conclusions</h3><p>Our results indicated a consistent preference for services provided by mental health professionals across demographics and regardless of previous mental health service experience. We also found a preference for personalized services, highlighting a shift towards customization in healthcare and emphasizing the need to customize digital mental health services.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"325 - 341"},"PeriodicalIF":3.3,"publicationDate":"2025-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145538885","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":"Data-Related and Methodological Challenges in Assessing the Cost-Effectiveness of Traditional and Genomic Newborn Screening Programs","authors":"Ramesh Lamsal,&nbsp;Hadley Stevens Smith,&nbsp;Scott D. Grosse","doi":"10.1007/s40258-025-01013-3","DOIUrl":"10.1007/s40258-025-01013-3","url":null,"abstract":"<div><p>As newborn screening (NBS) programs consider adding conditions, policymakers would benefit from evidence showing whether early screening offers greater economic and health benefits than standard clinical diagnosis and treatment. However, economic evaluations have not been consistently used to guide decisions on expansion of NBS programs globally. This narrative review discusses data-related and methodological challenges in conducting cost-effectiveness analyses (CEAs) of NBS programs including (1) difficulties in identifying appropriate comparators and related data, (2) gaps in understanding and limited data on health outcomes and costs of care among children with newborn-screened conditions who received effective treatment after clinical diagnosis without screening, (3) lack of data on short- and longer-term costs and outcomes for affected individuals identified through screening, (4) challenges in distinguishing the effects of screening from improvements in treatment over time, and (5) limited data on costs outside of the healthcare system, limiting the ability to conduct evaluations from the societal perspective. Advancements in genomic technologies, such as genomic sequencing and gene-targeted therapies, are expanding the number of conditions that are potential targets for NBS. Genomic NBS poses additional challenges for CEAs that stem from the ability to simultaneously identify hundreds or even thousands of conditions and limitations of knowledge of genotype-phenotype associations. The data-related challenges discussed herein may encourage cross-disciplinary collaboration to improve data collection. Addressing these methodological issues is imperative for generating reliable evidence on the costs and benefits of NBS program expansion, thereby supporting informed policymaking on adding new conditions to NBS programs.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"271 - 286"},"PeriodicalIF":3.3,"publicationDate":"2025-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145494242","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-Utility Analysis of TAVI Versus Surgery in Low-Risk Patients with Severe Aortic Stenosis in the UK","authors":"Nick Curzen,&nbsp;Pascal Candolfi,&nbsp;Philip MacCarthy,&nbsp;Clinton T. Lloyd,&nbsp;Muhammad Aetesam-Ur-Rahman,&nbsp;Tom Bromilow,&nbsp;Valentina Sellitto,&nbsp;Daniel J. Blackman","doi":"10.1007/s40258-025-01012-4","DOIUrl":"10.1007/s40258-025-01012-4","url":null,"abstract":"<div><h3>Background and Objective</h3><p>European guidelines recommend transcatheter aortic valve implantation (TAVI; class IA) for symptomatic severe aortic stenosis (sSAS) in patients aged ≥ 75 years, if a transfemoral approach is possible. Recent cost-utility analyses based on the PARTNER 3 trial have suggested that TAVI with the SAPIEN 3 device is cost effective versus surgical aortic valve replacement (SAVR) in patients at low risk of surgical mortality in several European countries. This cost-utility analysis compares TAVI with SAPIEN 3 versus SAVR in patients with sSAS at low risk of surgical mortality from the UK healthcare system perspective, using 5-year PARTNER 3 trial data.</p><h3>Methods</h3><p>A two-stage, Markov-based, cost-utility analysis was performed using published and validated methodology to estimate changes in both direct healthcare costs and health-related quality of life for TAVI with SAPIEN 3 versus SAVR in patients with sSAS at low surgical risk from the perspective of the UK healthcare system. The model used a lifetime horizon with a 3.5% yearly discounting factor. Uncertainty was addressed using deterministic and probabilistic sensitivity analyses.</p><h3>Results</h3><p>Transcatheter aortic valve implantation with SAPIEN 3 generated an incremental health benefit of 0.47 (90% credible interval 0.35–0.60) quality-adjusted life-years per patient compared with SAVR, at an increased cost of £7999 (£852–£15,035 90% credible interval) per patient over a lifetime horizon (incremental cost-effectiveness ratio: £16,979 per quality-adjusted life-year gained). Transcatheter aortic valve implantation has a 63–90% probability of cost effectiveness based on a £20,000–£30,000 willingness-to-pay threshold. Transcatheter aortic valve implantation remained cost effective across most deterministic sensitivity analyses, confirming the relative robustness of the results.</p><h3>Conclusions</h3><p>Transcatheter aortic valve implantation with SAPIEN 3 has a 63–90% probability of being cost effective compared with SAVR for low-surgical-risk patients with sSAS. These findings may inform policy decision making in the management of this patient group.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"365 - 378"},"PeriodicalIF":3.3,"publicationDate":"2025-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12924842/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145470511","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":"Budget Impact Analysis of Implementing C-Reactive Protein Point-of-Care Testing to Aid Antibiotic Prescribing in Belgian Primary Care","authors":"Salima Azahaf,&nbsp;Erinn D’hulster,&nbsp;Margo Van Genechten,&nbsp;Thaddee Ding,&nbsp;Tibo Wynant,&nbsp;Jeroen Luyten,&nbsp;Jan Y. Verbakel","doi":"10.1007/s40258-025-01011-5","DOIUrl":"10.1007/s40258-025-01011-5","url":null,"abstract":"<div><h3>Background</h3><p>The inappropriate use of antibiotics is a key driver of antimicrobial resistance (AMR) and can lead to various adverse side effects. C-reactive protein (CRP) point-of-care testing (POCT) in primary care has shown potential as an effective strategy to reduce inappropriate antibiotic prescribing. This study evaluates the budget impact of introducing CRP POCT in Belgian primary care over a 5-year time horizon.</p><h3>Methods</h3><p>A decision tree model was developed to compare the budget impact of implementing CRP POCT in primary care versus usual care (without CRP POCT) based on 712,102 acute cough patients annually. Cost and probability parameters were derived through literature review and primary data collection. The robustness of the findings was assessed using univariate and probabilistic sensitivity analyses. Additionally, alternative scenarios were explored by modifying key assumptions in the base-case scenario to evaluate the effect on the results.</p><h3>Results</h3><p>Over a 5-year period, implementing CRP POCT in Belgian primary care for the management of 712,102 acute cough patients annually is estimated to result in an incremental cost of approximately €12.8 million (95% credible interval: 12,699,459 to 12,924,898), primarily driven by capital expenditures for analysers and CRP test expenses. Scenario analyses showed substantial variation in budget impact depending on implementation choices, ranging from €13.6 million to €39.6 million, with the highest costs when general practitioners’ (GPs’) compensation was included. Reduced inappropriate antibiotic use, adverse side effects, and AMR rates would decrease costs, with these savings expected to grow over time as CRP POCT becomes more widely adopted. However, the proportion of these savings relative to total expenditure is limited.</p><h3>Conclusion</h3><p>The implementation of CRP POCT entails a significant financial investment. Our scenario analyses indicate a substantial increase in budget impact when GPs’ compensation was included. However, a well-designed implementation strategy with an appropriate GP compensation structure may be crucial to avoid inefficiencies from over- or underuse of CRP testing and achieve the anticipated health benefits.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"393 - 405"},"PeriodicalIF":3.3,"publicationDate":"2025-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145470465","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":"Which Factors are Holding up the Integration of the Environmental Impact of Health Technologies in Health Technology Assessment? Insights from a Multi-Stakeholder Interview Study","authors":"Vittoria Ardito,&nbsp;Helen Banks,&nbsp;Rosanna Tarricone","doi":"10.1007/s40258-025-01010-6","DOIUrl":"10.1007/s40258-025-01010-6","url":null,"abstract":"<div><h3>Objectives</h3><p>The integration of environmental impact into health technology assessment (HTA) is increasingly discussed, but its practical implementation remains uncertain. This study explores multi-stakeholder perspectives on the progress, challenges, and equity implications of incorporating environmental considerations into HTA.</p><h3>Methods</h3><p>As part of the Horizon Europe project HI-PRIX (ID: 101095593), 13 semi-structured interviews were conducted with experts across academia, HTA agencies, regulatory bodies, and industry in seven European countries. Interview transcripts were analyzed through a thematic analysis conducted manually, using a grounded-theory approach. From the interviews, 101 quotes corresponding to 37 codes were extracted and organized in eight analytical themes.</p><h3>Results</h3><p>Stakeholders agreed that environmental impact is relevant but under-addressed in HTA due to fragmented data, methodological uncertainty, and lack of regulatory guidance. Many emphasized that a health system perspective is too narrow, where methods conventionally used to assess health technologies are not necessarily appropriate to also assess environmental impact, calling for a societal approach that reflects lifecycle impacts. Equity consequences linked to the inclusion of environmental impact of health technologies in HTA were specifically recognized as both reasons for its incorporation and potential barriers. At this stage, procurement was identified as a practical approach to account for the environmental dimension.</p><h3>Conclusions</h3><p>Integrating environmental impact into HTA will require shifting toward a societal perspective, stronger collaboration across stakeholders, and development of EU-level guidance, expert panels, and standardized methodologies. Methodologies incorporating equity into economic analyses should be explored as a potential direction for future research. Until frameworks mature, environmental impact is likely to remain informative but not yet decisive in HTA.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"379 - 391"},"PeriodicalIF":3.3,"publicationDate":"2025-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12924823/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145386132","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":"Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations","authors":"Adéla Bártová,&nbsp;Jiří Samek,&nbsp;Vera Pinheiro,&nbsp;Barbora Říhová,&nbsp;João Vasco Santos","doi":"10.1007/s40258-025-01008-0","DOIUrl":"10.1007/s40258-025-01008-0","url":null,"abstract":"<div><h3>Objectives</h3><p>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><h3>Methods</h3><p>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><h3>Results</h3><p>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><h3>Conclusion</h3><p>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></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"24 2","pages":"343 - 364"},"PeriodicalIF":3.3,"publicationDate":"2025-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12924838/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}