Journal of market access & health policy最新文献

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Role of General Practitioners in European Health Technology Assessment, EU-HTA Innovation in Oncology as an Example. 全科医生在欧洲卫生技术评估中的作用,以欧盟- hta在肿瘤学中的创新为例。
Journal of market access & health policy Pub Date : 2026-09-21 DOI: 10.3390/jmahp14030057
Daniel Widmer, Hermenegildo Marcos Carrera, Jörg Ruof, Gunasekara Vidanamestrige Chamath Fernando, Patrick Ouvrard, Marie-Christine Bonnamour, Mary McCarthy, Shrikant Atreya
{"title":"Role of General Practitioners in European Health Technology Assessment, EU-HTA Innovation in Oncology as an Example.","authors":"Daniel Widmer, Hermenegildo Marcos Carrera, Jörg Ruof, Gunasekara Vidanamestrige Chamath Fernando, Patrick Ouvrard, Marie-Christine Bonnamour, Mary McCarthy, Shrikant Atreya","doi":"10.3390/jmahp14030057","DOIUrl":"10.3390/jmahp14030057","url":null,"abstract":"<p><strong>Introduction: </strong>Different from established clinical guideline workstreams, the participation of general practitioners (GPs) in the health technology assessment (HTA) process at the European level is new. There is a research gap in this domain. The European Union of General Practitioners (UEMO) is the political association of GPs in Europe and has been collaborating as a stakeholder in the creation of the European Regulation on HTA since 2017. The aim of our research is to provide a framework for GPs'role in EU HTA.</p><p><strong>Methods: </strong>Mixed methods: questionnaire and focus groups.</p><p><strong>Participants: </strong>Representatives of national medical/GP associations of 17 Member States of the EU and five EU non-member countries.</p><p><strong>Results: </strong>Most respondents supported GP involvement in the development of clinical recommendations relevant to general practice. Respondents emphasized that UEMO must turn to WONCA-Europe, the academic society of GPs, when scientific input is required. Among the ten priority topics for HTA in oncology, four emerged as most relevant for the GPs: 1. Conscientious prescription of interventions considering over- and under-medicalization, 2. establishing a system for continuity of care integrating palliative care, 3. ensuring evidence-based medicine, and 4. promoting collaborative care and considering workload.</p><p><strong>Discussion and conclusions: </strong>The GP can contribute to the debate by defending these four priorities. Presenting the difficulties in this debate can contribute to a better understanding of the implementation of new technology in the real world.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608814/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Parent-Reported Satisfaction, Perceived Child Acceptance, and Ease of Administration of a Strawberry-Flavoured Ibuprofen Suspension for Children: A Cross-Sectional Survey of Experienced Users in Germany. 家长报告的满意度,感知到的儿童接受度,以及儿童草莓味布洛芬悬浮液的管理便利性:一项对德国有经验用户的横断面调查。
Journal of market access & health policy Pub Date : 2026-09-18 DOI: 10.3390/jmahp14030056
Kerstin Weber, Spasenija Bradic, Gábor Kertes, Marcel Konrad, Karel Kostev
{"title":"Parent-Reported Satisfaction, Perceived Child Acceptance, and Ease of Administration of a Strawberry-Flavoured Ibuprofen Suspension for Children: A Cross-Sectional Survey of Experienced Users in Germany.","authors":"Kerstin Weber, Spasenija Bradic, Gábor Kertes, Marcel Konrad, Karel Kostev","doi":"10.3390/jmahp14030056","DOIUrl":"10.3390/jmahp14030056","url":null,"abstract":"<p><strong>Background: </strong>Palatability and ease of administration are recognised as key determinants of medication adherence in paediatric populations. Oral ibuprofen suspensions are among the most commonly used analgesic and antipyretic medications for children in Germany, yet parental and parent-perceived child perspectives on these formulation attributes remain underreported for the German market.</p><p><strong>Methods: </strong>A cross-sectional, online market-research survey, commissioned to substantiate potential product communication claims, was conducted among 1005 parents residing in Germany, each of whom had administered a strawberry-flavoured ibuprofen suspension (Nurofen Junior Fever and Pain Suspension Strawberry, Reckitt Benckiser) to at least one child aged 3 months to 12 years within the preceding 12 months. Closed-ended items assessed overall satisfaction, willingness to recommend, parent-perceived child acceptance, perceived ease of administration, and taste satisfaction; all product-specific statements (Q17 and Q18) used a binary agree/do-not-agree format without a neutral option.</p><p><strong>Results: </strong>Overall satisfaction with the product was reported by 99% of respondents (995/1005), and 98% (985/1005) stated they would recommend it to other parents. Agreement rates exceeded 90% for all primary claim statements (Q18): easy and safe to administer (96%), satisfied with how well the child takes the medicine (92%), well accepted by child (92%), satisfied with flavour (92%), good feeling child takes medicine without problems (91%), child finds taste pleasant (90%), child likes the taste (90%), and child takes it without problems (88%). For comparative acceptance statements (Q17), agreement ranged from 83% to 93%. Fever was the primary indication for use (91%), and the product was used a mean of 3.3 times per year.</p><p><strong>Conclusions: </strong>These findings provide consumer-reported, real-world survey data indicating that, among surveyed parents with recent personal experience of using the product, reported satisfaction and perceived child acceptance were high. Because participation required experience with the product within the previous 12 months, while allowing concomitant experience with other paediatric products, and because fever and pain episodes commonly occur more than once over a year in children, the study relied on parent-reported outcomes. The study was designed as a descriptive survey of user experiences and was not intended as a comparative evaluation; accordingly, no comparator was included. The findings should therefore be interpreted as descriptive of this study population and cannot be used to infer comparative performance, improved adherence, or better clinical outcomes; they may, however, help identify taste and ease of administration as candidate domains of paediatric acceptability to be examined in future, more rigorously designed studies.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608591/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042591","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Post-Authorisation Maze: Mapping of European Pharmaceutical Market Access Pathways in the Early JCA Era. 授权后的迷宫:早期JCA时代欧洲药品市场准入路径的映射。
Journal of market access & health policy Pub Date : 2026-09-17 DOI: 10.3390/jmahp14030055
Lisa-Maria Hagemann, Karolin Eberle, Jana Maurer, Alexandra Carls, Andreas D Meid, Eva-Maria Reuter
{"title":"The Post-Authorisation Maze: Mapping of European Pharmaceutical Market Access Pathways in the Early JCA Era.","authors":"Lisa-Maria Hagemann, Karolin Eberle, Jana Maurer, Alexandra Carls, Andreas D Meid, Eva-Maria Reuter","doi":"10.3390/jmahp14030055","DOIUrl":"10.3390/jmahp14030055","url":null,"abstract":"<p><p>The Regulation (EU) 2021/2282 on health technology assessment (HTAR) harmonises clinical evidence assessment across Europe. However, national appraisal, pricing, and reimbursement processes remain divergent and could affect the efficiency of decision-making and patient access. Here we mapped the sequencing of national assessment, pricing, and reimbursement steps across 30 European countries. National pathways from marketing authorisation to reimbursed access were systematically identified and coded as categorical process sequences. Inpatient and outpatient pathways were analysed separately. Pathway dissimilarities were calculated using Optimal Matching and grouped by hierarchical clustering. We further explored whether clusters differed with regard to country-level indicators, including medicine availability, average time to access, gross domestic product, and healthcare expenditure. We found substantial procedural heterogeneity across countries in both inpatient and outpatient settings. Nevertheless, recurring pathway structures were identified. Across settings, the dominant distinction was between assessment-led pathways, in which evidence assessment precedes pricing and reimbursement steps, and pricing-led pathways, in which price formation occurs prior to assessment and reimbursement-related steps. Cluster membership was not associated with any country-level indicators. Our findings provide policymakers, agencies, and industry with a baseline for understanding national procedural heterogeneity and for evaluating how market access pathways evolve as the JCA is implemented.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608677/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Shelf Life of Epinephrine Auto-Injectors in Canada: Implications for Renewal Frequency and Patient Burden. 加拿大肾上腺素自动注射器的实际保质期:更新频率和患者负担的影响。
Journal of market access & health policy Pub Date : 2026-09-14 DOI: 10.3390/jmahp14030054
John Papastergiou, Mille Vang Lybech, Christoffer Mertz, Anne Danø, Jess Finney, David Grabowski
{"title":"Real-World Shelf Life of Epinephrine Auto-Injectors in Canada: Implications for Renewal Frequency and Patient Burden.","authors":"John Papastergiou, Mille Vang Lybech, Christoffer Mertz, Anne Danø, Jess Finney, David Grabowski","doi":"10.3390/jmahp14030054","DOIUrl":"10.3390/jmahp14030054","url":null,"abstract":"<p><p>Although patients in Canada at risk of anaphylaxis are recommended to maintain access to two in-date epinephrine auto-injectors (EAIs) at all times, underuse during emergencies remains common. Multiple factors contribute to this gap, but the impact of real-world shelf life has been understudied. This study examines the remaining in-pharmacy shelf life of EAIs in Canada and considers its potential implications for patient burden, renewal frequency, and anaphylaxis preparedness. In this cross-sectional study, 50 licensed Canadian pharmacists from chain, independent, grocery/mass merchandise, and hospital pharmacies completed an online questionnaire and assessed EAI stock. Participating pharmacists verified all EAI stock on site, including batch numbers and expiry dates, and reported patient disposal practices. Of the 50 participating pharmacists, 49 reported 411 EAI devices across 98 batches. The mean in-pharmacy shelf life at dispensing was 12.8 months, with 47.4% of devices having 12 months or less remaining shelf life. Disposal practices varied and expiry was the most cited reason for disposal. Shortened in-pharmacy shelf life may increase renewal frequency, cost, waste, and the risk that patients carry expired devices or no device during anaphylaxis. Longer-shelf-life epinephrine treatment options or improved distribution chain practices may help reduce patient burden and enhance anaphylaxis preparedness in Canada.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608879/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Analysis of Factors Influencing Drug Prices Differently in Official Drug Pricing and Cost-Effectiveness Evaluation. 官方药品定价差异影响因素分析及成本-效果评价。
Journal of market access & health policy Pub Date : 2026-08-31 DOI: 10.3390/jmahp14030053
Mariko Hara, Hiroshi Nakamura
{"title":"Analysis of Factors Influencing Drug Prices Differently in Official Drug Pricing and Cost-Effectiveness Evaluation.","authors":"Mariko Hara, Hiroshi Nakamura","doi":"10.3390/jmahp14030053","DOIUrl":"10.3390/jmahp14030053","url":null,"abstract":"<p><strong>Background: </strong>In Japan, official prices of pharmaceuticals have been determined by efficacy and safety. In 2019, Cost-Effectiveness Evaluation (CEE) was introduced to adjust these prices according to Incremental Cost-Effectiveness Ratios (ICERs).</p><p><strong>Objectives: </strong>To analyze the differences in the factors influencing drug price between the initial official drug pricing and subsequent price adjustments based on CEE.</p><p><strong>Methods: </strong>Twenty-three drugs evaluated in Japan between 2019 and May 2025 were descriptively analyzed using public reports on official drug pricing and CEE.</p><p><strong>Results: </strong>Four differences were identified: (1) Official initial drug pricing grants premiums for certain characteristics that are not directly associated with improvements in Health-Related Quality of Life (HRQoL), whereas such premiums are not considered in CEE. (2) Official drug pricing limits comparators to existing drugs, whereas CEE did not impose such restrictions. (3) Official drug pricing excludes older drugs as comparators to reward innovation, whereas they were not excluded in CEE. (4) CEE requires sufficient data for CE analysis, such as clinical trial or real-world data, for comparators, unlike official drug pricing. Factors (1) and (3) were most frequent.</p><p><strong>Conclusion: </strong>Reducing these differences and maintaining consistent innovation evaluations may enhance price predictability and support sustainable pharmaceutical investments in the Japanese market.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042624","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Procedural Theatre: EU-HTA Between Ritual and Purpose-Why the Buyer's Audit Has Outgrown the Transaction. 程序性戏剧:仪式与目的之间的EU-HTA——为什么买方的审计已经超越了交易。
Journal of market access & health policy Pub Date : 2026-08-29 DOI: 10.3390/jmahp14030052
Mondher Toumi, Maarten Jacobus Postma, Bruno Falissard, Frank-Ulrich Fricke, Stefano Capri, Jürgen Wasem, Steven Simoens, Renato Bernardini, Malgorzata Wojtal, Anna Kapuśniak, Oriol Sola Morales, Laurent Boyer, Jaime Espin, Pascal Auquier
{"title":"Procedural Theatre: EU-HTA Between Ritual and Purpose-Why the Buyer's Audit Has Outgrown the Transaction.","authors":"Mondher Toumi, Maarten Jacobus Postma, Bruno Falissard, Frank-Ulrich Fricke, Stefano Capri, Jürgen Wasem, Steven Simoens, Renato Bernardini, Malgorzata Wojtal, Anna Kapuśniak, Oriol Sola Morales, Laurent Boyer, Jaime Espin, Pascal Auquier","doi":"10.3390/jmahp14030052","DOIUrl":"10.3390/jmahp14030052","url":null,"abstract":"<p><p>Health technology assessment is a discipline that has developed, over five decades, an elaborate scientific and institutional identity centered on the evaluation of clinical evidence, comparative effectiveness, and economic value [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Model-Based Public-Payer Investment Appraisal of a National Home Hemodialysis Program in Greece: A Net Present Value Analysis. 基于模型的公共支付者对希腊国家家庭血液透析项目的投资评估:净现值分析。
Journal of market access & health policy Pub Date : 2026-08-27 DOI: 10.3390/jmahp14030051
Vasileios Zavvos, John Fanourgiakis, Michael A Talias, Christos Iatrou, Christos Ntais
{"title":"A Model-Based Public-Payer Investment Appraisal of a National Home Hemodialysis Program in Greece: A Net Present Value Analysis.","authors":"Vasileios Zavvos, John Fanourgiakis, Michael A Talias, Christos Iatrou, Christos Ntais","doi":"10.3390/jmahp14030051","DOIUrl":"10.3390/jmahp14030051","url":null,"abstract":"<p><strong>Background: </strong>In-center hemodialysis is the dominant kidney replacement therapy modality in Greece and generates substantial recurring expenditure for the public payer. Home hemodialysis is not currently implemented at national scale, but it may reduce long-term public expenditure if early investment in training capacity, equipment and home support is recovered over time.</p><p><strong>Objective: </strong>To evaluate, from the Greek public-payer perspective, the discounted budget impact and net present value (NPV) of implementing a national home hemodialysis program for 300 patients.</p><p><strong>Methods: </strong>We developed a deterministic investment-appraisal model comparing gradual implementation of home hemodialysis with continued in-center hemodialysis for the same projected cohort over 10 years. The in-center comparator was informed by a 2022 Greek patient-level micro-costing study. Home hemodialysis expenditure was constructed from explicit patient-flow equations, resource quantities, unit costs, capital purchases and hospital-tariff offsets. Annual incremental savings were discounted at 3% in the base case. Alternative discount rates, deterministic one-way sensitivity analyses and program-scale scenarios were examined. Fiscal benefit-cost ratio (BCR) and return on investment (ROI) were also calculated.</p><p><strong>Results: </strong>Undiscounted 10-year public expenditure was EUR 69,681,804 for home hemodialysis and EUR 86,700,267 for continued in-center hemodialysis, yielding savings of EUR 17,018,463. During the first 5 years, the program required EUR 1,724,012 in additional expenditure. At a 3% discount rate, NPV was EUR 12,696,564, the fiscal BCR was 2.89, fiscal ROI was 189.3% and discounted payback occurred during year 6. NPV remained positive at 5% (EUR 10,391,382) and across all tested one-way scenarios (range EUR 706,179 to EUR 24,686,948).</p><p><strong>Conclusions: </strong>The modeled national home hemodialysis program generated a positive 10-year public-payer NPV under the base-case and tested sensitivity assumptions. A positive NPV is not, however, a formal Greek health-system decision rule and does not capture health outcomes, patient and family costs, or equity. The findings support staged pilot implementation and prospective collection of Greek real-world data before wider rollout.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13608129/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042615","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
From Joint Clinical Assessment to National Decisions: EU HTA Implementation and Implications for Patient Access. 从联合临床评估到国家决策:欧盟HTA的实施和对患者获取的影响。
Journal of market access & health policy Pub Date : 2026-08-19 DOI: 10.3390/jmahp14030050
Kalpana D'Oca, Ada Adriano, Eline Darquennes, Natalie Steck
{"title":"From Joint Clinical Assessment to National Decisions: EU HTA Implementation and Implications for Patient Access.","authors":"Kalpana D'Oca, Ada Adriano, Eline Darquennes, Natalie Steck","doi":"10.3390/jmahp14030050","DOIUrl":"10.3390/jmahp14030050","url":null,"abstract":"<p><p>The introduction of Joint Clinical Assessments (JCAs) under the European Union Health Technology Assessment Regulation (EU HTAR) represents a major structural reform aimed at reducing fragmentation and duplication in clinical evidence assessment across Member States. While JCAs are intended to support national health technology assessment (HTA) processes through a common EU-level clinical evaluation, their integration into established national HTA and reimbursement systems remains untested at this early stage of implementation. This paper reports findings from two surveys conducted in 2025 among local affiliate representatives with expertise in relevant HTA and market access activities across 25 European countries (comprising 24 EU Member States and Norway), capturing early national perspectives on key aspects of JCA implementation. The surveys explored anticipated impact on national reimbursement timelines, current opportunities for early HTA advice and PICO input, approaches to handling post-JCA data availability, and nationally prioritised policy and implementation issues. Results indicate mixed expectations regarding the potential impact of JCA on reimbursement timelines, with perceived risks of delay largely viewed as possibly transitional and mitigable through national process adaptation. Respondents emphasised the importance of transparency in PICO consolidation and clarity on the use of JCA reports in national appraisals. Overall, the findings suggest that JCA may represent a reconfiguration rather than a centralisation of evidence assessment, with national influence exercised earlier in the assessment lifecycle. These insights provide policy-relevant input to inform ongoing EU HTA implementation and future refinement of the JCA framework.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148833984","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Health-Related Quality of Life of Patients with Epidermolysis bullosa and Carers Using a Time Trade-Off Approach in the UK General Population to Elicit Utilities for Health Technology Assessment. 大疱性表皮松解症患者和护理人员的健康相关生活质量在英国普通人群中使用时间权衡方法,以引出健康技术评估的效用。
Journal of market access & health policy Pub Date : 2026-08-11 DOI: 10.3390/jmahp14030048
Luke Stainer, George Morgan, Thomas Snell, Claire Mather, Sagair Hussain, Keith Tolley
{"title":"Health-Related Quality of Life of Patients with <i>Epidermolysis bullosa</i> and Carers Using a Time Trade-Off Approach in the UK General Population to Elicit Utilities for Health Technology Assessment.","authors":"Luke Stainer, George Morgan, Thomas Snell, Claire Mather, Sagair Hussain, Keith Tolley","doi":"10.3390/jmahp14030048","DOIUrl":"10.3390/jmahp14030048","url":null,"abstract":"<p><strong>Background: </strong>Epidermolysis bullosa (EB) is a complex group of rare, inherited skin disorders that blister easily, imposing substantial burden on patients and carers. There is a lack of published evidence in the UK quantifying health-related quality of life in EB patients and carers as utility values suitable for use in Health Technology Assessment (HTA).</p><p><strong>Methods: </strong>A time trade-off (TTO) study was conducted with 120 UK general public members to estimate utility values for EB patient and primary carer vignettes depicting varying EB severity. An exploratory sub-study involving six carer and clinical experts was conducted to estimate utility values of second carers.</p><p><strong>Results: </strong>115 general public participants were included in the final TTO analysis. Mean utility values declined as disease severity increased, from 0.82 in the least severe state, to 0.54 in the most severe. Carer utilities were higher than the equivalent patient health states, but with a similar pattern of decreasing utility with increasing EB severity, ranging from 0.85 to 0.64. The secondary carer sub-study, in six respondents estimated a second care burden factor relative to the primary carer of 77%.</p><p><strong>Conclusions: </strong>The utilities estimated for both EB patients and their carers have relevance for future economic evaluations and HTA of new EB therapies.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13511220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834047","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment Preferences in Acute Allergic Reactions-How Can the Context Impact the Preference for a Corticosteroid Mouth Film Versus Conventional Tablets? 急性过敏反应的治疗偏好-环境如何影响皮质类固醇口腔膜与常规片剂的偏好?
Journal of market access & health policy Pub Date : 2026-08-11 DOI: 10.3390/jmahp14030049
Andrea Karadak, Karin Wahlberg, Göran Tornling, James Kereki, Leif Bjermer, Jonas Hjelmgren
{"title":"Treatment Preferences in Acute Allergic Reactions-How Can the Context Impact the Preference for a Corticosteroid Mouth Film Versus Conventional Tablets?","authors":"Andrea Karadak, Karin Wahlberg, Göran Tornling, James Kereki, Leif Bjermer, Jonas Hjelmgren","doi":"10.3390/jmahp14030049","DOIUrl":"10.3390/jmahp14030049","url":null,"abstract":"<p><strong>Background: </strong>The objective was to assess how preferences for a self-dissolving dexamethasone mouth film versus conventional tablets for acute allergic reactions may vary according to population characteristics and the level of outcome knowledge for the intervention (i.e., ex ante, interim, and ex post).</p><p><strong>Methods: </strong>We used preference data from two studies: a questionnaire study with a choice between mouth film and tablets based on a description of the mouth film (ex ante), and a clinical trial with ratings of accessibility and perceived security when the mouth film was available (interim) or used for acute allergic reactions (ex post). Associations between preferences and participant characteristics (demographics and clinical history) were analyzed using logistic regression models.</p><p><strong>Results: </strong>Preference for the mouth film over tablets remained strong as outcome information increased from ex ante to ex post and was consistent across study populations. Higher relative preference was associated with female sex, longer disease duration, older age, and prior severe reactions, whereas satisfaction with current treatment and regular allergy check-ups were linked to weaker relative preference.</p><p><strong>Conclusions: </strong>Preference for the mouth film over tablets was stable, but the degree of preference may depend on characteristics that influence sensitivity to treatment challenges in acute situations.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13511562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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