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Validation of the Cancer-Specific Preference-Based Measure EORTC QLU-C10D against the Generic Instruments EQ-5D-5L and SF-6Dv2 in a Prospectively Collected Sample of Patients with Cancer in Austria and France. 在奥地利和法国前瞻性癌症患者样本中验证基于癌症特异性偏好的EORTC qu - c10d对通用仪器EQ-5D-5L和SF-6Dv2的有效性。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-04-27 DOI: 10.1007/s40273-025-01501-3
Micha J Pilz, Simone Seyringer, Virginie Nerich, Madeleine T King, Richard Norman, Eva M Gamper
{"title":"Validation of the Cancer-Specific Preference-Based Measure EORTC QLU-C10D against the Generic Instruments EQ-5D-5L and SF-6Dv2 in a Prospectively Collected Sample of Patients with Cancer in Austria and France.","authors":"Micha J Pilz, Simone Seyringer, Virginie Nerich, Madeleine T King, Richard Norman, Eva M Gamper","doi":"10.1007/s40273-025-01501-3","DOIUrl":"10.1007/s40273-025-01501-3","url":null,"abstract":"<p><strong>Background: </strong>The Quality of Life Utility - Core 10 Dimensions (QLU-C10D) is a disease-specific preference-based measure (PBM) designed to obtain health state utility values from patients with cancer. Previously, satisfactory psychometric properties were established from retrospective trial analyses using clinical anchors. This study aimed to validate the QLU-C10D against two generic PBMs in a prospective sample of Austrian and French patients with cancer using patient-reported anchors.</p><p><strong>Methods: </strong>Patients completed the European Organisation of Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30), EQ-5D-5L and Short Form 36 (SF-36) at study baseline (any time during anti-cancer treatment) plus a follow-up assessment 3-6 months later. Sociodemographic and clinical characteristics were assessed. QLU-C10D and SF-6Dv2 utilities were calculated from QLQ-C30 and SF-36 data, respectively. German, French and UK value sets were applied for all three PBMs. Floor and ceiling effects were assessed. Known-group validity (independent t-test) and responsiveness (paired t-tests) were assessed respectively by ability to detect health status differences and changes over time according to patient-rated overall quality of life/health perception assessed by the QLQ-C30 Global Health Status scale, the EQ-5D-5L VAS and the SF-36 General Health scale.</p><p><strong>Results: </strong>A total of 465 patients were included in the analysis. QLU-C10D index scores (intra-class correlation) and domains (Pearson) were correlated with EQ-5D-5L and Short-Form Six Dimensions (SF-6Dv2) conceptual counterparts. Correlation coefficients for the index scores of QLU-C10D and the generic PBMs ranged from 0.63 to 0.81. The QLU-C10D detected statistically significant differences between groups at baseline in 100% of tests performed (n = 27). For changes over time, QLU-C10D detected expected effects in 68% of cases (n = 29). In comparison with the generic PBMs, QLU-C10D detected differences and changes with a higher statistical efficiency in 76% of cases (77 of 102).</p><p><strong>Conclusions: </strong>The QLU-C10D is a fit-for-purpose ready-to-use PBM to estimate health state utilities of patients with cancer. This study adds to evidence that QLU-C10D has appropriate psychometric properties and appears to have higher statistical efficiency than generic PBMs in cancer.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"937-953"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031413","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}
引用次数: 0
Under-reporting of Validation Efforts for Health Economic Models Persists Despite the Availability of Validation Tools: A Systematic Review. 尽管有验证工具,但对卫生经济模型验证工作的低报告仍然存在:一项系统回顾。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-04-28 DOI: 10.1007/s40273-025-01491-2
Katharina Abraham, Isaac Corro Ramos, C Louwrens Braal, Talitha Feenstra, Anne Kleijburg, George A K van Voorn, Carin Uyl-de Groot
{"title":"Under-reporting of Validation Efforts for Health Economic Models Persists Despite the Availability of Validation Tools: A Systematic Review.","authors":"Katharina Abraham, Isaac Corro Ramos, C Louwrens Braal, Talitha Feenstra, Anne Kleijburg, George A K van Voorn, Carin Uyl-de Groot","doi":"10.1007/s40273-025-01491-2","DOIUrl":"10.1007/s40273-025-01491-2","url":null,"abstract":"<p><strong>Objective: </strong>In this study we aimed to identify possible changes over time in validation efforts and the way in which they are reported for model-based health economic (HE) evaluations, given the introduction of several new validation tools and methods in the past decade.</p><p><strong>Methods: </strong>A systematic review was conducted using PubMed and Embase on published HE models for early breast cancer (EBC) for the period 2016 to 2024. AdViSHE-consisting of four validation categories that cover the main HE model aspects-was utilized to systematically evaluate the reported evaluation efforts. The percentage of studies reporting validation per category was compared with the review by de Boer et al. that covers the years 2008 to 2015.</p><p><strong>Results: </strong>Of the 2199 records, 78 studies fulfilled the eligibility criteria. Reported validation efforts did not significantly improve compared with the previous period, except for the validation of input data by experts. Reporting on the validation of the conceptual model remained low with around 10% of the studies providing validation. Validation of the computerized model and validation against outcomes using alternative input data were the most underreported validation categories with < 4% of the studies. The validation of model outcomes, specifically cross validity and the comparison with empirical data, remained the most reported categories in this review also, with 52% and 36%, respectively. When validation efforts were reported, this was done in a non-systematic manner, and the tests and results were rarely detailed.</p><p><strong>Conclusion: </strong>Overall reporting of validation efforts for model-based HE evaluations in the past decade did not significantly change compared with the previous decade.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"849-858"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255658/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008428","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}
引用次数: 0
The Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review. 健康经济学中离散选择实验的演变图景:系统回顾。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-21 DOI: 10.1007/s40273-025-01495-y
Sven Petrus Henricus Nouwens, Stella Maria Marceta, Michael Bui, Daisy Maria Alberta Hendrika van Dijk, Catharina Gerarda Maria Groothuis-Oudshoorn, Jorien Veldwijk, Janine Astrid van Til, Esther Wilhelmina de Bekker-Grob
{"title":"The Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review.","authors":"Sven Petrus Henricus Nouwens, Stella Maria Marceta, Michael Bui, Daisy Maria Alberta Hendrika van Dijk, Catharina Gerarda Maria Groothuis-Oudshoorn, Jorien Veldwijk, Janine Astrid van Til, Esther Wilhelmina de Bekker-Grob","doi":"10.1007/s40273-025-01495-y","DOIUrl":"10.1007/s40273-025-01495-y","url":null,"abstract":"<p><strong>Introduction: </strong>Stakeholder preference evaluations are increasingly emphasized in healthcare policy and health technology assessment. Discrete choice experiments (DCEs) are the most common method for quantifying preferences among patients, the public, and healthcare professionals. While prior reviews (1990-2017) have examined DCE trends, no comprehensive synthesis exists for studies published since 2018. This updated review (2018-2023) provides critical insights into evolving methodologies and global trends in health-related DCEs.</p><p><strong>Methods: </strong>A systematic search (2018-2023) of Medline, Embase, and Web of Science identified relevant studies. Studies were screened for inclusion and data were extracted, including details on DCE design and analysis. To enable trend comparisons, the search strategy and extraction items aligned with previous reviews.</p><p><strong>Results: </strong>Of 2663 identified papers, 1279 met the inclusion criteria, reflecting a significant rise in published DCEs over time. DCEs were conducted globally, with a remarkable increase in publications from Asia and Africa compared with previous reviews. Experimental designs and econometric models have advanced, continuing prior trends. Notably, most recent DCEs were administered online.</p><p><strong>Discussion: </strong>The rapid growth of DCE applications underscores their importance in health research. While the methodology is advancing rapidly, it is crucial that researchers provide full transparency in reporting their methods, particularly in detailing experimental designs and validity tests, which are too often overlooked. Key recommendations include improving reporting of experimental designs, applying validity tests, following good practices for presenting benefit-risk attributes, and adopting open science practices. Ensuring methodological rigor will maximize the impact and reproducibility of DCE research in health economics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"879-936"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255651/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111597","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}
引用次数: 0
The Use of EQ-5D in the Middle East and North Africa Region: A Systematic Literature Review. EQ-5D在中东和北非地区的使用:系统的文献综述。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-10 DOI: 10.1007/s40273-025-01483-2
Abeer Al Rabayah, Sibylle Puntscher, Fatima Al Sayah, Razan Sawalha, Elly Stolk, Judit Simon, Michael Drummond, Uwe Siebert
{"title":"The Use of EQ-5D in the Middle East and North Africa Region: A Systematic Literature Review.","authors":"Abeer Al Rabayah, Sibylle Puntscher, Fatima Al Sayah, Razan Sawalha, Elly Stolk, Judit Simon, Michael Drummond, Uwe Siebert","doi":"10.1007/s40273-025-01483-2","DOIUrl":"10.1007/s40273-025-01483-2","url":null,"abstract":"<p><strong>Introduction: </strong>The EQ-5D is the most commonly used preference-based measure of health-related quality of life. There is limited evidence about the use of the EQ-5D in the Middle East and North Africa (MENA) region. This study aimed to systematically identify, review, summarize, and synthesize the published literature on using the EQ-5D in this region.</p><p><strong>Methods: </strong>A systematic literature review was conducted, according to the PRISMA 2020 guidelines, using PubMed, Cochrane, PsycINFO, and CINAHL and covering the period up to 30 August 2024. Studies using any version of the EQ-5D in adults or youth in the MENA region were included. Pilot studies, guidelines, study protocols, and reviews were excluded. Key study characteristics and outcomes assessed included study design, clinical area, population, type of EQ-5D data reported, reference value set used, and mode of administration. Title/abstract screening was conducted independently by two reviewers to assess eligibility for inclusion. Two researchers completed full-text screening and extracted data using a standardized form. Disagreements were referred to a third reviewer if not resolved by discussion. Results were summarized in systematic evidence tables.</p><p><strong>Results: </strong>After removing duplicates, 18,034 references were considered for title/abstract screening. In total, 184 studies were included with a total sample size of 128,164 subjects. Of the included single-country studies, 42% were reported in Iran, 20% in Saudi Arabia, and 11% in Jordan. Patient populations were investigated in 86% of the studies, 23% of which targeted endocrine diseases. Study design was observational in 57% and experimental in 14% of the studies. Only 10% of the included studies applied the EQ-5D in an economic evaluation. The EQ-5D-3L version was used in 40% of the studies. However, the trend is towards a greater use of the 5L version in more recent years. Twenty percent of the studies reported EQ-5D results using the index score, frequencies of severity levels per dimension, and visual analog scale scores. EQ-5D modes of administration and funding sources were not reported in 16% and 20% of the studies, respectively.</p><p><strong>Conclusion: </strong>There is an increased use of the EQ-5D in the MENA region, especially since 2020. In the region, the use of the EQ-5D is more prevalent in clinical studies than in economic evaluation studies. The reporting heterogeneity indicates the need for guidance in reporting EQ-5D study results in this region.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"859-877"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255660/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143992196","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}
引用次数: 0
Societal Versus Healthcare Perspectives on the Cost Effectiveness of Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in Aotearoa New Zealand. Ocrelizumab治疗新西兰原发性进行性多发性硬化症的成本效益的社会与医疗视角
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-07 DOI: 10.1007/s40273-025-01486-z
Richard J Milne, Carsten Schousboe, Julie A Campbell, John Mottershead
{"title":"Societal Versus Healthcare Perspectives on the Cost Effectiveness of Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in Aotearoa New Zealand.","authors":"Richard J Milne, Carsten Schousboe, Julie A Campbell, John Mottershead","doi":"10.1007/s40273-025-01486-z","DOIUrl":"10.1007/s40273-025-01486-z","url":null,"abstract":"<p><strong>Objectives: </strong>Multiple sclerosis (MS) is a progressive, degenerative, autoimmune neuronal disease. This study compares the impact of a societal versus a healthcare perspective on the cost effectiveness of treatment of primary progressive MS (PPMS) with ocrelizumab (OCR) versus best supportive care (BSC) in New Zealand.</p><p><strong>Methods: </strong>The analysis utilises a lifetime cohort Markov model based on ten Expanded Disability Status Scale (EDSS) states, plus death. It has two structurally identical arms, with forward transition probabilities in the treatment arm obtained by multiplying forward transition probabilities (converted to rates, and back to probabilities) in the control arm by the 12-week disability progression hazard ratio in the clinical trial ORATORIO. Direct and indirect costs of MS were estimated from a 2017 Australian survey and converted to 2024 NZ dollars using purchasing power parity and the NZ consumer price index. Future costs and benefits were discounted at 3.5% per annum. The model is calibrated to NZ mortality for PPMS, and therapy ends when wheelchair dependence (EDSS7) is reached.</p><p><strong>Results: </strong>For a modelled cohort 40 years of age starting at the ORATORIO distribution of EDSS, at 50% of the list price (viz. 50% rebate on $NZ37,384 per patient per annum), the incremental cost-effectiveness ratio (ICER) of OCR versus BSC is $NZ114,427 per QALY ($US64,651) from a societal perspective or $NZ146,711 ($US82,892) from a healthcare perspective. From a societal perspective, at Treasury's willingness to pay (WTP) criterion of $NZ120,200, an acquisition cost up to 56% of list price ($NZ20,935; 44% rebate) is justifiable. From a healthcare payer perspective, at Treasury's implied WTP of $NZ43,313, an acquisition cost up to 10% of list price ($NZ3738; 90% rebate) is justifiable. Based on this metric, a 5.6-fold higher investment in OCR can be justified from a societal perspective compared with a healthcare perspective. Alternatively, an acquisition cost up to 37.9% of list price (viz. 62.1% rebate or $NZ14,169) could be justified if the criterion of one GDP per capita ($NZ83,011) is used as a societal funding threshold. These results are sensitive to the cost of BSC from a societal perspective but not from a healthcare perspective. From both perspectives the cost effectiveness is sensitive to the acquisition cost and efficacy of OCR, potential waning of efficacy, the age and EDSS state when therapy begins, the cost and timing of entry of a biosimilar pharmaceutical and the discount rate.</p><p><strong>Conclusions: </strong>Treatment of PPMS with OCR is more cost effective from a societal than a healthcare perspective, therefore prioritisation of public funding of novel pharmaceuticals for MS and other resource intensive chronic health conditions will depend critically upon the study perspective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"969-985"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255576/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143975564","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}
引用次数: 0
Does the Informal Carer Identification Method Matter? Evidence from Self-Declaration and Time Diary Approaches. 非正式照顾者识别方法重要吗?来自自我陈述和时间日记方法的证据。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-16 DOI: 10.1007/s40273-025-01506-y
Sean Urwin, Charles Smith, Matt Sutton
{"title":"Does the Informal Carer Identification Method Matter? Evidence from Self-Declaration and Time Diary Approaches.","authors":"Sean Urwin, Charles Smith, Matt Sutton","doi":"10.1007/s40273-025-01506-y","DOIUrl":"10.1007/s40273-025-01506-y","url":null,"abstract":"<p><strong>Objectives: </strong>Impacts on informal carers are increasingly being incorporated into cost-of-illness and cost-effectiveness analyses. However, little is known about whether the method used to identify carers affects the estimated impacts. We compare a novel time diary technique to a common self-declaration question for identifying carers. We investigate whether it: (1) detects more and different carers, and (2) if carers across identification techniques have different mental health outcomes.</p><p><strong>Methods: </strong>We use the Innovation Panel component of the UK Household Longitudinal Study, which records all activities performed in two 24-h periods and contains a rich set of individual characteristics. We use regression analysis to compare the number and characteristics of carers identified across the two methods. We then use the doubly robust approach of entropy balancing combined with regression adjustment to estimate the mental health impacts of caregiving across both methods.</p><p><strong>Results: </strong>Among 1055 individuals, we identify 261 carers by at least one method. The self-declaration method fails to classify 16% of individuals identified as carers through time diary data. We find that carers identified by the time diary have a 1.24 (p < 0.05) higher score on the 36-point General Health Questionnaire (GHQ) scale in the subsequent survey wave compared with similar non-carers. For self-declared carers, the estimated difference in GHQ score is 0.36 (p > 0.1), a smaller and statistically non-significant association compared with that observed among time diary-identified carers CONCLUSIONS: The mental health impacts of caregiving may be underestimated when carers are identified by self-declaration. Supplementing self-declaration with time diaries may offer a means of including more carers. Future research, if only one method is applied, should more carefully consider the means of identifying informal carers and the implications that the use of one method may have on conclusions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"987-997"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086395","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}
引用次数: 0
Cost-Effectiveness of a Digital Leakage Notification System (Heylo™) for People with Ileostomies or Colostomies in the United Kingdom. 英国回肠造口术或结肠造口术患者的数字泄漏通知系统(hello™)的成本效益
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-06 DOI: 10.1007/s40273-025-01498-9
Esben Bo Boisen, Matthew Cawson, Lasse de Fries Jensen, Stuart Mealing, Naomi van Hest
{"title":"Cost-Effectiveness of a Digital Leakage Notification System (Heylo™) for People with Ileostomies or Colostomies in the United Kingdom.","authors":"Esben Bo Boisen, Matthew Cawson, Lasse de Fries Jensen, Stuart Mealing, Naomi van Hest","doi":"10.1007/s40273-025-01498-9","DOIUrl":"10.1007/s40273-025-01498-9","url":null,"abstract":"<p><strong>Background: </strong>People with stomas report a substantial negative impact of stomal effluent leaking outside the ostomy baseplate and subsequent complications in their professional and social lives, causing immense worry. However, many patients are not able to recognize leakages in a timely manner. We conducted a cost-effectiveness study to evaluate the impact of a digital leakage notification system (DLNS) to reduce leakages outside the baseplate (LOB) and worry about leakage for people with intestinal stomas from a UK National Health Service and Personal Social Services perspective.</p><p><strong>Methods: </strong>A Markov model for ostomy care was used to compare health-related quality of life and costs for adults with ileostomies or colostomies using UK standard of care ostomy products with the DLNS (intervention) or without the DLNS (comparator). The base case model used a 3-year time horizon with 1-week cycles and an annual 3.5% discounting of utilities and costs. Patients in all health states experience LOB events and/or worry about leakage as events associated with one-time utility decrements and costs of additional healthcare provider visits and ostomy product use. Probabilities of LOB (DLNS, 46.5%; comparator, 78.6%) and worry about leakage (DLNS, 39.1%; comparator, 78.6%) were based on clinical trial results. Peristomal skin complications were included in the model with the same probabilities of occurrence in the intervention and comparator arms. Sensitivity and scenario analyses were performed to test the robustness of the base case model assumptions.</p><p><strong>Results: </strong>In the base case analysis, the DLNS arm had 49.81 fewer LOB events per person than the comparator arm, resulting in 0.309 incremental quality-adjusted life-years (QALYs) and cost savings of British Pound Sterling (GBP) £1703 per person over 3 years (2023/2024 costing year). The DLNS arm had 56.98 fewer worry about leakage events per person, resulting in cost savings of £403 per person. Total costs and QALYs were £18,600 and 1.818 for the intervention arm, respectively, and £18,566 and 1.509 for the comparator arm. Overall, the DLNS provided 0.309 incremental QALYs at an incremental cost of £34 versus the comparator arm for an incremental cost-effectiveness ratio of £110/QALY gained, well below a willingness-to-pay threshold of £20,000/QALY. Sensitivity analyses showed the DLNS was cost-effective in 97.6% of simulations.</p><p><strong>Conclusions: </strong>This analysis suggests that the DLNS added to standard of care ostomy products is a cost-effective intervention to help prevent LOB events and reduce worry about leakage for people with stomas in the UK. Results of the present study suggest that timely awareness of leakage has a positive impact on the health-related quality of life of people with stomas and on the direct costs of stoma management in the UK.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"955-968"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986686","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}
引用次数: 0
When Evidence Falls Short-Belzutifan for Treating Tumours Associated with von Hippel-Lindau Disease: An External Assessment Group Perspective on a NICE Single Technology Appraisal. 当证据不足时,贝祖替芬治疗与希佩尔-林道病相关的肿瘤:NICE单一技术评估的外部评估小组视角
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-08-01 Epub Date: 2025-05-07 DOI: 10.1007/s40273-025-01505-z
Isaac Corro Ramos, Susan O'Meara, Mubarak Patel, Lisa Stirk, Jiongyu Chen, Xiaoyu Tian, Nigel Armstrong
{"title":"When Evidence Falls Short-Belzutifan for Treating Tumours Associated with von Hippel-Lindau Disease: An External Assessment Group Perspective on a NICE Single Technology Appraisal.","authors":"Isaac Corro Ramos, Susan O'Meara, Mubarak Patel, Lisa Stirk, Jiongyu Chen, Xiaoyu Tian, Nigel Armstrong","doi":"10.1007/s40273-025-01505-z","DOIUrl":"10.1007/s40273-025-01505-z","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"845-848"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255580/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037254","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}
引用次数: 0
Correction: Exploring the Economic Burden of Pulmonary Arterial Hypertension and Its Relation to Disease Severity and Treatment Escalation: A Systematic Literature Review. 更正:探讨肺动脉高压的经济负担及其与疾病严重程度和治疗升级的关系:一项系统的文献综述。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-07-10 DOI: 10.1007/s40273-025-01518-8
Gautam Ramani, Vishal Bali, Heather Black, Danny Bond, Ina Zile, Ashley C Humphries, Dominik Lautsch
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引用次数: 0
Correction: A Framework for Using Cost‑effectiveness Analysis to Support Pricing and Reimbursement Decisions for New Pharmaceuticals in a Context of Evolving Treatments, Prices, and Evidence. 更正:在不断发展的治疗、价格和证据背景下,使用成本效益分析来支持新药定价和报销决策的框架。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-07-04 DOI: 10.1007/s40273-025-01513-z
Beth Woods, Alfredo Palacios, Mark Sculpher
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引用次数: 0
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