Arthur E Attema, Marcello Antonini, Mesfin Genie, Aleksandra Torbica, Francesco Paolucci
{"title":"Time preferences and COVID-19 vaccination uptake.","authors":"Arthur E Attema, Marcello Antonini, Mesfin Genie, Aleksandra Torbica, Francesco Paolucci","doi":"10.1007/s10198-025-01801-7","DOIUrl":"https://doi.org/10.1007/s10198-025-01801-7","url":null,"abstract":"","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295366","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}
Matthew Taylor, Joe Goldbacher, Charlotte Graham, James Mahon, Mike Chambers
{"title":"Clinical equivalence and non-inferiority within health technology assessment.","authors":"Matthew Taylor, Joe Goldbacher, Charlotte Graham, James Mahon, Mike Chambers","doi":"10.1007/s10198-025-01803-5","DOIUrl":"https://doi.org/10.1007/s10198-025-01803-5","url":null,"abstract":"<p><strong>Background: </strong>Non-inferiority and clinical equivalence clinical trials can be used to determine whether a health technology is no worse than an existing treatment. This study identified international guidance for conducting non-inferiority and clinical equivalence trials and investigated the current practices in conducting and reporting such trials, especially in the context of Health Technology Assessment (HTA).</p><p><strong>Methods: </strong>A pragmatic approach was used to identify international guidelines and published literature reporting approaches for the conduct and reporting of non-inferiority or clinical equivalence studies. Guidelines from both HTA and regulatory bodies were considered, and literature reviews from 2010 to 2023 were identified. The results of the reviews were supplemented by stakeholder interviews and synthesised to form a series of recommendations for the UK National Institute for Health and Care Excellence in the appraisal of non-inferiority and equivalence trials.</p><p><strong>Results and conclusion: </strong>The majority of guidelines (13/15) discussed methods to determine the non-inferiority margin and how the analysis should be conducted. Despite this, the quality of reporting in non-inferiority and clinical equivalence trials is consistently poor. Prior to presentation of trial evidence, HTA submissions that claim non-inferiority or equivalence should present the technical, biological and/or pharmacokinetic reasonings that support the claim.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144287107","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}
V Bonnemains, Y Foucher, P Tessier, C David, M Giral, E Dantan
{"title":"Predicting EQ-5D-3L utility values from clinical data in a prospective cohort of kidney transplant recipients.","authors":"V Bonnemains, Y Foucher, P Tessier, C David, M Giral, E Dantan","doi":"10.1007/s10198-025-01802-6","DOIUrl":"https://doi.org/10.1007/s10198-025-01802-6","url":null,"abstract":"<p><strong>Objectives: </strong>Modelling health-state utility values (HSUVs) from clinical data offers a means to conduct retrospective cost-effectiveness analyses using clinical studies that did not collect direct HSUV measures. Such studies can support the efficient allocation of resources in kidney transplantation (KT). We aim to model KT recipients' EQ-5D-3L HSUVs using routinely collected clinical data.</p><p><strong>Methods: </strong>From a French observational multicentric prospective cohort, we included 2,787 adult recipients of a first or second single renal graft transplanted between January 2014 and December 2021 who completed 5,679 EQ-5D-3L questionnaires post-KT, from which the HSUVs were calculated. Considering two time periods before and after 1-year post-KT, we estimated a linear mixed effect model (LME), a mixed adjusted limited dependent variable mixture model, and beta and two-part beta mixed models. We compared their predictive performances in terms of precision and calibration.</p><p><strong>Results: </strong>In each model, recipient age, female sex, higher body mass index, presence of comorbidities and time spent on dialysis prior to KT were associated with lower HSUVs. The predicted HSUVs increased during the first year post-KT before slowly decreasing afterwards. The two-part beta mixed model resulted in the most precise predictions but showed poor calibration. The LME was associated with better calibration than the other models.</p><p><strong>Conclusions: </strong>Our study illustrates the importance of estimating longitudinal predictive algorithms to consider possible time variations in HSUVs. We provide an online calculator for predicting the HSUVs of KT recipients over time. Future studies in international cohorts are important to support the external validity of our results.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144267877","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}
Kenny Haining, Elizabeth Lemmon, Peter Hall, Nazir I Lone
{"title":"Long-term costs associated with healthcare use of people with cancer in Scotland.","authors":"Kenny Haining, Elizabeth Lemmon, Peter Hall, Nazir I Lone","doi":"10.1007/s10198-025-01800-8","DOIUrl":"https://doi.org/10.1007/s10198-025-01800-8","url":null,"abstract":"<p><strong>Background: </strong>Evidence for the long-term costs of cancer is limited, particularly in the Scottish population. Our aim was to better understand the long-term healthcare use and associated costs of cancer in Scotland, and their relationship with cancer survival.</p><p><strong>Methods: </strong>This was a retrospective study using routine healthcare data to measure inpatient, outpatient, community prescription use and their costs from a national health service perspective. Per-episode incidence costs were assigned using reference costs and charted over eight years during the period 2009 to 2018 by year and phase of care. Risk factors for survival and costs were analysed using Cox regression and generalised linear model regression.</p><p><strong>Results: </strong>In total, 55,807 adults with cancer were followed over eight years after their diagnosis. Trajectories indicated a complex relationship with survival. Mean cumulative per-patient costs for all cancers were £29,460 at 2017/18 price levels (95% CI £29,199 to £29,720). Considerable variation was observed between cancer types with the highest costs in non-Hodgkin lymphoma at £47,672 (95%CI £45,500 to £49,843) and the lowest in malignant melanoma of skin at £19,217, (95%CI £18,251 to £20,184). Variables negatively associated with costs tended to be positively associated with hazard of death. Only screening was significantly associated with both lower costs (adjusted cost ratio 0.85, p < 0.001) and lower hazard of death (adjusted hazard ratio 0.30, p < 0.001).</p><p><strong>Conclusions: </strong>Substantial costs were observed in all cancer types studied, with the highest costs measured in the year following diagnosis. Screening was associated with both lower costs and better survival, supporting the focus on early detection.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144259362","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}
Robert Wittram, Léon Kreis, Hans-Helmut König, Christian Brettschneider
{"title":"Economic evaluations of early detection strategies for pancreatic cancer: a systematic review.","authors":"Robert Wittram, Léon Kreis, Hans-Helmut König, Christian Brettschneider","doi":"10.1007/s10198-025-01793-4","DOIUrl":"10.1007/s10198-025-01793-4","url":null,"abstract":"<p><strong>Objectives: </strong>The early detection of pancreatic cancer is an important step in reducing mortality by offering patients curative treatment. The aim of this study was to synthesize available evidence on the costs and cost-effectiveness of strategies for early pancreatic cancer detection.</p><p><strong>Methods: </strong>The electronic databases PubMed, Web of Science, and EconLit were searched for peer-reviewed and published papers in English until April 2024 with no date or contextual restrictions. Economic evaluations of early pancreatic cancer detection strategies compared to alternative or no detection strategies were criteria for inclusion.</p><p><strong>Results: </strong>Thirty-one articles were included, 22 were full and nine were partial economic evaluations. Fifteen studies screened target populations with pancreatic cancer-associated risk factors and 16 conducted surveillance of patients with precancerous lesions. Six studies found early detection strategies to be cost-effective, one did not, and thirteen reported partially cost-effective results. In all studies, populations of interest had an elevated pancreatic cancer risk compared to the general population. Endoscopic ultrasound, magnetic resonance imaging, and computed tomography were the most frequently evaluated imaging modalities. Patient engagement, valuation of outcomes and choice of discount rates were among incomplete reporting categories, and narrow evaluation perspectives may have biased the results.</p><p><strong>Conclusions: </strong>Early detection strategies for pancreatic cancer may be cost-effective for certain high-risk patient groups. However, evaluations so far have applied heterogeneous methods, used different modalities, had various target groups and screened at different frequencies. Further evaluations will be required to systematically synthesize economic evidence regarding specific early detection strategies.</p><p><strong>Registration: </strong>PROSPERO registration CRD42023475348.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227513","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}
Tianxin Pan, Misel Trajanovska, Nathan Kwong, Sebastian K King, Ilias Goranitis
{"title":"Psychometric comparison of CHU9D and PedsQL 4.0 proxy version administered to parents of children with congenital colorectal conditions in Australia.","authors":"Tianxin Pan, Misel Trajanovska, Nathan Kwong, Sebastian K King, Ilias Goranitis","doi":"10.1007/s10198-025-01797-0","DOIUrl":"https://doi.org/10.1007/s10198-025-01797-0","url":null,"abstract":"<p><strong>Objective: </strong>To assess the psychometric performance of the Child Health Utility (CHU9D) proxy version compared with the Pediatric Quality of Life Inventory (PedsQL) in Australian children aged 0-7 years with anorectal malformations (ARM) or Hirschsprung disease (HD).</p><p><strong>Methods: </strong>Parents of children with ARM or HD were identified from a patient database managed by a tertiary paediatric hospital in Australia over the past 20 years. Since 2020, CHU9D and PedsQL proxy report versions were administered to parents via telephone interview. Using data collected between 2020 and 2022, we assessed the feasibility, ceiling and floor effects, known-group validity and convergent validity for both instruments in the total sample, by conditions and child age.</p><p><strong>Results: </strong>The study included 145 children with ARM or HD, among which, 13.1% had missing values on the CHU9D schoolwork dimension, and 20.7% had missing values on the PedsQL school functioning domain (2-4 year old version). The CHU9D and PedsQL did not demonstrate ceiling effects. The CHU9D showed stronger effect size (ES) in differentiating children with ARM (ES = 0.32) or HD (ES = 0.90) with healthy children compared to the PedsQL. We did not find statistically significant differences in CHU9D or PedsQL scores between ARM and HD. There were moderate to strong correlations in most theoretically related dimensions of the CHU9D and PedsQL.</p><p><strong>Conclusion: </strong>The CHU9D and PedsQL demonstrated comparable and acceptable psychometric properties in Australian children aged 2 years and above with ARM or HD. However, the validity of the CHU9D in children under 2 years old needs to be further explored and modification may be needed.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227514","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}
Marcel A N de Bruijn, Emily Z Boersma, Lysanne van Silfhout, Tjarda N Tromp, Eddy M M Adang, Erik van de Krol, Michael J R Edwards, Vincent M A Stirler, Erik Hermans
{"title":"One week versus three to five weeks of plaster cast immobilization for nonreduced distal radius fractures, a cost effectiveness analysis embedded in a stepped wedge cluster randomized controlled trial.","authors":"Marcel A N de Bruijn, Emily Z Boersma, Lysanne van Silfhout, Tjarda N Tromp, Eddy M M Adang, Erik van de Krol, Michael J R Edwards, Vincent M A Stirler, Erik Hermans","doi":"10.1007/s10198-025-01795-2","DOIUrl":"https://doi.org/10.1007/s10198-025-01795-2","url":null,"abstract":"<p><strong>Objective: </strong>Distal radius fractures are commonly seen at the Emergency Department. In the Netherlands, non- or minimally displaced fractures are immobilized for 3-5 weeks. However, evidence suggests shorter immobilization yields similar or better functional outcome. There is a lack of cost-effectiveness studies investigating shorter duration of cast immobilization. This study investigates the cost-effectiveness of one week of plaster cast immobilization versus three to five weeks.</p><p><strong>Methods: </strong>Cost-effectiveness data was collected as part of the Cast-OFF 2 study which started the 1st of January 2022. A randomized stepped wedge cluster design was used with 11 hospitals, and 10 clusters, including patients with an isolated non- or minimally displaced distal radius fracture without fracture reduction. Costs on medical consumption, and productivity were scored with the local Electronical Patient Record, and questionnaires at week one, six, month six, and twelve. Cost-effectiveness was reported per Quality-Adjusted Life Year (QALY).</p><p><strong>Results: </strong>A total of 402 patients were included (control n = 197 vs intervention n = 205). No differences in QALY were observed (+ 0.02, CI [-0.02, 0.06]). Cost savings for the intervention group ranged from €31.94 to €322.41 depending on different scenarios. The future perspective scenario with reduction of one outpatient clinic visit showed a significant cost saving of €254.27 (CI [-467.33, -41.21]). No significant differences were observed in baseline characteristics.</p><p><strong>Conclusion: </strong>One week of plaster cast immobilization for non- or minimally displaced distal radius fractures results in comparable or better cost savings compared to usual care. Adopting one week of cast immobilization as the new standard-of-care could reduce healthcare costs.</p><p><strong>Trial registration: </strong>Netherlands Trial Register NL9278. CMO: 2-21-7308.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144210214","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Strategic behavior and entry deterrence by branded drug firms: the case of authorized generic drugs.","authors":"Lu Yao, Mengde Liu","doi":"10.1007/s10198-024-01721-y","DOIUrl":"10.1007/s10198-024-01721-y","url":null,"abstract":"<p><p>Pharmaceutical firms that market brand-name drugs lose substantial market share to generic manufacturers after patent expiration. As a response to the threat of generic competition, branded manufacturers pursue defensive strategies. One such strategy is the launch of authorized generic drugs. Authorized generic drugs are produced by branded manufacturers to compete against other generic drug entrants. Such competition may lower the expected profits of generic drug manufacturers and hence deter future generic drug entry. This paper models and empirically examines whether the introduction of authorized generic drugs changes the independent generic firms' decisions on entering the market. We use an instrumental variable approach to evaluate the effect of authorized generic drugs on the responses of generic manufacturers. The results show that the entry of authorized generic drugs deters and delays the entry of generic drugs.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":"561-575"},"PeriodicalIF":3.1,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142309102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Comparison of the EQ-5D-Y and the CHU-9D instruments in a general child population based on self-reports and proxy-reports.","authors":"Yan Li, Yanqiu Chen, Jize Sun, Mingyu Jiang, Aixia Ma, Tiantian Tao, Pingyu Chen","doi":"10.1007/s10198-024-01722-x","DOIUrl":"10.1007/s10198-024-01722-x","url":null,"abstract":"<p><strong>Objective: </strong>This study utilized the EQ-5D-Y and the Child Health Utility 9D (CHU-9D) instruments to empirically investigate a general child population aged 7-8 years in China, with the aim of assessing and comparing the performance, correlation, and agreement between these two instruments. Both self-reported and proxy-reported versions of the instruments were considered in the analysis.</p><p><strong>Methods: </strong>Data were collected from 7-8-year-old students in the second grade from four schools in Guangxi and Guiyang provinces, China. Children and their proxies independently completed their respective versions of the questionnaires, including the EQ-5D-Y, the CHU-9D, and other socio-demographic information. The psychometric properties of the EQ-5D-Y and the CHU-9D were assessed, including ceiling effects, internal consistency, and known-group validity. Spearman's correlation coefficient, Intraclass Correlation Coefficient (ICC), and Bland-Altman plots were calculated and plotted to assess the correlation and agreement between the EQ-5D-Y and CHU-9D.</p><p><strong>Results: </strong>A total of 369 pairs of valid questionnaires were collected from both children and proxies. Due to the study's focus on a general child population, both EQ-5D-Y and CHU-9D yielded high utility values, with a significant ceiling effect observed, particularly in the EQ-5D-Y proxy-reported results. Compared to the EQ-5D-Y, the CHU-9D demonstrated a higher Cronbach's alpha coefficient and better internal consistency. Both instruments also demonstrated known-group validity, distinguishing different health status groups, except for EQ-5D-Y proxy-reported results. Spearman's correlation coefficient indicated some correlations in similar dimensions and utility values between the EQ-5D-Y and CHU-9D. The ICC of the EQ-5D-Y and CHU-9D utility values was 0.290 for self-reports and 0.383 for proxy-reports, indicating poor agreement between the two instruments. The Bland-Altman plots showed that the mean utility values obtained from EQ-5D-Y were significantly higher than those from CHU-9D.</p><p><strong>Conclusion: </strong>The EQ-5D-Y and the CHU-9D demonstrated acceptable performance within the general child population aged 7-8 years in China, except for the EQ-5D-Y proxy-reported version. It suffered from a notable ceiling effect, poor internal consistency, as well as weak known-group validity and discriminative ability. Moreover, although there existed a certain degree of correlation between the EQ-5D-Y and CHU-9D, their utility values exhibited significant differences. Therefore, these instruments are not interchangeable in practice.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":"577-588"},"PeriodicalIF":3.1,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332148","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The societal cost of 'unwanted' loneliness in Spain.","authors":"Bruno Casal, Eva Rodríguez-Miguez, Berta Rivera","doi":"10.1007/s10198-024-01724-9","DOIUrl":"10.1007/s10198-024-01724-9","url":null,"abstract":"<p><p>Unwanted loneliness negatively affects people's health and quality of life, increasing morbidity and the risk of premature death; this situation can generate major social costs. The aim of this study is to estimate the social costs of loneliness in Spain for 2021: both tangible costs -monetary value of health costs and production losses- and intangible costs -Quality Adjusted Life Years (QALYs). To estimate costs not derived from mortality, information from a sample of 400 people with unwanted loneliness was compared with that derived from two samples of the general population obtained from the Spanish National Health Surveys. To estimate the costs associated with premature deaths, the population attributable fraction was calculated using the relative risks estimated in previous survival studies. In the baseline scenario, the tangible costs of unwanted loneliness are estimated to be around 14,129 million euros in 2021, representing 1.2% of Spain's GDP. Approximately 56.8% of the tangible costs correspond to production losses due to reduced working time, and 43.2% are due to healthcare costs related with increased consultation frequency in healthcare services and higher consumption of medicines related to loneliness. In addition, loneliness generates a reduction in quality of life equivalent to 1.04 million QALYs, which corresponds to 2.8% of the total stock of QALYs of the Spanish population over 15 years of age.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":"605-617"},"PeriodicalIF":3.1,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332149","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}