PharmacoEconomics最新文献

{"title":"Correction: Health-Related Quality-of-Life Outcomes of Very Preterm or Very Low Birth Weight Adults: Evidence From an Individual Participant Data Meta-Analysis.","authors":"Corneliu Bolbocean, Sylvia van der Pal, Stef van Buuren, Peter J Anderson, Peter Bartmann, Nicole Baumann, Jeanie L Y Cheong, Brian A Darlow, Lex W Doyle, Kari Anne I Evensen, John Horwood, Marit S Indredavik, Samantha Johnson, Neil Marlow, Marina Mendonça, Yanyan Ni, Dieter Wolke, Lianne Woodward, Erik Verrips, Stavros Petrou","doi":"10.1007/s40273-025-01502-2","DOIUrl":"https://doi.org/10.1007/s40273-025-01502-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128231","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":"Are Economic Evaluations of Task Shifting Too Narrow in Focus? A Rapid Review.","authors":"Peter Murphy, Susan Griffin, Helen Fulbright, Simon Walker","doi":"10.1007/s40273-025-01507-x","DOIUrl":"https://doi.org/10.1007/s40273-025-01507-x","url":null,"abstract":"<p><strong>Background and objectives: </strong>Task shifting between different cadres of health worker has been proposed as an approach to address workforce shortages. Whether such reallocation is a useful strategy for a health system depends on the potential costs and consequences. Too narrow a focus has implications for population health as resources could be incorrectly directed towards inefficient activities owing to important costs and/or benefits being omitted from the evaluation. We aim to identify the key issues when evaluating the value for money of task shifting and review the applied literature to determine whether it is fit for purpose.</p><p><strong>Methods: </strong>We developed an a priori logic model of task shifting and searched five databases (MEDLINE, Embase, EconLit, Social Sciences Citation Index and CEA Registry) for economic evaluations of task shifting published between 2014 and 2024. We performed forwards and backwards citation searching. We considered the scope of the evaluations with respect to the ability to capture key costs and outcomes of task shifting from the logic model. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist.</p><p><strong>Results: </strong>The rapid review identified 26 studies for inclusion covering 16 countries. Studies evaluated task shifting to community health workers and lay health workers as well as from doctors to radiographers, non-physician clinicians and nurse-midwives. The studies included health costs and outcomes but few included changes in the capacity of the workforce to undertake tasks, access, waiting times, productivity, burden on other staff, patient satisfaction, patient productivity and health equity concerns. There was a predominance for cost-effectiveness analysis to be used to assess the value for money of task shifting but the literature did include a cost-benefit analysis, a cost-consequence analysis and an extended cost-effectiveness analysis.</p><p><strong>Conclusions: </strong>The majority of studies identified a range of costs and consequences that may only be appropriate for resource allocation under the strong assumption that all longer term costs and consequences would be unaffected by the task shift.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128211","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":"Anchoring the Hemophilia Joint Health Score for Conventional Value Assessment in the Care of People Living with Severe Hemophilia A.","authors":"Satoko Ito, Manraj Sra, George Goshua","doi":"10.1007/s40273-025-01508-w","DOIUrl":"https://doi.org/10.1007/s40273-025-01508-w","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132696","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":"Reply to Comment on \"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria\".","authors":"Daniel Gallacher","doi":"10.1007/s40273-025-01499-8","DOIUrl":"https://doi.org/10.1007/s40273-025-01499-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119445","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":"Comment on \"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria\".","authors":"Benjamin Farrar, Daniel Gladwell, Matthew Woods","doi":"10.1007/s40273-025-01500-4","DOIUrl":"https://doi.org/10.1007/s40273-025-01500-4","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119512","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 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":"https://doi.org/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":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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":"https://doi.org/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":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-16","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}

{"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":"https://doi.org/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":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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":"https://doi.org/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":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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":"https://doi.org/10.1007/s40273-025-01505-z","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}