PharmacoEconomics最新文献

{"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}

{"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}

{"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}

{"title":"Microsimulation Modeling for Health Decision Sciences Using C++: A Tutorial.","authors":"Aku-Ville Lehtimäki, Janne Martikainen","doi":"10.1007/s40273-025-01526-8","DOIUrl":"https://doi.org/10.1007/s40273-025-01526-8","url":null,"abstract":"<p><p>Microsimulation models have become increasingly common in the field of decision modeling for health. Because microsimulation models are computationally more demanding than traditional Markov cohort models, the use of computer programming languages in their development has become more common. C++ is a programming language that has gained widespread recognition in computationally intensive fields, including systems modeling and performance-critical applications. It offers powerful tools for building high-performance microsimulation models, outpacing many traditional modeling software solutions, such as native R, in terms of speed and control over memory management. However, there is limited accessible guidance for implementing microsimulation models in C++. This tutorial offers a step-by-step approach to constructing microsimulation models in C++ and demonstrates its application through simplified but adaptable example decision models. We walk the reader through essential steps and provide generic C++ code that is flexible and suitable for adapting to a range of models. Finally, we present the standalone C++ models and their Rcpp counterparts run within R, and compare their performance to equivalent R implementations in terms of speed and memory efficiency.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732669","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":"Defining Biological and Clinical Plausibility: The DICSA Framework for Protocolized Assessment in Survival Extrapolations Across Therapeutic Areas.","authors":"Bart Heeg, Dawn Lee, Jane Adam, Maarten Postma, Mario Ouwens","doi":"10.1007/s40273-025-01485-0","DOIUrl":"10.1007/s40273-025-01485-0","url":null,"abstract":"<p><strong>Background: </strong>Numerous health technology assessment guidance documents emphasize the importance of biological/clinical plausibility of modeled lifetime incremental survival without clearly defining it.</p><p><strong>Objectives: </strong>This paper defines biologically and clinically plausible lifetime survival extrapolations and proposes a framework to systematically assess this by comparing survival expectations estimated premodeling, with the final modeled survival extrapolations. This framework is embedded in a survival extrapolation protocol template, which ensures that both the expectations and extrapolations are based on unified, comprehensive evidence.</p><p><strong>Methods: </strong>A targeted review was conducted of 29 guidance documents from National Institute for Health and Care Excellence, Pharmaceutical Benefits Advisory Committee, Haute Autorité de Santé, Canada's Drug Agency, and European joint clinical assessment, focusing on survival analysis, evidence synthesis, cost-effectiveness modeling methods, and use of observational data.</p><p><strong>Results: </strong>Survival extrapolations are biologically/clinically plausible when \"predicted survival estimates that fall within the range considered plausible a-priori, obtained using a-priori justified methodology.\" These a priori expectations should utilize the totality of evidence available and take into account local target setting (i.e., survival-influencing aspects such as patient population, treatment pathway, and country). Pre-protocolized biologically/clinically plausible survival extrapolation was operationalized in a five-step DICSA approach: (1) Describe the target setting as defined by all relevant treatment and disease aspects that influence survival; (2) collect Information from relevant sources; (3) Compare survival-influencing aspects across information sources; (4) Set pre-protocolized survival expectations and plausible ranges; and (5) Assess how trial-based extrapolations align with the set expectations by comparing modeled survival extrapolations to the range of values a priori considered to be plausible.</p><p><strong>Conclusion: </strong>The definition of plausibility of survival extrapolations, the operationalization of its assessment, and the corresponding extrapolation protocol template can contribute to the transparent development of biologically/clinically plausible survival extrapolations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"793-803"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167276/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743541","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}

{"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":"10.1007/s40273-025-01499-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"839-840"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","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":"10.1007/s40273-025-01500-4","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"835-837"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","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":"How do Health State Values Differ When Respondents Consider Adults Versus Children Living in Those States? A Systematic Review.","authors":"Ashwini De Silva, Alexander van Heusden, Zhongyu Lang, Nancy Devlin, Richard Norman, Kim Dalziel, Tessa Peasgood, Tianxin Pan","doi":"10.1007/s40273-025-01493-0","DOIUrl":"10.1007/s40273-025-01493-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This systematic review examines how different perspectives influence the valuation of child health-related quality of life (HRQoL). Specifically, it explores differences in values when health states are assessed by children, adolescents, or adults (or some combination of these), from the perspective of the first person (self) or the third person (other), and whether specifying (or not) the age of the person living the described health state affects the valuations. Recent studies suggest discrepancies for descriptively similar health states potentially owing to differences in respondents' willingness to trade length-of-life for quality-of-life for children, though findings are inconsistent. This review aims to assess: (1) differences in peoples' willingness to trade, (2) differences between the relative importance of dimensions, and (3) factors influencing these differences.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This systematic review follows PRISMA guidelines. A search in Ovid MEDLINE, Ovid Embase, and EconLit up to November 2024 was undertaken. We included studies where different perspectives and different valuation instruments were considered. We extracted information on study characteristics, instruments, valuation methods, perspective, study design, analytical methods, sample characteristics, differences in values by respondents, and perspective. A multi-level meta-regression assessed the impact of factors affecting the mean differences between perspectives.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;In total, 24 studies were included, which were from 2004 to 2024. Studies used a range of preference elicitation methods and nearly half (38%) used mixed valuation methods. Most studies (71%) used the EQ- 5D-Y- 3L instrument. Overall, 54% of studies compared adults valuing health states for themselves, or other adult versus adults valuing for other children or themselves as children. The multi-level meta-regression found that the severity of the health state and the valuation method has a significant impact on the mean differences between child and adult values for child health states. In most of the studies when adults are respondents, pain or discomfort was considered as the most important dimension. When adolescent respondents value health states the results are mixed. Qualitative studies identified respondents' difficulty imagining a child in ill health and becoming emotional while thinking about child poor health and early death as potential reasons behind differences in child values versus adult values.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;The evidence suggests that differences in mean values arise when different perspectives are used in valuing severe child health states by adults. These differences are influenced by factors such as health state severity and valuation method. While the review identified the key factors influencing the differences in mean values, an uncertainty remains regarding the optimal choice of preferenc","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"723-740"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167273/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986868","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}

{"title":"Do the Age of Children and Parental Status Matter in Valuing the Child Health Utility 9D (CHU9D)?","authors":"Xiuqin Xiong, Li Huang, Natalie Carvalho, Kim Dalziel, Nancy Devlin","doi":"10.1007/s40273-025-01494-z","DOIUrl":"10.1007/s40273-025-01494-z","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to test whether preferences for children's health states differ (a) when considering those aged 2-4 years compared with older children and (b) by parental status; we also aim to provide a value set that can be used for 2-4 years old.</p><p><strong>Methods: </strong>Health states were described using the Child Health Utility 9D (CHU9D). A discrete choice experiment (DCE) survey was administered between September 2023 and March 2024 to a representative sample of the Australian general adult population which included a 20% quota of parents of 0-18-year-old children. Participants were randomly allocated to two study arms considering the health of a 2-4-year-old or a 10-year-old child. A conditional logit model was used to obtain the latent values from the choice responses. The differences in latent values between the two ages and between parental status were analyzed using relative attribute importance (RAI), poolability test, and pooled model with interaction terms. Visual Analogue Scale (VAS) responses were used to anchor the latent values onto a 0-1 utility scale.</p><p><strong>Results: </strong>In all, 3112 participants were included. Results suggested that the preferences between the two age perspectives were similar, with only 1 out of 36 interaction terms being significant. Preferences of parents of children aged 0-18 years differed from those who were not, as indicated by three significant interaction terms and failure in poolability testing, having smaller disutility for severe health states in the Pain, Tired, and Joining in Activities dimensions.</p><p><strong>Conclusion: </strong>In the valuation of CHU9D health states, asking respondents to consider a 2-4-year-old compared with a 10-year-old did not influence adults' preferences; however, the preferences of respondents who were parents of 0-18-year-olds at that time differed from those who were not. Two CHU9D value sets are reported for children 2 years and older, one derived from the general adult population and the other from parents.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"819-833"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144009590","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}

{"title":"The Potential Health Economic Value of Adding Magnetomotive Ultrasound to Current Diagnostic Methods for Detecting Lymph Node Metastases in Rectal Cancer.","authors":"Emelie Andersson, Ulrika Axelsson, Carl-Fredrik Rönnow, Henrik Thorlacius, Linda Persson, Adam Fridhammar","doi":"10.1007/s40273-025-01490-3","DOIUrl":"10.1007/s40273-025-01490-3","url":null,"abstract":"<p><strong>Background: </strong>Local resection of early rectal cancer (RC) is a desirable treatment option compared with surgery, offering reduced morbidity, mortality, health care costs and avoidance of stoma. However, local resection is restricted to cases without suspicion of lymph node metastases (LNM). Current methods to diagnose LNM and risk estimations based on histopathology cannot reliably identify patients eligible for local resection. The NanoEcho diagnostic system is based on a novel method for lymph node staging in RC. The aim of this study was to perform a health economic analysis at an early stage of clinical development to estimate the potential value of adding NanoEcho diagnostics to current diagnostic methods in RC.</p><p><strong>Methods: </strong>A Markov model for RC diagnosis was developed where the costs and health outcomes, including quality-adjusted life years (QALYs), for adding the NanoEcho diagnostics to current diagnostic methods were compared with current diagnostic methods alone. The diagnostic performance of the NanoEcho diagnostic system is still unknown and the base-case analysis was performed at an assumed 85% sensitivity and 85% specificity. Two testing strategies corresponding to two alternative ways of implementing the diagnostic test in clinic were evaluated: (1) examine all patients diagnosed with RC and (2) examine only patients diagnosed with clinical stages T1 and T2.</p><p><strong>Results: </strong>Adding the NanoEcho diagnostic system resulted in a gain of 0.032 life years and 0.124 QALYs per patient in the target population compared with current diagnostic methods alone. At a cost-neutral level, the estimated justifiable price of NanoEcho diagnostics was SEK 6995 in the first testing strategy and SEK 50,658 in the second testing strategy. The justifiable price of the NanoEcho diagnostics at a willingness to pay of 500,000 SEK/QALY was SEK 10,654 in the first testing strategy and SEK 65,132 in the second testing strategy.</p><p><strong>Conclusion: </strong>The results indicate that adding NanoEcho diagnostics to standard of care can potentially reduce healthcare costs and increase quality of life in RC patients, assuming a sensitivity and specificity of 85%.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"779-791"},"PeriodicalIF":4.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12167262/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973431","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}