PharmacoEconomics最新文献

{"title":"Challenges in the Assessment of a Disease Model in the NICE Single Technology Appraisal of Tirzepatide for Treating Type 2 Diabetes: An External Assessment Group Perspective.","authors":"Mirre Scholte, Bram Ramaekers, Evangelos Danopoulos, Sabine E Grimm, Andrea Fernandez Coves, Xiaoyu Tian, Thomas Debray, Jiongyu Chen, Lisa Stirk, Rachel Croft, Manuela Joore, Nigel Armstrong","doi":"10.1007/s40273-024-01394-8","DOIUrl":"10.1007/s40273-024-01394-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"829-832"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11249712/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140877024","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":"A Systematic Review of Methods and Practice for Integrating Maternal, Fetal, and Child Health Outcomes, and Family Spillover Effects into Cost-Utility Analyses.","authors":"Ramesh Lamsal, E Ann Yeh, Eleanor Pullenayegum, Wendy J Ungar","doi":"10.1007/s40273-024-01397-5","DOIUrl":"10.1007/s40273-024-01397-5","url":null,"abstract":"<p><strong>Background: </strong>Maternal-perinatal interventions delivered during pregnancy or childbirth have unique characteristics that impact the health-related quality of life (HRQoL) of the mother, fetus, and newborn child. However, maternal-perinatal cost-utility analyses (CUAs) often only consider either maternal or child health outcomes. Challenges include, but are not limited to, measuring fetal, newborn, and infant health outcomes, and assessing their impact on maternal HRQoL. It is also important to recognize the impact of maternal-perinatal health on family members' HRQoL (i.e., family spillover effects) and to incorporate these effects in maternal-perinatal CUAs.</p><p><strong>Objective: </strong>The aim was to systematically review the methods used to include health outcomes of pregnant women, fetuses, and children and to incorporate family spillover effects in maternal-perinatal CUAs.</p><p><strong>Methods: </strong>A literature search was conducted in Medline, Embase, EconLit, Cochrane Collection, Cumulative Index to Nursing and Allied Health Literature (CINAHL), International Network of Agencies for Health Technology Assessment (INAHTA), and the Pediatric Economic Database Evaluation (PEDE) databases from inception to 2020 to identify maternal-perinatal CUAs that included health outcomes for pregnant women, fetuses, and/or children. The search was updated to December 2022 using PEDE. Data describing how the health outcomes of mothers, fetuses, and children were measured, incorporated, and reported along with the data on family spillover effects were extracted.</p><p><strong>Results: </strong>Out of 174 maternal-perinatal CUAs identified, 62 considered the health outcomes of pregnant women, and children. Among the 54 quality-adjusted life year (QALY)-based CUAs, 12 included fetal health outcomes, the impact of fetal loss on mothers' HRQoL, and the impact of neonatal demise on mothers' HRQoL. Four studies considered fetal health outcomes and the effects of fetal loss on mothers' HRQoL. One study included fetal health outcomes and the impact of neonatal demise on maternal HRQoL. Furthermore, six studies considered the impact of neonatal demise on maternal HRQoL, while four included fetal health outcomes. One study included the impact of fetal loss on maternal HRQoL. The remaining 26 only included the health outcomes of pregnant women and children. Among the eight disability-adjusted life year (DALY)-based CUAs, two measured fetal health outcomes. Out of 174 studies, only one study included family spillover effects. The most common measurement approach was to measure the health outcomes of pregnant women and children separately. Various approaches were used to assess fetal losses in terms of QALYs or DALYs and their impact on HRQoL of mothers. The most common integration approach was to sum the QALYs or DALYs for pregnant women and children. Most studies reported combined QALYs and incremental QALYs, or DALYs and incremental DALYs, ","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"843-863"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11249496/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141180378","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":"Incorporating Real Option Value in Valuing Innovation: A Way Forward.","authors":"Meng Li, Louis P Garrison","doi":"10.1007/s40273-024-01352-4","DOIUrl":"10.1007/s40273-024-01352-4","url":null,"abstract":"<p><strong>Background: </strong>Considerable progress has been made in defining and measuring the real option value (ROV) of medical technologies. However, questions remain on how to estimate (1) ROV outside of life-extending oncology interventions; (2) the impact of ROV on costs and cost effectiveness; and (3) potential interactions between ROV and other elements of value.</p><p><strong>Methods: </strong>We developed a 'minimal modeling' approach for estimating the size of ROV that does not require constructing a full, formal cost-effectiveness model. We proposed a qualitative approach to assessing the level of uncertainty in the ROV estimate. We examined the potential impact of ROV on the incremental cost-effectiveness ratio as well as on the potential interactions between ROV and other elements of value. Lastly, we developed and presented a 15-item checklist for reporting ROV in value assessment.</p><p><strong>Results: </strong>The minimal modeling approach uses estimates on the efficacy of current treatment and potential future innovation, as well as success rate and length of new treatment development, and can be applied to all types of ROV across disease areas. ROV may interact with the conventional value, value of hope, productivity effects, and insurance value. The impact of ROV on cost effectiveness can be evaluated via threshold analysis.</p><p><strong>Conclusion: </strong>The minimal modeling approach and the checklist developed in this paper simplifies and standardizes the estimation and reporting of ROV in value assessment. Systematically including and reporting ROV in value assessment will minimize bias and improve transparency, which will help improve the credibility of ROV research and acceptance by stakeholders.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"199-210"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11230964/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139681436","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":"Estimating and Rewarding the Value of Healthcare Interventions Beyond the Healthcare Sector: A Conceptual Framework.","authors":"Askal Ayalew Ali, Amit Kulkarni, Sandipan Bhattacharjee, Vakaramoko Diaby","doi":"10.1007/s40273-024-01392-w","DOIUrl":"10.1007/s40273-024-01392-w","url":null,"abstract":"<p><strong>Background: </strong>Evaluating healthcare interventions for their impacts beyond health outcomes may result in recognition of changes in human capital, income level, tax revenue, and government spending, which could affect economic growth and population health. In this paper, we document instances where current health technology assessment (HTA) practices fail to account for the impacts of healthcare interventions on broader society beyond the healthcare sector.</p><p><strong>Methods: </strong>We propose a novel conceptual framework, highlighting its three components (distributional cost-effectiveness analysis [DCEA], input-output model, and voting scheme) and their contributions to capturing the economic and societal ripple effects of healthcare interventions. This manuscript also outlines a case study in which the framework is applied to the reassessment of a previously evaluated digital health therapeutic for the treatment of opioid use disorder (OUD) compared with standard of care, demonstrating its practical application.</p><p><strong>Results: </strong>The DCEA health value metric indicates that digital therapeutic is more equitable, favoring socioeconomically disadvantaged groups, while standard of care exacerbates health inequality by benefiting the already advantaged. Additionally, digital therapeutic shows potential for boosting productivity, raising income, and creating jobs, supporting its consideration by employer-sponsored health plans to optimize resource allocation for treating OUD.</p><p><strong>Conclusion: </strong>The conceptual framework provides insights for enhancing HTAs to incorporate the broader economic and societal impacts of healthcare interventions. By integrating DCEA, extended HTA analysis with input-output modeling, and a voting scheme, decision makers can make informed choices aligned with societal priorities, although further research and validation are necessary for practical implementation across diverse healthcare contexts.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"211-224"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11230979/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958740","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-of-Illness of Skin Cancer: A Systematic Review.","authors":"Annick Meertens, Laura Van Coile, Tijs Van Iseghem, Lieve Brochez, Nick Verhaeghe, Isabelle Hoorens","doi":"10.1007/s40273-024-01389-5","DOIUrl":"10.1007/s40273-024-01389-5","url":null,"abstract":"<p><strong>Background: </strong>Skin cancer's rising incidence demands understanding of its economic impact. The current understanding is fragmented because of the various methodological approaches applied in skin cancer cost-of-illness studies.</p><p><strong>Objective: </strong>This study systematically reviews melanoma and keratinocyte carcinoma cost-of-illness studies to provide an overview of the applied methodological approaches and to identify the main cost drivers.</p><p><strong>Methods: </strong>This systematic review was conducted adhering to the 2020 PRISMA guidelines. PubMed, Embase, and Web of Science were searched from December 2022 until December 2023 using a search strategy with entry terms related to the concepts of skin cancer and cost of illness. The records were screened on the basis of the title and abstract and subsequently on full text against predetermined eligibility criteria. Articles published before 2012 were excluded. A nine-item checklist adapted for cost-of-illness studies was used to assess the methodological quality of the articles.</p><p><strong>Results: </strong>This review included a total of 45 studies, together evaluating more than half a million patients. The majority of the studies (n = 36) focused on melanoma skin cancer, a few (n = 3) focused on keratinocyte carcinomas, and 6 studies examined both. Direct costs were estimated in all studies, while indirect costs were only estimated in nine studies. Considerable heterogeneity was observed across studies, mainly owing to disparities in study population, methodological approaches, included cost categories, and differences in healthcare systems. In melanoma skin cancer, both direct and indirect costs increased with progressing tumor stage. In advanced stage melanoma, systemic therapy emerged as the main cost driver. In contrast, for keratinocyte carcinoma no obvious cost drivers were identified.</p><p><strong>Conclusions: </strong>A homogeneous skin cancer cost-of-illness study design would be beneficial to enhance between-studies comparability, identification of cost drivers, and support evidence-based decision-making for skin cancer.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"751-765"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958704","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":"Bayesian Meta-Analysis of Health State Utility Values: A Tutorial with a Practical Application in Heart Failure.","authors":"Joseph Alvin Ramos Santos, Robert Grant, Gian Luca Di Tanna","doi":"10.1007/s40273-024-01387-7","DOIUrl":"10.1007/s40273-024-01387-7","url":null,"abstract":"<p><p>Researchers incorporate health state utility values as inputs to inform economic models. However, for a particular health state or condition, multiple utility values derived from different studies typically exist and a single study is often insufficient to represent the best available source of utility needed to inform policy decisions. The purpose of this paper is to provide an introductory guidance for conducting Bayesian meta-analysis of health state utility values to generate a single parameter input for economic evaluation, using R. The tutorial is illustrated using data from a systematic review of health state utilities of patients with heart failure, with 21 studies that reported utilities measured using the EuroQol-5D (EQ-5D). Explanations, key considerations and suggested readings are provided for each step of the tutorial, adhering to a clear workflow for conducting Bayesian meta-analysis: (1) setting-up the data; (2) employing methods to impute missing standard deviations; (3) defining the priors; (4) fitting the model; (5) diagnosing model convergence; (6) interpreting the results; and (7) performing sensitivity analyses. The posterior distributions for the pooled effect size (i.e. mean health state utility) and between-study heterogeneity are discussed and interpreted in light of the data, priors and models used. We hope that this tutorial will foster interest in Bayesian methods and their applications in the meta-analysis of utilities.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"721-735"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11180161/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141065652","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":"Evaluating the Validation Process: Embracing Complexity and Transparency in Health Economic Modelling.","authors":"Isaac Corro Ramos, Talitha Feenstra, Salah Ghabri, Maiwenn Al","doi":"10.1007/s40273-024-01364-0","DOIUrl":"10.1007/s40273-024-01364-0","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"715-719"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11180005/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140158690","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 Fremanezumab in Episodic and Chronic Migraine Patients from a Japanese Healthcare Perspective.","authors":"Takao Takeshima, Fumihiko Sakai, Xinyu Wang, Kentaro Yamato, Yoshitsugu Kojima, Yilong Zhang, Craig Bennison, Martijn J H G Simons","doi":"10.1007/s40273-024-01380-0","DOIUrl":"10.1007/s40273-024-01380-0","url":null,"abstract":"<p><strong>Background and objectives: </strong>Fremanezumab is an effective treatment for episodic (EM) and chronic migraine (CM) patients in Japan, but its cost effectiveness remains unknown. The objective of this study was to determine the cost effectiveness of fremanezumab compared with standard of care (SOC) in previously treated EM and CM patients from a Japanese healthcare perspective.</p><p><strong>Methods: </strong>Estimated regression models were implemented in a probabilistic Markov model to inform effectiveness and health-related quality-of-life data for fremanezumab and SOC. The model was further populated with data from the literature. The adjusted Japanese healthcare perspective included productivity losses. The main model outcomes were quality-adjusted life-years (QALYs), costs (2022 Japanese Yen [¥]), and incremental outcomes including the incremental cost-effectiveness ratio (ICER). Analyses were performed separately for the EM and CM patients and combined. Costs and effects were discounted at an annual rate of 2.0%.</p><p><strong>Results: </strong>The mean QALYs over a 25-year time horizon for the EM and CM populations combined were 13.03 for SOC and 13.15 for fremanezumab. The associated costs were ¥27,550,292 for SOC and ¥28,371,048 for fremanezumab. QALYs were higher and costs lower for EM patients compared with CM patients for both fremanezumab and SOC. The deterministic ICERs of fremanezumab versus SOC were ¥6,334,861 for EM, ¥7,393,824 for CM, and ¥6,530,398 for EM and CM combined. Indirect costs and choice of mean migraine days model distribution had a substantial impact on the ICER.</p><p><strong>Conclusion: </strong>Using fremanezumab in a heterogeneous mixture of Japanese EM and CM patients resulted in a reduction of monthly migraine days and thus more QALYs compared with SOC. The cost effectiveness of fremanezumab versus SOC in EM and CM patients resulted in an ICER of ¥6,530,398, from an adjusted Japanese public healthcare perspective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"811-822"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11180151/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070928","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":"Advancing Methods to Measure and Reward Healthcare Innovation.","authors":"Richard Chapman, Richard Xie","doi":"10.1007/s40273-024-01408-5","DOIUrl":"10.1007/s40273-024-01408-5","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"183-185"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11230982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141446709","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":"PharmacoEconomics Supplement : Advancing methods to measure and reward healthcare innovation: results from the valuing innovation project.","authors":"","doi":"10.1007/s40273-024-01407-6","DOIUrl":"10.1007/s40273-024-01407-6","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"181"},"PeriodicalIF":4.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141327764","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}