PharmacoEconomics最新文献

{"title":"A Systematic Review of Modeling Approaches to Evaluate Treatments for Relapsed Refractory Multiple Myeloma: Critical Review and Considerations for Future Health Economic Models.","authors":"Andreas Freitag, Grammati Sarri, An Ta, Laura Gurskyte, Dasha Cherepanov, Luis G Hernandez","doi":"10.1007/s40273-024-01399-3","DOIUrl":"10.1007/s40273-024-01399-3","url":null,"abstract":"<p><strong>Background and objective: </strong>Multiple myeloma is a rare incurable hematological cancer in which most patients relapse or become refractory to treatment. This systematic literature review aimed to critically review the existing economic models used in economic evaluations of systemic treatments for relapsed/refractory multiple myeloma and to summarize how the models addressed differences in the line of therapy and exposure to prior treatment.</p><p><strong>Methods: </strong>Following a pre-approved protocol, literature searches were conducted on 17 February, 2023, in relevant databases for models published since 2014. Additionally, key health technology assessment agency websites were manually searched for models published as part of submission dossiers since 2018. Reported information related to model conceptualization, structure, uncertainty, validation, and transparency were extracted into a pre-defined extraction sheet.</p><p><strong>Results: </strong>In total, 49 models assessing a wide range of interventions across multiple lines of therapy were included. Only five models specific to heavily pre-treated patients and/or those who were refractory to multiple treatment classes were identified. Most models followed a conventional simple methodology, such as partitioned survival (n = 28) or Markov models (n = 9). All included models evaluated specific interventions rather than the whole treatment sequence. Where subsequent therapies were included in the model, these were generally only considered from a cost and resource use perspective. The models generally used overall and progression-free survival as model inputs, although data were often immature. Sensitivity analyses were frequently reported (n = 41) whereas validation was only considered in less than half (n = 19) of the models.</p><p><strong>Conclusions: </strong>Published economic models in relapsed/refractory multiple myeloma rarely followed an individual patient approach, mainly owing to the higher need for complex data assumptions compared with simpler modeling approaches. As many patients experience disease progression on multiple treatment lines, there is a growing need for modeling complex treatment strategies, leading to more sophisticated approaches in the future. Maintaining transparency, high reporting standards, and thorough analyses of uncertainty are crucial to support these advancements.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"955-1002"},"PeriodicalIF":4.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11343819/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141451125","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 Model-Based Economic Evaluation of Hypothetical Treatments for Amyotrophic Lateral Sclerosis in the UK: Implications for Pricing of New and Emerging Health Technologies.","authors":"Paul Tappenden, Orla Hardiman, Sun-Hong Kwon, Mon Mon-Yee, Miriam Galvin, Christopher McDermott","doi":"10.1007/s40273-024-01395-7","DOIUrl":"10.1007/s40273-024-01395-7","url":null,"abstract":"<p><strong>Background: </strong>Amyotrophic lateral sclerosis (ALS) is a devastating disease which leads to loss of muscle function and paralysis. Historically, clinical drug development has been unsuccessful, but promising disease-modifying therapies (DMTs) may be on the horizon.</p><p><strong>Objectives: </strong>The aims of this study were to estimate survival, quality-adjusted life-years (QALYs) and costs under current care, and to explore the conditions under which new therapies might be considered cost effective.</p><p><strong>Methods: </strong>We developed a health economic model to evaluate the cost effectiveness of future ALS treatments from a UK National Health Service and Personal Social Services perspective over a lifetime horizon using data from the ALS-CarE study. Costs were valued at 2021/22 prices. Two hypothetical interventions were evaluated: a DMT which delays progression and mortality, and a symptomatic therapy which improves utility only. Sensitivity analysis was conducted to identify key drivers of cost effectiveness.</p><p><strong>Results: </strong>Starting from King's stage 2, patients receiving current care accrue an estimated 2.27 life-years, 0.75 QALYs and lifetime costs of £68,047. Assuming a 50% reduction in progression rates and a UK-converted estimate of the price of edaravone, the incremental cost-effectiveness ratio for a new DMT versus current care is likely to exceed £735,000 per QALY gained. Symptomatic therapies may be more likely to achieve acceptable levels of cost effectiveness.</p><p><strong>Conclusions: </strong>Regardless of efficacy, DMTs may struggle to demonstrate cost effectiveness, even at a low price. The cost effectiveness of DMTs is likely to be strongly influenced by drug price, the magnitude and durability of relative treatment effects, treatment starting/stopping rules and any additional utility benefits over current care.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1003-1016"},"PeriodicalIF":4.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141180376","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":"Estimating Risk Factor Time Paths Among People with Type 2 Diabetes and QALY Gains from Risk Factor Management.","authors":"Ni Gao, Helen A Dakin, Rury R Holman, Lee-Ling Lim, José Leal, Philip Clarke","doi":"10.1007/s40273-024-01398-4","DOIUrl":"10.1007/s40273-024-01398-4","url":null,"abstract":"<p><strong>Objectives: </strong>Most type 2 diabetes simulation models utilise equations mapping out lifetime trajectories of risk factors [e.g. glycated haemoglobin (HbA_1c)]. Existing equations, using historic data or assuming constant risk factors, frequently underestimate or overestimate complication rates. Updated risk factor time path equations are needed for simulation models to more accurately predict complication rates.</p><p><strong>Aims: </strong>(1) Update United Kingdom Prospective Diabetes Study Outcomes Model (UKPDS-OM2) risk factor time path equations; (2) compare quality-adjusted life-years (QALYs) using original and updated equations; and (3) compare QALY gains for reference case simulations using different risk factor equations.</p><p><strong>Methods: </strong>Using pooled contemporary data from two randomised trials EXSCEL and TECOS (n = 28,608), we estimated: dynamic panel models of seven continuous risk factors (high-density lipoprotein cholesterol, low density lipoprotein cholesterol, HbA_1c, haemoglobin, heart rate, blood pressure and body mass index); two-step models of estimated glomerular filtration rate; and survival analyses of peripheral arterial disease, atrial fibrillation and albuminuria. UKPDS-OM2-derived lifetime QALYs were extrapolated over 70 years using historical and the new risk factor equations.</p><p><strong>Results: </strong>All new risk factor equation predictions were within 95% confidence intervals of observed values, displaying good agreement between observed and estimated values. Historical risk factor time path equations predicted trial participants would accrue 9.84 QALYs, increasing to 10.98 QALYs using contemporary equations.</p><p><strong>Discussion: </strong>Incorporating updated risk factor time path equations into diabetes simulation models could give more accurate predictions of long-term health, costs, QALYs and cost-effectiveness estimates, as well as a more precise understanding of the impact of diabetes on patients' health, expenditure and quality of life.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT01144338 and NCT00790205.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1017-1028"},"PeriodicalIF":4.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344020/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141451126","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 an EQ-5D-Y-3L Value Set for Brazil.","authors":"Caique Melo Espirito Santo, Gisela Cristiane Miyamoto, Verônica Souza Santos, Ângela Jornada Ben, Aureliano Paolo Finch, Bram Roudijk, Fabianna Resende de Jesus-Moraleida, Airton Tetelbom Stein, Marisa Santos, Tiê Parma Yamato","doi":"10.1007/s40273-024-01404-9","DOIUrl":"10.1007/s40273-024-01404-9","url":null,"abstract":"<p><strong>Introduction: </strong>The EQ-5D-Y-3L is a generic measure of health-related quality of life in children and adolescents. Although the Brazilian-Portuguese EQ-5D-Y-3L version is available, there is no value set for it, hampering its use in economic evaluations. This study aimed to elicit a Brazilian EQ-5D-Y-3L value set based on preferences of the general adult population.</p><p><strong>Methods: </strong>Two independent samples of adults participated in an online discrete choice experiment (DCE) survey and a composite time trade-off (cTTO) face-to-face interview. The framing was \"considering your views for a 10-year-old child\". DCE data were analyzed using a mixed-logit model. The 243 DCE predicted values were mapped into the observed 28 cTTO values using linear and non-linear mapping approaches with and without intercept. Mapping approaches' performance was assessed to estimate the most valid method to rescale DCE predicted values using the model fit (R²), Akaike Information Criteria (AIC), root mean squared error (RMSE), and mean absolute error (MAE).</p><p><strong>Results: </strong>A representative sample of 1376 Brazilian adults participated (DCE, 1152; cTTO, 211). The linear mapping without intercept (R² = 96%; AIC, - 44; RMSE, 0.0803; MAE, - 0.0479) outperformed the non-linear without intercept (R² = 98%; AIC, - 63; RMSE, 0.1385; MAE, - 0.1320). Utilities ranged from 1 (full health) to - 0.0059 (the worst health state). Highest weights were assigned to having pain or discomfort (pain/discomfort), followed by walking about (mobility), looking after myself (self-care), doing usual activities (usual activities), and feeling worried, sad, or unhappy (anxiety/depression).</p><p><strong>Conclusion: </strong>This study elicited the Brazilian EQ-5D-Y-3L value set using a mixed-logit DCE model with a power parameter based on a linear mapping without intercept, which can be used to estimate the quality-adjusted life-years for economic evaluations of health technologies targeting the Brazilian youth population.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1047-1063"},"PeriodicalIF":4.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11343814/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141493000","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":"NICE Approaches to Expert Opinion Evidence in Highly Specialised Technologies: Time to Change? Evidence Assessment Group Perspective.","authors":"Najmeh Moradi, Nicole O'Connor, Katie H Thomson, Hosein Shabaninejad, Tumi Sotire, Madeleine Still, Cristina Fernandez-Garcia, Sheila A Wallace, Oleta Williams, Luke Vale, Gurdeep S Sagoo","doi":"10.1007/s40273-024-01405-8","DOIUrl":"10.1007/s40273-024-01405-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"913-917"},"PeriodicalIF":4.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141306503","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":"Could or Should We Use Cost-Effectiveness Thresholds in the French Value-Based Pricing Process for New Drugs?","authors":"Salah Ghabri","doi":"10.1007/s40273-024-01393-9","DOIUrl":"10.1007/s40273-024-01393-9","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"823-827"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140908951","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":"A Prototype Software Framework for Transferable Computational Health Economic Models and Its Early Application in Youth Mental Health.","authors":"Matthew P Hamilton, Caroline Gao, Glen Wiesner, Kate M Filia, Jana M Menssink, Petra Plencnerova, David G Baker, Patrick D McGorry, Alexandra Parker, Jonathan Karnon, Sue M Cotton, Cathrine Mihalopoulos","doi":"10.1007/s40273-024-01378-8","DOIUrl":"10.1007/s40273-024-01378-8","url":null,"abstract":"<p><p>We are developing an economic model to explore multiple topics in Australian youth mental health policy. To help make that model more readily transferable to other jurisdictions, we developed a software framework for authoring modular computational health economic models (CHEMs) (the software files that implement health economic models). We specified framework user requirements for: a simple programming syntax; a template CHEM module; tools for authoring new CHEM modules; search tools for finding existing CHEM modules; tools for supplying CHEM modules with data; reproducible analysis and reporting tools; and tools to help maintain a CHEM project website. We implemented the framework as six development version code libraries in the programming language R that integrate with online services for software development and research data archiving. We used the framework to author five development version R libraries of CHEM modules focussed on utility mapping in youth mental health. These modules provide tools for variable validation, dataset description, multi-attribute instrument scoring, construction of mapping models, reporting of mapping studies and making out of sample predictions. We assessed these CHEM module libraries as mostly meeting transparency, reusability and updatability criteria that we have previously developed, but requiring more detailed documentation and unit testing of individual modules. Our software framework has potential value as a prototype for future tools to support the development of transferable CHEMs.Code: Visit https://www.ready4-dev.com for more information about how to find, install and apply the prototype software framework.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"833-842"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11249430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141065650","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 for Estimating Productivity Losses due to Illness or Caregiving in Low- and Middle-Income Countries.","authors":"Ery Setiawan, Sarah A Cassidy-Seyoum, Kamala Thriemer, Natalie Carvalho, Angela Devine","doi":"10.1007/s40273-024-01402-x","DOIUrl":"10.1007/s40273-024-01402-x","url":null,"abstract":"<p><strong>Background: </strong>Productivity losses are often included in costing studies and economic evaluations to provide a comprehensive understanding of the economic burden of disease. Global guidance on estimating productivity losses is sparse, especially for low-and middle-income countries (LMICs) where informal and unpaid work remains dominant. This study aims to describe current practices for valuing productivity losses in LMICs.</p><p><strong>Methods: </strong>We performed a systematic review of studies published before April 2022 using three databases, including PubMed, Cochrane Library and Web of Science Core Collection. We included any costing or economic evaluation study conducted in a LMIC that provided methodological details on how the monetary value for productivity losses was estimated. Two reviewers independently screened articles for inclusion, extracted data and assessed the quality of the studies.</p><p><strong>Results: </strong>A total of 281 articles were included. While most studies did not specify the overall approach used to measure and value productivity losses (58%), the human capital approach was the most frequently used approach to measure productivity losses when this was clearly stated (39%). The most common methods to estimate a monetary value for productivity losses were market wages (51%), self-reported wages (28%) and macroeconomic measures (15%).</p><p><strong>Conclusion: </strong>Reporting standards for productivity losses in LMIC settings have room for improvement. While market wages were the most frequently used method to estimate the monetary value of productivity losses, this relies on context-specific data availability. Until a consensus is reached on if, when and how to include productivity losses in costing and economic evaluation studies, future studies could include a sensitivity analysis to explore the impact of different methods for estimating the monetary value of productivity losses.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"865-877"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11249595/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141317963","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":"Understanding Clinician Preferences for Treatment Attributes in Oncology: A Discrete Choice Experiment of Oncologists' and Urologists' Preferences for First-Line Treatment of Locally Advanced/Unresectable Metastatic Urothelial Carcinoma in Five European Countries.","authors":"Laura Panattoni, Mairead Kearney, Natalie Land, Thomas Flottemesch, Patrick Sullivan, Melissa Kirker, Murtuza Bharmal, Brett Hauber","doi":"10.1007/s40273-024-01359-x","DOIUrl":"10.1007/s40273-024-01359-x","url":null,"abstract":"<p><strong>Introduction: </strong>Prior discrete choice experiments (DCE) in oncology found that, on average, clinicians rank survival as the most important treatment attribute. We investigate heterogeneity in clinician preferences within the context of first-line treatment for advanced urothelial carcinoma in Spain, France, Italy, Germany, and the UK.</p><p><strong>Methods: </strong>The online DCE included 12 treatment choice tasks, each comparing two hypothetical therapy profiles defined by treatment attributes: grade 3/4 treatment-related adverse events (TRAEs), induction and maintenance administration schedules, progression-free survival, and overall survival (OS). We used a random parameters logit model to estimate attribute relative importance (RI) (0-100%) and generate preference shares for four treatment profiles. Results were stratified by country. Preference heterogeneity was evaluated by latent class analysis.</p><p><strong>Results: </strong>In August and September 2022, 498 clinicians (343 oncologists and 155 urologists) completed the DCE. OS had the strongest influence on clinicians' preferences [RI = 62%; range, 51.6% (Germany) to 63.7% (Spain)] followed by frequency of grade 3/4 TRAEs (RI = 27%). Among treatment profiles, the chemotherapy plus immune checkpoint inhibitor maintenance therapy profile had the largest preference share [51%; range, 38% (Italy) to 56% (UK)]. Four latent classes of clinicians were identified (N = 469), with different treatment profile preferences: survival class (30.1%), trade-off class (22.4%), no strong preference class (40.9%), and aggressive treatment class (6.6%). OS was not the most important attribute for 30.0% of clinicians.</p><p><strong>Conclusion: </strong>While average sample results were consistent with those of prior DCEs, this study found heterogeneity in clinician preferences within and across countries, highlighting the diversity in clinician decision making in oncology.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"895-909"},"PeriodicalIF":4.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140111084","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":"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}