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Economic Evaluation of Patiromer in Patients with Concomitant Heart Failure and Chronic Kidney Disease in Italy. 意大利并发心力衰竭和慢性肾脏疾病患者的Patiromer经济评价
IF 2
PharmacoEconomics Open Pub Date : 2025-05-12 DOI: 10.1007/s41669-025-00581-3
M Senni, E Paoletti, Ewa Stawowczyk, M Hale, A Ramirez de Arellano
{"title":"Economic Evaluation of Patiromer in Patients with Concomitant Heart Failure and Chronic Kidney Disease in Italy.","authors":"M Senni, E Paoletti, Ewa Stawowczyk, M Hale, A Ramirez de Arellano","doi":"10.1007/s41669-025-00581-3","DOIUrl":"https://doi.org/10.1007/s41669-025-00581-3","url":null,"abstract":"<p><strong>Background: </strong>Hyperkalaemia (HK), in patients with heart failure (HF) with and without chronic kidney disease (CKD), is potentially life-threatening. Risk of HK is further heightened in those patients receiving renin-angiotensin-aldosterone system inhibitors (RAASi), used to reduce cardiovascular morbidity and mortality in HF. Patiromer, an oral potassium (K<sup>+</sup>) binder, has been shown to reduce the risk of HK and enable optimal RAASi dosing. We evaluated the cost-effectiveness of patiromer in HF patients with CKD in the Italian setting, utilising results from the recent DIAMOND clinical trial, which assessed long-term use of patiromer in HK management.</p><p><strong>Methods: </strong>An established Markov model was adapted to include data from DIAMOND using the National Health Service (NHS) perspective. In DIAMOND, patients received patiromer during a run-in period (up to 12 weeks) to achieve optimal RAASi without HK. However, this led to low mean K<sup>+</sup> concentrations in the placebo arm, resulting from a legacy effect of patiromer in the run-in phase of the trial. Therefore, the DIAMOND population was adjusted to a real-world population to better represent the K<sup>+</sup> levels in the standard of care (SoC) arm. Mean K<sup>+</sup> concentration for baseline and the patiromer arm was calculated from the overall population at baseline (screening phase) and after treatment (end of run-in period), respectively. Lifetime trajectories were estimated for quality-adjusted life years (QALYs), life years (LYs) and costs.</p><p><strong>Results: </strong>The economic evaluation model calculated a discounted total average cost per patient of €109,900 for patiromer and €64,847 for SoC. Patiromer generated a gain of 1.97 LYs (1.55 QALYs) compared with SoC. The incremental cost-effectiveness ratio (ICER) for patiromer was €29,060/QALY gained versus SoC.</p><p><strong>Conclusion: </strong>Applying DIAMOND data, patiromer is deemed to be cost-effective at a willingness-to pay threshold of €40,000 per QALY gained in Italy.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Costs and Complications of Respiratory Syncytial Virus and Acute Respiratory Infections in the Adult Population: Analysis of a German Claims Database. 成人呼吸道合胞病毒和急性呼吸道感染的费用和并发症:德国索赔数据库的分析。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-03-19 DOI: 10.1007/s41669-025-00565-3
Pavo Marijic, Roman Kliemt, Martin Krammer, Nikolaus Kolb, Theo Last, Andreas Ambrosch, Santiago Ewig, Rembert Koczulla, Jörg Schelling, Claus Vogelmeier, Maria Waize, Manuela Stierl, Maria João Fonseca, Sara Pedron, Alen Marijam
{"title":"Costs and Complications of Respiratory Syncytial Virus and Acute Respiratory Infections in the Adult Population: Analysis of a German Claims Database.","authors":"Pavo Marijic, Roman Kliemt, Martin Krammer, Nikolaus Kolb, Theo Last, Andreas Ambrosch, Santiago Ewig, Rembert Koczulla, Jörg Schelling, Claus Vogelmeier, Maria Waize, Manuela Stierl, Maria João Fonseca, Sara Pedron, Alen Marijam","doi":"10.1007/s41669-025-00565-3","DOIUrl":"10.1007/s41669-025-00565-3","url":null,"abstract":"<p><strong>Background: </strong>Respiratory syncytial virus (RSV) infections pose health and economic burdens to adults. Using claims data, we estimated RSV-associated costs, healthcare resource utilization (HCRU), and complication rates from patients of a nationwide German health insurance database.</p><p><strong>Methods: </strong>We analyzed confirmed RSV, RSV-possible, and acute respiratory infection (ARI) cohorts, plus 1:1 matched control cohorts of individuals ≥ 18 years from 2010 to 2019. Matching was performed separately for patients 18-49, 50-59, and ≥ 60 years. Medical costs, HCRU, and sick leave were assessed for inpatients and outpatients. Complications were compared between cases and controls, and logistic regression assessed odds ratios (ORs) for risk.</p><p><strong>Results: </strong>Altogether, 2668 confirmed RSV index episodes occurred. In ≥ 60-year-olds, 862 episodes incurred mean excess costs of €3773 (95% confidence interval [CI]: €2956-€4591) per episode during the index quarter and €3286 (95% CI: €1841-€4732) in the following four quarters. Mean costs were €5553 per episode for inpatients and €116 for outpatients. In ≥ 60-year-olds, risk for congestive heart failure hospitalization (OR 2.3; 95% CI: 1.4-3.8), exacerbation of asthma (OR 6.0; 95% CI: 1.7-20.9), and chronic obstructive pulmonary disease (OR 3.9; 95% CI: 2.6-5.8) were higher for confirmed RSV than controls. In younger groups, costs, HCRU, and complications were also higher in cases than controls. The complication frequencies increased with age. RSV-possible episodes incurred mean excess costs of €615 (95% CI: €605-€626) during the index quarter and €610 (95% CI: €583-€637) during the following four quarters, while in the ARI cohort, the excess costs were €1003 (95% CI: €991-€1015) during the index quarter and €1003 (95% CI: €973-€1032) in the following four quarters. For all three cohorts, individuals who had comorbidities, were immunocompromised, or living in long-term care facilities incurred higher costs.</p><p><strong>Conclusions: </strong>Confirmed RSV is associated with high excess costs - especially in hospital settings - and HCRU. Complication risk increased with RSV presence.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"445-459"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037943/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664032","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost Effectiveness of Left Ventricular Assist Devices (LVADs) as Destination Therapy: A Systematic Review. 左心室辅助装置(lvad)作为目的治疗的成本效益:一项系统综述。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-02-20 DOI: 10.1007/s41669-025-00564-4
Tuba Saygın Avşar, Louise Jackson, Pelham Barton, Sophie Beese, Okeke Ogwulu Chidubem, Sern Lim, David Quinn, Malcolm J Price, David J Moore
{"title":"Cost Effectiveness of Left Ventricular Assist Devices (LVADs) as Destination Therapy: A Systematic Review.","authors":"Tuba Saygın Avşar, Louise Jackson, Pelham Barton, Sophie Beese, Okeke Ogwulu Chidubem, Sern Lim, David Quinn, Malcolm J Price, David J Moore","doi":"10.1007/s41669-025-00564-4","DOIUrl":"10.1007/s41669-025-00564-4","url":null,"abstract":"<p><strong>Objectives: </strong>Left ventricular assist devices (LVADs) can extend life and improve quality of life among advanced heart failure patients ineligible for transplantation (destination therapy). High-quality evidence on the cost effectiveness of LVADs compared with optimal medical management is needed to inform policy. This study identifies economic evaluations of LVADs for destination therapy and assesses their methodological quality.</p><p><strong>Methods: </strong>The review followed Centre for Review and Dissemination guidelines for methods, and PRISMA standards for reporting, and was registered on PROSPERO (CRD42020158987). Six databases were searched for studies published up to October 2024. Full economic evaluations of LVADs for destination therapy were included. Two reviewers independently conducted study selection, data extraction and quality assessment using validated tools.</p><p><strong>Results: </strong>The study identified 14 economic evaluations, including 10 modelling studies. Most studies were from the US and UK. There was substantial variation in model structure, methods, and cost estimates. Only seven studies used a lifetime horizon. Resource use was typically estimated based on data from small single-centre samples. Overall quality was moderate due to key limitations such as insufficient time horizons, omitting complications and costs, and limited consideration of uncertainty. Only two studies examined severity, and none assessed cost effectiveness by patient age. Most studies found LVADs not to be cost effective compared with medical management except for two UK-based evaluations.</p><p><strong>Conclusion: </strong>This review reveals important limitations in the current evidence on the cost effectiveness of LVADs as destination therapy. More comprehensive, robust evaluations are needed to inform policy decisions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"351-363"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037950/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468715","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Economic Burden of Acute Myeloid Leukemia in European Union: Results from a Systematic Review of Literature. 欧盟急性髓性白血病的经济负担:来自文献系统综述的结果。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-04-01 DOI: 10.1007/s41669-024-00554-y
Alisha Saeed, Zermina Tasleem, Sohail Ayaz Muhammad, Anees Ur Rehman, Shahid Shah, Qurratul Ain Jamil, Hajra Siddiqui, Hidayah Karuniawati, Saleh Karamah Al-Tamimi
{"title":"Economic Burden of Acute Myeloid Leukemia in European Union: Results from a Systematic Review of Literature.","authors":"Alisha Saeed, Zermina Tasleem, Sohail Ayaz Muhammad, Anees Ur Rehman, Shahid Shah, Qurratul Ain Jamil, Hajra Siddiqui, Hidayah Karuniawati, Saleh Karamah Al-Tamimi","doi":"10.1007/s41669-024-00554-y","DOIUrl":"10.1007/s41669-024-00554-y","url":null,"abstract":"<p><strong>Background: </strong>Acute myeloid leukemia (AML) is a heterogenous malignancy whose management is associated with considerable healthcare resource utilization and high expenditures because of recurrent and extended hospitalizations, multiple outpatient visits, and a wide range of supportive care. Modern therapies with improved safety profiles may assist in reducing healthcare costs; however, they are usually more expensive than standard chemotherapies. Few studies have addressed the expenses and burden of AML. Most of these studies were conducted in the USA. Very little research is available from the European Union (EU).</p><p><strong>Objectives: </strong>The aim of this study was to assess the economic impact of AML and determine the major cost-driving factors for its treatment in the EU.</p><p><strong>Methods: </strong>This systematic review is in accordance with PRISMA guidelines. A systematic search was conducted using PubMed, Embase, ScienceDirect, SCOPUS, and Google Scholar databases to identify relevant studies on the economic impact of AML in various countries of the EU, published before April 15, 2024. Original studies investigating direct costs including expenses for treatment and healthcare services, or resource utilization for AML management were included. The systematic review excluded commentaries, editorials, and pharmacoeconomic modeling studies. Two reviewers independently performed data extraction and quality assessment, and the third reviewer resolved disagreements. We employed the Allison Larg Cost-of-Illness Studies evaluation checklist to assess the risk of bias. The mean cost per patient for induction, consolidation, and transplantation was calculated, and the results were converted into 2024 Euros.</p><p><strong>Results: </strong>Twenty-eight studies met our inclusion criteria, with the sample size of AML patients ranging from 12 to 39,568. The calculated per-patient direct costs of induction chemotherapy in Spain, France, Netherlands, Germany, and Italy were €92,378, €77,844, €61,643, €46,113, and €20,254, respectively. The mean per-patient direct cost of consolidation chemotherapy in the Netherlands and Germany was €42,137, and €32,220, respectively. The mean per-patient direct costs of transplantation in Sweden, Austria, France, Netherlands, and Spain were €192,628, €188,453, €132,352, €122,760, and €47,968, respectively. The cost-driving factors associated with AML treatment were inpatient hospitalization and medication costs.</p><p><strong>Conclusion: </strong>AML seems to incur substantial direct economic expenses. Reducing the days of hospitalization can significantly decrease the economic burden of AML in the European Union. Moreover, there is a necessity for studies that comprehensively evaluate the economic implications, particularly concerning total and indirect costs.</p><p><strong>Registration: </strong>Registered in PROSPERO under the registration number 'CRD42024537725'.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"365-378"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037953/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764038","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Semaglutide 2.4 mg versus Liraglutide 3 mg for the Treatment of Obesity in Greece: A Short-Term Cost-Effectiveness Analysis. 塞马鲁肽2.4 mg与利拉鲁肽3 mg治疗希腊肥胖:短期成本-效果分析
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-01-23 DOI: 10.1007/s41669-025-00561-7
Panagiotis Papantoniou, Nikolaos Maniadakis
{"title":"Semaglutide 2.4 mg versus Liraglutide 3 mg for the Treatment of Obesity in Greece: A Short-Term Cost-Effectiveness Analysis.","authors":"Panagiotis Papantoniou, Nikolaos Maniadakis","doi":"10.1007/s41669-025-00561-7","DOIUrl":"10.1007/s41669-025-00561-7","url":null,"abstract":"<p><strong>Background: </strong>Obesity is a global health issue with significant economic implications for health systems. Pharmacotherapy, including semaglutide 2.4 mg and liraglutide 3 mg, offers a treatment option for weight management; however, its cost-effectiveness requires evaluation. This study assesses the short-term cost-effectiveness of semaglutide 2.4 mg versus liraglutide 3 mg in achieving clinically relevant weight loss targets at 68 weeks in Greece.</p><p><strong>Methods: </strong>A short-term cost-effectiveness analysis was conducted from the perspective of the Greek third-party payer [National Organization for the Provision of Health Services (EOPYY)], comparing costs and outcomes for semaglutide 2.4 mg and liraglutide 3 mg over a 68-week horizon. Effectiveness was measured by the proportion of patients achieving weight loss targets of ≥ 5%, ≥ 10%, ≥ 15%, and ≥ 20%, using efficacy data from the STEP-8 head-to-head trial, a 68-week, randomized, double-blind study conducted in the USA, comparing semaglutide 2.4 mg versus liraglutide 3 mg in adults who were overweight or had obesity without diabetes. Only direct medical costs were included, reflecting the payer perspective, and no discounting was applied owing to the short time horizon. Deterministic and probabilistic sensitivity analyses assessed the results' robustness.</p><p><strong>Results: </strong>Semaglutide 2.4 mg had higher treatment costs (€3285.55) compared with liraglutide 3 mg (€2742.47) but demonstrated greater efficacy and a lower cost of control across all weight loss targets. The cost per patient achieving ≥ 5% weight loss was €3768.72 for semaglutide and €4718.66 for liraglutide, corresponding to a difference of €949.95 per patient. The cost difference widened at higher weight loss targets, with semaglutide showing differences of €6064.20 for ≥ 10% weight loss, €17,005.23 for ≥ 15%, and €37,296.00 for ≥ 20%. These findings were consistent across sensitivity analyses.</p><p><strong>Conclusions: </strong>Semaglutide 2.4 mg is likely to be a short-term, cost-effective treatment option for adults who are overweight or have obesity without diabetes in Greece.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"487-497"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037448/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024279","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
From Vision to Reality: The EU's Pharmaceutical Reforms and the Path to Improved Access. 从愿景到现实:欧盟的药品改革和改善获取途径。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-01-25 DOI: 10.1007/s41669-024-00556-w
Caitlin Main, Cathrin Schäfer, Panos Kanavos
{"title":"From Vision to Reality: The EU's Pharmaceutical Reforms and the Path to Improved Access.","authors":"Caitlin Main, Cathrin Schäfer, Panos Kanavos","doi":"10.1007/s41669-024-00556-w","DOIUrl":"10.1007/s41669-024-00556-w","url":null,"abstract":"<p><p>Disparities in access to oncology medicines in European Union (EU) member states can impact patient outcomes profoundly, with availability and timely access varying significantly across and within member states. This paper discusses the intersection of the new European Health Technology Assessment Regulation (HTAR), the provisions of the proposed pharmaceutical legislation and their potential impacts on access to oncology medicines across EU member states. The HTAR, seeking to standardise the clinical evaluation of new medicines, has the potential to streamline the evaluation process but also risks oversimplifying diverse national healthcare needs. While the HTAR may accelerate access in countries with less-developed health technology assessment systems, it could potentially conflict with established practices in countries with advanced assessment systems, resulting in both joint and national clinical evaluations becoming necessary. The proposed pharmaceutical legislation reform, in both initial and updated forms, aims to incentivise an EU-wide launch of new medicines that challenges the feasibility for manufacturers, particularly in the context of diverse and complex national pricing and reimbursement systems. Both initiatives mark a significant shift towards more collaborative European healthcare policy yet faces the potential of unintended consequences owing to an apparent lack of pragmatism, such as delays in access because of increased administrative burdens and possible deterrents for innovation in Europe. The paper underscores the need for policy adaptation and multi-stakeholder collaboration to ensure the legislative changes achieve equitable and timely access to oncology treatments across the EU.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"331-339"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037959/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Predicting Danish EQ-5D-5L Utilities Based on United Kingdom EQ-5D-3L Utilities for Use in Health Economic Models. 基于英国EQ-5D-3L用于卫生经济模型的效用预测丹麦EQ-5D-5L效用
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-02-22 DOI: 10.1007/s41669-025-00562-6
Einar B Torkilseng, Nathan Clarke, Liza Sopina, Lars Oddershede, Rasmus Trap Wolf, Rachael Lawrance, Andrew Trigg, Bryan Bennett, James W Shaw
{"title":"Predicting Danish EQ-5D-5L Utilities Based on United Kingdom EQ-5D-3L Utilities for Use in Health Economic Models.","authors":"Einar B Torkilseng, Nathan Clarke, Liza Sopina, Lars Oddershede, Rasmus Trap Wolf, Rachael Lawrance, Andrew Trigg, Bryan Bennett, James W Shaw","doi":"10.1007/s41669-025-00562-6","DOIUrl":"10.1007/s41669-025-00562-6","url":null,"abstract":"<p><strong>Objectives: </strong>Since 2021, the Danish Medicines Council recommends the use of the Danish EQ-5D-5L value set when estimating utilities. The aim of this research was to develop and validate an algorithm that can accurately predict mean Danish EQ-5D-5L utilities based on published mean UK EQ-5D-3L utilities.</p><p><strong>Methods: </strong>The study design incorporated a secondary analysis of patient-level UK EQ-5D-3L utility index scores from 11 oncology clinical trials. The EQ-5D-3L responses were mapped to EQ-5D-5L responses with the van Hout and Shaw preferred mapping algorithm. Model fitting and internal cross-validation were completed on a pooled dataset formed from eight trials including a total of 30,755 EQ-5D-3L responses. Three other trials were used for external validation (21,587 EQ-5D-3L observations).</p><p><strong>Results: </strong>From the model fitting phase, a simple linear model for mean utility scores exhibited good fit and was selected as the optimal prediction algorithm. External validation using the algorithm to predict mean Danish EQ-5D-5L utilities was excellent, with the largest absolute prediction error being 0.020 (observed UK EQ-5D-3L means: 0.628-0.835).</p><p><strong>Conclusions: </strong>The prediction algorithm developed in this research can increase analysts' ability to apply utilities aligned with the Danish EQ-5D-5L value set and guideline recommendations, reducing decision uncertainty. Many health technology assessment (HTA) institutions are transitioning from the EQ-5D-3L to the EQ-5D-5L in the coming years; therefore, prediction algorithms are likely of interest to additional HTA institutions in the near future. This study can provide a blueprint for future studies.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"433-443"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037449/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476537","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness Analysis of Daridorexant for the Pharmacological Treatment of Chronic Insomnia Disorder in Adults. 用于成人慢性失眠症药物治疗的 Daridorexant 成本效益分析。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-03-28 DOI: 10.1007/s41669-025-00567-1
Andrew H Briggs, François-Xavier Chalet, Jacie Cooper, Peter Graham, Stephen Palmer, Paul Miller, Andrew Walker, Berkeley Greenwood, Charles M Morin
{"title":"Cost-Effectiveness Analysis of Daridorexant for the Pharmacological Treatment of Chronic Insomnia Disorder in Adults.","authors":"Andrew H Briggs, François-Xavier Chalet, Jacie Cooper, Peter Graham, Stephen Palmer, Paul Miller, Andrew Walker, Berkeley Greenwood, Charles M Morin","doi":"10.1007/s41669-025-00567-1","DOIUrl":"10.1007/s41669-025-00567-1","url":null,"abstract":"<p><strong>Objective: </strong>Daridorexant 50 mg is recommended for treating chronic insomnia in England, Wales (NICE, 2023) and Scotland (Scottish Medicines Consortium, 2024). This study examines the model and cost-effectiveness profile that led to these positive reimbursements.</p><p><strong>Methods: </strong>The cost-effectiveness model integrated data from daridorexant 50 mg phase III trials (studies 301 and 303) and the National Health and Wellness Survey (NHWS). Clinical parameters were the Insomnia Severity Index (ISI) score and adverse events. Using the NHWS, ISI data were mapped to utility, healthcare resource use, and work productivity. Daridorexant 50 mg was priced at £1.40/day. The base-case time horizon was 1 year. A lifetime model explored long-term effects. Parameters, data inputs, structural uncertainty, and alternative scenarios are all presented.</p><p><strong>Results: </strong>In the 12-months model compared with placebo, daridorexant was estimated to have an incremental cost of £389 and generate an additional 0.024 quality-adjusted life-years (QALYs), resulting in an incremental cost-effectiveness ratio (ICER) of £16,300 per additional QALY from a health service perspective. Due to selective attrition, the ICER improved to £9580 per QALY for those continuing treatment for >12 months. Adopting a societal productivity perspective, daridorexant was estimated to offer £596 (£330-£896) total productivity savings versus £411/year in treatment costs, leading to a situation of dominance. Lifetime modeling improved the long-term cost effectiveness of daridorexant under the assumption that any waning of treatment effect led to further dropout.</p><p><strong>Conclusion: </strong>Daridorexant 50 mg is estimated to be a cost-effective pharmacological treatment for chronic insomnia disorder in adult patients.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"379-397"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037965/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143736117","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Publisher Correction: Pricing for Multi-Indication Drugs in the Italian Regulatory Context. 出版商更正:在意大利监管背景下多适应症药物的定价。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 DOI: 10.1007/s41669-025-00566-2
Maria Grazia Ursino, Annalisa Milano, Filippo Viti De Angelis, Eva Alessi, Francesco Trotta
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引用次数: 0
Burden of Disease and Cost of Illness of Triple-Negative Breast Cancer in Portugal. 葡萄牙三阴性乳腺癌的疾病负担和疾病费用。
IF 2
PharmacoEconomics Open Pub Date : 2025-05-01 Epub Date: 2025-02-11 DOI: 10.1007/s41669-024-00552-0
Joana Silva, Gabriela Sousa, Luís Costa, Margarida Brito, Sónia Oliveira, Bernardo Rodrigues, João Ferreira, Margarida Borges, Luís Miguel
{"title":"Burden of Disease and Cost of Illness of Triple-Negative Breast Cancer in Portugal.","authors":"Joana Silva, Gabriela Sousa, Luís Costa, Margarida Brito, Sónia Oliveira, Bernardo Rodrigues, João Ferreira, Margarida Borges, Luís Miguel","doi":"10.1007/s41669-024-00552-0","DOIUrl":"10.1007/s41669-024-00552-0","url":null,"abstract":"<p><strong>Background: </strong>Triple-negative breast cancer accounts for 15% of all breast cancer cases, and it has a lower survival rate and higher incidence of early recurrence, particularly during the first 10 years after diagnosis.</p><p><strong>Objective: </strong>This study aimed to estimate the cost and burden of triple-negative breast cancer among the female population in 2019 in Portugal from a societal perspective.</p><p><strong>Methods: </strong>The prevalence of triple-negative breast cancer was calculated using a cumulative incidence model on the basis of national epidemiological data. The burden of disease was expressed as disability-adjusted life years, including the years lost due to disability and years of life lost. Healthcare resource utilization was quantified with input from an expert panel, and costs were estimated on the basis of diagnosis-related groups. Indirect costs were established following the human capital approach and supported by inputs from an expert panel.</p><p><strong>Results: </strong>Considering a prevalence of 7052 cases of triple-negative breast cancer in 2019, the expert panel confirmed that approximately 24%, 29%, 28% and 19% of the patients were in stages I, II, III and IV, respectively. The burden of this disease in Portugal was estimated at 22,566 disability-adjusted life years per year, 94% of which resulted from premature deaths. The total annual cost was equal to €50,351,934, with direct and indirect costs representing 56% and 44%, respectively. The average cost per patient with triple-negative breast cancer was €7140. Direct costs accounted for €28 million and were associated mainly with triple-negative breast cancer locoregional stage treatment and follow-up (65%). Indirect costs represented €22 million and were largely linked to withdrawal from the job market (94%).</p><p><strong>Conclusion: </strong>Triple-negative breast cancer is an impactful disease with high humanistic and economic costs at the national level. The high mortality and low survival rates of this subtype mean that most disability-adjusted life years are due to years of life lost rather than years lost due to disability. Its prevalence is greater among women aged 45-49 years, suggesting a considerable burden regarding labour absenteeism and withdrawal from the job market.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"423-431"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037440/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143391333","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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