PharmacoEconomics Open最新文献

{"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}

{"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}

{"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}

{"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}

{"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}

{"title":"Publisher Correction: Pricing for Multi-Indication Drugs in the Italian Regulatory Context.","authors":"Maria Grazia Ursino, Annalisa Milano, Filippo Viti De Angelis, Eva Alessi, Francesco Trotta","doi":"10.1007/s41669-025-00566-2","DOIUrl":"10.1007/s41669-025-00566-2","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"499"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037920/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143425936","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}

{"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}

{"title":"Very Early Health Technology Assessment for Potential Predictive Biomarkers in the Treatment of Advanced Non-Small Cell Lung Cancer.","authors":"Leila-Sophie Otten, Alessandra I G Buma, Berber Piet, Rob Ter Heine, Michel M van den Heuvel, Valesca P Retèl","doi":"10.1007/s41669-025-00557-3","DOIUrl":"10.1007/s41669-025-00557-3","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;Immune checkpoint inhibitor (ICI)-containing treatment is currently prescribed as first-line treatment for all patients with advanced non-small cell lung cancer (NSCLC) without targetable driver mutations. However, only 30-45% of patients show no progression within 12 months after treatment start. Various biomarkers are being studied to save costly and potentially harmful treatment in non-responders. We evaluated the cost-effectiveness of implementing a hypothetical predictive biomarker for ICI-containing treatment response compared with standard of care (e.g., no implemented biomarker) for pembrolizumab-containing treatment in patients with advanced NSCLC in the Netherlands.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Materials and methods: &lt;/strong&gt;Standard-of-care-based and predictive-biomarker-based strategies were compared using Markov models for three first-line pembrolizumab-containing treatments depending on a patient's tumor programmed cell death ligand-1 (PD-L1) expression and histology. A Dutch healthcare system perspective was adopted. Assuming a receiver operating characteristic-area under the curve of 1.0 in identifying responders, alternative treatments were offered for non-responders in the predictive-biomarker-based strategy. Parameters and assumptions were based on real-world data from surveys, literature using a targeted search, expert opinion, and registries. Outcomes included differences in costs, survival (life years (LYs)), and survival corrected for health-related quality of life (QoL) quality-adjusted life-years (QALYs) between the predictive-biomarker- and standard-of-care-based strategy.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Implementing a predictive biomarker in pembrolizumab-carboplatin-paclitaxel treatment led to a mean survival reduction of 24 days (- 0.067 LYs) (18 days corrected for QoL (- 0.049 QALYs)), with cost savings of €22,606 compared with standard of care. Pembrolizumab monotherapy and pembrolizumab-pemetrexed-platinum treatments showed survival reductions of 4.5 and 3.9 months, respectively (3.6 and 2.8 months corrected for QoL), with cost savings of €24,345 and €28,456. Sensitivity analyses confirmed consistent cost savings and survival reductions. Survival losses were mainly observed due to the lower survival rates associated with the alternative first-line treatment options available for non-responders in the predictive-biomarker-based strategy within each pembrolizumab-containing treatment regimen. Pembrolizumab-carboplatin-paclitaxel treatment also showed survival gains under certain conditions related to QoL and survival estimates.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Our study highlights the importance of careful de-implementation of ICI-treatments in advanced NSCLC, balancing costs reductions and side effects without comprising survival. In the pembrolizumab-carboplatin-paclitaxel treatment regimen, the survival loss could be considered negligible. Future research should define acceptable tradeoffs and","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"471-485"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037958/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143060133","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}

{"title":"Pricing for Multi-Indication Drugs in the Italian Regulatory Context.","authors":"Maria Grazia Ursino, Annalisa Milano, Filippo Viti De Angelis, Eva Alessi, Francesco Trotta","doi":"10.1007/s41669-024-00555-x","DOIUrl":"10.1007/s41669-024-00555-x","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The authorization of new therapeutic indications for drugs already reimbursed by the Italian National Health Service (NHS) represents a matter of importance. This study aims to estimate the additional discount attributed to the extension of indications (EoIs) to explore the potential correlation between spending and negotiated discounts and to find specific factors (determinants) that impact on discount.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The study focused on drugs approved in Italy between 2003 and 2017 with at least four therapeutic indications, including the first approved and EoIs, with follow-up extended until 2021 to acquire all the information on the negotiation process that has been completed. Data were obtained from reimbursement and pricing dossiers, and negotiation assessments. Trends in the number of EoIs submitted and the additional discounts negotiated were analyzed, along with the relationship between the negotiated discount and subsequent drug expenditure. Determinants influencing the extent of the negotiated discount were assessed, including drug type, orphan drug designation, innovativeness status, number of EoIs, disease incidence and prevalence, estimated number of patients, revenue projections, availability of therapeutic alternatives, and efficacy outcomes. A Wilcoxon nonparametric test was used to evaluate the associations between determinants and the negotiated additional discount, with a significance level of 0.05.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The study identified nine medicines: five of these were used in onco-hematologic therapeutic areas, while the remaining four were immunosuppressants for dermatologic and/or rheumatologic conditions. These nine active substances accounted for 65 approved therapeutic indications, of which 50 were reimbursed by the Italian NHS, including the first indication; the analysis focused only on 40 reimbursed EoIs. The additional discount obtained for EoIs averages approximately 12.5% (95% CI 9.4-16.6%), with a median value of approximately 11%. This latter value was used as the threshold in the analysis of the determinants potentially impacting the negotiated discount amount. Discounts greater than 11% were significantly associated with EoI beyond the fifth and oncology drugs. The additional discount seemed small when compared with the increased spending.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;The study provides valuable insights into the negotiation outcomes for medicines with multiple therapeutic indications, particularly in onco-hematologic and immunosuppressive areas. The analysis revealed that additional discounts for EoIs averaged 12.5%, with a median of 11%, a value used to assess the impact of specific determinants. A discount higher than 11% was statistically correlated with drugs having more than five indications and oncology treatments, showing their influence in negotiations. However, the savings from discounts were modest relative to the increased drug","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"415-422"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024275","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}

{"title":"So Many Choices, So Little Value: Potential Savings from Selecting Cost-Effective Proton Pump Inhibitors.","authors":"Hye-Young Kwon","doi":"10.1007/s41669-025-00563-5","DOIUrl":"10.1007/s41669-025-00563-5","url":null,"abstract":"<p><strong>Background: </strong>Given that Korea lacks measures to promote cost-effective prescribing, this study investigated the efficiency of prescribers' decision-making when faced with the choice of 398 products containing 15 proton pump inhibitors (PPI).</p><p><strong>Objective: </strong>This study aimed to explore PPI prescribing patterns in ambulatory care over 10 years and elucidate prescribers' practices. Further analysis was conducted to estimate the achievable potential savings, assuming that the recommendation of first-choice drugs is based on the rational use of medicines.</p><p><strong>Methods: </strong>Retrospective PPI prescribing data from the ambulatory sector pertaining to medical services provided to the entire South Korean population from 2013 to 2022 were extracted from the Korean National Health Information Database; the data were analyzed to identify annual trends in PPI spending and utilization volume according to defined daily doses (DDDs). Unit price per DDD was calculated, and a preferred cost-effective choice with identical efficacy and safety profile was selected among PPIs. The potential savings were then simulated using Dirichlet distribution.</p><p><strong>Results: </strong>In ambulatory care, PPI drug costs increased by 13.0% per annum over 10 years. The preferred substances were esomeprazole (31.6%), rabeprazole (23.2%), and tegoprazan (16.5%), which are among the most expensive PPIs. However, the most cost-effective substance was dexlansoprazole (South Korean won [KRW] 522.7; standard deviation [SD] = 72.2; and median = 583). If dexlansoprazole is recommended as the first-choice PPI in the Korean context, the estimated cost savings would be 404.24 billion KRW, equivalent to 47.0% of the current PPI spending in ambulatory care.</p><p><strong>Conclusions: </strong>This study identified opportunities for substantial cost savings related to PPI prescribing. Guided decision-making toward cost-effective PPI prescribing would increase the efficiency of prescribing and the sustainability of the healthcare system.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"461-469"},"PeriodicalIF":2.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12037450/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414767","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}