PharmacoEconomics Open最新文献

{"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468715","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}

{"title":"Do We Understand Unmet Need? A Proposal to Use Length-Of-Life Equivalent (LOLE) as a Patient-Centric Measure of Unmet Need.","authors":"Kevin Marsh, Robert F Reynolds, Linda Nelsen, Stephen Watt, Omar A Escontrías, Brett Hauber","doi":"10.1007/s41669-025-00560-8","DOIUrl":"https://doi.org/10.1007/s41669-025-00560-8","url":null,"abstract":"<p><p>Many decision-makers have emphasized the importance of leveraging patient experience data to measure unmet need. However, there is no standardized, patient-centric unmet need measure that formalizes how the value judgements inherent in such a measure should be made. Several initiatives have identified measuring unmet need as one of the primary uses of patient preference data. After reviewing how decision-makers define unmet need, this paper proposes that a thresholding method could be used to generate a standardized, patient-centric, disease-agnostic, quantitative unmet need estimate, length of life equivalent (LOLE). LOLE would address some of the limitations of current methods, including facilitating capture of the impact of disease beyond health-related quality of life, and being more sensitive to the impact of a disease on patients. However, the acceptability of LOLE raises questions for decision-makers, including: Is length of life equivalence the best common metric in which to express unmet need? Is it appropriate to rate a disease as having no unmet need if patients are unwilling to trade off life expectancy for improvements in their quality of life? Can LOLE be estimated for more complex disease profiles? Is thresholding the appropriate method to use to estimate LOLE? How should LOLE be integrated into decision-making, including the level of LOLE that defines different levels of unmet need? Further work could usefully address these questions with decision-makers.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441786","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}

{"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":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143425936","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}

{"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414767","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}

{"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143391333","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}

{"title":"COVID-19 Vaccine Preferences in China: A Comparison of Discrete Choice Experiment and Profile Case Best-Worst Scaling.","authors":"Enxue Chang, Yanni Jia, Xiaoying Zhu, Lunan Wang, Ying Yan, Kejun Liu, Weidong Huang","doi":"10.1007/s41669-025-00559-1","DOIUrl":"https://doi.org/10.1007/s41669-025-00559-1","url":null,"abstract":"<p><strong>Objectives: </strong>Little is known about the diversity of residents' preferences for COVID-19 vaccines during the time when COVID-19 management was downgraded in China. This study aims to investigate these preferences using discrete choice experiment (DCE) and profile case best-worst scaling (BWS-2), and to assess the concordance between these two methods.</p><p><strong>Methods: </strong>Chinese residents recruited for the online survey were asked to evaluate COVID-19 vaccine profiles through both DCE and BWS-2 from April to July 2023. Attributes included effectiveness, duration of protection, risk of severe adverse events (degree), the total out-of-pocket (OOP) cost, brand, and the vaccination method. We utilized conditional regression and mixed logit regression models to estimate the preference levels for potential attributes. To assess preference concordance between the two methods, re-scaling and the Spearman correlation test were used. Additionally, subgroup analysis was conducted to determine the most suitable method for different population groups, categorized by vaccine hesitancy and risk level.</p><p><strong>Results: </strong>A total of 438 (71.22%) respondents were included. A similar pattern was found in the DCE and BWS-2 methods, with the respondents having a strong preference for 90% vaccine effectiveness. However, the methods diverged in other preferences; DCE favored domestic brands and low severe adverse event risk, while BWS-2 preferred moderate risk and three years of protection. Concordance assessment, including Spearman's correlation and linear regression, showed no significant correlation and poor concordance between the methods, underscoring these differences. Preference heterogeneity is revealed among different groups; however, effectiveness remained the most important attribute for all subgroups of the population. Oral vaccination was the preferred option for both the vaccine-hesitant and high-risk groups.</p><p><strong>Conclusion: </strong>This study offers new insights into the varying preferences for COVID-19 vaccines among Chinese residents following the downgrading of pandemic management measures. The findings underscore the need for diverse strategies in vaccine policy design. Special emphasis should be placed on vaccine attributes that align with public priorities, such as high effectiveness and low risk levels, to enhance vaccine uptake.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075287","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}

{"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":""},"PeriodicalIF":2.0,"publicationDate":"2025-01-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143060133","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}

{"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040664","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}

{"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024279","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}

{"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":""},"PeriodicalIF":2.0,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024275","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}