PharmacoEconomics Open最新文献

{"title":"Modernizing Newborn Screening in the Genomic Era: Importance of Health-Related Quality of Life.","authors":"Ellen Kim DeLuca, Ann Chen Wu, Kurt D Christensen, Davene R Wright, Jennifer Yeh, Hadley Stevens Smith","doi":"10.1007/s41669-024-00528-0","DOIUrl":"10.1007/s41669-024-00528-0","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"787-792"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499486/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142366102","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 of Dupilumab and Oral Janus Kinase Inhibitors for the Treatment of Moderate-to-Severe Atopic Dermatitis in Singapore.","authors":"Clarence Ong, Jamaica Briones, Zhi Zhen Lim, Nisha Suyien Chandran, Haur Yueh Lee, Benny Kaihui Li, Yik Weng Yew, Hwee-Lin Wee","doi":"10.1007/s41669-024-00507-5","DOIUrl":"10.1007/s41669-024-00507-5","url":null,"abstract":"<p><strong>Background: </strong>Atopic dermatitis (AD) affects both adults and children, impacting their quality of life and productivity; however, traditional systemic treatments such as cyclosporine have limitations. Emerging novel systemic interventions, including monoclonal antibodies and Janus kinase (JAK) inhibitors, have been shown to improve patient outcomes.</p><p><strong>Objective: </strong>This study evaluates the cost-effectiveness of novel systemic interventions for moderate-to-severe AD in adults compared with the best supportive care (BSC) in Singapore.</p><p><strong>Methods: </strong>The economic evaluation used a hybrid model consisting of a decision tree and Markov model. Treatment responses at 16 weeks were based on a network meta-analysis that was developed specifically for this study. Long-term response, discontinuation rates, episodes of flares and treatment-emergent adverse events were obtained from key dupilumab, abrocitinib, baricitinib and upadacitinib trials. The study had a 5-year time horizon and considered the healthcare payer's perspective. Sensitivity and scenario analyses were performed as well.</p><p><strong>Results: </strong>Baricitinib 4 mg and 2 mg have the lowest incremental cost-effectiveness ratios, at Singapore dollars (S$) 60,730/quality-adjusted life-year (QALY) and S$66,842/QALY, respectively. Upadacitinib 30 mg offers the highest incremental QALY gain, while baricitinib 2 mg offers the least. The cost of the intervention drugs accounted for the highest proportion of the overall expenses (68-93%) for those in the maintenance state. Other influential factors within the model included (1) the incremental utility derived from intervention response; (2) the probability of achieving Eczema Area and Severity Index 75 (EASI-75) with BSC; and (3) the relative risk of achieving EASI-75 with the interventions. In a scenario where the cost of all drugs is matched to the lowest-priced drug, the top three cost-effectiveness interventions are dupilumab, upadacitinib 30 mg and abrocitinib 200 mg, respectively.</p><p><strong>Conclusion: </strong>The interventions are not found to be cost-effective at their existing prices when compared with BSC. Ideally, a composite score of treatment success and quality-of-life scores ought to be included, but such data were unavailable. Future research should consider conditional discontinuation data and long-term outcomes when such data become accessible.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"809-822"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499508/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141603925","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":"Estimation of the Clinical, Economic, and Social Burden of Stage IV Non-Small Cell Lung Cancer in Mexico.","authors":"Denisse Añorve Bailon, Javier Picó-Guzmán, Sergio Cifuentes, Rogelio Trejo, Jeronimo Rodríguez Cid, Juan Jose Juarez-Vignon Whaley, Alan Alexis Heredia Zepeda, Raquel Gerson, Christian Patricio Camacho-Limas, José Fabián Martínez-Herrera, Diana Bonilla Molina, Efraín Camarín Sánchez, Daniela Shveid Gerson","doi":"10.1007/s41669-024-00514-6","DOIUrl":"10.1007/s41669-024-00514-6","url":null,"abstract":"<p><strong>Introduction: </strong>Lung cancer continues to be the leading cause of death among cancer patients worldwide. This study aimed to estimate the clinical, economic, and social burdens of stage IV non-small cell lung cancer (NSCLC) in private and public healthcare centers in Mexico, utilizing real-world evidence.</p><p><strong>Methods: </strong>The study population included patients >18 years of age diagnosed with stage IV NSCLC who received cancer treatment at the Centro Médico Nacional Siglo XXI (IMSS), the Centro Médico Nacional \"20 de Noviembre\" (ISSSTE), the Mexican Institute of Respiratory Diseases (INER), and the Medical Center ABC (American British Cowdray) from 1 January 2019 to 31 December 2020. The analysis included evaluation of epidemiological data, treatment regimens, and clinical outcomes, and emphasized pharmacological and non-pharmacological treatments, including detailed follow-up investigations, as part of comprehensive clinical management. Additionally, the study assessed the social burden through variables such as working-age absenteeism and presenteeism and caregiver productivity loss, as well as economic burden, considering both clinical and social components, with costs adjusted to 2022 Mexican pesos (MXN) values.</p><p><strong>Results: </strong>A total of 188 patients with metastatic NSCLC were studied. The main type of NSCLC tumor found in the sample was adenocarcinoma (81%). Treatment regimens included pharmacological treatments (78%), non-pharmacological treatments (25%), and palliative care (24%). Complications were present in 73% of the cohort, while 60% presented adverse events. Clinical management costs of up to MXN1,001,579 per patient in the public sector and MXN2,140,604 in the private sector were reported. It was estimated that working-age patients lose 84-335 days yearly due to absenteeism and presenteeism, while caregivers report a productivity loss equivalent to 13-30 days due to the management of NSCLC patients. These indirect costs of NSCLC contribute to the social burden. A working-age patient with stage IV disease is associated with an average indirect cost of MXN49,731-178,287 in public institutions, while in private institutions, the cost elevates to MXN438,103.</p><p><strong>Conclusions: </strong>This study highlights the substantial clinical, economic, and social burdens of stage IV NSCLC in Mexico, revealing significant disparities between public and private healthcare sectors. It underscores the urgent need for standardized practices and equitable care across all systems.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"869-885"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499576/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897955","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":"Economic Impact of Bladder Cancer in the USA.","authors":"Otavio Clark, Tulio Sarmento, Anthony Eccleston, Julia Brinkmann, Renato Picoli, Vamsi Daliparthi, Jorine Voss, Sanjana Chandrasekar, Allison Thompson, Jane Chang","doi":"10.1007/s41669-024-00512-8","DOIUrl":"10.1007/s41669-024-00512-8","url":null,"abstract":"<p><strong>Introduction: </strong>Incidence and mortality for bladder cancer has changed very little over the past 20 years. Approximately 40% of patients with high-risk nonmuscle invasive bladder cancer eventually recur/progress. It is important to understand the economic impact of disease recurrence/progression in bladder cancer. Our aim was to estimate and understand the direct costs associated with the treatment of bladder cancer from the payer's perspective in the USA, in the year of 2021, including costs for both newly diagnosed bladder cancer (stages 0a-IV) and recurrent patients.</p><p><strong>Methods: </strong>An economic model was constructed to calculate the number of patients receiving each treatment modality at every stage of disease and their respective costs. Epidemiological data were based on the CancerMPact Patient Metrics (PM) database and treatment modality data retrieved from CMP Treatment Architecture (TA), 2021 version. Resource utilization and costs were obtained from medical literature and public data sources. Only direct costs were considered.</p><p><strong>Results: </strong>There were an estimated 83,532 newly diagnosed patients with bladder cancer of all stages in 2021 with a projected total cost of treatment of ~$2.6 billion. Average cost per newly diagnosed patient varied from $19,521 (stage 0a) to $169,533 (metastatic disease). Cost profile differed substantially among the stages of disease. For the 75,760 patients that were expected to have a recurrence in 2021, an additional cost of ~$3.9 billion was estimated at an average cost per patient of $52,179. The expected total cost to treat newly diagnosed and newly recurrent patients is reported in this model, with the total cost in 2021 estimated to exceed $6.5 billion.</p><p><strong>Conclusions: </strong>Treatment and resource costs increase for bladder cancer as the disease recurs/progresses. More effective treatments that can delay recurrence/progression may reduce the economic burden associated with bladder cancer.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"837-845"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499469/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141996295","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":"The Economic Impact of Introducing RefluxStop for Refractory Gastroesophageal Reflux Disease on the Italian Healthcare System.","authors":"Sam Harper, Muralikrishnan Kartha, Stuart Mealing, Maurizio Pavanello, Luigi Bonavina","doi":"10.1007/s41669-024-00521-7","DOIUrl":"10.1007/s41669-024-00521-7","url":null,"abstract":"<p><strong>Introduction: </strong>Gastroesophageal reflux disease (GERD) is a common ailment associated with troublesome symptoms. The standard of care in Italy involves initial treatment with proton pump inhibitor (PPI)-based medical management or laparoscopic Nissen fundoplication (LNF) for patients unwilling to continue or intolerant of long-term PPI therapy. RefluxStop is a novel medical device, intended for laparoscopic implantation, that has recently proven to be an efficacious and cost-effective treatment option for patients with GERD. This analysis aims to describe the short-term budget impact of introducing RefluxStop as a GERD treatment option within the Italian National Health Service (SSN).</p><p><strong>Methods: </strong>A model adherent to international best practice recommendations was developed to estimate the budget impact of introducing RefluxStop over a 5-year time horizon. Two scenarios were considered: one without RefluxStop (i.e., comprising PPI therapy, LNF, and magnetic sphincter augmentation using the LINX system); and one with RefluxStop (i.e., addition of RefluxStop to the three treatment options previously mentioned). Clinical benefits and costs associated with each intervention were included in the analysis.</p><p><strong>Results: </strong>Over 5 years, the introduction of RefluxStop resulted in avoidance of 95 surgical failures, 11 reoperations, and 64 endoscopic esophageal dilations. Introduction of RefluxStop resulted in an almost neutral impact on the existing budget with a 0.316% increase in the annual Italian SSN spending on GERD treatment.</p><p><strong>Conclusion: </strong>Introduction of RefluxStop as a GERD treatment option in Italy is likely to be associated with substantial clinical benefits and a marginal budget impact.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"935-943"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499547/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142073460","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":"Clinicians' Preferences for Sphingosine-1-Phosphate Receptor Modulators in Multiple Sclerosis Based on Clinical Management Considerations: A Choice Experiment.","authors":"Alexander Keenan, Chiara Whichello, Hoa H Le, David M Kern, Gabriela S Fernandez, Vicky Turner, Anup Das, Matt Quaife, Amy Perrin Ross","doi":"10.1007/s41669-024-00510-w","DOIUrl":"10.1007/s41669-024-00510-w","url":null,"abstract":"<p><strong>Background: </strong>Four sphingosine-1-phosphate receptor (S1PR) modulators are currently available in the USA for treating relapsing forms of multiple sclerosis (MS). These S1PR modulators have similar efficacy. Clinicians may therefore consider other factors, such as clinical management considerations, when distinguishing among treatments. This study estimated which S1PR modulator clinicians would choose on the basis of a treatment's clinical management and quantified how individual aspects of clinical management might drive this choice.</p><p><strong>Methods: </strong>A multi-criteria decision analysis (MCDA) was conducted on the basis of clinical management preferences elicited in a discrete choice experiment (DCE) and real-world clinical management profiles of the S1PR modulators currently available to treat relapsing forms of MS (fingolimod, ozanimod, ponesimod, siponimod). The DCE was completed by neurologists in the USA experienced in treating MS and included eight clinical management attributes: first-dose observations, genotyping, liver function tests, eye exams, drug-drug interactions, interactions with antidepressants, interactions with foods high in tyramine, and immune system recovery time. Attribute levels were selected on the basis of S1PR modulator product labels. In the MCDA, partial MCDA scores were created for each attribute and summed to produce an overall MCDA score for each S1PR modulator.</p><p><strong>Results: </strong>The DCE was completed by 200 neurologists. The overall MCDA score was highest for ponesimod (4.78 points), followed by siponimod (4.10 points), fingolimod (3.61 points), and ozanimod (2.38 points). Having fewer drug-drug interactions contributed most to the overall scores (up to 1.56 points), followed by having no first-dose observations (0.95 points), the shortest immune system recovery time (0.94 points), and not interacting with foods high in tyramine (0.86 points).</p><p><strong>Conclusion: </strong>When considering clinical management convenience, the average US-based neurologist treating MS is likely to choose ponesimod over siponimod, fingolimod, or ozanimod. The strongest driver of preferences was the number of drug-drug interactions. This information can help inform recommendations for the treatment of MS and facilitate shared decision-making between clinicians and patients.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"857-867"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142081126","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-Utility Analysis of Adjuvant Olaparib for Germline BRCA1/2-Mutated, High-Risk HER2-Negative Early Breast Cancer in Spain.","authors":"Sergio Cedillo, Almudena González-Domínguez, Yoana Ivanova-Markova, Rafael López López, Sara López-Tarruella Cobo, José Alberto Peña Pedrosa","doi":"10.1007/s41669-024-00518-2","DOIUrl":"10.1007/s41669-024-00518-2","url":null,"abstract":"<p><strong>Objective: </strong>Here we estimate the cost-effectiveness of olaparib in the Spanish National Health Service (SNHS) as adjuvant treatment of early germline mutations in the BRCA1/2 genes (gBRCAm) HER2-negative (HER2neg) breast cancer (BC) with high risk of recurrence.</p><p><strong>Methods: </strong>A semi-Markov model was adapted to the Spanish healthcare setting, using the perspective of the SNHS, and a lifetime horizon. Two scenarios were compared: receiving olaparib versus standard of care (SoC) treatment. The model comprised five health states and included the clinical results of the OlympiA trial, along with the direct healthcare costs associated with the use of early BC and subsequent treatment resources (€2023). A discount rate of 3% was applied for future cost and quality-of-life outcomes. A probabilistic sensitivity analysis (PSA) was carried out.</p><p><strong>Results: </strong>The introduction of olaparib as adjuvant treatment for patients with early gBRCAm HER2neg BC with high risk of recurrence could involve an incremental cost of €44,273 and €50,164, with an improvement of 1.14 and 1.28 quality-adjusted life years (QALYs) for hormone receptor-positive (HR⁺) and triple-negative (TN) patients, respectively. Therefore, adjuvant olaparib could be cost-effective for early gBRCAm HER2neg BC, with an incremental cost-effectiveness ratio of €38,839/QALY and €39,084/QALY for HR⁺ and TN patients, respectively. The results from the PSA showed that 75.7% and 82.2% of the simulations fell below the €60,000/QALY threshold.</p><p><strong>Conclusions: </strong>Olaparib as adjuvant treatment could be cost-effective in gBRCAm patients with early HER2neg BC in Spain.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"887-896"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499548/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004967","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":"Comparison of Two Financial Incentives to Encourage the Use of Adalimumab Biosimilars: Results of a French Experiment Close to Clinicians.","authors":"Marion Tano, Pascal Paubel, Matthieu Ribault, Albane Degrassat-Théas","doi":"10.1007/s41669-024-00505-7","DOIUrl":"10.1007/s41669-024-00505-7","url":null,"abstract":"<p><strong>Background: </strong>In 2018, the French government introduced two mutually exclusive financial incentives to increase etanercept and insulin glargine biosimilar use. The general case redirects 20% of the price difference between the reference product and its biosimilars to hospitals for every biosimilar dispensed in community pharmacies from hospital prescriptions. The experimental case redirects 30% to prescribing clinical units after hospital selection. Adalimumab was added to these incentives in 2019, after its first biosimilar was launched.</p><p><strong>Objective: </strong>This retrospective observational study aimed to compare both general and experimental incentives after 19 months to assess the impact of directly incentivizing clinical units for adalimumab biosimilars.</p><p><strong>Method: </strong>The monthly number of adalimumab boxes dispensed in community pharmacies was linked to the corresponding hospital's prescription using IQVIA Xponent data from November 2017 to October 2020. The monthly mean rate of adalimumab biosimilars was compared between incentive groups and subgroups depending on hospitals' prior experience with the etanercept experimental case.</p><p><strong>Results: </strong>General case hospitals had a significantly lower mean biosimilar uptake (16.7% vs 23.2%, p = 0.029) than those included in the experimental case. Twenty-three of the 40 hospitals in the experimental case and ten out of the 91 hospitals studied in the general case had already taken part in the etanercept experiment. Biosimilar uptake was higher, but not statistically significant, for hospitals with prior experience in the adalimumab general case group (p = 0.086).</p><p><strong>Conclusions: </strong>This study confirmed that incentivizing close to physicians was more effective in increasing the biosimilar rate. It also suggested that previous incentive experience positively influenced biosimilar penetration.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"897-909"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499575/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141492949","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 of Three Different New-Generation Drug-Eluting Stents in the Randomised BIO-RESORT Trial at 3 Years.","authors":"Eline H Ploumen, Martijn J Oude Wolcherink, Rosaly A Buiten, Tineke H Pinxterhuis, Carine J M Doggen, Carl E Schotborgh, Peter W Danse, Martijn Scholte, K Gert van Houwelingen, Paolo Zocca, Xavier G L V Pouwels, Clemens von Birgelen","doi":"10.1007/s41669-024-00539-x","DOIUrl":"https://doi.org/10.1007/s41669-024-00539-x","url":null,"abstract":"<p><strong>Background and objective: </strong>Evidence on health economic outcomes for percutaneous coronary intervention (PCI) comparing different contemporary drug-eluting stents (DES) with each other is scarce, as most previous randomised DES trials did not assess such aspects. This prespecified health economic evaluation of the Comparison of Biodegradable Polymer and Durable Polymer Drug-Eluting Stents in an All Comers Population (BIO-RESORT) trial aimed to compare at 3-year follow-up both health effects and costs of PCI with one of three new-generation drug-eluting stents (DES) in patients with obstructive coronary artery disease.</p><p><strong>Methods: </strong>The randomised BIO-RESORT trial assessed in 3514 patients the ultrathin-strut biodegradable polymer Orsiro sirolimus-eluting stent (SES) and very-thin-strut Synergy everolimus-eluting (EES) stent versus the thin-strut durable polymer Resolute Integrity zotarolimus-eluting stent (ZES). In the current analysis, we used the perspective of a health insurer in the Netherlands. The main endpoints were quality-adjusted life years (QALYs), and costs for each treatment strategy. Bootstrapping with 5000 resamples was performed to capture the uncertainty of results.</p><p><strong>Results: </strong>Mean QALYs for each stent group were 2.566 for the SES, 2.551 for the EES, and 2.550 for the ZES. Mean costs per strategy were €14,670 for the SES, €14,946 for the EES, and €15,069 for the ZES. The SES had the highest probability of being cost-effective for every willingness-to-pay threshold up to €100,000 per QALY. Furthermore, in 79% of modelling scenarios, the SES was more effective and cheaper than ZES.</p><p><strong>Conclusion: </strong>At 3-year follow-up, PCI with the SES had the highest probability of being cost-effective due to greater effectiveness and lower costs compared with the ZES and EES. These findings suggest that, due to the overall high volume of coronary stenting in clinical practice, use of this SES could result in substantial cost savings, complemented by slight additional health benefits.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142546692","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":"Mortality, Clinical Complications, and Healthcare Resource Utilization Associated with Managing Transfusion-Dependent β-Thalassemia and Sickle Cell Disease with Recurrent Vaso-occlusive Crises in Italy.","authors":"Chuka Udeze, Melania Dovizio, Chiara Veronesi, Luca Degli Esposti, Nanxin Li, Thi Xuan Mai Patricia Dang, Gian Luca Forni","doi":"10.1007/s41669-024-00532-4","DOIUrl":"https://doi.org/10.1007/s41669-024-00532-4","url":null,"abstract":"<p><strong>Objective: </strong>To examine the clinical burden and healthcare resource utilization (HCRU) among patients with transfusion-dependent β-thalassemia (TDT) and patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in Italy.</p><p><strong>Methods: </strong>Eligible patients were identified from an administrative claims database from 1 January 2010 and 1 February 2019. Patients with TDT had ≥ 1 iron chelation treatment, ≥ 8 red blood cell transfusions (RBCTs) during any 12-month period, and ≥ 12 months of available data pre- and post-index (i.e., first RBCT claim). Patients with SCD with recurrent VOCs had ≥ 2 VOCs/year in ≥ 2 consecutive years and ≥ 12 months of available data pre- and post-index (second VOC claim in the second of 2 consecutive years). Patients were propensity score matched to five controls by age, sex, geographic area, and index year. Clinical and HCRU outcomes were evaluated post-index.</p><p><strong>Results: </strong>In total, 214 patients with TDT and 111 patients with SCD with recurrent VOCs were matched to 1070 and 555 controls, respectively. Both patient groups had substantially higher mortality rates than controls (TDT: 4.8 versus 0.8 deaths per 100 person-years; SCD: 1.6 versus 0.4 deaths per 100 person-years). Clinical complications were prevalent in both patient groups. Compared with controls, both patient groups had significantly higher mean rates of all-cause hospitalizations (TDT: 1.4 versus 0.1; SCD: 2.0 versus 0.1) and outpatient services (TDT: 21.9 versus 1.6; SCD: 6.2 versus 1.0) per patient per year (all: p < 0.05).</p><p><strong>Conclusions: </strong>Management of TDT and SCD in Italy is associated with significant clinical and health system burden, highlighting the need for new treatments that eliminate RBCTs and VOCs.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142522637","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}