PharmacoEconomics Open最新文献

{"title":"Measuring the Socioeconomic Impact of Cancer: A Systematic Review and Standardized Assessment of Patient-Reported Outcomes (PRO) Instruments.","authors":"Phu Duy Pham, Jasper Ubels, Rachel Eckford, Michael Schlander","doi":"10.1007/s41669-025-00568-0","DOIUrl":"https://doi.org/10.1007/s41669-025-00568-0","url":null,"abstract":"<p><strong>Background: </strong>A number of instruments have been developed to measure the socioeconomic impact (SEI) of cancer. A standardized comparison of the quality and content validity of these instruments is lacking. This study aimed to (1) conduct a standardized assessment of the quality of SEI instruments and (2) assess the content validity of these instruments using the conceptual framework developed by the Organization of European Cancer Institutes (OECI) for SEI analysis.</p><p><strong>Method: </strong>We identified articles measuring the SEI of cancer with ad hoc and/or validated instruments from an existing database. These articles were the initial pearls in a systematic review of published articles that applied and validated these instruments using the pearl-growing search strategy in PubMed, Web of Science, and Google Scholar databases. The Evaluating the Measurement of Patient-Reported Outcomes (EMPRO) tool was utilized to provide quantitative assessment and comparison of the quality of identified instruments. To examine content validity, we allocated each instrument's items against the themes and sub-themes of the established conceptual framework for SEI analysis.</p><p><strong>Results: </strong>We identified and investigated 21 validation studies using nine original instruments. The number of articles varied significantly among the identified instruments. The COmprehensive Score for financial Toxicity (COST) instrument was the most frequently used, validated in ten different settings, whereas some newer instruments have not been applied yet. This variation resulted in significant differences in EMPRO overall scores among these instruments. Regarding content validity, we found that not all themes of the OECI framework were covered by the content of the instruments.</p><p><strong>Conclusion: </strong>The quality and the application of instruments measuring the SEI of cancer varied significantly. The content of the instruments seems not to cover all related themes of the applied OECI framework in this study. Further studies are warranted to confirm the quality and content validity of the instruments measuring the SEI of cancer.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143780613","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":"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764038","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":"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143736117","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":"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":"https://doi.org/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":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664032","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":"Cost-Benefit Analysis of the Enhancing Men's Awareness of Testicular diseases (E-MAT) Feasibility Trial: A Virtual Reality Experience to Increase Testicular Knowledge and Self-Examination among Male Athletes.","authors":"Aileen Murphy, Ann Kirby, Federica De Blasio, Megan McCarthy, Frances Shiely, Josephine Hegarty, Martin P Davoren, Janas M Harrington, Gillian W Shorter, David Murphy, Billy O'Mahony, Eoghan Cooke, Michael J Rovito, Steve Robertson, Serena FitzGerald, Alan O Connor, Mícheál O Riordan, Mohamad M Saab","doi":"10.1007/s41669-025-00571-5","DOIUrl":"https://doi.org/10.1007/s41669-025-00571-5","url":null,"abstract":"<p><strong>Background: </strong>Virtual reality (VR) is potentially effective in raising awareness of testicular diseases, promoting self-examination and early help-seeking among men. This paper presents an early economic evaluation exploring the potential cost-effectiveness of Enhancing Men's Awareness of Testicular diseases (E-MAT)_VR, a VR interactive experience compared with E-MAT_E, electronic information, among male athletes Results from this economic evaluation will inform and support the design of a future randomized controlled trial (RCT).</p><p><strong>Methods: </strong>Results from an Irish feasibility trial (ClinicalTrials.gov identifier: NCT05146466) with 74 participants conducted in 2022 were employed. Benefits were measured in monetary units whereby the contingent valuation method was used to elicit participants' preferences through willingness-to-pay measures. A micro-cost analysis estimated the costs of the intervention and comparator and subsequent resource use. The costs and benefits of E-MAT_VR and E-MAT_E were compared to determine the net benefit. Sensitivity analyses were also conducted.</p><p><strong>Results: </strong>Base case analysis suggests participants were willing to pay €21.88 for E-MAT_VR and €11.16 for E-MAT_E. The total cost of E-MAT_VR was €104.09 and of E-MAT_E was €22.75 per participant. These estimates include capital and delivery costs, of which delivery costs were €25.02 and €22.40 for E-MAT_VR and E-MAT_E, respectively. A negative net benefit indicates E-MAT_VR was not cost-beneficial as delivered in the feasibility trial. Scenario analyses demonstrated reducing costs via delivery modifications increased the probability of E-MAT_VR being considered cost-effective. The cost-benefit analysis was feasible, response rates were acceptable, and willingness-to-pay estimates were stable.</p><p><strong>Conclusions: </strong>Economic evaluations alongside feasibility trials enable early economic evaluations, informing the design and conduct of a future RCT. E-MAT_VR had higher expected benefits (WTP) and costs than E-MAT_E, yielding a negative net benefit. Given the high cost of digital health interventions, investigating their cost-effectiveness early is important to inform and optimize resource allocation decisions. We present a series of scenarios to demonstrate how delivery modifications to reduce costs could improve the likelihood of E-MAT_VR being considered cost-effective.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664018","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":"Effects of Generic Entry on Market Shares and Prices of Originator Drugs: Evidence from Chinese Pharmaceutical Market.","authors":"Weimiao Li, Xiaoyu Li, Yaoguang He, Luwen Shi, Jing Chen","doi":"10.1007/s41669-025-00570-6","DOIUrl":"https://doi.org/10.1007/s41669-025-00570-6","url":null,"abstract":"<p><strong>Objectives: </strong>This article analyzes how market shares and prices for brand-name drugs are affected by generic competition in China.</p><p><strong>Methods: </strong>Data were collected for originator drugs that experienced initial generic entry between 2006 and 2016 from China Medicine Economic Information (CMEI), a large database of drug procurement records covering 699 tertiary hospitals across 28 provinces in mainland China. Quarterly utilization and expenditure data were collected. We compared the change of market share and price of originator drugs eight quarters after the first generic entry. General linear regression was performed to analyze the factors that influence the market share and price of originator drugs.</p><p><strong>Results: </strong>A total of 15 of 27 originator drugs maintained over 70% market share eight quarters after the first generic entry. In addition, 24 brand-name companies lowered prices with an average price decrease of 3% eight quarters after the first generic competitor appeared; prices for 3 drugs rose by an average of 0.62%. The median price ratio between originator and generic drugs was 1.76 when the first generic substitution entered, and the ratio became 2.00 eight quarters later. Regression showed that the number of generic manufacturers and time interval since the first generic entry exhibited a negative relation with the market share of originator drugs (P < 0.01; P < 0.01), and no relation with the prices of originator drugs (P = 0.61; P = 0.42).</p><p><strong>Conclusions: </strong>Generic medicines in China had only modest market penetration and little effect on originator drugs' prices eight quarters after first generic entry.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664043","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":"Healthcare Expenditure Trajectories in the Last 5 Years of Life: A Retrospective Cohort Study of Decedents with Advanced Cancer and End-Stage Organ Diseases.","authors":"Sheryl Hui-Xian Ng, Palvinder Kaur, Laurence Lean Chin Tan, Mervyn Yong Hwang Koh, Andy Hau Yan Ho, Allyn Hum, Woan Shin Tan","doi":"10.1007/s41669-025-00573-3","DOIUrl":"https://doi.org/10.1007/s41669-025-00573-3","url":null,"abstract":"<p><strong>Background: </strong>Patients with nonmalignant end-stage organ diseases often incur healthcare costs in the last year of life that are disproportionately higher than in the period prior. Studies on healthcare expenditure (HCE) trends in these patients have largely focused on the final year of life, and examining a longer-term trajectory could better support healthcare professionals to target the timing and methods of care management. In this study, we aim to describe the HCE trajectories of end-stage organ disease (ESOD) over the last 5 years of life, compared against advanced cancer (AC).</p><p><strong>Methods: </strong>We conducted a retrospective cohort study to profile decedents who had either a primary or secondary diagnosis of AC, advanced dementia, severe liver disease, as well as heart failure (HF), end-stage renal failure (ESRF), or respiratory failure (RF) in the last 5 years of their lives using a regional health system database in Singapore. Hospital-based HCE and utilization for each diagnosis group was reported cumulatively for 5 years, by year and by month. The proportion of the 5-year HCE incurred in each year was also reported.</p><p><strong>Results: </strong>Across all conditions, monthly HCE started to increase rapidly around 3 years prior to death, with 80% of the 5-year expenditure incurred in the same period. Expenditure among patients with ESODs other than dementia was £12,787 to £21,019 higher in comparison with patients with AC. Patients with RF incurred the highest 5-year HCE, driven by inpatient admissions.</p><p><strong>Conclusions: </strong>Our findings highlight the importance of examining HCE trends in ESOD and their cost drivers over multiple years prior to the last year of life to inform healthcare policy and to review care processes to ensure appropriate and efficient end-of-life (EOL) care.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664067","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":"Cost-Utility Analysis of LifeVest® in Post-Myocardial Infarction Patients at Risk of Sudden Cardiac Death in England.","authors":"Vasileios Kontogiannis, Farai Goromonzi, Brigitte Both, Frank Semrau, Michael Branagan-Harris, Jowan Atkinson, Paul R Roberts, Mehdi Javanbakht","doi":"10.1007/s41669-024-00553-z","DOIUrl":"10.1007/s41669-024-00553-z","url":null,"abstract":"<p><strong>Background: </strong>Patients with a left ventricular ejection fraction ≤ 35% are at increased risk of sudden cardiac death (SCD) within the first months after a myocardial infarction (MI). The wearable cardioverter defibrillator (WCD) is an established, safe and effective solution which can protect patients from SCD during the first months after an MI, when the risk of SCD is at its peak. This study aimed to evaluate the cost-effectiveness of WCD combined with guideline-directed medical therapy (GDMT) compared to GDMT alone, after MI in the English National Health Service (NHS).</p><p><strong>Methods: </strong>A multi-state Markov model, with a hypothetical cohort of 1000 patients, was developed to evaluate WCD + GDMT compared with GDMT alone, over a lifetime time horizon. Model input parameters were obtained from the pivotal randomised controlled trial and literature. The outcomes were costs and quality-adjusted life-years (QALYs), discounted at 3.5% annually, with overall results presented as an incremental cost-effectiveness ratio (ICER).</p><p><strong>Results: </strong>The cost-effectiveness analysis demonstrated that WCD + GDMT is potentially a cost-effective option with an ICER of £23,024 per QALY gained, which is in the acceptable willingness to pay threshold (WTP) range of £20,000-£30,000 set by the National Institute for Health and Care Excellence (NICE) in England. Results of probabilistic sensitivity analysis (PSA) indicated that WCD + GDMT has 89.3% and 23% probability of being cost-effective at WTP thresholds of £30,000 and £20,000, respectively.</p><p><strong>Conclusions: </strong>Implementation of WCD in patients post-MI is potentially a cost-effective use of resources for the NHS and improves clinical outcomes amongst adherent patients and in circumstances where implantable cardioverter defibrillators are not indicated by the guidelines.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"301-312"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11865419/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029183","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":"Health-Economic Modelling of Improved Behavior in Insulin Injection Technique in Belgium.","authors":"Kristof Theys, Sofie Vermander, Lieven Annemans, Christophe De Block, Michel P Hermans, Imke Matthys, Frank Nobels, Trung Nguyen, Vanessa Preumont, Katerina Zakrzewska, Frank Vanderdonck","doi":"10.1007/s41669-024-00547-x","DOIUrl":"10.1007/s41669-024-00547-x","url":null,"abstract":"<p><strong>Background: </strong>Adequate insulin injection technique (IIT) is crucial to optimize the efficacy of diabetes therapy. Widespread non-practice of injection-site rotation and frequent reuse of insulin pen needles (PN) promote high rates of lipohypertrophy (LH) among people living with diabetes (PwD). LH is associated with increased insulin requirement and suboptimal insulin absorption leading to worsened glycemic control and increased risk for hypoglycemia. Avoiding out-of-the-pocket patient costs of PN could reduce PN reuse, thereby limiting its contribution to LH occurrence.</p><p><strong>Objectives: </strong>A model was developed to compute the impact of a behavior shift in reuse on clinical and economic outcomes for type 1 and insulin-treated type 2 diabetes populations in Belgium.</p><p><strong>Methods: </strong>Patient populations were characterized by treatment-specific characteristics and grouped by their frequency of PN replacement. The intervention was modelled to cause a change in reuse frequency, with the effects propagating downstream of the model. Model and input parameters were based on literature research and expert opinions from a Delphi panel, since available data was found to be limited, incomplete or inconsistent and assumptions were needed.</p><p><strong>Results: </strong>Using the current situation as comparator, this analysis showed a reduction of healthcare expenditures following an improvement in IIT. Considering a 5-year time horizon, this study yields potential savings of 52.6 million euros (28.1-77.9 million euros) when 55% of PwD improve PN reuse behavior.</p><p><strong>Conclusion: </strong>Our model shows that even in an era of technological advances and established diabetes care, lack of adherence to correct IIT has an important impact on economic and health outcomes of PwD in Belgium.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"259-270"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11865415/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142829627","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":"Clinical Complications and Healthcare Resource Utilization Associated with Conventional Management of Sickle Cell Disease with Recurrent Vaso-occlusive Crises and Transfusion-Dependent β-Thalassemia in Germany.","authors":"Chuka Udeze, Nanxin Li, Colin Kunzweiler, Jessica Baldwin, Petra Tuzin, Sebastian Dietmar Zingel, Céline Vetter, Silvia Dombrowski, Elena Georgiadou-Schmidt, Aranzazu Alba, Roland Meisel","doi":"10.1007/s41669-024-00550-2","DOIUrl":"10.1007/s41669-024-00550-2","url":null,"abstract":"<p><strong>Objective: </strong>The purpose of this study was to describe clinical complications and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and patients with transfusion-dependent β-thalassemia (TDT) in Germany.</p><p><strong>Methods: </strong>The Betriebskrankenkasse (BKKs) Database was used to identify patients with SCD or TDT. To be eligible for inclusion, patients with SCD were required to have ≥ 2 VOCs/year in any two consecutive years and ≥ 12 months of available data before and after the index date (second VOC in the second consecutive year). Patients with TDT were required to have ≥ 8 red blood cell transfusions (RBCTs) in any 12-month period and ≥ 12 months of available data after the index date (first RBCT). Clinical and HCRU outcomes were analyzed during follow-up.</p><p><strong>Results: </strong>Overall, 84 patients with SCD with recurrent VOCs and 68 patients with TDT were identified in the BKKs database. Among patients with SCD with recurrent VOCs, the most prevalent complications were retinopathy (45.2%), multisystem organ disease/failure (40.5%), and mental health complications (31.0%); among patients with TDT, they were endocrine (69.1%) and cardiopulmonary (55.9%) complications and malignancies (44.1%). Patients with SCD experienced a mean of 4.0 (standard deviation [SD] 3.9) VOCs and 1.9 (SD 2.5) hospitalizations per patient per year (PPPY) during follow-up. Patients with TDT had a mean (SD) of 16.4 (11.2) RBCTs and 59.4 (40.8) outpatient visits PPPY.</p><p><strong>Conclusions: </strong>Patients with SCD with recurrent VOCs or TDT in Germany experience significant clinical complications and HCRU.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"291-300"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11865411/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142927647","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}