Pub Date : 2025-09-17eCollection Date: 2025-01-01DOI: 10.36469/001c.143065
Maria J Ospina-Fadul, Pedro Kremer, Florence Haruna, Fred Adomako-Boateng, Kenneth Fosu Oteng, Diana N Tsali
Background: In sub-Saharan Africa, low antenatal care (ANC) coverage and limited access to facility-based deliveries remain key drivers of adverse maternal and newborn health (MNH) outcomes. Inadequate service provision at health facilities and insufficient care-seeking behavior are exacerbated by supply chain inefficiencies that restrict access to essential maternal health commodities. Aerial logistics (centralized storage and drone delivery) has shown promise as a novel approach to addressing these logistical challenges and supporting maternal health service delivery, but its cost-effectiveness has not been evaluated.
Objectives: This study evaluates the cost-effectiveness of aerial logistics as an intervention for MNH. It builds on previously observed programmatic effects (increases in ANC visits, facility-based deliveries, and reductions in maternal mortality in Ghana's Ashanti Region) to model downstream health outcomes and estimate incremental economic value.
Methods: Using microsimulation and published epidemiological parameters, the study models additional health outcomes resulting from increased service utilization among 11 249 pregnant women, including reductions in low birth weight, postpartum hemorrhage, neonatal mortality, and early-onset neonatal sepsis. Alongside the observed maternal mortality reduction, all outcomes are translated into life-years saved and discounted disability-adjusted life-years (DALYs) averted. Cost estimates are based on real-world aerial logistics operations and national data on health system expenditures and household out-of-pocket costs. Incremental cost-effectiveness ratios (ICERs) are calculated from both health system and societal perspectives. Uncertainty is addressed through one-way and probabilistic sensitivity analyses.
Results: The intervention averted 3754.99 discounted DALYs at a net cost of US 106.79 per DALY averted. From the societal perspective, the ICER was US
{"title":"Cost-Effectiveness of Aerial Logistics for Maternal and Newborn Health: A Simulation-Based Analysis Grounded in Real-World Evidence from the Ashanti Region in Ghana.","authors":"Maria J Ospina-Fadul, Pedro Kremer, Florence Haruna, Fred Adomako-Boateng, Kenneth Fosu Oteng, Diana N Tsali","doi":"10.36469/001c.143065","DOIUrl":"10.36469/001c.143065","url":null,"abstract":"<p><strong>Background: </strong>In sub-Saharan Africa, low antenatal care (ANC) coverage and limited access to facility-based deliveries remain key drivers of adverse maternal and newborn health (MNH) outcomes. Inadequate service provision at health facilities and insufficient care-seeking behavior are exacerbated by supply chain inefficiencies that restrict access to essential maternal health commodities. Aerial logistics (centralized storage and drone delivery) has shown promise as a novel approach to addressing these logistical challenges and supporting maternal health service delivery, but its cost-effectiveness has not been evaluated.</p><p><strong>Objectives: </strong>This study evaluates the cost-effectiveness of aerial logistics as an intervention for MNH. It builds on previously observed programmatic effects (increases in ANC visits, facility-based deliveries, and reductions in maternal mortality in Ghana's Ashanti Region) to model downstream health outcomes and estimate incremental economic value.</p><p><strong>Methods: </strong>Using microsimulation and published epidemiological parameters, the study models additional health outcomes resulting from increased service utilization among 11 249 pregnant women, including reductions in low birth weight, postpartum hemorrhage, neonatal mortality, and early-onset neonatal sepsis. Alongside the observed maternal mortality reduction, all outcomes are translated into life-years saved and discounted disability-adjusted life-years (DALYs) averted. Cost estimates are based on real-world aerial logistics operations and national data on health system expenditures and household out-of-pocket costs. Incremental cost-effectiveness ratios (ICERs) are calculated from both health system and societal perspectives. Uncertainty is addressed through one-way and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>The intervention averted 3754.99 discounted DALYs at a net cost of US <math><mn>400</mn> <mrow><mo> </mo></mrow> <mn>987</mn> <mi>f</mi> <mi>r</mi> <mi>o</mi> <mi>m</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>g</mi> <mi>o</mi> <mi>v</mi> <mi>e</mi> <mi>r</mi> <mi>n</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>s</mi> <mi>p</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>v</mi> <mi>e</mi> <mo>,</mo> <mi>y</mi> <mi>i</mi> <mi>e</mi> <mi>l</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>a</mi> <mi>n</mi> <mi>I</mi> <mi>C</mi> <mi>E</mi> <mi>R</mi> <mi>o</mi> <mi>f</mi> <mi>U</mi> <mi>S</mi></math> 106.79 per DALY averted. From the societal perspective, the ICER was US <math><mn>377.82</mn> <mo>.</mo> <mi>T</mi> <mi>h</mi> <mi>e</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>p</mi> <mi>r</mi> <mi>e</mi> <mi>m</mi> <mi>a</mi> <mi>t</mi> <mi>u</mi> <mi>r</mi> <mi>e</mi> <mi>d</mi> <mi>e</mi> <mi>a</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>v</mi> <mi>e</mi> <mi>r</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>w</m","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"143065"},"PeriodicalIF":2.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12448434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113507","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}
Background: Cystic fibrosis (CF) is a rare genetic disorder that places a substantial financial burden on patients, families, and the healthcare system. This study aimed to estimate the economic impact of CF in southern Iran.
Methods: A cross-sectional, prevalence-based cost-of-illness study was conducted from a societal perspective, using a bottom-up approach and the human capital method. Data were collected through insurance records and a standardized cost questionnaire. Mean annual per-patient costs were calculated, and cost determinants were analyzed using the Mann-Whitney and Kruskal-Wallis tests.
Results: The average annual cost per CF patient was US $4070, with 67% attributed to direct medical costs, 20% to direct nonmedical costs, and 13% to indirect costs. Higher total costs were significantly associated with disease severity, hospitalization history, and absence of supplementary insurance.
Conclusion: CF imposes a considerable economic burden in Iran, predominantly driven by drug and hospitalization expenses. Direct nonmedical costs and indirect costs also contribute meaningfully. These findings highlight the need for improved access to specialized CF care, enhanced insurance coverage, and stronger support for informal caregivers to alleviate the financial pressure on affected families.
{"title":"Societal Economic Burden of Cystic Fibrosis in Iran: A Cost-of-Illness Study.","authors":"Hassan Karami, Shideh Rafati, Maryam Shirvani Shiri, Ali Mouseli, Hedayat Salari, Amin Ghanbarnejad, Mitra Nowrouzpour, Fatemeh Noroozian, Ali Alizadeh, Fatemeh Asadi, Narges Salehi","doi":"10.36469/001c.143266","DOIUrl":"10.36469/001c.143266","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis (CF) is a rare genetic disorder that places a substantial financial burden on patients, families, and the healthcare system. This study aimed to estimate the economic impact of CF in southern Iran.</p><p><strong>Methods: </strong>A cross-sectional, prevalence-based cost-of-illness study was conducted from a societal perspective, using a bottom-up approach and the human capital method. Data were collected through insurance records and a standardized cost questionnaire. Mean annual per-patient costs were calculated, and cost determinants were analyzed using the Mann-Whitney and Kruskal-Wallis tests.</p><p><strong>Results: </strong>The average annual cost per CF patient was US $4070, with 67% attributed to direct medical costs, 20% to direct nonmedical costs, and 13% to indirect costs. Higher total costs were significantly associated with disease severity, hospitalization history, and absence of supplementary insurance.</p><p><strong>Conclusion: </strong>CF imposes a considerable economic burden in Iran, predominantly driven by drug and hospitalization expenses. Direct nonmedical costs and indirect costs also contribute meaningfully. These findings highlight the need for improved access to specialized CF care, enhanced insurance coverage, and stronger support for informal caregivers to alleviate the financial pressure on affected families.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"116-123"},"PeriodicalIF":2.3,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12425157/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145064885","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}
Pub Date : 2025-09-08eCollection Date: 2025-01-01DOI: 10.36469/001c.143489
David Shin, Carson Cummings, David Cheng, Chandler Dinh, Daniel Im, Timothy Tang, Isabella Oh, Lauren Han, Patricia Carlson, Gideon Harianja, Jacob Razzouk, Olumide Danisa, Wayne Cheng
<p><strong>Background: </strong>The cost of medical procedures in the United States varies dramatically depending on the payment system, including Medicare, Medi-Cal (California's Medicaid program), private insurance, or lien-based payment models used in personal injury cases. Cost discrepancies can discourage physician participation in Medicare and Medi-Cal, potentially limit access to care for vulnerable patient populations, and complicate the determination of proper compensation in court.</p><p><strong>Objectives: </strong>To survey healthcare providers to determine reasonable costs for medical procedures, potentially aligning legal standards with healthcare costs.</p><p><strong>Methods: </strong>An anonymous, 8-question electronic survey was distributed through Survey Legend® between February and September 2023 to providers in orthopedic surgery, neurosurgery, anesthesiology, interventional radiology (IR), physical medicine and rehabilitation (PMR), pain management, and physician assistants (PAs) or nurse practitioners (NPs). Three procedures-epidural injection, facet injection/medial branch block, and radiofrequency ablation-were included, with participants selecting from 5 cost categories: < <math><mn>1000</mn> <mo>,</mo></math> 1000- <math><mn>4999</mn> <mo>,</mo></math> 5000- <math><mn>9999</mn> <mo>,</mo></math> 10000- <math><mn>19999</mn> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mo>></mo></math> 20,000. Additional questions explored participant insight into discounts for cash and lien-based payments.</p><p><strong>Results: </strong>For all procedures and participants, the most common value was <math><mn>1000</mn> <mo>-</mo></math> 4999. Neurosurgery selected significantly higher epidural values than pain management (<i>P</i>=.025), PMR (<i>P</i>=.029), and PA/NP (<i>P</i>=.04); higher facet injection/medial branch block values than PMR (<i>P</i>=.03) and PA/NPs (<i>P</i>=.01); and higher radiofrequency ablation values than PA/NPs (<i>P</i>=.02). Physicians not accepting lien payments showed significantly lower values across all specialties and procedures.</p><p><strong>Discussion: </strong>The range of reported reasonable costs by respondents reflects a discrepancy between physician expectations and existing reimbursement models, indicating a lack of a standardized value for procedural pricing. Medicare's estimated <math><mn>500</mn> <mi>r</mi> <mi>e</mi> <mi>i</mi> <mi>m</mi> <mi>b</mi> <mi>u</mi> <mi>r</mi> <mi>s</mi> <mi>e</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>f</mi> <mi>o</mi> <mi>r</mi> <mi>e</mi> <mi>p</mi> <mi>i</mi> <mi>d</mi> <mi>u</mi> <mi>r</mi> <mi>a</mi> <mi>l</mi> <mi>i</mi> <mi>n</mi> <mi>j</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mi>s</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>f</mi> <mi>a</mi> <mi>c</mi> <mi>e</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>j</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mrow><mo>/</mo></mrow> <mi>m</mi> <mi>e</mi> <mi
背景：美国医疗程序的费用因支付系统的不同而有很大差异，包括医疗保险、Medi-Cal（加州医疗补助计划）、私人保险或人身伤害案件中使用的基于留置权的支付模式。费用差异会阻碍医生参与医疗保险和Medi-Cal，可能会限制弱势患者群体获得医疗服务，并使法庭上适当赔偿的确定复杂化。目的：调查医疗保健提供者，以确定医疗程序的合理成本，可能使法律标准与医疗保健成本保持一致。方法：在2023年2月至9月期间，通过survey Legend®对骨科、神经外科、麻醉学、介入放射学（IR）、物理医学与康复学（PMR）、疼痛管理、医师助理（PAs）或执业护士（NPs）的提供者进行了一项8题的匿名电子调查。包括三种手术-硬膜外注射，关节突注射/内侧分支阻滞和射频消融，参与者从5个成本类别中选择：< 1000,1000- 4999,5000- 9999,10000- 19999，和bbb20,000。其他问题探讨了参与者对现金和留置权支付折扣的见解。结果：对于所有程序和参与者，最常见的值是1000 - 4999。神经外科选择的硬膜外值明显高于疼痛管理（P= 0.025）、PMR （P= 0.029）和PA/NP (P= 0.04)；关节突注射/内侧分支阻滞值高于PMR （P=.03）和PA/NPs (P=.01)；射频消融值高于PA/NPs （P= 0.02）。不接受留置权支付的医生在所有专业和程序中都显示出明显较低的价值。讨论：受访者报告的合理费用范围反映了医生期望与现有报销模式之间的差异，表明缺乏程序定价的标准化值。医疗保险的大约500 r e i m b u r s e m e n t f o r e p i d u r l i n j e c t i o n s n d f c e t i n j e c t i o n / m e d l b r n c h b l o c k s n d 1000射频消融术低于physician-perceived合理成本和膨胀的指控在lien-based案件中经常发现。相比之下，硬膜外注射的个人伤害费用可能高达20,000美元，只有2.9%的受访者选择这一费用类别。结论：这项调查突出了医疗保健提供者对合理费用的看法，可能有助于改进报销模式，确保法律程序的一致性，并为患者和提供者保持适当的可及性和补偿。
{"title":"Reasonable Cost for Procedures: An Anonymous Survey of Healthcare Providers.","authors":"David Shin, Carson Cummings, David Cheng, Chandler Dinh, Daniel Im, Timothy Tang, Isabella Oh, Lauren Han, Patricia Carlson, Gideon Harianja, Jacob Razzouk, Olumide Danisa, Wayne Cheng","doi":"10.36469/001c.143489","DOIUrl":"10.36469/001c.143489","url":null,"abstract":"<p><strong>Background: </strong>The cost of medical procedures in the United States varies dramatically depending on the payment system, including Medicare, Medi-Cal (California's Medicaid program), private insurance, or lien-based payment models used in personal injury cases. Cost discrepancies can discourage physician participation in Medicare and Medi-Cal, potentially limit access to care for vulnerable patient populations, and complicate the determination of proper compensation in court.</p><p><strong>Objectives: </strong>To survey healthcare providers to determine reasonable costs for medical procedures, potentially aligning legal standards with healthcare costs.</p><p><strong>Methods: </strong>An anonymous, 8-question electronic survey was distributed through Survey Legend® between February and September 2023 to providers in orthopedic surgery, neurosurgery, anesthesiology, interventional radiology (IR), physical medicine and rehabilitation (PMR), pain management, and physician assistants (PAs) or nurse practitioners (NPs). Three procedures-epidural injection, facet injection/medial branch block, and radiofrequency ablation-were included, with participants selecting from 5 cost categories: < <math><mn>1000</mn> <mo>,</mo></math> 1000- <math><mn>4999</mn> <mo>,</mo></math> 5000- <math><mn>9999</mn> <mo>,</mo></math> 10000- <math><mn>19999</mn> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mo>></mo></math> 20,000. Additional questions explored participant insight into discounts for cash and lien-based payments.</p><p><strong>Results: </strong>For all procedures and participants, the most common value was <math><mn>1000</mn> <mo>-</mo></math> 4999. Neurosurgery selected significantly higher epidural values than pain management (<i>P</i>=.025), PMR (<i>P</i>=.029), and PA/NP (<i>P</i>=.04); higher facet injection/medial branch block values than PMR (<i>P</i>=.03) and PA/NPs (<i>P</i>=.01); and higher radiofrequency ablation values than PA/NPs (<i>P</i>=.02). Physicians not accepting lien payments showed significantly lower values across all specialties and procedures.</p><p><strong>Discussion: </strong>The range of reported reasonable costs by respondents reflects a discrepancy between physician expectations and existing reimbursement models, indicating a lack of a standardized value for procedural pricing. Medicare's estimated <math><mn>500</mn> <mi>r</mi> <mi>e</mi> <mi>i</mi> <mi>m</mi> <mi>b</mi> <mi>u</mi> <mi>r</mi> <mi>s</mi> <mi>e</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>f</mi> <mi>o</mi> <mi>r</mi> <mi>e</mi> <mi>p</mi> <mi>i</mi> <mi>d</mi> <mi>u</mi> <mi>r</mi> <mi>a</mi> <mi>l</mi> <mi>i</mi> <mi>n</mi> <mi>j</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mi>s</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>f</mi> <mi>a</mi> <mi>c</mi> <mi>e</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>j</mi> <mi>e</mi> <mi>c</mi> <mi>t</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mrow><mo>/</mo></mrow> <mi>m</mi> <mi>e</mi> <mi","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"108-115"},"PeriodicalIF":2.3,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12422406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040309","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}
Pub Date : 2025-08-29eCollection Date: 2025-01-01DOI: 10.36469/001c.142771
David Waterhouse, Iris Li, Laura Morrison, Bruno Emond, Marie-Hélène Lafeuille, Annalise Hilts, Jill Korsiak, Patrick Lefebvre, Pratyusha Vadagam, Dexter Waters
<p><strong>Background: </strong>Approximately 17% of patients with non-small cell lung cancer (NSCLC) have epidermal growth factor receptor-mutated (EGFRm) NSCLC, 84% of which are exon 19 deletions (Ex19del)/exon 21 substitutions (L858R). Unmet needs for patients treated with tyrosine kinase inhibitors (TKIs) for EGFRm (Ex19del/L858R) advanced NSCLC, including osimertinib, are relevant to US population health decision makers.</p><p><strong>Objectives: </strong>To describe healthcare resource utilization (HRU) and costs by line of therapy (LOT) among patients with EGFRm (Ex19del/L858R) advanced NSCLC initiating first-line (1L) treatment.</p><p><strong>Methods: </strong>IBM MarketScan® Research Databases (1/1/2010-1/31/2023) were used to select adult patients with advanced NSCLC initiating an EGFR-TKI during any LOT on/after 4/18/2018 (osimertinib approval; EGFRm Ex19del/L858R proxy). Per-patient-per-month (PPPM) all-cause HRU and costs were described in 1L, second-line (2L), and third-line (3L) overall and among subgroups receiving 1L osimertinib monotherapy or platinum-based chemotherapy (PBC) without immunotherapy, separately.</p><p><strong>Results: </strong>The study included 409 patients with EGFRm advanced NSCLC (mean age, 60.5 years; 70.2% female). In 1L, 72.9% initiated osimertinib-based therapy (2L, 45.9%; 3L, 41.2%), 21.0% initiated chemotherapy (2L, 30.0%; 3L, 36.5%), 4.6% initiated another EGFR-TKI (2L, 12.9%; 3L, 12.9%), and 1.5% initiated immunotherapy (2L, 11.2%; 3L, 9.4%). Overall, 170 patients (41.6%) progressed to 2L among whom 85 (50.0%) progressed to 3L. Mean LOT duration decreased with each successive LOT (1L, 10.2 months; 2L, 8.7 months; 3L, 8.0 months). Across LOTs, patients had a mean of >4 outpatient visits PPPM (1L, 4.79; 2L, 4.26; 3L, 4.40), and the 1L osimertinib monotherapy subgroup (n = 279) had a mean of 0.69 inpatient days PPPM during 1L (2L, 0.82; 3L, 0.74). Mean all-cause costs PPPM were <math><mn>27</mn> <mrow><mo> </mo></mrow> <mn>751</mn> <mi>i</mi> <mi>n</mi> <mn>1</mn> <mi>L</mi> <mo>,</mo></math> 28 971 in 2L, and <math><mn>31</mn> <mrow><mo> </mo></mrow> <mn>251</mn> <mi>i</mi> <mi>n</mi> <mn>3</mn> <mi>L</mi> <mo>.</mo> <mi>A</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>g</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mn>1</mn> <mi>L</mi> <mi>o</mi> <mi>s</mi> <mi>i</mi> <mi>m</mi> <mi>e</mi> <mi>r</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>i</mi> <mi>b</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>o</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>p</mi> <mi>y</mi> <mi>s</mi> <mi>u</mi> <mi>b</mi> <mi>g</mi> <mi>r</mi> <mi>o</mi> <mi>u</mi> <mi>p</mi> <mo>,</mo> <mi>m</mi> <mi>e</mi> <mi>a</mi> <mi>n</mi> <mi>P</mi> <mi>P</mi> <mi>P</mi> <mi>M</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mi>w</mi> <mi>e</mi> <mi>r</mi> <mi>e</mi></math> 27 610 in 1L, <math><mn>35</mn> <mrow><mo> </mo></mrow> <mn>501</mn> <mi>i</mi> <mi>n</mi> <mn>2</mn> <mi>L</mi> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi><
背景：约17%的非小细胞肺癌（NSCLC）患者为表皮生长因子受体突变（EGFRm）型NSCLC，其中84%为外显子19缺失(Ex19del)/外显子21替换（L858R）。EGFRm （Ex19del/L858R）晚期NSCLC患者未满足的酪氨酸激酶抑制剂（TKIs）治疗需求，包括奥西替尼，与美国人口健康决策者相关。目的：描述EGFRm （Ex19del/L858R）晚期NSCLC患者开始一线（1L）治疗的医疗资源利用率（HRU）和费用（LOT）。方法：使用IBM MarketScan®研究数据库（2010年1月1日- 2023年1月31日）选择2018年4月18日/之后任何LOT期间启动EGFR-TKI的晚期NSCLC成年患者（奥西替尼批准；EGFRm Ex19del/L858R代理）。每个患者每月（PPPM）的全因HRU和成本分别在1L、二线（2L）和三线（3L）总体上和接受1L奥西替尼单药治疗或铂基化疗（PBC）不进行免疫治疗的亚组中进行描述。结果：该研究纳入409例EGFRm晚期NSCLC患者（平均年龄60.5岁，70.2%为女性）。在1L中，72.9%的患者开始了基于奥西替尼的治疗（2L, 45.9%; 3L, 41.2%）， 21.0%的患者开始了化疗（2L, 30.0%; 3L, 36.5%）， 4.6%的患者开始了另一种EGFR-TKI (2L, 12.9%; 3L, 12.9%)， 1.5%的患者开始了免疫治疗（2L, 11.2%; 3L, 9.4%）。总体而言，170例（41.6%）进展为2L，其中85例（50.0%）进展为3L。平均LOT持续时间随每次LOT的延长而减少（1L， 10.2个月；2L， 8.7个月；3L， 8.0个月）。在所有批次中，患者的平均门诊次数PPPM为4840次（1L, 4.79; 2L, 4.26; 3L, 4.40）， 1L单药治疗亚组（n = 279）在1L期间的平均住院天数PPPM为0.69天（2L, 0.82; 3L, 0.74）。平均全因成本PPPM为27 751 i / 1 L， 28 971 / 2L, 31 251 / 3 L。m o n g t h e 1 L o s i m e r t i n i b m o n o t h e r p y s u b g r o p m e n p p p m c o s t s w e r e 27 610年1 L,我35 501 n 2 L, n d 36 618 3 L。在1L PBC亚组（n = 58）中，1L的平均PPPM成本为23 820美元，2L为24 788美元，3L为23 348美元。讨论：在EGFRm （Ex19del/L858R）晚期NSCLC患者中，每次连续LOT的时间更短，费用更高。结论：研究结果强调了使用最有效的1L治疗来延缓疾病进展、降低HRU和成本的重要性。
{"title":"Healthcare Resource Use, Healthcare Costs, and Unmet Needs Among Patients Treated for EGFR-Mutated Advanced or Metastatic Non-small Cell Lung Cancer.","authors":"David Waterhouse, Iris Li, Laura Morrison, Bruno Emond, Marie-Hélène Lafeuille, Annalise Hilts, Jill Korsiak, Patrick Lefebvre, Pratyusha Vadagam, Dexter Waters","doi":"10.36469/001c.142771","DOIUrl":"10.36469/001c.142771","url":null,"abstract":"<p><strong>Background: </strong>Approximately 17% of patients with non-small cell lung cancer (NSCLC) have epidermal growth factor receptor-mutated (EGFRm) NSCLC, 84% of which are exon 19 deletions (Ex19del)/exon 21 substitutions (L858R). Unmet needs for patients treated with tyrosine kinase inhibitors (TKIs) for EGFRm (Ex19del/L858R) advanced NSCLC, including osimertinib, are relevant to US population health decision makers.</p><p><strong>Objectives: </strong>To describe healthcare resource utilization (HRU) and costs by line of therapy (LOT) among patients with EGFRm (Ex19del/L858R) advanced NSCLC initiating first-line (1L) treatment.</p><p><strong>Methods: </strong>IBM MarketScan® Research Databases (1/1/2010-1/31/2023) were used to select adult patients with advanced NSCLC initiating an EGFR-TKI during any LOT on/after 4/18/2018 (osimertinib approval; EGFRm Ex19del/L858R proxy). Per-patient-per-month (PPPM) all-cause HRU and costs were described in 1L, second-line (2L), and third-line (3L) overall and among subgroups receiving 1L osimertinib monotherapy or platinum-based chemotherapy (PBC) without immunotherapy, separately.</p><p><strong>Results: </strong>The study included 409 patients with EGFRm advanced NSCLC (mean age, 60.5 years; 70.2% female). In 1L, 72.9% initiated osimertinib-based therapy (2L, 45.9%; 3L, 41.2%), 21.0% initiated chemotherapy (2L, 30.0%; 3L, 36.5%), 4.6% initiated another EGFR-TKI (2L, 12.9%; 3L, 12.9%), and 1.5% initiated immunotherapy (2L, 11.2%; 3L, 9.4%). Overall, 170 patients (41.6%) progressed to 2L among whom 85 (50.0%) progressed to 3L. Mean LOT duration decreased with each successive LOT (1L, 10.2 months; 2L, 8.7 months; 3L, 8.0 months). Across LOTs, patients had a mean of >4 outpatient visits PPPM (1L, 4.79; 2L, 4.26; 3L, 4.40), and the 1L osimertinib monotherapy subgroup (n = 279) had a mean of 0.69 inpatient days PPPM during 1L (2L, 0.82; 3L, 0.74). Mean all-cause costs PPPM were <math><mn>27</mn> <mrow><mo> </mo></mrow> <mn>751</mn> <mi>i</mi> <mi>n</mi> <mn>1</mn> <mi>L</mi> <mo>,</mo></math> 28 971 in 2L, and <math><mn>31</mn> <mrow><mo> </mo></mrow> <mn>251</mn> <mi>i</mi> <mi>n</mi> <mn>3</mn> <mi>L</mi> <mo>.</mo> <mi>A</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>g</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mn>1</mn> <mi>L</mi> <mi>o</mi> <mi>s</mi> <mi>i</mi> <mi>m</mi> <mi>e</mi> <mi>r</mi> <mi>t</mi> <mi>i</mi> <mi>n</mi> <mi>i</mi> <mi>b</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>o</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>p</mi> <mi>y</mi> <mi>s</mi> <mi>u</mi> <mi>b</mi> <mi>g</mi> <mi>r</mi> <mi>o</mi> <mi>u</mi> <mi>p</mi> <mo>,</mo> <mi>m</mi> <mi>e</mi> <mi>a</mi> <mi>n</mi> <mi>P</mi> <mi>P</mi> <mi>P</mi> <mi>M</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mi>w</mi> <mi>e</mi> <mi>r</mi> <mi>e</mi></math> 27 610 in 1L, <math><mn>35</mn> <mrow><mo> </mo></mrow> <mn>501</mn> <mi>i</mi> <mi>n</mi> <mn>2</mn> <mi>L</mi> <mo>,</mo> <mi>a</mi> <mi>n</mi> <mi>d</mi><","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"98-107"},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12399230/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957076","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}
Pub Date : 2025-08-26eCollection Date: 2025-01-01DOI: 10.36469/001c.142444
Srujitha Marupuru, Kristin Moore, Desiree Hall, Sarah Aurit, Gretchen Hultman, Noah Webb, Yong Zhu, Gieira Jones
Background: Patients with ovarian cancer incur substantial economic burdens. However, little is known about the differences in metrics such as treatment patterns, healthcare resource utilization (HCRU), and costs between those with BRCA mutant (BRCAm) and BRCA wildtype (BRCAwt) tumors.
Objective: This study assessed demographic and clinical characteristics, treatment patterns, and HCRU and costs among patients diagnosed with ovarian cancer, stratified by BRCA testing status and result.
Methods: This retrospective study included patients with ovarian cancer between Jan. 1, 2017, and June 30, 2022, with electronic health record (EHR) and administrative claims data in Optum's Clinical EHR and claims databases. Data collected included baseline characteristics, lines of therapy (LOTs) (captured at 6, 12, and 24 months follow-up), HCRU (captured for 12-month baseline and follow-up periods), and costs (captured for 6-month baseline and 12-month follow-up periods). Patients were stratified by the presence or absence of a BRCA test and by BRCA testing results.
Results: A total of 13 981 patients were included in the sample; 23.3% had a BRCA test and 76.7% did not. Among those with a BRCA test, 62.0% were BRCAm and 35.8% were BRCAwt. Patients who did not receive BRCA testing were more likely to be non-Hispanic African American and to live in the South (all P < .001). Patients who received testing were more likely to progress to a subsequent LOT but also more likely to receive BRCA-targeted therapies. The median per-patient-per-month (PPPM) total costs were 62% higher in BRCA-tested patients than those without tests ( 3845). Similarly, median PPPM ambulatory visits cost and pharmacy cost were 81% and 137% higher in those with BRCA tests than those without tests ( 1232, and 335, respectively).
Conclusions: Approximately one-fourth of patients received BRCA testing. Disparities existed between those who received testing and those who did not. Patients who were tested had higher costs than those who were not; this difference was driven mostly by ambulatory visits and pharmacy costs, potentially due to increased clinical encounters and higher costs of targeted treatments.
背景：卵巢癌患者承受着巨大的经济负担。然而，对于BRCA突变型（BRCAm）和BRCA野生型（BRCAwt）肿瘤患者在治疗模式、医疗资源利用（HCRU）和成本等指标上的差异知之甚少。目的：本研究评估卵巢癌患者的人口学和临床特征、治疗模式、HCRU和成本，并根据BRCA检测状态和结果进行分层。方法：本回顾性研究纳入了2017年1月1日至2022年6月30日期间的卵巢癌患者，这些患者在Optum的临床EHR和索赔数据库中使用电子健康记录（EHR）和行政索赔数据。收集的数据包括基线特征、治疗线（批次）（随访6个月、12个月和24个月）、HCRU（12个月基线和随访期）和成本（6个月基线和12个月随访期）。根据是否进行BRCA检测和BRCA检测结果对患者进行分层。结果：共纳入13 981例患者；23.3%的人有BRCA检测，76.7%的人没有。在BRCA检测中，62.0%为BRCAm， 35.8%为brcat。未接受BRCA检测的患者更有可能是非西班牙裔非裔美国人，并且生活在南方（所有P 6242 vs s 3845）。同样，进行BRCA检测的患者的PPPM门诊费用和药房费用中位数比未进行BRCA检测的患者高81%和137%（分别为2236 v和1232 v， 793 v和335 v）。结论：大约四分之一的患者接受了BRCA检测。接受测试的人和没有接受测试的人之间存在差异。接受检测的患者比未接受检测的患者花费更高；这种差异主要是由门诊就诊和药房费用造成的，可能是由于临床就诊次数增加和靶向治疗费用增加。
{"title":"Real-World Treatment Patterns and Cost of Care in US Ovarian Cancer Patients Undergoing BRCA Testing.","authors":"Srujitha Marupuru, Kristin Moore, Desiree Hall, Sarah Aurit, Gretchen Hultman, Noah Webb, Yong Zhu, Gieira Jones","doi":"10.36469/001c.142444","DOIUrl":"10.36469/001c.142444","url":null,"abstract":"<p><strong>Background: </strong>Patients with ovarian cancer incur substantial economic burdens. However, little is known about the differences in metrics such as treatment patterns, healthcare resource utilization (HCRU), and costs between those with BRCA mutant (BRCAm) and BRCA wildtype (BRCAwt) tumors.</p><p><strong>Objective: </strong>This study assessed demographic and clinical characteristics, treatment patterns, and HCRU and costs among patients diagnosed with ovarian cancer, stratified by BRCA testing status and result.</p><p><strong>Methods: </strong>This retrospective study included patients with ovarian cancer between Jan. 1, 2017, and June 30, 2022, with electronic health record (EHR) and administrative claims data in Optum's Clinical EHR and claims databases. Data collected included baseline characteristics, lines of therapy (LOTs) (captured at 6, 12, and 24 months follow-up), HCRU (captured for 12-month baseline and follow-up periods), and costs (captured for 6-month baseline and 12-month follow-up periods). Patients were stratified by the presence or absence of a BRCA test and by BRCA testing results.</p><p><strong>Results: </strong>A total of 13 981 patients were included in the sample; 23.3% had a BRCA test and 76.7% did not. Among those with a BRCA test, 62.0% were BRCAm and 35.8% were BRCAwt. Patients who did not receive BRCA testing were more likely to be non-Hispanic African American and to live in the South (all <i>P</i> < .001). Patients who received testing were more likely to progress to a subsequent LOT but also more likely to receive BRCA-targeted therapies. The median per-patient-per-month (PPPM) total costs were 62% higher in BRCA-tested patients than those without tests ( <math><mn>6242</mn> <mi>v</mi> <mi>s</mi></math> 3845). Similarly, median PPPM ambulatory visits cost and pharmacy cost were 81% and 137% higher in those with BRCA tests than those without tests ( <math><mn>2236</mn> <mi>v</mi> <mi>s</mi></math> 1232, and <math><mn>793</mn> <mi>v</mi> <mi>s</mi></math> 335, respectively).</p><p><strong>Conclusions: </strong>Approximately one-fourth of patients received BRCA testing. Disparities existed between those who received testing and those who did not. Patients who were tested had higher costs than those who were not; this difference was driven mostly by ambulatory visits and pharmacy costs, potentially due to increased clinical encounters and higher costs of targeted treatments.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"85-97"},"PeriodicalIF":2.3,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12393876/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144956997","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}
[This corrects the article DOI: 10.36460/jheor.2025.141471.].
[这更正了文章DOI: 10.36460/jheor.2025.141471.]。
{"title":"Erratum: Effect of Fresh Frozen Plasma Infusion on Hospital Length of Stay for Patients With Hereditary Angioedema.","authors":"Subhan Khalid, Alan T Hitch","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.36460/jheor.2025.141471.].</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"143440"},"PeriodicalIF":2.3,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12374767/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957003","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}
Pub Date : 2025-08-21eCollection Date: 2025-01-01DOI: 10.36469/001c.143450
Subhan Khalid, Alan T Hitch
[This corrects the article DOI: 10.36469/jheor.2025.141747.].
[这更正了文章DOI: 10.36469/jheor.2025.141747.]。
{"title":"Erratum: Characteristics and Comorbidities Influencing Mortality Risk Among Hereditary Angioedema Patients.","authors":"Subhan Khalid, Alan T Hitch","doi":"10.36469/001c.143450","DOIUrl":"10.36469/001c.143450","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.36469/jheor.2025.141747.].</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"143450"},"PeriodicalIF":2.3,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12374766/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957049","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}
<p><strong>Background: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with C5 inhibitors (C5i), eculizumab and ravulizumab, being part of current treatment options.</p><p><strong>Objectives: </strong>To estimate the 5-year prevalence of PNH and describe the healthcare resource utilization and direct healthcare costs associated with C5i among commercially insured patients with PNH treated with C5i in the US.</p><p><strong>Methods: </strong>The 5-year prevalence of adults with PNH in IQVIA PharMetrics® Plus was estimated (2018-2022). A retrospective cohort study (2011-2022) was also conducted in adults with PNH treated with C5i and ≥3 months of continuous health plan coverage following the first claim for C5i (index date). PNH-related health resource utilization and direct healthcare costs were assessed from index date until earliest of treatment discontinuation/end of data/end of continuous health plan coverage (follow-up period).</p><p><strong>Results: </strong>The 5-year prevalence of PNH was 2.4 per 100 000 persons in commercial claims. A total of 371 patients treated with C5i (median age: 40 years; female: 55.3%; eculizumab: 53.9%; ravulizumab: 46.1%) were followed for a mean ± SD [median] of 19.3 ± 16.9 [14.7] months. Annual incidence rates of PNH-related blood transfusion and breakthrough hemolysis (BTH) among patients treated with C5i were 1.2 (eculizumab: 1.3; ravulizumab: 1.0) and 4.5 (eculizumab: 5.2; ravulizumab: 3.3) per person per year (PPPY), respectively. In patients treated with eculizumab and ravulizumab, respectively, PNH-related blood transfusion was required by 46.2% and 11.9% of patients in the first 6 months post-index, and over the follow-up period, transfusion avoidance was observed in 46.2% and 78.2% of patients. The 6- and 12-month rates of PNH-related thrombosis were 8.0% and 10.6% for eculizumab and 6.1% and 11.6% for ravulizumab, respectively. Among patients treated with C5i, estimated annual total PNH-related costs PPPY were <math><mn>660</mn> <mrow><mo> </mo></mrow> <mn>533</mn> <mo>(</mo> <mi>e</mi> <mi>c</mi> <mi>u</mi> <mi>l</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mo>:</mo></math> 697 459; ravulizumab: <math><mn>612</mn> <mrow><mo> </mo></mrow> <mn>522</mn> <mo>)</mo> <mi>f</mi> <mi>o</mi> <mi>r</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>f</mi> <mi>i</mi> <mi>r</mi> <mi>s</mi> <mi>t</mi> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 633 984 (eculizumab: <math><mn>691</mn> <mrow><mo> </mo></mrow> <mn>022</mn> <mo>;</mo> <mi>r</mi> <mi>a</mi> <mi>v</mi> <mi>u</mi> <mi>l</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mo>:</mo></math> 570 832) for subsequent years, with treatment costs accounting for 94.3% to 94.6% of total costs.</p><p><strong>Discussion: </strong>Despite treatment with C5i, patients with PNH still exhibited BTH, required blood transfusions, and experienced thrombosis.</p><p><str
背景：阵发性夜间血红蛋白尿（PNH）是一种罕见的血液疾病，C5抑制剂（C5i）， eculizumab和ravulizumab是目前治疗方案的一部分。目的：估计5年PNH的患病率，并描述在美国商业保险的接受C5i治疗的PNH患者中与C5i相关的医疗资源利用和直接医疗费用。方法：估计IQVIA PharMetrics®Plus中成人PNH的5年患病率（2018-2022）。一项回顾性队列研究（2011-2022）也对接受C5i治疗的PNH成人患者进行了研究，该患者在首次申请C5i（索引日期）后连续健康计划覆盖≥3个月。从索引日期到最早停止治疗/数据终止/连续健康计划覆盖结束（随访期），评估了与pnh相关的卫生资源利用和直接卫生保健费用。结果：商业索赔中PNH的5年患病率为2.4 / 100,000 万人。共371例接受C5i治疗的患者（中位年龄：40岁，女性：55.3%,eculizumab: 53.9%, ravulizumab: 46.1%）被随访，平均±SD[中位数]为19.3±16.9[14.7]个月。在接受C5i治疗的患者中，pnh相关输血和突破性溶血（BTH）的年发生率分别为每人每年1.2 （eculizumab: 1.3; ravulizumab: 1.0）和4.5 (eculizumab: 5.2; ravulizumab: 3.3) （PPPY）。在分别接受eculizumab和ravulizumab治疗的患者中，46.2%和11.9%的患者在指数后的前6个月内需要与pnh相关的输血，在随访期间，46.2%和78.2%的患者观察到输血避免。eculizumab组6个月和12个月pnh相关血栓发生率分别为8.0%和10.6%，ravulizumab组为6.1%和11.6%。C5i患者中,估计每年总PNH-related成本PPPY 660 533 (e c u l z u m b: 697 459;ravulizumab: 612 522)o r t h e f ir s t y e r n d 633 984 (v eculizumab: 691 022;r u l z u m b: 570 832)随后几年,治疗成本占总成本的94.3%到94.6%。讨论：尽管使用C5i治疗，PNH患者仍然表现出BTH，需要输血，并经历血栓形成。结论：本研究强调了对更有效的PNH治疗的需求，以解决与PNH相关的经济和临床负担，并改善患者的疾病控制。
{"title":"Real-World Prevalence and Outcomes of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors in the US: A Retrospective Claims Database Analysis.","authors":"Srinivas K Tantravahi, Dominick Latremouille-Viau, Raj Desai, Soyon Lee, Jincy Paulose, Anumaxine Geevarghese, Annie Guérin, Shravanthi Seshasayee, Mohin Chanpura, Glorian Yen","doi":"10.36469/001c.142049","DOIUrl":"10.36469/001c.142049","url":null,"abstract":"<p><strong>Background: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with C5 inhibitors (C5i), eculizumab and ravulizumab, being part of current treatment options.</p><p><strong>Objectives: </strong>To estimate the 5-year prevalence of PNH and describe the healthcare resource utilization and direct healthcare costs associated with C5i among commercially insured patients with PNH treated with C5i in the US.</p><p><strong>Methods: </strong>The 5-year prevalence of adults with PNH in IQVIA PharMetrics® Plus was estimated (2018-2022). A retrospective cohort study (2011-2022) was also conducted in adults with PNH treated with C5i and ≥3 months of continuous health plan coverage following the first claim for C5i (index date). PNH-related health resource utilization and direct healthcare costs were assessed from index date until earliest of treatment discontinuation/end of data/end of continuous health plan coverage (follow-up period).</p><p><strong>Results: </strong>The 5-year prevalence of PNH was 2.4 per 100 000 persons in commercial claims. A total of 371 patients treated with C5i (median age: 40 years; female: 55.3%; eculizumab: 53.9%; ravulizumab: 46.1%) were followed for a mean ± SD [median] of 19.3 ± 16.9 [14.7] months. Annual incidence rates of PNH-related blood transfusion and breakthrough hemolysis (BTH) among patients treated with C5i were 1.2 (eculizumab: 1.3; ravulizumab: 1.0) and 4.5 (eculizumab: 5.2; ravulizumab: 3.3) per person per year (PPPY), respectively. In patients treated with eculizumab and ravulizumab, respectively, PNH-related blood transfusion was required by 46.2% and 11.9% of patients in the first 6 months post-index, and over the follow-up period, transfusion avoidance was observed in 46.2% and 78.2% of patients. The 6- and 12-month rates of PNH-related thrombosis were 8.0% and 10.6% for eculizumab and 6.1% and 11.6% for ravulizumab, respectively. Among patients treated with C5i, estimated annual total PNH-related costs PPPY were <math><mn>660</mn> <mrow><mo> </mo></mrow> <mn>533</mn> <mo>(</mo> <mi>e</mi> <mi>c</mi> <mi>u</mi> <mi>l</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mo>:</mo></math> 697 459; ravulizumab: <math><mn>612</mn> <mrow><mo> </mo></mrow> <mn>522</mn> <mo>)</mo> <mi>f</mi> <mi>o</mi> <mi>r</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>f</mi> <mi>i</mi> <mi>r</mi> <mi>s</mi> <mi>t</mi> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 633 984 (eculizumab: <math><mn>691</mn> <mrow><mo> </mo></mrow> <mn>022</mn> <mo>;</mo> <mi>r</mi> <mi>a</mi> <mi>v</mi> <mi>u</mi> <mi>l</mi> <mi>i</mi> <mi>z</mi> <mi>u</mi> <mi>m</mi> <mi>a</mi> <mi>b</mi> <mo>:</mo></math> 570 832) for subsequent years, with treatment costs accounting for 94.3% to 94.6% of total costs.</p><p><strong>Discussion: </strong>Despite treatment with C5i, patients with PNH still exhibited BTH, required blood transfusions, and experienced thrombosis.</p><p><str","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"66-74"},"PeriodicalIF":2.3,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12358179/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144873505","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}
Pub Date : 2025-08-12eCollection Date: 2025-01-01DOI: 10.36469/001c.143106
Niodita Gupta-Werner, Vipin Khare, Brian Macomson, Rohan Medhekar
[This corrects the article DOI: 10.36469/001c.141714.].
[这更正了文章DOI: 10.36469/ 001c141714 .]。
{"title":"Correction: Cost of Anti-CD38 Monoclonal Antibodies in Combination With Bortezomib, Lenalidomide and Dexamethasone for the Frontline Treatment of Transplant-Ineligible Patients With Newly Diagnosed Multiple Myeloma in the US.","authors":"Niodita Gupta-Werner, Vipin Khare, Brian Macomson, Rohan Medhekar","doi":"10.36469/001c.143106","DOIUrl":"10.36469/001c.143106","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.36469/001c.141714.].</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"62-66"},"PeriodicalIF":2.3,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12352406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144873495","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}
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