Journal of Health Economics and Outcomes Research最新文献

{"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}

{"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":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To survey healthcare providers to determine reasonable costs for medical procedures, potentially aligning legal standards with healthcare costs.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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: &lt; &lt;math&gt;&lt;mn&gt;1000&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt;&lt;/math&gt; 1000- &lt;math&gt;&lt;mn&gt;4999&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt;&lt;/math&gt; 5000- &lt;math&gt;&lt;mn&gt;9999&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt;&lt;/math&gt; 10000- &lt;math&gt;&lt;mn&gt;19999&lt;/mn&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mo&gt;&gt;&lt;/mo&gt;&lt;/math&gt; 20,000. Additional questions explored participant insight into discounts for cash and lien-based payments.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;For all procedures and participants, the most common value was &lt;math&gt;&lt;mn&gt;1000&lt;/mn&gt; &lt;mo&gt;-&lt;/mo&gt;&lt;/math&gt; 4999. Neurosurgery selected significantly higher epidural values than pain management (&lt;i&gt;P&lt;/i&gt;=.025), PMR (&lt;i&gt;P&lt;/i&gt;=.029), and PA/NP (&lt;i&gt;P&lt;/i&gt;=.04); higher facet injection/medial branch block values than PMR (&lt;i&gt;P&lt;/i&gt;=.03) and PA/NPs (&lt;i&gt;P&lt;/i&gt;=.01); and higher radiofrequency ablation values than PA/NPs (&lt;i&gt;P&lt;/i&gt;=.02). Physicians not accepting lien payments showed significantly lower values across all specialties and procedures.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;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 &lt;math&gt;&lt;mn&gt;500&lt;/mn&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;j&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;j&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mrow&gt;&lt;mo&gt;/&lt;/mo&gt;&lt;/mrow&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;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}

{"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":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;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 &gt;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 &lt;math&gt;&lt;mn&gt;27&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;751&lt;/mn&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mn&gt;1&lt;/mn&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt;&lt;/math&gt; 28 971 in 2L, and &lt;math&gt;&lt;mn&gt;31&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;251&lt;/mn&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mn&gt;3&lt;/mn&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mo&gt;.&lt;/mo&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mn&gt;1&lt;/mn&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;P&lt;/mi&gt; &lt;mi&gt;P&lt;/mi&gt; &lt;mi&gt;P&lt;/mi&gt; &lt;mi&gt;M&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt;&lt;/math&gt; 27 610 in 1L, &lt;math&gt;&lt;mn&gt;35&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;501&lt;/mn&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mn&gt;2&lt;/mn&gt; &lt;mi&gt;L&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt;&lt;","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}

{"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}

{"title":"Costs of First-Line Treatment With FOLFIRINOX, Modified FOLFIRINOX, and Gemcitabine With Nab-Paclitaxel in Metastatic Pancreatic Ductal Adenocarcinoma.","authors":"Syvart Dennen, Marty Masek, Paul Cockrum, Elizabeth Nagelhout, Ravi Paluri","doi":"10.36469/001c.142403","DOIUrl":"10.36469/001c.142403","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Further research is needed to determine real-world costs of first-line (1L) treatment of metastatic pancreatic ductal adenocarcinoma (mPDAC) with FOLFIRINOX (FFX), modified FFX (mFFX), and gemcitabine with nab-paclitaxel (GnP).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To describe healthcare costs by treatment regimen, stratified by commercial and Medicare Advantage insurance.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This retrospective cohort study of adult patients with mPDAC utilized Optum's de-identified Market Clarity Dataset. Demographics, clinical characteristics, and 1L unadjusted all-cause healthcare costs were examined. Total all-cause costs included costs from inpatient, outpatient, chemotherapy drug and administration, granulocyte colony-stimulating factor (G-CSF), radiation therapy, and other outpatient and pharmacy costs.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 3115 patients met the criteria for inclusion and received 1L treatment with either FFX, mFFX, or GnP. Among those, 1703 had commercial insurance (FFX, 536; mFFX, 673; GnP, 494) and 1412 had Medicare Advantage (FFX, 201; mFFX, 317; GnP, 894). Total cost of care (mean [SD]) was similar between regimens for each insurance cohort (mean [SD] commercial: FFX, &lt;math&gt;&lt;mn&gt;137&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;813&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 127 504]; mFFX, &lt;math&gt;&lt;mn&gt;120&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;109&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 112 208]; GnP, &lt;math&gt;&lt;mn&gt;133&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;042&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 154 248]; Medicare Advantage: FFX, &lt;math&gt;&lt;mn&gt;110&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;788&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 98 492]; mFFX, &lt;math&gt;&lt;mn&gt;98&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;667&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 83 437]; GnP, &lt;math&gt;&lt;mn&gt;110&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;211&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 100 150]). For both insurance cohorts, chemotherapy drug costs were highest for GnP (mean [SD] commercial: FFX, &lt;math&gt;&lt;mn&gt;10&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;916&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 21 647]; mFFX, &lt;math&gt;&lt;mn&gt;7653&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 10 054]; GnP, &lt;math&gt;&lt;mn&gt;60&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;466&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 112 589]; Medicare Advantage: FFX, &lt;math&gt;&lt;mn&gt;8028&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 11 044]; mFFX, &lt;math&gt;&lt;mn&gt;6016&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 7688]; GnP, &lt;math&gt;&lt;mn&gt;49&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;263&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 49 373]), while chemotherapy administration costs were higher for FFX and mFFX (commercial: FFX, &lt;math&gt;&lt;mn&gt;25&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;458&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 33 350]; mFFX, &lt;math&gt;&lt;mn&gt;22&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;795&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 24 309]; GnP &lt;math&gt;&lt;mn&gt;12&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;206&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 15 766]; Medicare Advantage: FFX, &lt;math&gt;&lt;mn&gt;25&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;512&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 36 352]; mFFX, &lt;math&gt;&lt;mn&gt;21&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;524&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 22 317]; GnP &lt;math&gt;&lt;mn&gt;11&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;103&lt;/mn&gt; &lt;mo&gt;[&lt;/mo&gt;&lt;/math&gt; 13 089]). G-CSF costs were also higher ","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"75-84"},"PeriodicalIF":2.3,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12375408/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957042","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":"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":2.5,"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}

{"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}

{"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":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with C5 inhibitors (C5i), eculizumab and ravulizumab, being part of current treatment options.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;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 &lt;math&gt;&lt;mn&gt;660&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;533&lt;/mn&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;z&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mo&gt;:&lt;/mo&gt;&lt;/math&gt; 697 459; ravulizumab: &lt;math&gt;&lt;mn&gt;612&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;522&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt;&lt;/math&gt; 633 984 (eculizumab: &lt;math&gt;&lt;mn&gt;691&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;022&lt;/mn&gt; &lt;mo&gt;;&lt;/mo&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;z&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mo&gt;:&lt;/mo&gt;&lt;/math&gt; 570 832) for subsequent years, with treatment costs accounting for 94.3% to 94.6% of total costs.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;Despite treatment with C5i, patients with PNH still exhibited BTH, required blood transfusions, and experienced thrombosis.&lt;/p&gt;&lt;p&gt;&lt;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}

{"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}

{"title":"Cost per Response of Acthar® Gel vs Standard of Care for the Treatment of Proteinuria in Nephrotic Syndrome Due to Idiopathic Membranous Nephropathy Among Adults from the US Healthcare Perspective.","authors":"Jas Bindra, Ishveen Chopra, Kyle Hayes, John Niewoehner, Mary P Panaccio, George J Wan","doi":"10.36469/001c.142078","DOIUrl":"10.36469/001c.142078","url":null,"abstract":"<p><p><b>Background:</b> Proteinuria, a critical marker of glomerulosclerosis, poses a challenge in idiopathic membranous nephropathy (iMN), particularly when standard treatments fail. Acthar® Gel, a US Food and Drug Administration-approved treatment option, may offer an alternative for managing refractory proteinuria in nephrotic syndrome (NS) due to iMN where multiple treatments have failed. <b>Objective:</b> The cost per response of Acthar® Gel vs standard of care (SoC; cyclophosphamide or rituximab) for treatment of proteinuria in NS due to iMN was evaluated among adults who had failed multiple treatments from a US payer perspective over a 1- to 3-year horizon. <b>Methods:</b> A probabilistic, cohort-level state-transition model simulated patient progression through various health states using 6-month cycles. Patients began in a relapse phase and received either Acthar® Gel or SoC. Transition probabilities determined whether patients achieved a response, experienced no response, progressed to renal failure, or remained in relapse. Responders could potentially maintain their response or relapse, while nonresponders risked renal failure, with potential mortality from any state. Clinical, healthcare resource utilization, and cost data were derived from published literature. Drug prices were based on wholesale acquisition costs. <b>Results:</b> Over 1 year, Acthar® Gel showed a lower cost per response ( <math><mn>377</mn> <mrow><mo> </mo></mrow> <mn>185</mn> <mo>)</mo> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>n</mi> <mi>c</mi> <mi>y</mi> <mi>c</mi> <mi>l</mi> <mi>o</mi> <mi>p</mi> <mi>h</mi> <mi>o</mi> <mi>s</mi> <mi>p</mi> <mi>h</mi> <mi>a</mi> <mi>m</mi> <mi>i</mi> <mi>d</mi> <mi>e</mi> <mo>(</mo></math> 551 687) and rituximab ($741 373). This cost advantage of Acthar® Gel was maintained over 2 and 3 years. Acthar® Gel had higher drug acquisition costs than cyclophosphamide and rituximab but resulted in lower overall medical costs and higher response rates within 1 year, without additional treatment-related costs. Over 2 and 3 years, Acthar® Gel had a lower overall cost of care and higher response rates than SoC, establishing it as a dominant treatment option. <b>Conclusions:</b> Based on current model assumptions and clinical inputs, Acthar® Gel may potentially be a cost-effective and value-based treatment strategy vs unapproved SoCs for adults with refractory proteinuria in NS due to iMN, particularly for those who have not responded to conventional therapies over a 1- to 3-year period within a US payer context. These results may inform clinical and payer decision-making in cases when other standard therapies fail to achieve desired outcomes for a specific population.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"50-61"},"PeriodicalIF":2.3,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12333935/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144816818","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}