Journal of Health Economics and Outcomes Research最新文献

{"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}

{"title":"Prostate-Specific Antigen Reduction After Androgen Receptor Pathway Inhibitor Initiation: Real-World Comparison of Disease Progression Among Patients With Metastatic Castration-Sensitive Prostate Cancer.","authors":"Shawn Du, Carmine Rossi, Ibrahim Khilfeh, Porpong Boonmak, Gordon Wong, Dominic Pilon, Lorie Ellis","doi":"10.36469/001c.141170","DOIUrl":"10.36469/001c.141170","url":null,"abstract":"<p><p><b>Background:</b> Prostate-specific antigen (PSA) has been used as both a screening tool and a marker for treatment response for advanced prostate cancer. With the introduction of androgen receptor pathway inhibitor (ARPI)-based treatment for metastatic castration-sensitive prostate cancer (mCSPC), there is a need to understand the impact that early treatment response, as measured by PSA, has on long-term clinical outcomes. <b>Objectives:</b> To assess whether long-term indicators of treatment success differ among ARPI-naïve patients with mCSPC who did or did not attain ≥90% reduction in PSA levels within 6 months of treatment initiation. <b>Methods:</b> Patients with mCSPC initiating a first ARPI (ie, apalutamide, enzalutamide, abiraterone acetate, darolutamide) were identified using electronic medical record data linked to insurance claims in the United States (1/1/2016-9/30/2022). Eligible patients were classified based on whether they achieved ≥90% reduction in PSA measured between pre-treatment and a window of 30 to 180 days after ARPI initiation. Cohorts were balanced using inverse probability of treatment weighting. Weighted Kaplan-Meier analysis was used to compare overall survival and castration-resistance-free survival by 36 months post-index between those with and without ≥90% PSA reduction. <b>Results:</b> Weighted cohorts included 1192 patients with early PSA reduction ≥90% and 699 without. By 36 months, significantly better overall survival was observed in those with early PSA reduction ≥90% than in those without (71.5% vs 54.7%; hazard ratio [HR]: 0.40, 95% confidence interval [CI]: 0.31, 0.50; <i>P</i><.001). Similarly, significantly better castration-resistance-free survival was observed in those with early PSA reduction ≥90% than in those without (53.3% vs 36.8%; HR: 0.51, 95% CI: 0.43, 0.60; <i>P</i>< .001). <b>Discussion:</b> Early reduction of PSA levels by ≥90% within 6 months of ARPI initiation among patients with mCSPC in the real world is a robust indicator of treatment success, with improved long-term clinical outcomes, including survival and reduction in disease progression. <b>Conclusions:</b> These findings corroborate those of clinical trials and highlight the long-term benefits of an early and deep PSA response to ARPIs among real-world patients with mCSPC in the United States.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"41-49"},"PeriodicalIF":2.3,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12313024/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144760303","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 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.141714","DOIUrl":"10.36469/001c.141714","url":null,"abstract":"<p><p><b>Background:</b> The efficacy of the combination of bortezomib, lenalidomide, and dexamethasone with daratumumab (DVRd) or isatuximab (IsaVRd) for the frontline treatment of transplant-ineligible (TIE) newly diagnosed multiple myeloma (NDMM) has been demonstrated in clinical trials. However, the treatment cost for DVRd and IsaVRd has not been compared. <b>Objectives:</b> To compare the drug acquisition costs (DAC) of DVRd vs IsaVRd in the first 2 years of frontline treatment for TIE patients with NDMM in the United States. <b>Methods:</b> Dosing schedules from the CEPHEUS and IMROZ clinical US trials were used for this analysis. AnalySource® was utilized to access the First Databank drug pricing database to collect current US DACs. Drug administration time and costs were identified and weighted, assuming 40% and 60% received the drug in a hospital outpatient and community oncology setting, respectively. Total costs were calculated by adding DACs and drug administration costs. <b>Results:</b> The DAC was <math><mn>200</mn> <mrow><mo> </mo></mrow> <mn>866</mn> <mi>i</mi> <mi>n</mi> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mn>1</mn> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 137 434 in year 2 for daratumumab and <math><mn>212</mn> <mrow><mo> </mo></mrow> <mn>421</mn> <mi>i</mi> <mi>n</mi> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mn>1</mn> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 144 143 in year 2 for isatuximab. The DAC of daratumumab was <math><mn>18</mn> <mrow><mo> </mo></mrow> <mn>264</mn> <mo>(</mo> <mn>5.4</mn></math> 17 269 and <math><mn>17</mn> <mrow><mo> </mo></mrow> <mn>327</mn> <mi>l</mi> <mi>e</mi> <mi>s</mi> <mi>s</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>n</mi> <mi>I</mi> <mi>s</mi> <mi>a</mi> <mi>V</mi> <mi>R</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>s</mi> <mo><</mo> <mn>75</mn> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mi>s</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mo>≥</mo> <mn>75</mn> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mi>s</mi> <mi>o</mi> <mi>l</mi> <mi>d</mi> <mo>,</mo> <mi>r</mi> <mi>e</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> <mi>l</mi> <mi>y</mi> <mo>.</mo> <mi>I</mi> <mi>n</mi> <mi>y</mi> <mi>e</mi> <mi>a</mi> <mi>r</mi> <mn>2</mn> <mo>,</mo> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>t</mi> <mi>o</mi> <mi>t</mi> <mi>a</mi> <mi>l</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>o</mi> <mi>f</mi> <mi>D</mi> <mi>V</mi> <mi>R</mi> <mi>d</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>w</mi> <mi>a</mi> <mi>s</mi></math> 10 444 and <math><mn>10</mn> <mrow><mo> </mo></mrow> <mn>553</mn> <mi>l</mi> <mi>e</mi> <mi>s</mi> <mi>s</mi> <mi>t</mi> <mi>h</mi> <mi>a</mi> <mi>n</mi> <mi>I</mi> <mi>s</mi> <mi>a</mi> <mi>V</mi> <mi>R</mi> <mi>d</mi> <mi>i</mi> <mi>n</mi> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"27-31"},"PeriodicalIF":2.3,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12285652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144698653","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":"Optimizing Oncology Generic Medication Selection in the Gulf Region: Expert Consensus and MCDA Tool Development.","authors":"Anas Hamad, Omar AbdulAziz, Osama Abu Tabar, Sara Al Balushi, Sana Alblooshi, Mohamad Alfar, Eren Dawoud, Fahmeeda Khan, Abdulrahman Nabeel, Wael Sayam, Ahmad ElSheashaey","doi":"10.36469/001c.140955","DOIUrl":"10.36469/001c.140955","url":null,"abstract":"<p><p><b>Background:</b> Cancer remains a leading global health challenge, with high mortality rates and increasing financial burdens on healthcare systems. In the Gulf Cooperation Council countries, generic oncology medications offer a cost-effective alternative to patented treatments. However, disparities in regulatory frameworks and concerns about quality and supply reliability hinder their widespread adoption. Addressing these challenges is crucial to ensuring optimal access to oncology treatments. <b>Methods:</b> This study employed multicriteria decision analysis to evaluate key factors influencing the selection of off-patent oncology drugs. A structured survey was conducted among 10 formulary management experts from the United Arab Emirates, Qatar, Kuwait, Bahrain, and Oman. Participants engaged in a 3-hour workshop to assess and prioritize critical decision-making criteria through guided discussions, case studies, and weighting surveys. <b>Results:</b> The analysis identified manufacturing quality (30.8%), cost (20%), and use in reference countries (14.6%) as the most critical factors in selecting generic oncology mediations. Supply reliability (13%), regulatory aspects (8%), and pharmacovigilance services and extra services (6.8% each) also played supporting roles. The structured evaluation framework developed through this study provides insights into the factors shaping formulary decisions in the Gulf Cooperation Council region and highlights areas requiring regulatory and logistical improvements. <b>Conclusion:</b> This expert consensus underscores the need for a balanced approach that ensures quality, affordability, and accessibility in adopting oncology generics. Strengthening regulatory frameworks, improving pharmacovigilance, and enhancing stakeholder education are essential steps to optimizing the integration of oncology drugs into regional healthcare systems. These findings provide a foundation for policy recommendations aimed at improving generic drug adoption and patient outcomes in oncology care.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"21-26"},"PeriodicalIF":2.3,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12276732/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144674964","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":"Characteristics and Comorbidities Influencing Mortality Risk Among Hereditary Angioedema Patients.","authors":"Subhan Khalid, Alan T Hitch","doi":"10.36469/001c.141747","DOIUrl":"10.36469/001c.141747","url":null,"abstract":"<p><p><b>Background:</b> Patients with hereditary angioedema (HA) face a heightened mortality risk due to multiple factors. <b>Objective:</b> The purpose of this study was to identify patient demographics or comorbidities associated with higher mortality risk using Bayesian network analysis. <b>Methods:</b> Data from the 2021 Nationwide Inpatient Sample were used to identify hospitalized patients with HA. Patient demographics, comorbidities, and severity measures were analyzed, and a Bayesian network model was developed to assess factors contributing to mortality risk. Structure learning was performed using a directed acyclic graph and probability estimating using Bayesian inference. Model performance was validated using a 70/30 training-testing split and assessed via area under the curve. <b>Results:</b> Older HA patients and those with autoimmune conditions, hypertension, or low income were at higher risk of mortality. Elevated risk was also observed across certain racial groups, insurance types, and income levels. Notably, older Black patients from the Midwest exhibited the highest estimated mortality risk. <b>Conclusion:</b> The Bayesian network demonstrated strong predictive performance, highlighting its potential for identifying high-risk subgroups and supporting targeted clinical interventions.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"11-20"},"PeriodicalIF":2.3,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12276015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144674963","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":"Effect of Fresh Frozen Plasma Infusion on Hospital Length of Stay for Patients With Hereditary Angioedema.","authors":"Subhan Khalid, Alan T Hitch","doi":"10.36469/001c.141171","DOIUrl":"10.36469/001c.141171","url":null,"abstract":"<p><p><b>Background:</b> Patients with hereditary angioedema treated with fresh frozen plasma (FFP) infusion face complications and risk of side effects. <b>Objective:</b> To study the effect of FFP infusion on hospital length of stay for patients with hereditary angioedema. <b>Methods:</b> Data from the 2021 Nationwide Inpatient Sample were used to identify hospitalized patients with hereditary angioedema. Patient demographics, comorbidities, and severity measures were analyzed, and a Bayesian additive regression tree model was used to assess factors contributing to length of stay. <b>Results:</b> FFP infusion was found to be associated with increased length of stay for patients with risk factors such as respiratory, cardiovascular disease, or urticaria. <b>Conclusions:</b> Caution is recommended when planning to use FFP, to ensure that underlying patient conditions and risk factors are thoroughly understood. The findings emphasize the need for personalized treatment plans based on individual risk factors, with a recommendation for prioritizing C1-inhibitor therapy over FFP.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 2","pages":"1-10"},"PeriodicalIF":2.3,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12251561/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626531","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":"Characteristics, Treatment Patterns, Healthcare Resource Utilization, and Costs Among Patients with Multifocal Motor Neuropathy: A US Claims Database Cohort Study.","authors":"Nikhil Khandelwal, Marie Sanchirico, Ade Ajibade, Kiraat Munshi, Michelle Vu, Nicole Engel-Nitz, Christina Steiger, Amy J Anderson, Chafic Karam","doi":"10.36469/001c.140817","DOIUrl":"10.36469/001c.140817","url":null,"abstract":"<p><p><b>Background:</b> Multifocal motor neuropathy (MMN) is a rare, slowly progressive nerve disorder characterized by asymmetric limb weakness without sensory abnormalities. MMN is often misdiagnosed due to similarities in clinical symptoms with conditions including amyotrophic lateral sclerosis (ALS), making diagnosis and treatment challenging. <b>Objectives:</b> This study assessed patient characteristics, treatment patterns, and the economic burden of MMN in the United States. <b>Methods:</b> Using the Optum Research Database, this retrospective analysis included patients with ≥1 diagnostic or nondiagnostic medical claim with an MMN diagnosis code between 2016 and 2020 (date of first diagnosis-related claim =index date), and continuous enrollment for 12 months preindex and postindex. Patients with MMN within this group were identified using more specific criteria; ≥2 MMN nondiagnostic claims separated by ≥30 days, with no subsequent ALS diagnosis during follow-up. All patients who did not meet these criteria were included in the MMN-mimic cohort. Outcomes included treatment patterns, differential diagnoses, healthcare utilization, and costs. <b>Results:</b> Of 904 patients identified, 37% had MMN and 63% had an MMN-mimic condition. Patients with MMN were significantly younger than patients in the MMN-mimic cohort (mean, 64.9 vs 66.8 years; <i>P</i> = .047). At preindex, significantly more patients with MMN received MMN-related medications than patients in the MMN-mimic cohort (20.5% vs 9.0%, respectively; <i>P</i> < .001). Intravenous immunoglobulin (IVIG) was the most common MMN-related medication. At postindex, more patients with MMN used IVIG (28.0%) compared with preindex (16.4%). In the 12 months preindex and postindex, >70% of patients had ≥1 differential diagnosis. The MMN cohort had higher all-cause total costs than the MMN-mimic cohort (mean preindex, <math><mn>58</mn> <mrow><mo> </mo></mrow> <mn>974</mn> <mi>v</mi> <mi>s</mi></math> 48 132, respectively [<i>P</i> = .100]; mean postindex, <math><mn>74</mn> <mrow><mo> </mo></mrow> <mn>187</mn> <mi>v</mi> <mi>s</mi></math> 50 652 [P = .002]); they also had significantly higher MMN-related healthcare costs (mean preindex, <math><mn>23</mn> <mrow><mo> </mo></mrow> <mn>625</mn> <mi>v</mi> <mi>s</mi></math> 12 890 [<i>P</i> = .011]; mean postindex, <math><mn>39</mn> <mrow><mo> </mo></mrow> <mn>521</mn> <mi>v</mi> <mi>s</mi></math> 11 938 [<i>P</i> < .001]). <b>Discussion:</b> This study showed that most patients with initial MMN diagnoses had an alternative disorder after subsequent evaluation/follow-up, and patients with MMN incurred higher costs. Many patients with MMN did not receive IVIG, suggesting that undertreatment or misattribution of diagnosis codes are common. <b>Conclusions:</b> Further education is needed regarding accurate diagnosis of MMN to ensure patient access to guideline-recommended treatment.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"261-268"},"PeriodicalIF":2.3,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12205905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144528271","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":"Size and Treatment Outcomes of HR+, HER2- Early Breast Cancer Population With High Risk of Recurrence: A Real-World Cohort Study With Danish Breast Cancer Cooperative Group Registry Data.","authors":"Heidi Loponen, Juha Mehtälä, Laila Mehkri, Astrid Torstensson, Anna Emde, Tero Ylisaukko-Oja, Walid Fakhouri","doi":"10.36469/001c.137277","DOIUrl":"10.36469/001c.137277","url":null,"abstract":"<p><p><b>Background:</b> While the prognosis is generally good for hormone receptor-positive (HR+), human epidermal growth factor-negative (HER2-) early breast cancer (EBC) patients, up to 30% of patients with high-risk clinical and/or pathologic features experience recurrence. <b>Objectives:</b> This retrospective cohort study was designed to estimate the proportion of BC patients meeting the high-risk criteria used in monarchE, a phase III study of abemaciclib, and to describe the characteristics, survival, and disease recurrence in a Danish patient population. <b>Methods:</b> The study cohort included all women with BC diagnosis registered in the Danish Breast Cancer Cooperative Group registry, and lumpectomy or mastectomy performed between January 1, 2010, and December 31, 2019. The patient characteristics and survival outcomes were compared between high-risk patients (≥4 positive lymph nodes or 1-3 positive nodes and grade 3 and/or primary tumor size ≥5 cm), low/moderate-risk patients, and patients with triple-negative EBC (TNBC). <b>Results:</b> A total of 13.0% of the HR+, HER2- EBC patients met the high-risk criteria. Five-year invasive disease-free survival (IDFS) and distant recurrence-free survival rates (DRFS) were significantly lower in the high-risk group (73.9% and 75.9%, respectively) and the TNBC group (73.0% and 76.5%, respectively), than the low/moderate-risk group (86.1% and 87.7%, respectively) (P < .0001). <b>Discussion:</b> This study is in line with earlier observations showing that HR+, HER2- is the most common subtype, accounting for over 70% of all BC cases. The size of the monarchE-like high-risk group aligns with previous evidence from large US cohort studies. We observed that the proportion of TNBC among all EBC patients showed a decreasing trend between 2010-2019, consistent with earlier reports. The 5-year IDFS and DRFS rates of high-risk patients observed in this study are in line with the evidence from a large US cohort study, however, slightly lower IDFS and DRFS rates at 5 years for the low/moderate-risk group were observed here. <b>Conclusion:</b> About 13.0% of the HR+, HER2- EBC patient population has a high risk of recurrence and would likely benefit from novel treatment strategies targeted for patients with a high risk of recurrence.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"252-260"},"PeriodicalIF":2.3,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12201145/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144505932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Impact of Enhanced Behavioral Health Services on Total Healthcare Costs Among US Employers: A Site-Level Analysis of 19 Cohort Studies.","authors":"Matt Hawrilenko, Casey Smolka, Emily Ward, RuthAnne Kavelaars, Millard Brown, Adam M Chekroud","doi":"10.36469/001c.138634","DOIUrl":"10.36469/001c.138634","url":null,"abstract":"<p><p><b>Background:</b> The return on investment (ROI) of mental health care is a critical metric in an era of cost-conscious healthcare decision-making. However, selective reporting of positive study results may inflate ROI estimates. <b>Objective:</b> To quantify the mean and variation in employer-level ROI outcomes for a comprehensive behavioral health benefit program. <b>Methods:</b> Data were obtained from 19 employer-specific studies conducted between May 2023 and December 2024. Sources included medical claims data spanning 12 months pre- and post-program launch, and program billing records of clinical and nonclinical costs. Studies were included if they were conducted by a single behavioral health benefit where the full sample of studies was known. The population included 19 US employers where employees and dependents received up to 12 free psychotherapy or medication management sessions. All studies used the same inclusion and exclusion criteria, retrospective matched cohort design, and difference-in-differences analysis. Data were abstracted following PRISMA guidelines. ROI was estimated using a difference-in-differences model to control for baseline medical spending and pooled using inverse variance weighting with a random effects structure. The primary outcome was the ROI multiple, defined as the ratio of gross per-member-per-month savings to total program spending. <b>Results:</b> The meta-analysis included 42 148 participants (14 645 program users and 27 503 matched controls) across a range of employer sizes and industries. The pooled ROI multiple was 2.3 (95% CI, 1.9-2.8), corresponding to net savings of <math><mn>159</mn> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>m</mi> <mi>e</mi> <mi>m</mi> <mi>b</mi> <mi>e</mi> <mi>r</mi> <mi>p</mi> <mi>e</mi> <mi>r</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>t</mi> <mi>h</mi> <mo>(</mo> <mn>95</mn></math> 111-$207). Significant heterogeneity was observed (I² = 67.8%; t² = 0.646; <i>P</i> < .001). A sensitivity analysis including nonclinical costs yielded a pooled ROI of 1.8. <b>Conclusion:</b> This meta-analysis, the largest of its kind, demonstrates that a centralized behavioral health benefit can consistently generate net savings across varied employer settings. These findings provide robust evidence to support the adoption of comprehensive mental health programs as an effective strategy for reducing overall medical spending in employer-sponsored health plans.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"246-251"},"PeriodicalIF":2.3,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182909/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475602","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}