Journal of Health Economics and Outcomes Research最新文献

{"title":"We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It?","authors":"Rebecca Smith, Samara Eisenberg, Aaron Turner-Pfifer, Jacqueline LeGrand, Sarah Pincus, Yousra Omer, Fei Wang, Bruce Pyenson","doi":"10.36469/001c.124604","DOIUrl":"10.36469/001c.124604","url":null,"abstract":"<p><p><b>Background:</b> Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients' access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. <b>Objectives:</b> This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. <b>Methods:</b> We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. <b>Results:</b> We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. <b>Discussion:</b> Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. <b>Conclusions:</b> Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"145-153"},"PeriodicalIF":2.3,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11566618/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142648041","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":"Retrospective Analysis of RSV Infection in Pediatric Patients: Epidemiology, Comorbidities, Treatment, and Costs in Dubai (2014-2023).","authors":"Jean Joury, Nawal Al Kaabi, Sara Al Dallal, Bassam Mahboub, Mostafa Zayed, Mohamed Abdelaziz, Jennifer Onwumeh-Okwundu, Mark A Fletcher, Subramanyam Kumaresan, Badarinath C Ramachandrachar, Mohamed Farghaly","doi":"10.36469/001c.123889","DOIUrl":"10.36469/001c.123889","url":null,"abstract":"<p><p><b>Background:</b> Infections attributable to respiratory syncytial virus (RSV) are a major cause of hospitalization among young children worldwide. Despite substantial clinical and economic burden, real-world data associated with RSV infections in the United Arab Emirates (UAE) are limited. <b>Objectives:</b> This study aimed to assess among children (<18 years) diagnosed with RSV the epidemiology, seasonality, comorbidities, treatment patterns, length of hospital stay, healthcare resource utilization (HCRU), and costs associated with pediatric infection in Dubai, UAE. <b>Methods:</b> This 10-year retrospective cohort study (Jan. 1, 2014-Sept. 30, 2023) utilized Dubai Real-World Database, a private insurance claims database. Patients aged <18 years with a first-episode diagnosis claim (primary or secondary, or a hospital admission) for RSV any time during the index period (Jan. 1, 2014-June 30, 2023) were included. Outcomes were analyzed during a 3-month follow-up. Patients were stratified into 3 cohorts: Cohort 1 (<2 years), Cohort 2 (2 to <6 years), and Cohort 3 (6 to <18 years). <b>Results:</b> Of 28 011 patients identified, 25 729 were aged <18 years with RSV infection. An increasing trend in reported cases was observed from 2014 to 2022, with an average annual increase of 55%. Half of study patients (49.3%) belonged to Cohort 1, with a mean age of 0.6 years, while less than 2% had known risk factors and 22% of the patients in cohort 1 were hospitalized. In Cohort 1, 32.0% had upper respiratory tract infections, 39.4% had lower respiratory tract infections, and 44.4% of patients had an \"other respiratory disease.\" The average length of hospitalization was about 4 days across all cohorts. The total hospitalization cost was highest in patients <2 years, amounting to US <math><mn>9</mn> <mrow><mo> </mo></mrow> <mn>798</mn> <mrow><mo> </mo></mrow> <mn>174</mn> <mo>(</mo> <mi>m</mi> <mi>e</mi> <mi>d</mi> <mi>i</mi> <mi>a</mi> <mi>n</mi> <mo>,</mo> <mi>U</mi> <mi>S</mi></math> 2241.30). <b>Conclusion:</b> Among the RSV patients, 49.3% were <2 years of age and few had recognized risk factors. Among patients <2 years, 22% were hospitalized, with an average hospital stay of 4 days; the cost of hospitalization totaled US $9 798 174. These findings can inform healthcare stakeholders about future policy measures and the need for effective preventive strategies.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"133-144"},"PeriodicalIF":2.3,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11580195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142687047","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":"Assessing the Fiscal Burden of Overweight and Obesity in Japan through Application of a Public Economic Framework.","authors":"Ataru Igarashi, Cillian Copeland, Nikos Kotsopoulos, Riku Ota, Silvia Capucci, Daisuke Adachi","doi":"10.36469/001c.123991","DOIUrl":"https://doi.org/10.36469/001c.123991","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Introduction:&lt;/b&gt; Obesity continues to represent a significant public health concern, with a broad impact from both a health and economic perspective. &lt;b&gt;Objective:&lt;/b&gt; This analysis assesses the fiscal consequences of overweight and obesity (OAO) in Japan by capturing obesity-attributable lost tax revenue and increased government transfers using a government perspective. &lt;b&gt;Methods:&lt;/b&gt; The fiscal burden of OAO was estimated using an age-specific prevalence model, which tracked the Japanese population across different body mass index (BMI) categories. The model was populated with fiscal data for Japan, including employment activity and government spending, to calculate tax revenue and transfer costs. A targeted literature review was conducted to identify data estimating the impact of OAO on employment, income, sick leave, retirement, and mortality. These modifiers were applied to Japanese epidemiological and fiscal projections to calculate government tax revenue and spending. The incremental impact of reducing OAO in the general population was subsequently calculated. Results were estimated based on the 2023 Japanese working-age population aged 18 to 70 years. &lt;b&gt;Results:&lt;/b&gt; The total fiscal burden of OAO in Japan, defined as BMI of at least 25, is estimated at US &lt;math&gt;&lt;mn&gt;13.41&lt;/mn&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;l&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;mo&gt;(&lt;/mo&gt; &lt;mrow&gt;&lt;mo&gt;¥&lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;1925&lt;/mn&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;l&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;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&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;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&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;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mn&gt;0.4&lt;/mn&gt;&lt;/math&gt; 6.3 billion (¥901 billion) and &lt;math&gt;&lt;mn&gt;1.2&lt;/mn&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;l&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;mo&gt;(&lt;/mo&gt; &lt;mrow&gt;&lt;mo&gt;¥&lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;179&lt;/mn&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;l&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;mo&gt;)&lt;/mo&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;r&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;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;r&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;t&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;x&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;p&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;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;o&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;mi&gt;m&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;y&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;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;g&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;s&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;k&lt;/mi&gt; &lt;mi&gt;l&lt;/mi","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"125-132"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11555824/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142621935","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 HbA1c Changes Among Type 2 Diabetes Mellitus Patients Initiating Treatment With a 1.0 Mg Weekly Dose of Semaglutide for Diabetes.","authors":"Noelle N Gronroos, Caroline Swift, Monica S Frazer, Andrew Sargent, Michael Leszko, Erin Buysman, Sara Alvarez, Tyler J Dunn, Josh Noone","doi":"10.36469/001c.124111","DOIUrl":"10.36469/001c.124111","url":null,"abstract":"<p><p><b>Background:</b> Medical management of patients with type 2 diabetes mellitus (T2DM) is complex because of the chronic nature of the disease and its associated comorbidities. Injectable once-weekly semaglutide for diabetes (OW sema T2D) is a type of glucagon-like peptide-1 receptor agonist approved for the treatment of patients with T2DM. <b>Objectives:</b> To describe patient characteristics and HbA1c changes for patients prescribed 1.0 mg maintenance dose OW sema T2D. <b>Methods:</b> This retrospective study included adult patients with T2DM with a pre-index glycated hemoglobin (HbA1c) of at least 7%, initiating treatment with OW sema T2D between January 1, 2018, and December 31, 2019, and prescribed a 1.0 mg maintenance dose. Patients were identified in the Optum Research Database and were included if they had continuous health plan enrollment for at least 12 months prior to (pre-index) and at least 12 months following (post-index) the date of the first OW sema T2D claim (index). Dose at initiation and prescriber specialty were captured. Change in HbA1c between the latest post-index and pre-index HbA1c measurement was calculated among all patients and among those with at least 90 days of continuous treatment (persistent patients). <b>Results:</b> A total of 2168 patients were included in this study. On average, patients were taking 13.5 different classes of medications. The majority of patients had lipid metabolism disorder (90.8%), hypertension (86.6%), diabetes with complications (86.8%), or other nutritional/endocrine/metabolic disorders (72.5%). The mean HbA1c reduction was 1.2% (P < .001). Patients persistent with OW sema T2D (n =1280) had a mean HbA1c reduction of 1.4% (P < .001). The mean (SD) days covered with a 1.0 mg maintenance dose was 236.1 (94.1) days. <b>Discussion:</b> Despite being medically complex, the patients in this real-world study experienced significant reductions in HbA1c following initiation of OW sema T2D. <b>Conclusions:</b> A 1.0 mg maintenance dose of OW sema T2D is an effective treatment for T2DM in the real world.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"118-124"},"PeriodicalIF":2.3,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11539928/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590761","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 Utilization Among Patients With Agitation in Alzheimer Dementia.","authors":"Christie Teigland, Zulkarnain Pulungan, David Bruhn, Steve Hwang","doi":"10.36469/001c.124455","DOIUrl":"10.36469/001c.124455","url":null,"abstract":"<p><p><b>Background:</b> Agitation in Alzheimer dementia is common, but the associated healthcare burden remains unclear. <b>Objective:</b> This retrospective analysis evaluated baseline characteristics, healthcare resource utilization, and costs among patients with agitation in Alzheimer dementia and those without agitation in Alzheimer dementia. <b>Methods:</b> Medicare beneficiaries from 100% of the Medicare Fee-for-Service claims database (2009-2016) with 2 or more claims 30 or more days apart for both Alzheimer's disease and dementia and continuous enrollment with medical/pharmacy coverage for 6 months before and 12 months after the index diagnosis were included. Patients with agitation in Alzheimer dementia were identified by 2 or more claims 14 or more days apart using <i>International Classification of Diseases-9-CM/-10-CM</i> codes based on the provisional International Psychogeriatric Association agitation definition. Patients with severe psychiatric disorders were excluded. Two cohorts of patients (with and without agitation) were then defined, and patient characteristics, healthcare resource utilization, and costs were compared in a descriptive exploratory analysis. <b>Results:</b> Of 2 684 704 Fee-for-Service patients with Alzheimer dementia, 769 141 met all inclusion criteria; among these, 281 042 (36.5%) had agitation. The mean age in patients with and without agitation in Alzheimer dementia was 83 years. Most patients in both groups were female, but the proportion of males was slightly higher in the agitation in Alzheimer dementia group (30.3% vs 28.2%, respectively). Patients with agitation in Alzheimer dementia were more likely than those without agitation in Alzheimer dementia to have lower socioeconomic status (dual eligibility for Medicaid, 45.0% vs 41.7%, respectively) or be disabled (10.5% vs 9.4%). Overall, healthcare costs were higher in the agitation in Alzheimer dementia population compared with those without agitation in Alzheimer dementia (mean cost PPPY, <math><mn>32</mn> <mrow><mo> </mo></mrow> <mn>322</mn> <mi>a</mi> <mi>n</mi> <mi>d</mi></math> 30 121, respectively), with the largest differences observed in inpatient and post-acute care costs. <b>Conclusions:</b> These exploratory findings underscore the substantial economic burden of agitation in Alzheimer dementia and highlight the need for treatment options for the agitation in Alzheimer dementia population to improve associated health outcomes.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"118-124"},"PeriodicalIF":2.3,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11539929/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590758","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 Potential Medication Wastage Due to Switching Treatment Among People With Multiple Sclerosis.","authors":"Darin T Okuda, Achal Patel, Robert Schuldt, Ibraheem Abioye, Nicole G Bonine","doi":"10.36469/001c.123336","DOIUrl":"10.36469/001c.123336","url":null,"abstract":"<p><p><b>Background:</b> Unused medications negatively impact healthcare resource utilization and environmental safety, contribute substantially to annual healthcare expenditures, and may ultimately affect patient health outcomes. People with multiple sclerosis (PwMS) commonly switch disease-modifying therapies (DMTs), leading to medication wastage and substantial costs for insurers and patients. <b>Objectives:</b> To estimate the cost associated with potential medication wastage (PMW) in a subcohort of PwMS receiving oral or self-injectable US Food and Drug Administration-approved DMTs who switched DMTs in a calendar year in the United States. <b>Methods:</b> This retrospective cohort study included adults with MS and used PharMetrics® Plus claims data from 2017 to 2021. PwMS were required to have 12 months of continuous eligibility for the entire year and a claim for at least 2 unique DMTs during the same calendar year. The PMW cohort was defined as those who had an aggregate overlap in days' supply across DMT switches within the year; those in the non-PMW cohort did not. The cost of PMW for insurers and PwMS due to overlap was calculated only at the point of switch to the new DMT and defined as the cost of the remaining days' supply of the prior DMT. <b>Results:</b> The number of PwMS meeting the inclusion criteria was 1762 in 2017, 1947 in 2018, 1679 in 2019, 1461 in 2020, and 1782 in 2021. Approximately 95% of PwMS switched DMTs once within single calendar years, and 25% (n = 381-464) contributed to PMW. For those who had overlapping DMT supply, it was estimated that 34% to 38% of the DMT being switched from was potentially wasted. The total cost of PMW paid by the insurer and PwMS ranged from <math><mn>1</mn> <mrow><mo> </mo></mrow> <mn>200</mn> <mrow><mo> </mo></mrow> <mn>866</mn> <mi>t</mi> <mi>o</mi></math> 1 489 859. While most of the total cost ( <math><mn>1</mn> <mrow><mo> </mo></mrow> <mn>172</mn> <mrow><mo> </mo></mrow> <mn>140</mn> <mo>-</mo></math> 1 450 328) was paid by the insurer, PwMS still owed substantial amounts ( <math><mn>28</mn> <mrow><mo> </mo></mrow> <mn>726</mn> <mo>-</mo></math> 74 578). Across all PwMS, the per person per year cost ranged from <math><mn>716</mn> <mi>t</mi> <mi>o</mi></math> 846. The estimated wastage and associated costs were consistent across all study years. <b>Conclusions:</b> DMT switching is common among PwMS, resulting in PMW and high costs to patients and insurers.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"103-108"},"PeriodicalIF":2.3,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545867","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":"Adherence to First-Line Intravesical Bacillus Calmette-Guérin Therapy in the Context of Guideline Recommendations for US Patients With High-Risk Non-muscle Invasive Bladder Cancer.","authors":"Franklin D Gaylis, Bruno Emond, Ameur M Manceur, Anabelle Tardif-Samson, Laura Morrison, Dominic Pilon, Patrick Lefebvre, Lorie A Ellis, Hiremagalur Balaji, Andrea Ireland","doi":"10.36469/001c.124208","DOIUrl":"10.36469/001c.124208","url":null,"abstract":"<p><p><b>Background:</b> Bacillus Calmette-Guérin (BCG) can reduce recurrence and delay progression among patients with high-risk non-muscle invasive bladder cancer (NMIBC), but is associated with a substantial emotional, physical, and social burden. <b>Objectives:</b> This study evaluated the adequacy of first-line intravesical BCG treatment among high-risk NMIBC patients in the United States, including the subgroup with carcinoma in situ (CIS) of the bladder. <b>Methods:</b> Adults with high-risk NMIBC treated with BCG were selected from de-identified MarketScan® Commercial, Medicare, and Medicaid Databases (1/1/2010-2/28/2021). Adequacy of BCG induction and maintenance was evaluated from the first BCG claim until the end of the patient's observation, using a previously published claims-based algorithm (induction: ≥5 instillations within 70 days; induction and maintenance: ≥7 instillations within 274 days of first instillation) and a definition based on the landmark Southwest Oncology Group (SWOG) trial (induction: ≥5 instillations without gaps >7 days; followed by ≥2 instillations at month 3, 6, and every 6 months thereafter). Proportions of patients with adequate BCG induction and maintenance were reported overall and compared between those with and without CIS. <b>Results:</b> Of 5803 high-risk NMIBC patients treated with first-line BCG (mean age, 67.3 years; 20.6% female), 930 (16.0%) had CIS. After first-line BCG, 56.6% received another treatment. Although 86.9% had adequate BCG induction based on the claims-based algorithm (SWOG, 73.6%), only 41.5% had adequate BCG induction and maintenance (SWOG, 1.6%). Similar trends were observed for patients with and without CIS, with higher adherence to guidelines for patients with CIS (adequate induction using claims-based algorithm: 90.3% vs 86.2%; adequate induction and maintenance: 50.8% vs 39.7%, all <i>P</i> < .001). A greater proportion of CIS patients than non-CIS patients had cystectomy (CIS, 14.4%, non-CIS, 8.5%; <i>P</i> < .001) after first-line BCG. <b>Discussion:</b> Among patients with NMIBC treated with first-line intravesical BCG, most received adequate BCG induction but less than half had adequate BCG maintenance. BCG treatment was also inadequate for patients with CIS, with only half of patients receiving adequate BCG maintenance and a higher proportion undergoing cystectomy following first-line BCG. <b>Conclusions:</b> Results emphasize the need for additional treatment options for patients with NMIBC.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"109-117"},"PeriodicalIF":2.3,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545866","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":"Patient Experience of Living With Hemophilia A: A Conceptual Model of Humanistic and Symptomatic Experience in Adolescents, Adults, and Children.","authors":"Zalmai Hakimi, Rakhee Ghelani, Linda Bystrická, Nana Kragh, Patrick Marquis, Jameel Nazir, Nadine McGale","doi":"10.36469/001c.123374","DOIUrl":"10.36469/001c.123374","url":null,"abstract":"<p><p><b>Background:</b> People living with hemophilia A face challenges impacting their daily lives despite treatment innovations. Previous studies have explored perceptions and treatment experiences; however, there is a lack of an evidence-based, comprehensive model to identify concepts (clinical, physical, and psychological functioning) relevant for people with hemophilia A (PwHA). <b>Objectives:</b> The aim of this qualitative study was to address the question: What is the humanistic and symptomatic experience of adolescents, adults, and children living with hemophilia A and what is the impact of hemophilia A on their quality of life? <b>Methods:</b> Participants, identified through patient associations in the UK, were male PwHA and caregivers of male PwHA receiving prophylactic treatment. Qualitative research was conducted involving semistructured telephone interviews with PwHA and caregivers between April 2020 and September 2020 in the UK. Standard analytical techniques of conceptual model development were used. <b>Results:</b> Of 30 participants, 23 were PwHA and 7 were caregivers. A conceptual model was produced describing patient experience of symptoms, physical functioning, treatment experiences, and the impact of symptoms and treatment on daily lives. Participants reported hemophilia-related symptoms, including bleeding, pain, and joint stiffness, as well as difficulties engaging with social and leisure activities. They also reported protection from bleeds provided by their treatment, relief from symptoms, and the resultant sense of normality. Concepts were broadly relevant across all age groups; however, psychological impacts were reported only by adult PwHA, and caregivers reported impacts related to outdoor activities, play, and education. Participants indicated that their ideal treatment would be delivered orally. <b>Discussion:</b> This study highlights the range of symptoms experienced by PwHA across a broad range of age groups, thus enabling the evaluation of relevant concepts across different stages of life. The research supports development of a conceptual model documenting symptoms, impacts, and treatment experience relevant to PwHA. <b>Conclusion:</b> Insights gathered through the interviews and resulting conceptual model support development of new therapies to address the physical and social challenges identified by PwHA and highlight a need for novel hemophilia A treatments that can ease treatment administration, provide adequate level of protection, and enable life to be lived normally.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"95-102"},"PeriodicalIF":2.3,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523570/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545868","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":"Artificial Intelligence in Identifying Patients With Undiagnosed Nonalcoholic Steatohepatitis.","authors":"Onur Baser, Gabriela Samayoa, Nehir Yapar, Erdem Baser","doi":"10.36469/001c.123645","DOIUrl":"10.36469/001c.123645","url":null,"abstract":"<p><p><b>Background:</b> Although increasing in prevalence, nonalcoholic steatohepatitis (NASH) is often undiagnosed in clinical practice. <b>Objective:</b> This study identified patients in the Veterans Affairs (VA) health system who likely had undiagnosed NASH using a machine learning algorithm. <b>Methods:</b> From a VA data set of 25 million adult enrollees, the study population was divided into NASH-positive, non-NASH, and at-risk cohorts. We performed a claims data analysis using a machine learning algorithm. To build our model, the study population was randomly divided into an 80% training subset and a 20% testing subset and tested and trained using a cross-validation technique. In addition to the baseline model, a gradient-boosted classification tree, naïve Bayes, and random forest model were created and compared using receiver operator characteristics, area under the curve, and accuracy. The best performing model was retrained on the full 80% training subset and applied to the 20% testing subset to calculate the performance metrics. <b>Results:</b> In total, 4 223 443 patients met the study inclusion criteria, of whom 4903 were positive for NASH and 35 528 were non-NASH patients. The remainder was in the at-risk patient cohort, of which 514 997 patients (12%) were identified as likely to have NASH. Age, obesity, and abnormal liver function tests were the top determinants in assigning NASH probability. <b>Conclusions:</b> Utilization of machine learning to predict NASH allows for wider recognition, timely intervention, and targeted treatments to improve or mitigate disease progression and could be used as an initial screening tool.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"86-94"},"PeriodicalIF":2.3,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11441708/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142348171","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial.","authors":"Alasdair MacCulloch, Alison Griffiths, Neil Johnson, Simon Shohet","doi":"10.36469/001c.121928","DOIUrl":"https://doi.org/10.36469/001c.121928","url":null,"abstract":"<p><p><b>Background:</b> Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzyme replacement therapy improves health outcomes for adults with late-onset Pompe disease (LOPD). PROPEL was a Phase 3, double-blind, randomized controlled trial, which evaluated cipaglucosidase alfa plus miglustat, vs alglucosidase alfa plus placebo in 123 adult patients with LOPD (clinicaltrials.gov: NCT03729362). <b>Objectives:</b> To analyze EQ-5D health-related quality of life (HRQoL) utility data from PROPEL. <b>Methods:</b> Multilevel modeling techniques (mixed regression methods) were used to analyze PROPEL EQ-5D-3L estimates and predict utility values for 7 health states previously identified in an economic evaluation for LOPD. In PROPEL, EQ-5D-5L values were assessed at screening and at weeks 12, 26, 38, and 52. EQ-5D-5L utility values were mapped to EQ-5D-3L values using the van Hout algorithm as recommended by the EuroQoL and the National Institute of Health and Care Excellence position statement at time of analysis. UK population tariffs were applied for all EQ-5D utility valuations. Utility values were predicted according to 6-minute walk distance (6MWD) and percent predicted sitting forced vital capacity. <b>Results:</b> The mixed model predicted that EQ-5D-3L utility values for patients who could walk >75 m with LOPD ranged between 0.55 and 0.67 according to patient 6MWD and respiratory function. In this analysis, patients with a 6MWD ≤75 m, consistent with a health state requiring wheelchair support in the economic analysis, had a predicted utility value of 0.49. There were few patients in PROPEL who could walk ≤75 m at any time point in the study, hence, these utility estimates should be interpreted with caution. EQ-5D-3L utility estimates from PROPEL were consistent with previously reported EQ-5D-3L values in LOPD. <b>Conclusions:</b> Overall, the results from our analysis indicate that important HRQoL losses are associated with reductions in mobility and respiratory function for patients with Pompe disease. The study provides important evidence of HRQoL utility values for patients with advanced LOPD, a population for whom published data are limited.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"80-85"},"PeriodicalIF":2.3,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11420789/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142348152","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}