Journal of Health Economics and Outcomes Research最新文献

{"title":"Are Trends in Economic Modeling of Pediatric Diabetes Mellitus up to Date with the Clinical Practice Guidelines and the Latest Scientific Findings?","authors":"Roque Cardona-Hernandez, Alberto de la Cuadra-Grande, Julen Monje, María Echave, Itziar Oyagüez, María Álvarez, Isabel Leiva-Gea","doi":"10.36469/001c.127920","DOIUrl":"10.36469/001c.127920","url":null,"abstract":"<p><p><b>Background:</b> Modeling techniques in the field of pediatrics present unique challenges beyond traditional model limitations, and sometimes difficulties in faithfully simulating the condition's evolution over time. <b>Objective:</b> This study aimed to identify whether economic modeling approaches in diabetes in pediatric patients align with the recommendations of clinical practice guidelines and the latest scientific evidence. <b>Methods:</b> A literature review was performed in March 2023 to identify modeling-based economic evaluations in diabetes in pediatric patients. Data were extracted and synthesized from eligible studies. Clinical practice guidelines for diabetes were gathered to compare their alignment with modeling strategies. Two endocrinology specialists provided insights on the latest findings in diabetes that are not yet included in the guidelines. A multidisciplinary group of experts agreed on the relevant themes to conduct the comparative analysis: parameter informing on glycemic control, diabetic ketoacidosis/hypoglycemia, C-peptide as prognostic biomarker, metabolic memory, age at diagnosis, socioeconomic status, pediatric-specific sources of risk equations, and pediatric-specific sources of utilities/disutilities. <b>Results:</b> Nineteen modeling-based studies (7 de novo, 12 predesigned models) and 34 guidelines were selected. Hemoglobin A1c was the main parameter to model the glycemic control; however, guidelines recommend the usage of complementary measures (eg, time in range) which are not included in economic models. Eight models included diabetic ketoacidosis (42.1%), 16 included hypoglycemia (84.2%), 2 included C-peptide (1 of those as prognostic factor) (10.5%) and 1 included legacy effect (5.3%). Neither guidelines nor models included recent findings, such as age at diagnosis or socioeconomic status, as prognostic factors. The lack of pediatric-specific sources for risk equations and utility/disutility values were additional limitations. <b>Discussion:</b> Economic models designed for assessing interventions in diabetes in pediatric patients should be based on pediatric-specific data and include novel adjuvant glucose-monitoring metrics and latest evidence on prognostic factors (C-peptide, legacy effect, age at diagnosis, socioeconomic status) to provide a more faithful reflection of the disease. <b>Conclusions:</b> Economic models represent useful tools to inform decision making. However, further research assessing the gaps is needed to enhance evidence-based health economic modeling that best represents reality.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"30-50"},"PeriodicalIF":2.3,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11797704/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255734","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 Associated with Leukopenia and Neutropenia in Kidney Transplant Recipients Receiving Valganciclovir in the United States.","authors":"Qinghua Li, Vladimir Turzhitsky, Pamela Moise, Harry Jin, Kaylen Brzozowski, Irina Kolobova","doi":"10.36469/001c.125097","DOIUrl":"10.36469/001c.125097","url":null,"abstract":"<p><p><b>Background:</b> Cytomegalovirus prophylaxis in kidney transplant recipients (KTRs) is limited by post-transplant neutropenia and leukopenia (PTN/PTL). Despite its clinical significance, the healthcare resource utilization (HCRU) related to PTN/PTL remains poorly characterized. <b>Objective:</b> To evaluate HCRU among KTRs taking valganciclovir during their first year post-transplant. <b>Methods:</b> Using TriNetX Dataworks-USA, a federated, de-identified electronic medical record database, we identified adult KTRs who underwent their first kidney transplant from January 2012 to September 2020. All eligible patients were followed for 1 year. PTN/PTL was defined as absolute neutrophil count less than 1000/μL or white blood cell count less than 3500/μL. Multivariable logistic/Poisson regression models were used to assess the association between PTN/PTL and various HCRU types. <b>Results:</b> A total of 8791 KTRs were identified, of whom 6219 (70.7%) developed PTN/PTL at a mean of 5.7 months post-transplantation. Hospitalizations, rehospitalizations, emergency room visits, outpatient appointments, packed red blood cell transfusions, and granulocyte-colony stimulating factor administration were more prevalent among KTRs with PTN/PTL (61.1% vs 49.5%, 24.5% vs 14.1%, 35.2% vs 28.9%, 30.4 vs 26.2 visits, 22.3% vs 17.6%, 23.4% vs 2.2%, respectively; P < .001). Adjusted analyses confirmed that PTN/PTL correlated with increased HCRU across all categories. <b>Conclusions:</b> KTRs who developed PTN/PTL had significantly higher HCRU. Further studies are needed to evaluate strategies addressing PTN/PTL for KTRs.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"22-29"},"PeriodicalIF":2.3,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11784900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080474","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":"Glycogen Storage Disease Type Ia: A Retrospective Claims Analysis of Complications, Resource Utilization, and Cost of Care.","authors":"Eliza Kruger, Justin Nedzesky, Nina Thomas, Jeffrey D Dunn, Andrew A Grimm","doi":"10.36469/001c.125886","DOIUrl":"10.36469/001c.125886","url":null,"abstract":"<p><p><b>Background:</b> Glycogen storage disease type Ia (GSDIa) is a rare inherited disorder resulting in potentially life-threatening hypoglycemia, metabolic abnormalities, and complications often requiring hospitalization. <b>Objective:</b> This retrospective database analysis assessed the complications, resource utilization, and costs in a large cohort of patients with GSDIa. <b>Methods:</b> We conducted a retrospective cohort study of GSDIa patients and matched non-GSDIa comparators utilizing the PharMetrics® Plus database. International Classification of Diseases, Tenth Revision (ICD-10) diagnosis codes in any billing position for inpatient and outpatient claims (January 2016-February 2020) were identified for complications related to GSDIa. Healthcare use and costs were assessed by setting of care (inpatient, outpatient, physician office, emergency department, and pharmacy). <b>Results:</b> Overall, 557 patients with GSDIa and 5570 matched comparators (male, 63%; adults, 67%) were identified. The most frequent complications in patients with GSDIa vs comparators included anemia due to enzyme disorders (odds ratio, 4.0 × 103; 95% confidence interval, 555.9-2.8 × 104), hepatocellular adenoma (305.9; 41.6-2.2 × 104), liver transplantation (164.6; 21.8-1.2 × 103), and gastrostomy (152.2; 61.1-379.2), as well as acidosis (45.5; 29.4-70.3), hepatomegaly (43.6; 29.1-65.3), hyperuricemia (23.6; 11.9-46.9), and hypoglycemia (20.2; 14.3-28.7). Chronic complications (eg, gout, osteoarthritis, chronic kidney disease, and neoplasms) were more common in adults with GSDIa, whereas acute complications (eg, poor growth, gastrostomy, seizure, and hypoglycemia) were more common in children with GSDIa. Patients with GSDIa more often required hospitalization (0.53 vs 0.06 hospitalizations per patient per year) vs comparators, including 2 or more hospitalizations (26.6% vs 2.3%), longer length of stay (3.1 vs 0.4 days), and more annual visits in all care settings, including 4.3 times more visits in the emergency department. Mean annual total healthcare costs were higher for GSDIa patients vs comparators ( <math><mn>33</mn> <mrow><mo> </mo></mrow> <mn>910</mn> <mi>v</mi> <mi>s</mi></math> 4410). <b>Discussion:</b> In this large, retrospective database analysis, complications observed among patients with GSDIa were consistent with prior reports and demonstrate the chronic and progressive nature of the disease. Resource utilization was substantial in GSDIa patients, and mean annual total healthcare costs were almost 8 times higher than those of comparators. <b>Conclusions:</b> GSDIa is associated with numerous potentially serious and sometimes fatal complications, extensive resource utilization, and high management costs.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"13-21"},"PeriodicalIF":2.3,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11716495/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142950020","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":"Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands.","authors":"Holly Lumgair, Lisa Bashorum, Alasdair MacCulloch, Elizabeth Minas, George Timmins, Drago Bratkovic, Richard Perry, Medi Stone, Vasileios Blazos, Elisabetta Conti, Raymond Saich","doi":"10.36469/001c.126018","DOIUrl":"https://doi.org/10.36469/001c.126018","url":null,"abstract":"<p><p><b>Background:</b> Late-onset Pompe disease (LOPD) is a rare, autosomal recessive metabolic disorder that is heterogeneous in disease presentation and progression. People with LOPD report a significantly lower physical, psychological, and social quality of life (QoL) than the general population. <b>Objectives:</b> This study investigated how individuals' self-reported LOPD status (improving, stable, declining) relates to their QoL. Participant experiences such as use of mobility or ventilation aids, caregivers, symptomology, and daily life impacts were also characterized. <b>Methods:</b> A 2-part observational study was conducted online between October and December 2023 using the 36-item short-form tool (SF-36) and a survey. Adults with LOPD (N=41) from Australia, France, Italy, and the Netherlands were recruited. <b>Results:</b> Participants reporting \"declining\" LOPD status (56%) had lower physical functioning SF-36 scores than those reporting as \"stable\" or \"improving.\" Those self-reporting as stable or improving often described an acceptance of declining health in their responses. Physical functioning scores were generally stable in respondents who had been receiving enzyme replacement therapy (ERT) for 1-15 years, but those who had received ERT for >15 years had lower scores. Requiring ventilation and mobility aids had additive negative impacts on physical functioning. Difficulty swallowing, speaking, and scoliosis were the most burdensome symptoms reported by those on ERT for >15-25 years. <b>Discussion:</b> These results demonstrate the humanistic burden of LOPD; through declining physical functioning SF-36 scores over increasing time and increased use of aids, and also through factors related to self-reported LOPD status (where declining status was associated with lower scores) and symptomology variances. Taken holistically, these areas are valuable to explore when informing optimized care. Among a largely declining cohort, even those not self-reporting decline often assumed future deterioration, highlighting the need for improved therapies and the potential to initiate or switch ERT based on evolving symptomology and daily life impacts. <b>Conclusion:</b> Our results indicate that progressing LOPD leads to loss of QoL in ways that relate to time, use of aids, evolving symptomology, and the patient's own perspective. A holistic approach to assessing the individual can help ensure relevant factors are investigated and held in balance, supporting optimized care.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"12 1","pages":"1-12"},"PeriodicalIF":2.3,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11699855/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142931935","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-Utility Analysis of Add-on Cannabidiol vs Usual Care Alone for the Treatment of Seizures in Patients With Treatment-Resistant Lennox-Gastaut Syndrome or Dravet Syndrome in the Netherlands.","authors":"Jamshaed Siddiqui, Sally Bowditch","doi":"10.36469/001c.126071","DOIUrl":"10.36469/001c.126071","url":null,"abstract":"<p><p><b>Background:</b> Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are severe, treatment-refractory, epileptic encephalopathies that often develop in infancy or early childhood. Since December 1, 2022, plant-derived highly purified cannabidiol (CBD) medicine (Epidyolex®; 100 mg/mL oral solution) has been reimbursed in the Netherlands for the adjunctive treatment of seizures associated with LGS or DS. <b>Objective:</b> To estimate the cost-effectiveness of CBD plus usual care vs usual care alone in patients with LGS or DS in the Netherlands. <b>Methods:</b> A cohort-based Markov model from a Dutch societal perspective, based on seizure frequency and seizure-free days, was developed for patients receiving CBD plus usual care (antiseizure medications, including clobazam) or usual care alone. Population characteristics, clinical inputs, and utility values were sourced from CBD clinical trials and quality-of-life studies. Drug acquisition, disease management, adverse events, and societal costs from published literature were included. A 2019/2020 price year in euros was used. The model used a mean dosage of 12 mg/kg/day, a lifetime (90-year) horizon, and a 3-month cycle length. Discount rates of 4.0% and 1.5% per annum were applied to costs and outcomes, respectively. Uncertainty was explored through deterministic and probabilistic sensitivity analyses. <b>Results:</b> In patients with LGS, CBD plus usual care led to additional costs of €28 338 and increased quality-adjusted life-years (QALYs) of 1.318 compared with usual care alone. The incremental cost-effectiveness ratio of €21 493/QALY in LGS is below the willingness-to-pay threshold of €80 000/QALY in the Netherlands. In patients with DS, CBD plus usual care dominated usual care alone, with cost savings of €23 642 and increased QALYs of 0.868. The probability that CBD plus usual care is cost-effective in the Netherlands compared with usual care alone is 96% and 99% in patients with LGS and DS, respectively. <b>Discussion:</b> Elicitation methods were used to address data gaps in model inputs (eg, healthcare resource utilization and utilities); Dutch clinical experts, sensitivity, and scenario analyses validated this approach. <b>Conclusions:</b> Based on a willingness-to-pay threshold of €80 000, the base case cost-utility analysis demonstrated the cost-effectiveness of CBD plus usual care in patients with treatment-refractory LGS or DS aged 2 years or older in the Netherlands.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"168-179"},"PeriodicalIF":2.3,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11686597/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142914987","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 Effects of Adverse Events and Associated Costs on Value-Based Care for Metastatic Pancreatic Ductal Adenocarcinoma.","authors":"Prachi Bhatt, Jared Hirsch, Paul Cockrum, George Kim, Gabriela Dieguez","doi":"10.36469/001c.124367","DOIUrl":"10.36469/001c.124367","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Background:&lt;/b&gt; Rising oncology healthcare costs have led to value-based care reimbursement models that coordinate care and improve quality while reducing overall spending. These models are increasingly important for traditional Medicare and other payers. &lt;b&gt;Objectives:&lt;/b&gt; To compare the incidence of adverse events (AEs), AE-associated excess costs, and total cost of care (TCOC) of 3 cohorts receiving first-line treatment for metastatic pancreatic ductal adenocarcinoma (mPDAC). &lt;b&gt;Methods:&lt;/b&gt; We conducted a retrospective analysis of administrative claims data from 2018 to 2022 using the Medicare 100% Research Identifiable Files. We examined 3 cohorts receiving mPDAC treatment: FOLFIRINOX (FFX) (oxaliplatin, irinotecan, leucovorin, 5-FU bolus and infusion); modified FFX, (5-FU infusion only); and gemcitabine/nab-paclitaxel (gem/abrax). We compared the incidence of clinically significant AEs, TCOC, components of TCOC, and costs related to AEs/treatment toxicity. &lt;b&gt;Results:&lt;/b&gt; Patient AE rates ranged from 6.2% to 51.7%. AEs occurred more frequently in patients receiving FFX with all 4 components. Patients receiving brand name gem/abrax had lower rates of febrile neutropenia (6.2%) and neutropenia (22.2%) than those receiving FFX with no 5-FU bolus (febrile neutropenia, 9.9%; neutropenia, 36.9%) and FFX with all 4 components (febrile neutropenia, 6.9%; neutropenia, 30.4%). Rates of most nonhematologic AEs were higher in patients receiving FFX with all 4 components, with diarrhea occurring in 28.3%, abdominal pain in 31.5%, and nausea/vomiting in 41.5% of patients. TCOC was lower in the gem/abrax cohort: &lt;math&gt;&lt;mn&gt;6505&lt;/mn&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;F&lt;/mi&gt; &lt;mi&gt;F&lt;/mi&gt; &lt;mi&gt;X&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;n&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mn&gt;5&lt;/mn&gt; &lt;mo&gt;-&lt;/mo&gt; &lt;mi&gt;F&lt;/mi&gt; &lt;mi&gt;U&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt;&lt;/math&gt; 6995) and FFX with all 4 components ( &lt;math&gt;&lt;mn&gt;7142&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;p&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;d&lt;/mi&gt; &lt;mi&gt;m&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;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&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;mo&gt;.&lt;/mo&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;d&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;l&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;p&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;o&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&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;g&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;E&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;t&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;a&lt;/mi&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;e&lt;/mi&gt; &lt;mi&gt;x&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;s&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;o&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt;&lt;/math&gt; 5993 per administration, ","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"161-167"},"PeriodicalIF":2.3,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664866/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882196","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":"Could the Inflation Reduction Act Maximum Fair Price Hurt Patients?","authors":"Anne M Sydor, Esteban Rivera, Robert Popovian","doi":"10.36469/001c.125251","DOIUrl":"10.36469/001c.125251","url":null,"abstract":"<p><p><b>Background:</b> The Inflation Reduction Act's Medicare Drug Price Negotiation Program allows the federal government to negotiate caps for select medications. These price caps may reduce revenue for the pharmacy benefit managers (PBMs) that negotiate the actual price paid for medicines in the U.S. To offset the resulting pressure on their profit margins, it is possible that PBMs would, in turn, increase patients' out-of-pocket costs for medicines with capped prices. The model presented here evaluates how increased out-of-pocket costs for the anticoagulants apixaban (Eliquis) and rivaroxaban (Xarelto) could impact patients financially and clinically. <b>Methods:</b> Copay distributions for all 2023 prescription fills for apixaban and rivaroxaban managed by the 3 largest PBMs, CVS Caremark, Express Scripts International, and Optum Rx, were used to approximate current copay costs. Increased out-of-pocket costs were modeled as a shift of all apixaban and rivaroxaban prescriptions to the highest copay tier. The known linear relationship between copay costs and treatment abandonment was used to calculate the potential resulting increase in treatment abandonment. Known rates of morbidity and mortality due to abandoning anticoagulants were used to estimate resulting increases in morbidity and mortality. <b>Results:</b> If the 3 largest PBMs all shifted costs onto patients by moving all apixaban and rivaroxaban prescriptions to the highest formulary tier, Tier 6, patients' copay amount would increase by <math><mn>235</mn> <mi>t</mi> <mi>o</mi></math> 482 million for apixaban and <math><mn>105</mn> <mi>t</mi> <mi>o</mi></math> 206 million for rivaroxaban. Such an increase could lead to 169 000 to 228 000 patients abandoning apixaban and 71 000 to 93 000 abandoning rivaroxaban. The resulting morbidity and mortality could include up to an additional 145 000 major cardiovascular events and up to 97 000 more deaths. <b>Conclusion:</b> The Medicare Price Negotiation Program could impact patients negatively if it causes PBMs to increase patients' out-of-pocket costs for medicines. Policymakers should closely monitor changes in overall affordability, including all patient out-of-pocket expenditures, for medications in the program. Preemptive measures should be considered to ensure that the most vulnerable citizens are not placed in precarious situations, leading to poorer health outcomes.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"11 2","pages":"154-160"},"PeriodicalIF":2.3,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11612897/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769717","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":"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}