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AIMS: To compare all-cause claims associated with the LATERA Absorbable Nasal Implant and surgical repair of nasal vestibular stenosis in patients with nasal valve collapse. METHODS: This retrospective cohort study utilized data from STATinMED RWD Insights. A defined set of HCPCS, ICD-10-CM and CPT codes were used to identify patients with ≥1 claim for a LATERA procedure, and patients with ≥1 claim for surgical repair between June 1, 2015- March 31, 2023. Patients with continuous capture for at least 12 months before and at least 6 months after the index date were selected. The index date was defined as earliest date of encounter for a LATERA or surgical repair procedure. Inverse probability of treatment weighting (IPTW) was used to ensure balance between cohorts. Descriptive analyses were provided for all claims data using standard summary statistics. All-cause claims were assessed during the baseline, index date, and follow-up period. Chi-squared tests and independent sample t-tests were used to assess differences in cohorts for categorical and continuous variables, respectively. RESULTS: The study population included 5,032 LATERA patients and 26,553 surgical repair patients. During the baseline and follow-up periods, the matched cohorts exhibited similar all-cause claims. On the index date, LATERA patients incurred lower claims vs. surgical repair, likely due to LATERA's ability to be implanted in the physician office setting. LATERA patients and surgical repair patients mean (SD) total costs were $9,612 [$14,930] vs $11,846 [$17,037] (p ≤ 0.0001), respectively. CONCLUSIONS: Treatment with the LATERA Absorbable Nasal Implant is a potentially cost saving option for payers on the index date compared to traditional surgical repair in patients with nasal valve collapse due to the ability to be performed in the office. All-cause claims were similar in the baseline and follow-up periods. When performed with concomitant procedures, all-cause claims during follow-up were similar between groups.
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Implantes Absorvíveis , Revisão da Utilização de Seguros , Humanos , Estudos Retrospectivos , Feminino , Masculino , Pessoa de Meia-Idade , Adulto , Obstrução Nasal/cirurgia , Idoso , Gastos em Saúde/estatística & dados numéricosRESUMO
AIMS: The Nagasaki Acute Myocardial Infarction Secondary Prevention Clinical Pathway (NASP), a guideline-based regional clinical pathway, was developed to manage low-density lipoprotein cholesterol levels for patients with acute myocardial infarction (AMI) in the Nagasaki prefecture in Japan. This study aimed to summarize the perceived best practices and barriers for the dissemination and operation of the NASP. METHODS: This exploratory sequential mixed methods study was developed around the RE-AIM (Reach, Effectiveness, Adoption, Implementation, Maintenance) framework. Focus group interviews were conducted with 24 physicians with experience treating AMI in alignment with the NASP at foundation hospitals. The identified themes and insights were integrated into the development of the questionnaire. The web-based, self-administered questionnaire with a cross-sectional study design was given to 62 physicians in the Nagasaki prefecture. Mixed-method data integration of the results from both study phases was conducted through meta-inferences made from the qualitative and quantitative data. RESULTS: The best practices included the development of multi-disciplinary operation teams at medical facilities in preparation for the implementation of the NASP, the simplification of the document preparation process, and the establishment of an additional medical fees policy for the utilization of the NASP instead of patient referral documents. Practices tailored to the type of medical institute such as instructing patients on the NASP regimen during index hospitalization for acute-care hospitals, and the development of NASP instructions and manuals for primary care hospitals/outpatient clinics were also recommended. In addition, barriers to the implementation of the NASP such as missed eligible AMI patients for the NASP and the inconsistent implementation to eligible AMI patients were identified. CONCLUSIONS: This study identified the perceived best practices and barriers for the NASP. This knowledge should be considered when expanding the NASP to other institutions across Japan.
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AIMS: This study assessed the budget impact of resmetirom as a treatment for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver fibrosis and estimated total costs for a hypothetical private payer in the United States. MATERIALS AND METHODS: A three-year budget impact analysis based on an open cohort state transition model was developed for a hypothetical one-million-member private health plan. The comparator was Standard of Care (SOC), defined as routine care for non-cirrhotic NASH patients with moderate-to-advanced liver fibrosis. Each year, the number of resmetirom treatment-eligible patients was estimated through prevalent, incident, and diagnostic rate estimates. Costs included resources incurred by the medical and pharmacy benefits of private payers, including resmetirom drug acquisition costs, diagnosis and monitoring, other medical and other prescription costs stratified by disease progression status (i.e., non-cirrhotic vs. cirrhotic/advanced liver diseases). Resmetirom adverse event management costs were included in sensitivity analysis. Drug costs were estimated based on the average wholesale acquisition cost as of March 2024. Other costs were based on published sources and inflated to 2023 US dollars. Budget impact outcomes were presented in aggregate, net, and on a per-member per-month (PMPM) basis. RESULTS: Compared with a scenario without resmetirom, the introduction of resmetirom yielded results ranging from 50 to 238 treated patients, net budget impact of $2.2 to $9.5 million, and PMPM from $0.19 to $0.80 over years one and three. Net costs excluding resmetirom declined over time. In sensitivity analyses, results were most sensitive to diagnostic and epidemiologic inputs. LIMITATIONS: Market shares are based on internal forecasts, a short time horizon, average treatment effects, and other limitations common to BIMs. CONCLUSION: The adoption of resmetirom on the formulary for the treatment of non-cirrhotic NASH with moderate-to-advanced liver fibrosis resulted in a moderate increase in budget impact with declining costs related to NASH progression.
Non-alcoholic steatohepatitis (NASH) is a serious liver disease that can lead to significant liver damage, other health complications, and increased healthcare costs. As the disease progresses, patients typically experience worsening health outcomes. Until recently, there were no Food and Drug Administration (FDA) approved treatments for NASH in the United States. However, in March 2024, the FDA approved REZDIFFRA™, a new drug specifically designed to treat NASH patients with moderate-to-advanced liver fibrosis (i.e., NASH with moderate-to-advanced scarring of the liver). Clinical trials have shown that REZDIFFRA™ can improve health outcomes in these patients.To identify patients who could benefit from REZDIFFRA™ and to estimate the associated costs, we developed a budget impact model. In this study, we detail the development of this model and present its findings. Our analysis revealed that, while REZDIFFRA™ is associated with higher overall costs, primarily due to the price of the drug itself, there are potential cost savings when considering the drug's ability to slow disease progression.
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AIMS AND BACKGROUND: Whole-genome sequencing (WGS) is increasingly applied in clinical practice and expected to replace standard-of-care (SoC) genetic diagnostics in hematological malignancies. This study aims to assess and compare the fully burdened cost ('micro-costing') per patient for Swedish laboratories using WGS and SoC, respectively, in pediatric and adult patients with acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). METHODS: The resource use and cost details associated with SoC, e.g. chromosome banding analysis, fluorescent in situ hybridization, and targeted sequencing analysis, were collected via activity-based costing methods from four diagnostic laboratories. For WGS, corresponding data was collected from two of the centers. A simulation-based scenario model was developed for analyzing the WGS cost based on different annual sample throughput to evaluate economy of scale. RESULTS: The average SoC total cost per patient was 2,465 for pediatric AML and 2,201 for pediatric ALL, while in adults, the corresponding cost was 2,458 for AML and 1,207 for ALL. The average WGS cost (90x tumor/30x normal; sequenced on the Illumina NovaSeq 6000 platform) was estimated to 3,472 based on an annual throughput of 2,500 analyses, however, with an annual volume of 7,500 analyses the average cost would decrease by 23% to 2,671. CONCLUSION: In summary, WGS is currently more costly than SoC, however the cost can be reduced by utilizing laboratories with higher throughput and by the expected decline in cost of reagents. Our data provides guidance to decision-makers for the resource allocation needed when implementing WGS in diagnostics of hematological malignancies.
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Testes Genéticos , Leucemia Mieloide Aguda , Leucemia-Linfoma Linfoblástico de Células Precursoras , Sequenciamento Completo do Genoma , Humanos , Suécia , Leucemia Mieloide Aguda/genética , Leucemia Mieloide Aguda/diagnóstico , Sequenciamento Completo do Genoma/economia , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Leucemia-Linfoma Linfoblástico de Células Precursoras/diagnóstico , Testes Genéticos/economia , Testes Genéticos/métodos , Adulto , Criança , Masculino , Feminino , Custos e Análise de CustoRESUMO
AIMS: Food allergies impose a large clinical and financial burden on patients and the health care system. However, little is known about the factors associated with health care resource use and costs. The aim of this study was to investigate health care resource use and costs in individuals with food allergies utilizing health care in the United States. METHODS: We conducted a retrospective analysis of insurance claims data from the Merative MarketScan Research Databases (indexed from 1 January 2015 to 30 June 2022). All-cause and food allergy-related health care resource use, direct medical, and out-of-pocket costs for medical services were estimated for 12 months post-index using International Classification of Diseases [ICD] codes. RESULTS: Of 355,520 individuals with food allergies continuously enrolled in a health insurance plan for ≥12 months pre- and post-index, 17% had a food allergy-related emergency department visit and 0.9% were hospitalized. The top patient characteristic associated with all-cause and food allergy-related hospitalizations, all-cause costs, and food allergy-related outpatient visit costs was a Charlson Comorbidity Index score of ≥2. Food allergy-related direct medical and out-of-pocket costs were high among patients with a food allergy-related visit. Out-of-pocket cost per patient per year for outpatient visits, emergency department visits, and hospitalizations had an estimated mean of $1631 for patients with food allergy-related visits, which is â¼11% of the total costs for these services ($14,395 per patient per year). LIMITATIONS: Study limitations are primarily related to the nature of claims databases, including generalizability and reliance on ICD codes. Nevertheless, MarketScan databases provide robust patient-level insights into health care resource use and costs from a large, commercially insured patient population. CONCLUSION: The health care resource use of patients with food allergies imposes a burden on both the health care system and on patients and their families, especially if patients had comorbidities.
Some people with food allergies might need extra visits to the doctor or hospital to manage allergic reactions to food, and these visits add to the cost of medical services for both families and for health care providers. Using records of health insurance claims, we looked into the factors affecting medical visits and costs in people with food allergies in the United States. For people with food allergies, having additional medical conditions (measured using the Charleson Comorbidity Index) were linked with extra medical visits and costs. Out-of-pocket costs were high for people who visited a doctor or hospital for their food allergies (costing each person more than $1,600 per year). The total medical cost of food allergy-related care was $14,395 per person per year, paid for by families and health care providers. Our findings might help to better manage and treat people with food allergies and reduce medical costs.
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Hipersensibilidade Alimentar , Gastos em Saúde , Revisão da Utilização de Seguros , Humanos , Hipersensibilidade Alimentar/economia , Masculino , Feminino , Estados Unidos , Estudos Retrospectivos , Adulto , Pessoa de Meia-Idade , Gastos em Saúde/estatística & dados numéricos , Adolescente , Adulto Jovem , Criança , Pré-Escolar , Lactente , Comorbidade , Idoso , Hospitalização/economia , Hospitalização/estatística & dados numéricos , Recursos em Saúde/economia , Recursos em Saúde/estatística & dados numéricos , Recém-NascidoRESUMO
AIM: To investigate hepatitis A-related healthcare resource use and costs in the US. METHODS: The Merative Marketscan Commercial Claims and Encounters database was retrospectively analyzed for hepatitis A-related inpatient, outpatient, and emergency department (ED) claims from January 1, 2012 to December 31, 2018. We calculated the hepatitis A incidence proportion per 100,000 enrollees, healthcare resource utilization, and costs (in 2020 USD). Results were stratified by age, gender, and select comorbidities. RESULTS: The overall hepatitis A incidence proportion was 6.1 per 100,000 enrollees. Among individuals with ≥1 hepatitis A-related claim, the majority (92.6%) had ≥1 outpatient visit related to hepatitis A; 9.1% were hospitalized and 4.2% had ≥1 ED visit. The mean (standard deviation [SD]) length of hospital stay was 5.2 (8.1) days; the mean (SD) number of outpatient and ED visits were 1.3 (1.3) and 1.1 (0.6), respectively. The incidence proportion per 100,000 was higher among adults than children (7.5 vs. 1.5), individuals with HIV than those without (126.7 vs. 5.9), and individuals with chronic liver disease than those without (143.6 vs. 3.8). The total mean (SD)/median (interquartile range, IQR) per-patient cost for hepatitis A-related care was $2,520 ($10,899)/$156 ($74-$529) and the mean cost of hospitalization was 18.7 times higher than that of outpatient care ($17,373 vs. $928). LIMITATIONS: The study data included only a commercially insured population and may not be representative of all individuals. CONCLUSIONS: In conclusion, hepatitis A is associated with a substantial economic burden among privately insured individuals in the US.
Hepatitis A is an acute liver infection caused by the hepatitis A virus. In the US, safe and effective vaccines for hepatitis A have been available since 1996. Vaccination recommendations include children (all children aged 1223 months and previously unvaccinated children aged 218 years old) and adults at risk of infection or severe disease (e.g. international travelers, men who have sex with men, persons experiencing homelessness, persons with chronic liver disease or persons with HIV infection). Since 2016, the US has experienced person-to-person outbreaks of hepatitis A, primarily affecting unvaccinated individuals who use drugs or are experiencing homelessness. To better understand the impact of hepatitis A in the US, we assessed healthcare resource use and costs in 15,435 patients with hepatitis A from 2012 to 2018 in the Merative Marketscan Commercial Claims and Encounters database. We found that slightly more than 6 per 100,000 enrollees had hepatitis A from 2012 to 2018 and the number of people treated for hepatitis A per 100,000 was highest for people living with HIV or with chronic liver disease. The majority (92.6%) of people reported at least an outpatient visit, 9.1% were hospitalized, and 4.2% had an emergency department visit. The average cost for hepatitis A-related care was $2,520 per patient and was 18.7 times higher for hospitalized patients ($17,373) than for patients treated in outpatient care ($928). Our results are limited by the generalizability of the dataset, which is a convenience sample of private insurance claims, and are therefore unlikely to capture groups at high-risk for hepatitis A, such as individuals experiencing homelessness. In conclusion, hepatitis A leads to considerable healthcare costs for privately insured individuals in the US.
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Hepatite A , Revisão da Utilização de Seguros , Aceitação pelo Paciente de Cuidados de Saúde , Humanos , Masculino , Estudos Retrospectivos , Feminino , Adulto , Estados Unidos , Pessoa de Meia-Idade , Hepatite A/economia , Hepatite A/epidemiologia , Adolescente , Adulto Jovem , Criança , Pré-Escolar , Lactente , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Fatores Etários , Tempo de Internação/economia , Tempo de Internação/estatística & dados numéricos , Incidência , Comorbidade , Fatores Sexuais , Gastos em Saúde/estatística & dados numéricos , Serviço Hospitalar de Emergência/estatística & dados numéricos , Serviço Hospitalar de Emergência/economia , Idoso , Hospitalização/economia , Hospitalização/estatística & dados numéricos , Recursos em Saúde/economia , Recursos em Saúde/estatística & dados numéricosRESUMO
AIMS: To describe healthcare resource utilization (HCRU) and associated costs after initiation of injectable glucagon-like peptide-1 receptor agonist (GLP-1 RA) therapy by adult patients with type 2 diabetes (T2D) in the prospective, observational, 24-month TROPHIES study in France, Germany, and Italy. MATERIALS AND METHODS: HCRU data for cost calculations were collected by treating physicians during patient interviews at baseline and follow-up visits approximately 6, 12, 18, and 24 months after GLP-1 RA initiation with once-weekly dulaglutide or once-daily liraglutide. Costs were evaluated from the national healthcare system (third-party payer) perspective and updated to 2018 prices. RESULTS: In total, 2,005 patients were eligible for the HCRU analysis (1,014 dulaglutide; 991 liraglutide). Baseline patient characteristics were generally similar between treatment groups and countries. The largest proportions of patients using ≥2 oral glucose-lowering medications (GLMs) at baseline (42.9-43.4%) and month 24 (44.0-45.1%) and using another injectable GLM at month 24 (15.3-23.2%) were in France. Mean numbers of primary and secondary healthcare contacts during each assessment period were highest in France (range = 4.0-10.7) and Germany (range = 2.9-5.7), respectively. The greatest proportions (≥60%) of mean annualized costs per patient comprised medication costs. Mean annualized HCRU costs per patient varied by treatment cohort and country: the highest levels were in the liraglutide cohort in France (909) and the dulaglutide cohort in Germany (883). LIMITATIONS: Limitations included exclusion of patients using insulin at GLP-1 RA initiation and collection of HCRU data by physician, not via patient-completed diaries. CONCLUSIONS: Real-world HCRU and costs associated with the treatment of adults with T2D with two GLP-1 RAs in TROPHIES emphasize the need to avoid generalization with respect to HCRU and costs associated with a particular therapy when estimating the impact of a new treatment in a country-specific setting.
Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have become frequent treatments of hyperglycemia in type-2 diabetes (T2D). Not all types of clinical study provide information about the cost of these treatments or the effects they might have on use of other medicines and equipment to control T2D or the need for visits to a doctor or nurse and different types of treatment in hospital. This study collected this information during the regular care of adults in France, Germany, or Italy who were prescribed either dulaglutide or liraglutide (both types of GLP-1 RAs) by their family doctor or a specialist in T2D. There were differences in costs and the need for other medicines and medical services between people using either dulaglutide or liraglutide and for people who were using the same GLP-1 RA in each of the three countries. The information from this study could be used to more accurately understand the overall costs and medical care needed when patients use dulaglutide or liraglutide in France, Germany, or Italy.
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Diabetes Mellitus Tipo 2 , Peptídeos Semelhantes ao Glucagon , Hipoglicemiantes , Fragmentos Fc das Imunoglobulinas , Liraglutida , Proteínas Recombinantes de Fusão , Humanos , Diabetes Mellitus Tipo 2/tratamento farmacológico , Diabetes Mellitus Tipo 2/economia , Liraglutida/uso terapêutico , Liraglutida/economia , Peptídeos Semelhantes ao Glucagon/análogos & derivados , Peptídeos Semelhantes ao Glucagon/uso terapêutico , Peptídeos Semelhantes ao Glucagon/economia , Peptídeos Semelhantes ao Glucagon/administração & dosagem , Fragmentos Fc das Imunoglobulinas/uso terapêutico , Fragmentos Fc das Imunoglobulinas/economia , Proteínas Recombinantes de Fusão/economia , Proteínas Recombinantes de Fusão/uso terapêutico , Proteínas Recombinantes de Fusão/administração & dosagem , Masculino , Hipoglicemiantes/uso terapêutico , Hipoglicemiantes/economia , Feminino , Estudos Prospectivos , Pessoa de Meia-Idade , Idoso , Recursos em Saúde/estatística & dados numéricos , Recursos em Saúde/economia , Modelos EconométricosRESUMO
OBJECTIVES: This study investigates the utilization of work absence benefits among United States (US) employees diagnosed with COVID-19, examining frequency, duration, cost, and types of work loss benefits used. METHODS: This retrospective analysis of the Workpartners Research Reference Database (RRDb) included employees eligible for short- and long-term disability (STD and LTD employer-sponsored benefits, respectively), and other paid work absence benefits from 2018 to 2022. Workpartners RRDb includes over 3.5 million employees from over 500 self-insured employers across the US. Employees were identified by codes from adjudicated medical and disability claims for COVID-19 (2020-2022) and influenza, as well as prescription claims for COVID-19 treatments. Associated payments were quantified for each absence reason. RESULTS: Approximately 1 million employees were eligible for employer-sponsored paid leave benefits between January 2018 and December 2022. The mean age was 37 years (22% >50 years), and 49.4% were females. COVID-19 was the 2nd most common reason for an STD claim (6.9% of all STD claims) and 13th for an LTD claim (1.7% of all LTD claims) from 2020-2022. The mean duration for COVID-19 STD claims was 24 days (N = 3,731, mean claim=$3,477) versus 10 days for influenza (N = 283, mean claim=$1,721). The mean duration for an LTD claim for COVID-19 was 153 days (N = 11, mean claim=$19,254). Only 21.5% of employees with STD claims in the COVID-19 cohort had prior COVID-19-associated medical or pharmacy claims; over half (range 53%-61%) had documented high risk factors for severe COVID-19. CONCLUSION: COVID-19 and influenza have the potential to cause work loss in otherwise healthy employees. In this analysis, COVID-19 was the second most frequent reason for an STD claim at the start of the pandemic and remained high (ranked 5th) in 2022. These results highlight the impact of COVID-19 on work loss beyond the acute phase. Comprehensively evaluating work loss implications may help employers prioritize strategies, such as vaccinations and timely treatments, to mitigate the impact of COVID-19 on employees and their companies.
COVID-19 results in short- and long-term symptoms that may affect employees' ability to work. Short- and long-term disability (STD and LTD, respectively), other work absences, and medical and pharmacy claims from the Workpartners Research Reference Database were analyzed for US adult (≥18 years) employees. COVID-19 claims were identified using the Center for Disease Control and Prevention recommended International Classification of Diseases codes during the analysis from 2020 to 2022. During 2020 to 2022, COVID-19 ranked as the second most frequent reason for STD claims and 13th most frequent among LTD claims. Influenza ranked 58th overall with no LTD claims (20182022). The average COVID-19 STD claim lasted 24 days and cost employers $3,477 per claim, and LTD claims averaged 153 days, costing $19,254. Only 21.5% of employees with STD claims in the COVID-19 cohort had prior COVID-19-associated medical or pharmacy claims, and over half (range 53%61%) had a documented high-risk factor for severe COVID-19. Our results highlight the ongoing and substantial impact of COVID-19 on work absence benefit utilization beyond the acute phase. This analysis demonstrates the need for employers and researchers to review all available medical, pharmacy, and disability claims to assess the acute and long-term impact of COVID-19 on employees and prioritize mitigation strategies to reduce the burden of the virus to their employees.
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COVID-19 , Licença Médica , Humanos , COVID-19/epidemiologia , COVID-19/economia , Estados Unidos , Estudos Retrospectivos , Masculino , Feminino , Adulto , Pessoa de Meia-Idade , Licença Médica/estatística & dados numéricos , Licença Médica/economia , SARS-CoV-2 , Revisão da Utilização de Seguros , Bases de Dados Factuais , Adulto Jovem , AbsenteísmoRESUMO
AIMS: Suboptimal treatment indicators, including treatment switch, are common among patients with Crohn's disease (CD), but little is known about their associated healthcare resource utilization (HRU) and costs. This study assessed the impact of suboptimal treatment indicators on HRU and costs among adults with CD newly treated with a first-line biologic. METHODS: Adult patients with CD were identified in the IBM MarketScan Commercial Subset (10/01/2015-03/31/2020). The index date was defined as initiation of the first-line biologic, and the study period was defined as the 12 months following the index date. Patients were classified into Suboptimal Treatment and Optimal Treatment cohorts based on observed indicators of suboptimal treatment during the study period. Patients in the Suboptimal Treatment Cohort with a treatment switch were classified into the Treatment Switch Cohort and compared to patients with no treatment switch. All-cause HRU and costs were measured during the study period and assessed for patients with suboptimal vs optimal treatment and patients with vs without a treatment switch. RESULTS: The study included 4,006 patients (Suboptimal Treatment: 2,091, Optimal Treatment: 1,915). Treatment switch was a common indicator of suboptimal treatment (Treatment Switch: 640, No Treatment Switch: 3,366). HRU and costs were significantly higher among patients with suboptimal treatment than those with optimal treatment (annual costs: $92,043 vs $73,764; p < 0.01), and among those with a treatment switch than those with no treatment switch (annual costs: $95,689 vs $81,027; p < 0.01). Increases in the number of suboptimal treatment indicators were associated with increased costs. LIMITATIONS: Claims data were used to identify suboptimal treatment indicators based on observed treatment patterns; reasons for treatment decisions could not be assessed. CONCLUSION: This study demonstrates that patients with suboptimal treatment indicators, including treatment switch, incur substantially higher HRU and costs compared to patients receiving optimal treatment and those that do not switch treatments.
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Doença de Crohn , Revisão da Utilização de Seguros , Humanos , Masculino , Doença de Crohn/tratamento farmacológico , Doença de Crohn/economia , Feminino , Adulto , Estudos Retrospectivos , Pessoa de Meia-Idade , Estados Unidos , Produtos Biológicos/uso terapêutico , Produtos Biológicos/economia , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Adulto Jovem , Gastos em Saúde/estatística & dados numéricos , Recursos em Saúde/estatística & dados numéricos , Recursos em Saúde/economia , AdolescenteRESUMO
AIMS: This study aimed to assess and compare the health care resource utilization (HCRU) and medical cost of metabolic dysfunction-associated steatohepatitis (MASH) by disease severity based on Fibrosis-4 Index (FIB-4) score among US adults in a real-world setting. MATERIALS AND METHODS: This observational cohort study used claims data from the Healthcare Integrated Research Database (HIRD) to compare all-cause, cardiovascular (CV)-related, and liver-related HCRU, including hospitalization, and medical costs stratified by FIB-4 score among patients with MASH (identified by International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] code K75.81). Hospitalization and medical costs were compared by FIB-4 score using generalized linear regression with negative binomial and gamma distribution models, respectively, while controlling for confounders. RESULTS: The cohort included a total of 5,104 patients with MASH and comprised 3,162, 1,343, and 599 patients with low, indeterminate, and high FIB-4 scores, respectively. All-cause hospitalization was significantly higher in the high FIB-4 cohort when compared with the low FIB-4 reference after covariate adjustment (rate ratio, 1.63; 95% CI, 1.32-2.02; p < .0001). CV-related hospitalization was similar across all cohorts; however, CV-related costs were 1.26 times higher (95% CI, 1.11-1.45; p < .001) in the indeterminate cohort and 2.15 times higher (95% CI, 1.77-2.62; p < .0001) in the high FIB-4 cohort when compared with the low FIB-4 cohort. Patients with indeterminate and high FIB-4 scores had 2.97 (95% CI, 1.78-4.95) and 12.08 (95% CI, 7.35-19.88) times the rate of liver-related hospitalization and were 3.68 (95% CI, 3.11-4.34) and 33.73 (95% CI, 27.39-41.55) times more likely to incur liver-related costs, respectively (p < .0001 for all). LIMITATIONS: This claims-based analysis relied on diagnostic coding accuracy, which may not capture the presence of all diseases or all care received. CONCLUSIONS: High and indeterminate FIB-4 scores were associated with significantly higher liver-related clinical and economic burdens than low FIB-4 scores among patients with MASH.
MASH is a serious liver disease that can lead to fibrosis, cirrhosis, and other complications. There is a need to understand the impact of disease severity on the burden of MASH. Health care claims data were used to assess the use of medical resources, including hospitalization, and medical costs among patients with 3 different levels of severity of MASH, as assessed via FIB-4 score. FIB-4 is a widely available non-invasive marker of severity. Rates of all-cause, cardiovascular-related and liver-related hospitalization and medical costs were several-fold higher in patients with high disease severity of MASH than those with low disease severity of MASH.
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Hospitalização , Revisão da Utilização de Seguros , Índice de Gravidade de Doença , Humanos , Masculino , Feminino , Pessoa de Meia-Idade , Hospitalização/economia , Hospitalização/estatística & dados numéricos , Adulto , Idoso , Gastos em Saúde/estatística & dados numéricos , Estados Unidos , Fígado Gorduroso/economia , Recursos em Saúde/estatística & dados numéricos , Recursos em Saúde/economia , Estudos Retrospectivos , Doenças Cardiovasculares/economia , Comorbidade , Doenças MetabólicasRESUMO
BACKGROUND: The economic and mortality burden of cancer is high worldwide. In Europe, cancer was responsible for 1.3 million deaths in 2020 and incurred an estimated cost of 50 billion from premature mortality. Human papillomavirus (HPV) and hepatitis B virus (HBV) are among the leading causes of infection-related cancers despite the availability of effective vaccines against these infections. This analysis estimated the mortality and productivity loss of HBV- and HPV-associated cancers that could be preventable through vaccination across European regions. MATERIALS AND METHODS: Institute for Health Metrics Evaluation (IHME) data were used to estimate mortality, years of life lost (YLL), and the value of years of life lost (VYLL) from five HBV- and HPV-related cancers (oral cavity, oropharynx, larynx, cervical, and liver cancers) across 40 European countries in 2019. Preventable deaths and YLL were estimated based on fractions attributable to infections. Data from the World Bank on GDP per capita were used to estimate the VYLL. The robustness of these results was explored with sensitivity and scenario analyses. RESULTS: In 2019, 31,906 cancer deaths resulted in an economic burden of 18,521,614,725 due to productivity losses across Europe. HPV-related cervical cancer had the highest mortality (19,473 deaths) and economic burden (10,706,253,185). HBV-related liver cancer and HPV-related larynx, oral cavity, and oropharynx cancers also had a substantial burden, particularly in males. Eastern Europe had the highest YLL (308,179; 39%) and Western Europe was responsible for the greatest VYLL (8,281,306,504; 45%), although the highest VYLL per death was in Northern Europe (923,638). HPV-related oropharynx cancer had the highest VYLL per death (656,607). CONCLUSION: HPV- and HBV-related cancer deaths are associated with substantial mortality and productivity losses in Europe, which could be reduced by the continued prioritization and implementation of prophylactic public health measures including systematic awareness, vaccination, and screening efforts.
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Efeitos Psicossociais da Doença , Infecções por Papillomavirus , Humanos , Europa (Continente)/epidemiologia , Feminino , Masculino , Infecções por Papillomavirus/prevenção & controle , Infecções por Papillomavirus/complicações , Pessoa de Meia-Idade , Hepatite B/prevenção & controle , Hepatite B/economia , Neoplasias/mortalidade , Neoplasias/economia , Adulto , Idoso , Vacinas contra Hepatite B/economia , Vacinas contra Hepatite B/administração & dosagem , Modelos Econométricos , Adulto Jovem , Vacinas contra Papillomavirus/economia , Vacinas contra Papillomavirus/administração & dosagemRESUMO
OBJECTIVES: Biosimilars improve patient access by providing cost-effective treatment options. This study assessed the potential for savings and expanded patient access with increased use of two biosimilar disease modifying anti-rheumatic drugs (DMARDs): (a) approved adalimumab biosimilars and (b) the first tocilizumab biosimilar, representing an established biosimilar field and a recent biosimilar entrant in France, Germany, Italy, Spain, and the United Kingdom (UK). METHODS: Separate ex-ante analyses were conducted for each country, parameterized using country-specific list prices, unit volumes annually, and market shares for each therapy. Discounting scenarios of 10%, 20%, and 30% were tested for tocilizumab. Outputs included direct cost-savings associated with drug acquisition or the incremental number of patients that could be treated if savings were redirected. Two biosimilar conversion scenarios were tested. RESULTS: Savings associated with a 100% conversion to adalimumab biosimilar ranged from 10.5 to 187 million (UK and Germany, respectively), or an additional 1,096 to 19,454 patients that could be treated using the cost-savings. Introduction of a tocilizumab biosimilar provided savings up to 29.3 million in the most conservative scenario. Exclusive use of tocilizumab biosimilars (at a 30% discount) could increase savings to 28.8 to 113 million or expand access to an additional 43% of existing tocilizumab users across countries. CONCLUSION: This study demonstrates the benefits that can be realized through increased biosimilar adoption, not only in an untapped tocilizumab market, but also through incremental increases in well-established markets such as adalimumab. As healthcare budgets continue to face downwards pressure globally, strategies to increase biosimilar market share could prove useful to help manage financial constraints.
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Adalimumab , Anticorpos Monoclonais Humanizados , Antirreumáticos , Medicamentos Biossimilares , Redução de Custos , Adalimumab/economia , Adalimumab/uso terapêutico , Medicamentos Biossimilares/economia , Medicamentos Biossimilares/uso terapêutico , Humanos , Anticorpos Monoclonais Humanizados/uso terapêutico , Anticorpos Monoclonais Humanizados/economia , Europa (Continente) , Antirreumáticos/economia , Antirreumáticos/uso terapêutico , Análise Custo-Benefício , Acessibilidade aos Serviços de Saúde/economia , Modelos EconométricosRESUMO
BACKGROUND: Infections are responsible for â¼13% of cancer cases worldwide, with human papillomavirus (HPV) and hepatitis B (HBV) among the infections associated with cancer for which vaccines are available. The aim of this study was to estimate the indirect cost of premature mortality related to cancers caused by HPV and HBV in Middle East and North Africa (MENA) countries. METHODS: The number of deaths and years of life lost (YLL) in 2019 from four HPV-related cancers: cervical cancer, oral cavity cancer, laryngeal cancer, and oropharynx cancer, as well as HBV-related liver cancer were sourced from the Institute for Health Metrics Evaluation (IHME) Global Burden of Disease database. HPV-attributable fractions were applied to deaths and YLL. The human capital approach was used to measure productivity loss, through value of YLL (VYLL), and estimated using gross domestic product per capita (World Bank; in USD). Seventeen countries in the MENA region were included. Four countries in the region were not included due to data availability. RESULTS: In 2019, there were 11,645 potentially vaccine-preventable cancer-related deaths across the MENA region. This resulted in an indirect cost of $1,688,821,605, with 76.1% of this accrued in the Middle East ($1,284,923,633). The number of deaths in the Middle East (5,986) were similar to Northern Africa (5,659) but YLL were higher in Northern Africa (179,425) compared to the Middle East (169,207). The highest indirect cost per death occurred in Qatar ($1,378,991), compared to $14,962 in Sudan. Oral cavity cancer had the highest VYLL per death ($186,084). CONCLUSIONS: There is a high burden of premature mortality and indirect costs of potentially vaccine-preventable cancer-related deaths in the MENA region. Improved vaccination program implementation, increased vaccine coverage of HPV and HBV vaccinations, and continued prioritization of public health measures, such as screening, could effectively reduce premature mortality and associated costs.
Assuntos
Neoplasias , Humanos , Oriente Médio/epidemiologia , África do Norte/epidemiologia , Feminino , Masculino , Neoplasias/mortalidade , Neoplasias/economia , Pessoa de Meia-Idade , Infecções por Papillomavirus/prevenção & controle , Adulto , Efeitos Psicossociais da Doença , Hepatite B/prevenção & controle , Hepatite B/economia , Vacinas contra Papillomavirus/economia , Vacinas contra Papillomavirus/administração & dosagem , Idoso , Mortalidade Prematura , Modelos EconométricosRESUMO
BACKGROUND: Hyperkalemia is an electrolyte abnormality with potentially life-threatening consequences. Published data have shown that potassium-binding polymer patiromer (Veltassa) is associated with reduced rates of severe edema and hospitalization for heart failure compared with sodium zirconium cyclosilicate (SZC, Lokelma) when treating hyperkalemia. The aim of this study was to evaluate the possible costs associated with these interventions in the Spanish and UK settings. METHODS: A cost-analysis model was developed in Microsoft Excel to compare the costs associated with patiromer and SZC for the management of hyperkalemia. Clinical event rates were taken from a published real-world comparative study, with the base case capturing the statistically significant reduction in severe edema with patiromer vs SZC and a sensitivity analysis also including the non-statistically significant reduction in hospitalization for heart failure. Country-specific costs, expressed in 2022 Euros (EUR) and British pounds sterling (GBP), were evaluated from a healthcare payer perspective and included pharmacy costs and costs of clinical events. RESULTS: Patiromer may be associated with cost savings of EUR 107 and GBP 630 per patient-year of treatment vs SZC in Spain and the UK, respectively. The majority of cost savings were due to the possible lower daily cost of patiromer compared with SZC. Including the difference in heart failure hospitalization rates in a sensitivity analysis led to greater cost savings with patiromer over SZC, increasing to EUR 460 and GBP 902 in Spain and the UK, respectively. Extrapolation of patient-level economic outcomes to a population level found that patiromer was associated with annual cost savings of EUR 30.6 million in Spain, and GBP 801.7 million in the UK vs SZC. CONCLUSIONS: Patiromer has the potential to be cost saving vs SZC for the treatment of hyperkalemia in Spain and the UK based on the results of a real-world evidence analysis.
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Hiperpotassemia , Polímeros , Silicatos , Hiperpotassemia/tratamento farmacológico , Hiperpotassemia/economia , Humanos , Espanha , Reino Unido , Polímeros/uso terapêutico , Silicatos/uso terapêutico , Silicatos/economia , Insuficiência Cardíaca/tratamento farmacológico , Insuficiência Cardíaca/economia , Análise Custo-Benefício , Hospitalização/economia , Hospitalização/estatística & dados numéricos , Custos e Análise de Custo , Modelos EconométricosRESUMO
AIMS: Patients with inborn errors of immunity (IEI) are predisposed to severe recurrent/chronic infections, and often require hospitalization, resulting in substantial burden to patients/healthcare systems. While immunoglobulin replacement therapies (IgRTs) are the standard first-line treatment for most forms of IEI, limited real-world data exist regarding clinical characteristics and treatment costs for patients with IEI initiating such treatment. This retrospective analysis examined infection and treatment characteristics in US patients with IEI initiating IgRT with immune globulin infusion (human), 10% (IG10%). Healthcare resource utilization (HCRU) and associated costs before and after treatment initiation were compared. Additionally, the impact of COVID-19 on infection diagnoses was evaluated. METHODS: Patients with IEI initiating IG10% between July 2012 and August 2019 were selected from Merative MarketScan Databases using diagnosis/prescription codes. Patients were followed 6 months before and after first IG10% claim date. Demographic and clinical characteristics were described. Treatment characteristics and HCRU before and after IG10% initiation were compared. Infection diagnoses during 2020 and 2019 (March-December) were compared. RESULTS: The study included 1,497 patients with IEI diagnoses (mean age = 43.4 years) initiating IG10%, with frequently reported comorbidities like asthma (32.1%). Following IG10% initiation, fewer severe infection diagnoses (11.6% vs 19.9%), fewer infection-related inpatient (10.8% vs 19.5%) and outpatient services (71.6% vs 79.9%), and lower infection-related total healthcare costs ($7,849 vs $13,995; p < 0.001)-driven by lower inpatient costs ($2,746 vs $9,900)-were observed than before. Fewer patients had infection diagnoses during COVID-19 (22.8%) than the prior year (31.2%). CONCLUSION: Patients with IEI are susceptible to severe infections leading to high disease burden and treatment costs. Following IG10% initiation, we observed fewer infections, lower infection-related treatment costs, and shift in care (inpatient to outpatient) leading to significant cost savings. Among patients with IEI, 27% fewer infection diagnoses were observed during the early COVID-19 lockdown period than the prior year.
Some people are born with inborn errors of immunity, or IEI. This study included 1,497 people with IEI who recently started taking a drug called immunoglobulin therapy. Before taking this drug, the participants got infections easily, were hospitalized often, and had to take other costly medicines. After starting this drug, they had fewer infections and could be treated at the doctor's office. They had fewer infections during the COVID-19 pandemic than before the pandemic.
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COVID-19 , Humanos , Masculino , Feminino , Estudos Retrospectivos , Adulto , Pessoa de Meia-Idade , Assistência Ambulatorial/economia , Estados Unidos , Aceitação pelo Paciente de Cuidados de Saúde/estatística & dados numéricos , Adulto Jovem , SARS-CoV-2 , Gastos em Saúde/estatística & dados numéricos , Hospitalização/economia , Hospitalização/estatística & dados numéricos , Recursos em Saúde/economia , Recursos em Saúde/estatística & dados numéricos , Adolescente , Índice de Gravidade de Doença , Comorbidade , Revisão da Utilização de Seguros , Imunoglobulinas Intravenosas/uso terapêutico , Imunoglobulinas Intravenosas/economiaRESUMO
BACKGROUND: Bioprostheses with RESILIA tissue demonstrate a reduction in calcification and improve health outcomes in pre-clinical and clinical studies. Prior economic analyses which relied on 5 years of evidence from the COMMENCE trial demonstrate financial savings for RESILIA tissue valves relative to mechanical valves after surgical aortic valve replacement (SAVR). Given the recent release of 7-year COMMENCE data, this economic evaluation updates the estimate for long-run savings of bioprosthetic valves with RESILIA. METHODS: Simulation models estimated disease progression across two hypothetical SAVR cohorts (tissue vs. mechanical) of 10,000 patients each in the US. The primary comparison calculated the SAVR-related expenditures associated with each valve type ($US, 2023). Health outcome probabilities were based on the COMMENCE trial though year 7 and projected for an additional 8 years based on prior studies of tissue and mechanical SAVR. Costs for key outcomes (mortality, reoperation, bleeding, thromboembolism, endocarditis) and anticoagulant monitoring were sourced from the literature. Incidence rates of health outcomes associated with mechanical valves relied on relative risks of tissue valve versus mechanical valve patients. RESULTS: Seven-year savings are $13,415 (95% CI = $10,472-$17,321) per patient when comparing RESILIA versus mechanical SAVR. Projected 15-year savings were $23,001 ($US, 2023; 95% CI = $17,802-$30,421). Most of the 15-year savings are primarily attributed to lower anti-coagulation monitoring costs ($21,073 in ACM savings over 15 years), but lower bleeding cost (savings: $2,294) and thromboembolism-related expenditures (savings: $852) also contribute. Reoperation and endocarditis expenditures were slightly larger in the RESILIA cohort. If reoperation relative risk reverts from 1.1 to 2.2 (the level in legacy tissue valves) after year 7, savings are $18,064. RESILIA SAVR also reduce costs relative to legacy tissue valves. CONCLUSION: Patients receiving RESILIA tissue valves are projected to have lower SAVR-related health expenditures relative to mechanical and legacy tissue valves.
Assuntos
Valva Aórtica , Bioprótese , Implante de Prótese de Valva Cardíaca , Próteses Valvulares Cardíacas , Humanos , Próteses Valvulares Cardíacas/economia , Implante de Prótese de Valva Cardíaca/economia , Implante de Prótese de Valva Cardíaca/métodos , Valva Aórtica/cirurgia , Bioprótese/economia , Redução de Custos , Análise Custo-Benefício , Reoperação/economia , Gastos em Saúde/estatística & dados numéricos , Endocardite/economia , Masculino , Feminino , Complicações Pós-Operatórias/economia , Desenho de Prótese , Progressão da Doença , Modelos Econométricos , Tromboembolia/economia , Tromboembolia/prevenção & controleRESUMO
AIMS: We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems. MATERIAL AND METHODS: This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation. RESULTS: People with CF (n = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls (n = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of 11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF (n = 100) had significantly increased lung function (mean change in ppFEV1 [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: -0·8, -0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9). CONCLUSION: CF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.
Cystic fibrosis (CF) is a disease caused by a single faulty gene called CFTR, which affects the lungs, pancreas, and other organs. Medications known as CFTR modulators help improve the function of this faulty gene and have shown benefits for people with CF. In Sweden, two such medicines, lumacaftor and ivacaftor (LUM/IVA), have been available since July 2018 for treating CF. This study looks at the impact of CF on patients, caregivers, and the healthcare system, as well as the benefits of CFTR modulators. Using data from Swedish national healthcare and social insurance registries, the study compared 743 people with CF in 2019 to about 7400 people without CF, matched by sex, birth year, and location. The findings show that people with CF had 24 times higher direct healthcare costs, including outpatient visits, hospitalizations, and CF-related medications, totaling 23,233 Euros. Indirect costs, such as work absences for those over 18 with CF anssd caregivers' absences to care for sick children, were 9,629 Euros, which is five times higher than the general population. Those over 6 years old treated with LUM/IVA showed improved lung health, reduced hospitalizations (though not significantly), and needed fewer antibiotics. Caregivers' work absences decreased, but there was no change in work absences for adults with CF. Overall, treatment with LUM/IVA improved clinical outcomes and reduced the burden on caregivers and society.
Assuntos
Aminofenóis , Aminopiridinas , Benzodioxóis , Efeitos Psicossociais da Doença , Regulador de Condutância Transmembrana em Fibrose Cística , Fibrose Cística , Combinação de Medicamentos , Quinolonas , Sistema de Registros , Humanos , Fibrose Cística/tratamento farmacológico , Masculino , Feminino , Suécia , Aminofenóis/uso terapêutico , Aminofenóis/economia , Quinolonas/uso terapêutico , Quinolonas/economia , Estudos Retrospectivos , Benzodioxóis/uso terapêutico , Benzodioxóis/economia , Aminopiridinas/uso terapêutico , Aminopiridinas/economia , Criança , Adolescente , Adulto , Adulto Jovem , Regulador de Condutância Transmembrana em Fibrose Cística/genética , Cuidadores , Pessoa de Meia-Idade , Absenteísmo , Gastos em Saúde/estatística & dados numéricos , Serviços de Saúde/estatística & dados numéricos , Serviços de Saúde/economiaRESUMO
BACKGROUND: Infections are responsible for approximately 13% of cancer cases worldwide and many of these infections can be prevented by vaccination. Human papillomavirus (HPV) and hepatitis B virus (HBV) are among the most common infections that cause cancer deaths globally, despite effective prophylactic vaccines being available. This analysis aims to estimate the global burden and economic impact of vaccine-preventable cancer mortality across World Health Organization (WHO) regions. METHODS: The number of deaths and years of life lost (YLL) due to five different vaccine-preventable cancer forms (oral cavity, liver, laryngeal, cervical, and oropharyngeal cancer) in each of the WHO regions (African, Eastern Mediterranean, European, the Americas, South-East Asia Pacific, and Western Pacific) were obtained from the Institute for Health Metrics Evaluation global burden of disease dataset. Vaccine-preventable mortality was estimated considering the fraction attributable to infection, to estimate the number of deaths and YLL potentially preventable through vaccination. Data from the World Bank on GDP per capita were used to estimate the value of YLL (VYLL). The robustness of these results was explored with sensitivity analysis. Given that several Epstein-Barr virus (EBV) vaccines are in development, but not yet available, the impact of a potential vaccine for EBV was evaluated in a scenario analysis. RESULTS: In 2019, there were 465,740 potentially vaccine-preventable cancer deaths and 14,171,397 YLL across all WHO regions. The estimated economic impact due to this mortality was $106.3 billion globally. The sensitivity analysis calculated a range of 403,025-582,773 deaths and a range in productivity cost of $78.8-129.0 billion. In the scenario analysis EBV-related cancer mortality increased the global burden by 159,723 deaths and $32.4 billion. CONCLUSION: Overall, the findings from this analysis illustrate the high economic impact of premature cancer mortality that could be potentially preventable by vaccination which may assist decision-makers in allocating limited resources among competing priorities. Improved implementation and increased vaccination coverage of HPV and HBV should be prioritized to decrease this burden.
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Saúde Global , Neoplasias , Humanos , Neoplasias/mortalidade , Neoplasias/economia , Feminino , Masculino , Carga Global da Doença , Efeitos Psicossociais da Doença , Doenças Preveníveis por Vacina/prevenção & controle , Doenças Preveníveis por Vacina/economia , Pessoa de Meia-Idade , Adulto , Modelos Econométricos , Infecções por Papillomavirus/prevenção & controle , Infecções por Papillomavirus/economia , Anos de Vida Ajustados por Qualidade de VidaRESUMO
INTRODUCTION: Etranacogene dezaparvovec (EDZ), Hemgenix, is a gene therapy recently approved for people with hemophilia B (PwHB). OBJECTIVE: To estimate long-term clinical impact and cost of EDZ in the United States (US). METHODS: A decision-analytic model was developed to evaluate the long-term impact of introducing EDZ for PwHB over a 20-year time horizon. Factor IX (FIX) prophylaxis comparator was a weighted average of different FIX prophylaxis regimens based on US market share data. We compared a scenario in which EDZ is introduced in the US versus a scenario without EDZ. Clinical inputs (annualized FIX-treated bleed rate; adverse event rates) were obtained from HOPE-B phase 3 trial. EDZ durability input was sourced from an analysis predicting long-term FIX activity with EDZ. EDZ one-time price was assumed at $3.5 million. Other medical costs, including FIX prophylaxis, disease monitoring, bleed management, and adverse events were from literature. The model estimated annual and cumulative costs, treated bleeds, and joint procedures over 20 years from EDZ introduction. RESULTS: Approximately 596 PwHB were eligible for EDZ. EDZ uptake was estimated to avert 11,282 bleeds and 64 joint procedures over 20 years. Although adopting EDZ resulted in an annual excess cost over years 1-5 (mean: $53 million annually, total $265 million), annual cost savings were achieved beginning in year 6 (mean: $172 million annually; total $2.58 billion in years 6-20). The total cumulative 20-year cost savings was $2.32 billion, with cumulative cost savings beginning in year 8. CONCLUSION: Introducing EDZ to treat PwHB is expected to result in cost savings and patient benefit over 20 years. Initiating PwHB on EDZ sooner can produce greater and earlier savings and additional bleeds avoided. These results may be a conservative estimate of the full value of EDZ, as PwHB would continue to accrue savings beyond 20 years.
This analysis assessed the long-term clinical and financial impact of introducing EDZ in the United States of America for people with severe or moderately severe hemophilia B. A decision-analytic model was developed comparing a scenario with EDZ and one without EDZ over 20 years. Introducing EDZ would avert 11,292 bleeds and 64 joint procedures over 20 years and would achieve cumulative cost savings in year 8, with a total cumulative 20-year cost saving of $2.32 billion.
Assuntos
Fator IX , Hemofilia B , Humanos , Hemofilia B/tratamento farmacológico , Hemofilia B/economia , Estados Unidos , Fator IX/economia , Fator IX/uso terapêutico , Hemorragia/economia , Terapia Genética/economia , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Adulto , Masculino , Criança , Adulto Jovem , AdolescenteRESUMO
AIMS: With recent European Union marketing authorization, tabelecleucel is the first off-the-shelf, allogeneic Epstein-Barr virus (EBV)-specific T-cell immunotherapy approved for the treatment of relapsed/refractory EBV-positive post-transplant lymphoproliferative disease (EBV+ PTLD). In the absence of a control arm, real-world evidence can provide a comparative benchmark for single-arm studies in ultra-rare populations. This study assessed the treatment effect of tabelecleucel in the single-arm phase 3 ALLELE study (NCT03394365) versus a treatment group from a multinational, multicenter retrospective chart review study (RS002) of patients with EBV+ PTLD. METHODS: In ALLELE, patients had disease relapsed/refractory to rituximab ± chemotherapy and received tabelecleucel 2x106 cells/kg on days 1, 8, and 15 in 35-day cycles. Patients in RS002 had disease relapsed/refractory to rituximab ± chemotherapy and received next line of systemic therapy between January 2000 and December 2018. Propensity score-based standardized mortality/morbidity ratio weighting was used to achieve balance between treatment and comparator arms. Kaplan-Meier estimators and Cox regression models were used to compare overall survival (OS) in the re-weighted sample. RESULTS: 30 patients (n = 14 hematopoietic cell transplant [HCT], n = 16 solid organ transplant [SOT]) from ALLELE (data cutoff: November 2021) and 84 patients (n = 36 HCT, n = 48 SOT) from RS002 (data lock: January 2021) were included. Median time from diagnosis to first tabelecleucel dose (ALLELE) or start date of next line of systemic therapy (RS002) was 3.6 months. Tabelecleucel was associated with a substantial OS benefit compared with current treatment, with an unadjusted HR of 0.47 (95% confidence interval [CI] 0.25-0.88) and adjusted HR of 0.37 (95% CI 0.20-0.71) when using the start date of the next line of therapy as the index date. Sensitivity analyses yielded consistent results. CONCLUSIONS: In this study of real-world data, tabelecleucel was associated with an OS benefit among patients with R/R EBV+ PTLD for whom there is high unmet need.