Trends in the Cost and Utilization of Omalizumab in the Medicare Population: 2013-2017.

Background: Omalizumab has been demonstrated to be effective in treating chronic spontaneous urticaria (CSU) and was FDA approved for this indication in 2014. Previous work has shown that access to injectable biologics varies across US counties. In the present study we evaluate geographic and temporal trends in the utilization of omalizumab in the Medicare population by dermatologists, with its use by allergists and pulmonologists as comparators. Methods: We analyzed year-over-year trends in omalizumab utilization across geographic regions using the Medicare Provider Utilization and Payment Data: Part D files. Results: Utilization of omalizumab by dermatologists increased rapidly after its FDA approval, from 0.08 claims/100,000 enrollees totaling $209/100,000 enrollees in 2014 to 1.45 claims/100,000 enrollees totaling $3115/100,000 enrollees in 2017. Nonetheless, prescribing dermatologists were present in only 2.8% (95% Confidence Interval (CI): 2.0%-3.9%) and 0.2% (95% CI: 0.0%-0.5%) of metropolitan and non-metropolitan counties, respectively, in 2017, demonstrating limited availability, especially in non-metropolitan counties. Similarly, prescribers of any specialty were available in 32.9% (95% CI: 30.2%-35.6%) and 3.8% (95% CI: 3.1%-4.8%) of metropolitan and non-metropolitan counties, respectively, in 2017. Conclusions: Our data suggest that despite increasing omalizumab utilization, there remains a lack of access across many counties, particularly in non-metropolitan regions. Efforts to expand omalizumab prescriber accessibility in these counties may improve outcomes for patients with CSU.

Clinical and economic burden of severe asthma among US patients treated with biologic therapies.

BACKGROUND: Patients with severe asthma may remain uncontrolled despite biologic therapy in addition to standard therapy, but this disease burden has not been quantified. OBJECTIVE: To estimate the clinical and economic burden in a US national sample. METHODS: Patients who have severe asthma with indicated biologic treatment (earliest use = index date) were selected from the MarketScan database between January 1, 2013, and June 30, 2018. Inclusion criteria were continuous enrollment for 12 months postindex with a minimum of 2 biologic fills, greater than or equal to 12 years of age, evidence of medium- to high-dose inhaled corticosteroids and long-acting ß-agonist combination before the index, and absence of other respiratory diagnoses and malignancies. Disease exacerbations (used to classify asthma control), health care costs, and treatment characteristics were reported during the 12-month postindex period. RESULTS: The sample included 3262 biologic patients; 88% with anti-immunoglobulin E therapy (omalizumab) and 12% non-anti-immunoglobulin E (reslizumab, mepolizumab, benralizumab). The mean age was 49 (±15) years; 64% were women. Prescriptions included inhaled corticosteroids and long-acting ß-agonist (82%), systemic corticosteroids (76%), and leukotriene receptor antagonists (68%). Notably, 63% of patients presented greater than or equal to 1 asthma exacerbation (mean 1.3 per patient/year). Furthermore, 35% of patients were categorized as having controlled asthma, whereas 28% were suboptimally controlled and 29% were uncontrolled. Patients with uncontrolled disease had higher all-cause and asthma-related costs ($69,206 and $45,693, respectively) than patients with suboptimally controlled ($59,407 and $40,793, respectively) or controlled disease ($53,083 and $38,393, respectively). Furthermore, 62% of newly treated patients were persistent with their index biologic. CONCLUSION: Biologic therapies are effective in reducing exacerbations, but a substantial proportion of patients with severe asthma treated with current biologics continue to experience uncontrolled disease, highlighting a remaining unmet need for patients with severe uncontrolled asthma.

Clinical and economic consequences of switching from omalizumab to mepolizumab in uncontrolled severe eosinophilic asthma.

Severe asthma is burdened by frequent exacerbations and use of oral corticosteroids (OCS), which worsen patients' health and increase healthcare spending. The aim of this study was to assess the clinical and economic impact of switching from omalizumab (OMA) to mepolizumab (MEP) in patients eligible for both biologics, but not optimally controlled by omalizumab. We retrospectively enrolled uncontrolled severe asthmatic patients who switched from OMA to MEP during the last two years. Information included blood eosinophil count, asthma control test (ACT), spirometry, serum IgE, fractional exhaled nitric oxide (FeNO), OCS intake, drugs, exacerbations/hospitalizations, visits and diagnostic exams. Within the perspective of Italian National Health System, a pre- and post-MEP 12-month standardized total cost per patient was calculated. 33 patients were enrolled: five males, mean age 57 years, disease onset 24 years. At OMA discontinuation, 88% were OCS-dependent with annual mean rate of 4.0 clinically significant exacerbations, 0.30 exacerbations needing emergency room visits or hospitalization; absenteeism due to disease was 10.4 days per patient. Switch to MEP improved all clinical outcomes, reducing total exacerbation rate (RR = 0.06, 95% CI 0.03-0.14), OCS-dependent patients (OR = 0.02, 95% CI 0.005-0.08), and number of lost working days (Δ = - 7.9, 95% CI - 11.2 to - 4.6). Pulmonary function improved, serum IgE, FeNO and eosinophils decreased. Mean annual costs were €12,239 for OMA and €12,639 for MEP (Δ = €400, 95% CI - 1588-2389); the increment due to drug therapy (+ €1,581) was almost offset by savings regarding all other cost items (- €1,181). Patients with severe eosinophilic asthma, not controlled by OMA, experienced comprehensive benefits by switching to MEP with only slight increases in economic costs.

The economic burden of severe asthma in children: a comprehensive study.

OBJECTIVE: The economic burden of severe asthma (SA) in children is poorly described. We aimed to determine the healthcare costs of SA in children for the French national health insurance (NHI). METHODS: Children (6-18 years of age) with physician-confirmed diagnoses of SA or non-SA (NSA) were identified. Direct and indirect expenditures related to asthma and associated co-morbidities in the previous six months were determined, based on a physician-guided parental questionnaire and confirmed by medical records. The costs for the French NHI were assessed per child over a six month period. RESULTS: Data from 74 children, including 48 with SA (22 requiring omalizumab) and 26 with NSA, were analyzed. The global cost of SA was €3,982 per child over a six-month period, including €3,821 direct costs and €161.9 indirect costs. The global cost was €6,716 (4,220) for those requiring omalizumab vs. €1,669 (3,108) for those who did not (p < 0.01). For children with SA, 65% of direct costs were attributed to medication. Among those on omalizumab, such treatment accounted for 93% of medication costs. The global cost was 10 times higher for children with SA than those with NSA (€3,982 (4,422) vs. €363.2 (352.6), p < 0.01), and 20 times higher for children with SA on omalizumab than those with NSA (p < 0.01). CONCLUSION: The economic burden of SA in children for the French NHI is substantial and mainly driven by the most severe children requiring biologics.

Cost-effectiveness of omalizumab in real world uncontrolled allergic asthma patients.

OBJECTIVE: To estimate the cost-effectiveness of omalizumab compared with standard of care in the treatment and control of severe persistent asthma, using the outcomes from the Portuguese subpopulation of the eXpeRience registry. METHODS: This was a pragmatic cost-effectiveness analysis based on real world data from the eXpeRience registry which recruited 62 patients with uncontrolled persistent allergic asthma from 20 participating centers in Portugal. Response to omalizumab treatment was measured prospectively up to 24 months by the physician's Global Evaluation of Treatment Effectiveness (GETE). Retrospective data on patients' clinical symptoms, asthma control, lung function, exacerbations, and healthcare utilization were available for up to 12 months before omalizumab initiation and served as the standard of care comparator. The number of exacerbations (severe and non-severe), the number of clinical episodes, the number of days absent from work and/or school, and GETE response to therapy were considered as effectiveness outcomes. Following a societal perspective, as cost indicators, both direct and indirect costs were considered. Direct costs relate to the cost of omalizumab, standard of care and clinical episodes (emergency room visits, hospitalizations, and unscheduled doctor visits). Indirect costs relate to the societal cost of work absenteeism. Unit costs for clinical episodes and drugs were taken from official sources within the Portuguese Health Authority. A univariate sensitivity analysis was performed. RESULTS: A rate of 1.5 exacerbations per patient-year was estimated following omalizumab treatment compared with 8.2 exacerbations per patient-year prior to omalizumab initiation, implying an 82.1% reduction in the incidence of exacerbations following omalizumab treatment relative to standard of care alone. A 54.1% reduction in GETE score was also observed in favor of omalizumab treatment. The mean cost per person-year was 3023є in the 12 months of standard of care prior to omalizumab and 16,111є in the period of treatment with omalizumab. The incremental cost-effectiveness ratios were 2244є/exacerbation avoided, and 1750є/unit decrease in GETE classification. CONCLUSION: Our results demonstrate that adding omalizumab to the treatment of patients with uncontrolled severe persistent asthma reduces the number of exacerbations, improving overall treatment effectiveness at an acceptable cost from a societal perspective.

Biologics and Allergy Immunotherapy in the Treatment of Allergic Diseases.

Allergic diseases represent some of the most chronic and costly chronic conditions. Medical management may require long-term pharmacotherapy, which is often associated with poor adherence. Although medications provide symptomatic control, they do not modify the allergic disease. Patients may prefer disease-modifying treatments that provide lasting benefits after discontinuation. To date, allergy immunotherapy is the only proved disease modification therapy associated with lasting benefits after discontinuation. However, allergy immunotherapy safety and efficacy has only been established in allergic rhinitis, mild to moderate asthma, and some patients with atopic dermatitis.

Cost-effectiveness of omalizumab for the treatment of inadequately controlled severe allergic asthma in Chinese children.

Objective: The addition of omalizumab to standard therapy has proven to be efficacious in children with severe allergic asthma. The goal of this study was to assess the cost-effectiveness of adding omalizumab to standard treatment for asthma in Chinese pediatric patients.Methods: A Markov model was constructed to project the health and economic outcomes in pediatric patients with severe allergic asthma. Model inputs were obtained from the literature. Cost and quality-adjusted life-years (QALYs) were measured over a five-year time horizon. One-way and probabilistic sensitivity analyses were conducted.Results: For the base-case analysis, the addition of omalizumab to standard therapy yielded an incremental cost of $49,047 for 0.232 incremental QALY, led to an incremental cost-effectiveness ratio of $211,217/QALY. Sensitivity analyses were robust for these results.Conclusions: This study found that the addition of omalizumab is not a cost-effective strategy compared with standard therapy for children with severe allergic asthma in China due to its high cost.

Estimation of Health and Economic Benefits of Clinic Versus Home Administration of Omalizumab and Mepolizumab.

BACKGROUND: Biologic therapy is a paradigm-shifting management strategy for many patients with asthma and chronic urticaria, but concerns for therapy-associated anaphylaxis may limit access to these therapies for patients unable to travel to medical clinics. OBJECTIVE: To characterize the cost-effectiveness of in-clinic versus at-home biologic therapy with omalizumab and mepolizumab. METHODS: Economic evaluation using microsimulations was performed from societal and health care sector perspectives for patients with asthma or chronic spontaneous urticaria receiving omalizumab or mepolizumab in an allergy clinic, primary care provider (PCP) office, or at home over a 1-year time horizon (12 injections per year in each base case with sensitivity analysis to 24 injections per year). Travel times and distances were applied to a population attending a tertiary care allergy clinic in Northern New England receiving omalizumab or mepolizumab, using a willingness-to-pay value of $10 million per death prevented and in-clinic administration reducing anaphylaxis fatality and hospitalization 10- to100-fold. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: One-way allergy clinic travel distances significantly exceeded local PCP offices (49 miles, 95% CI, 42-56, vs 12 miles, 95% CI, 10-15). In the omalizumab societal analysis, annual PCP and allergy clinic administration cost $1369.14 (mean) ± $51.33 (SD) and $1916.68 ± $40.86, respectively. Small reductions in medication-related fatalities with in-clinic administration were offset by the potential increase in automobile fatalities resulting from traveling to the allergy clinic (14.6 ± 15.0 per million person-years for this strategy). Compared with at-home administration, in-clinic omalizumab administration was not cost-effective, with an incremental cost-effectiveness ratio of $500,648,430 (PCP), and with allergy clinic administration dominated by higher costs and automobile-related fatalities. Routine mepolizumab clinic administration was dominated by at-home administration unless anaphylaxis rates or self-administration teaching costs were high. CONCLUSIONS: For many patients, at-home administration of omalizumab or mepolizumab may be a cost-effective strategy.

Evaluating direct costs and productivity losses of patients with asthma receiving GINA 4/5 therapy in the United States.

BACKGROUND: Despite the low prevalence for all patients with asthma, those with severe disease account for a disproportionately large economic burden. OBJECTIVE: To evaluate current direct health care and productivity loss costs associated with patients with asthma receiving Global Initiative for Asthma Step 4/5 therapy ("G4/5 asthma") in the United States. METHODS: Asthma patients aged 12 years or older were identified in the IBM MarketScan Research Databases between January 1, 2012 and December 31, 2015. Patients were indexed on their earliest medical claim for asthma and were required to have at last 2 years of continuous eligibility. The G4/5 asthma classification required 1 or more medium- or high-dosage inhaled corticosteroids (ICS)/long-acting beta-agonist (LABA) claims, 1 or more omalizumab claims, or systemic corticosteroids covering at least 50% of the 12-month baseline period. The European Respiratory Society/American Thoracic Society criteria for severe uncontrolled asthma were modified for claims data and used to identify patients with exacerbations or high rescue medication use ("Ex/R″). Direct health care costs and productivity loss costs attributable to workplace absence or short-term or long-term disability were measured during the 12-month post-index period. RESULTS: A total of 605,614 patients with asthma were identified. Annual health care costs were $4,384 greater for G4/5 asthma vs non-G4/5 patients with asthma; asthma-related costs contributed $2,183 of this difference (P < .001). Differences were primarily driven by G4/5 patients with asthma with Ex/R, whose costs were $5,019 greater than G4/5 patients without Ex/R (P < .001). For patients with 1 or more absences or short-term disability claims, G4/5 patients missed 7.2 more work hours for absence and had 3.9 more days of work lost for short-term disability than non-G4/5 patients with asthma, respectively (P < .05). CONCLUSION: G4/5 patients with asthma incurred significantly greater direct and indirect costs than non-G4/5 patients with asthma. Differences were largely driven by those with Ex/R.

Efectividad y análisis farmacoeconómico del tratamiento del asma grave con omalizumab en la práctica clínica / Effectiveness and pharmacoeconomic analysis of the treatment of severe asthma with omalizumab in clinical practice

Objetivo: Evaluar el gasto sociosanitario tras la incorporación de omalizumab al tratamiento estándar en el control del asma grave, así como analizar su efectividad, en condiciones de práctica clínica. Método: Estudio observacional, retrospectivo y multicéntrico realizado en 12 servicios de neumología de la Comunidad Valenciana. Se analizaron datos de 186 pacientes. Se compararon resultados del año previo y los cinco años posteriores a la incorporación de omalizumab. La efectividad se calculó a partir del incremento de tres puntos en el Asthma Control Test y la reducción del número de exacerbaciones anuales. La utilidad se calculó mediante el número de años de vida ajustados por calidad. En la evaluación económica se incluyeron costes directos e indirectos. Los resultados se expresaron en términos de relación coste-efectividad incremental y relación coste-utilidad incremental. Resultados: Se detectaron mejoras significativas en la función pulmonar, el control del asma, la calidad de vida y el número de años de vida ajustados por calidad, entre el año anterior y el posterior al inicio de omalizumab. Teniendo en cuenta los costes directos e indirectos, la relación coste-efectividad incremental por exacerbación evitada fue de 1.789,28 Euros (intervalo de confianza 95%: 1.019,13-3.038,12) y de 4.569,38 Euros (intervalo de confianza 95%: 3.442,86-6.075,05) por incremento de tres puntos en el Asthma Control Test. La relación coste-utilidad incremental por número de años de vida ajustados por calidad ganada fue de 50.239,98 Euros (intervalo de confianza 95%: 37.209,88-68.923,84). Conclusiones: La introducción de omalizumab en el tratamiento del asma grave es efectiva en condiciones de práctica clínica. Disminuye los costes directos e indirectos y proporciona mejoras significativas en el estado de salud de los pacientes

Objective: To assess socio-sanitary expenditure after the addition of omalizumab to standard treatment in the control of severe asthma and to analyse its effectiveness under standard clinical practice. Method: Observational retrospective multicentre study conducted in 12 pneumology services in the Valencian Community, Spain. Data from 186 patients were analysed. Results of the year before and after the addition of omalizumab were compared. Effectiveness was calculated based on a 3-point increase in the Asthma Control Test and a reduction in the number of annual exacerbations. Utility was calculated by the number of quality adjusted life years. The economic assessment included both direct and indirect costs and results were expressed in terms of incremental cost-effectiveness and incremental cost-utility ratio. Results: Significant improvements were found in lung function, asthma control, quality of life, and quality adjusted life years between the year before and after the introduction of omalizumab. Taking into account direct and indirect costs, the incremental cost-effectiveness for each avoided exacerbation was Euros 1,789.28 (95% CI: Euros 1,019.13-3,038.12) and Euros 4,569.38 (95% CI: 3,442.86-6,075.05) per 3-point increase in the Asthma Control Test score. The incremental cost-utility ratio per quality adjusted life years gained was Euros 50,239.98 (95% CI: 37,209.88-68,923.84). Conclusions: The addition of omalizumab to the treatment regime of patients with severe asthma is effective under standard clinical practice, decreases direct and indirect costs, and provides significant improvements in the health status of patients

Effectiveness and pharmacoeconomic analysis of the treatment of severe asthma with omalizumab in clinical practice.

OBJECTIVE: To assess socio-sanitary expenditure after the addition of omalizumab to standard treatment in the control of severe asthma and to analyse its effectiveness under standard clinical practice. METHOD: Observational retrospective multicentre study conducted in 12  pneumology services in the Valencian Community, Spain. Data from 186  patients were analysed. Results of the year before and after the addition of  omalizumab were compared. Effectiveness was calculated based on a 3-point  increase in the Asthma Control Test and a reduction in the number of  annual exacerbations. Utility was calculated by the number of quality  adjusted life years. The economic assessment included both direct and  indirect costs and results were expressed in terms of incremental cost- effectiveness and incremental cost-utility ratio. RESULTS: Significant improvements were found in lung function, asthma control, quality of life, and quality adjusted life years between the  year before and after the introduction of omalizumab. Taking into account  direct and indirect costs, the incremental cost-effectiveness for each avoided  exacerbation was € 1,789.28 (95% CI: € 1,019.13-3,038.12) and € 4,569.38 (95% CI: 3,442.86-6,075.05) per 3-point increase in the Asthma Control Test score. The incremental cost-utility ratio per quality adjusted life years gained was € 50,239.98 (95% CI: 37,209.88-68,923.84). CONCLUSIONS: The addition of omalizumab to the treatment regime of patients with severe asthma is effective under standard clinical practice, decreases direct and indirect costs, and provides significant  improvements in the health status of patients.

Objetivo: Evaluar el gasto sociosanitario tras la incorporación de  omalizumab al tratamiento estándar en el control del asma grave, así como analizar su efectividad, en condiciones de práctica clínica.Método: Estudio observacional, retrospectivo y multicéntrico realizado en 12  servicios de neumología de la Comunidad Valenciana. Se analizaron datos de 186 pacientes. Se compararon resultados del año previo  ylos cinco años posteriores a la incorporación de omalizumab. La efectividad se calculó a partir del incremento de tres puntos en el Asthma  Control Test y la reducción del número de exacerbaciones anuales. La  utilidad se calculó mediante el número de años de vida ajustados por calidad.  En la evaluación económica se incluyeron costes directos e  indirectos. Los resultados se expresaron en términos de relación coste- efectividad incremental y relación coste-utilidad incremental.Resultados: Se detectaron mejoras significativas en la función pulmonar, el  control del asma, la calidad de vida y el número de años de vida ajustados  por calidad, entre el año anterior y el posterior al inicio de omalizumab.  Teniendo en cuenta los costes directos e indirectos, la relación coste- efectividad incremental por exacerbación evitada fue de 1.789,28 €  (intervalo de confianza 95%: 1.019,13-3.038,12) y de 4.569,38 € (intervalo  de confianza 95%: 3.442,86-6.075,05) por incremento de tres puntos en el  Asthma Control Test. La relación coste-utilidad incremental por número de  años de vida ajustados por calidad ganada fue de 50.239,98 € (intervalo de  confianza 95%: 37.209,88-68.923,84).Conclusiones: La introducción de omalizumab en el tratamiento del asma  grave es efectiva en condiciones de práctica clínica. Disminuye los costes  directos e indirectos y proporciona mejoras significativas en el estado de  salud de los pacientes.

Efficacy and effectiveness of omalizumab in the treatment of childhood asthma.

INTRODUCTION: Omalizumab is a monoclonal antibody that binds and inhibits free serum immunoglobulin E, a mediator involved in the clinical manifestations of allergic asthma. Evidence for its efficacy and safety in the treatment of moderate-to-severe allergic asthma is based primarily on studies in adolescents and adults. However, there is increasing evidence of its utility in children with allergic asthma aged 6-12 years. Areas covered: This article reviews efficacy, safety, and effectiveness of omalizumab in the treatment of moderate-to-severe allergic asthma in children aged 6-12 years in clinical trials and in studies in clinical practice. Pharmacoeconomic aspects of its use among this population and the positioning of omalizumab in pediatric asthma management guidelines are also discussed. Additionally, an algorithm for the management of poorly controlled severe pediatric asthma in children older than 6 years is proposed. Electronic databases, such as PubMed, were searched for terms Asthma and Omalizumab and for asthma management guidelines. Expert commentary: Add-on omalizumab is an effective maintenance therapy in children aged 6-12 years with poorly controlled moderate-to-severe allergic asthma treated with medium-high inhaled corticosteroids doses and inhaled long-acting ß2-agonists. Omalizumab appears safe in children in both clinical trials and real-life setting and may be cost-effective.

Biomarkers for severe allergic asthma in children: could they be useful to guide disease control and use of omalizumab?

INTRODUCTION: Although symptom controls in asthmatic children can be achieved through compliant use of conventional medication, some children have uncontrolled severe persistent asthma, especially if they are allergic. For these children, omalizumab (approved by the EMA and FDA in children aged > 6 years) could be a therapeutic option. However, response to omalizumab varies from one child to another. Predictive biomarkers of omalizumab effectiveness could be useful to monitor response to treatment. Area covered: The authors searched in the PubMed database for publications related to the use of biomarkers in allergic asthma. Supported by their own experience in phenotyping asthma in children, they analyzed whether these biomarkers could be useful in assessing response to omalizumab. Expert commentary: Th2 inflammation in children with allergic asthma can be assessed by measuring several biomarkers (blood eosinophil, serum ECP or periostin, FeNO). While a single measurement may be insufficient, a combination of biomarkers assessments may improve the follow-up of children treated by omalizumab.

Clinical and economic impact of a one-year treatment with omalizumab in patients with severe allergic asthma within a drug programme in Poland.

BACKGROUND: Allergic asthma is the most prevalent phenotype of severe asthma where treatment with omalizumab (OMB) has been proven to be particularly beneficial. In Poland, OMB therapy is available and reimbursed within a drug programme where strict inclusion and exclusion criteria are defined. The objective of this study was to present a descriptive analysis regarding the trends in outcomes (clinical, quality of life, costs) among a cohort of patients who satisfy inclusion criteria for the initiation of OMB treatment and who successfully responded to OMB according to a set of objective criteria. METHODS: A retrospective analysis of data collected during the 52 weeks of OMB treatment was carried out. The study population was adolescents and adults with severe allergic asthma that was uncontrolled despite a combination of high-dose inhaled corticosteroids (ICS)/long-acting beta-agonists (LABA) and/or other controllers (leukotriene receptor antagonists (LTRA), sustained-release theophylline, and short- or long-acting muscarinic antagonists (SAMA/LAMA), who were the first to finish the one-year treatment. A clinical and cost analysis for patients included in the programme was conducted comparing the one-year pre-treatment period to the one-year treatment period outcomes. RESULTS: Data of 85 patients who completed the first year of therapy were reviewed and analysed. Add-on OMB treatment resulted in a median decrease in exacerbation rate of 66% relative to the baseline and a reduction in oral steroid (OCS) dose by an average of 7.7 mg. At the end of the 52 weeks of therapy the changes in the quality of life questionnaire (AQLQ) and the asthma control questionnaire (ACQ) scores were 1.86 and 1.45 points, respectively. The mean cost of asthma treatment increased by an average of 15,979 EUR per patient per year (baseline period - 802 EUR/patient/year; OMB treatment - 16,781 EUR/patient/year). The cost to avoid one exacerbation was 17721 EUR. CONCLUSION: The clinical outcomes for the observed subset of patients were highly improved. At the same time, costs of the treatment increased, mainly due to the high OMB costs. Other costs associated with a lower number of hospitalizations and ED and office visits and a reduction in OCS dose decreased. These descriptive data can be used for further investigation in defining patients who benefit the most from OMB treatment in clinical practice.

Cost-effectiveness of omalizumab for the treatment of chronic spontaneous urticaria.

BACKGROUND: Chronic spontaneous urticaria (CSU) is a skin disease with itchy hives and/or angio-oedema that last for at least 6 weeks without an obvious external trigger. OBJECTIVES: To determine the cost-effectiveness of omalizumab relative to standard of care (SoC; up to four times the daily dose of H1 -antihistamines) in the Netherlands from a societal perspective. METHODS: The Markov model used consisted of five health states based on Urticaria Activity Score over 7 days. Model settings and characteristics of the Dutch patient population were based on an online survey among clinical experts and were validated during an expert committee meeting. Transition probabilities were derived from the GLACIAL trial. Healthcare consumption, quality of life (using EuroQol-5D) and productivity losses were derived from a burden-of-illness study (ASSURE-CSU) among 93 Dutch patients. Healthcare consumption and productivity losses were evaluated using the Dutch costing manual. The comparator treatment was SoC, consisting of (updosed) antihistamines. A 10-year time horizon was used. RESULTS: The incremental cost-effectiveness ratio (ICER) of omalizumab vs. SoC was €17 502 per quality-adjusted life-year (QALY) gained. Productivity costs played an important role in the value of the ICER; discarding productivity costs resulted in an ICER of €85 310 per QALY. CONCLUSIONS: Omalizumab is cost-effective compared with SoC. The outcomes of this study were used to establish omalizumab as third-line therapy in the Dutch treatment guidelines for CSU.

Cost-effectiveness of omalizumab add-on to standard-of-care therapy in patients with uncontrolled severe allergic asthma in a Brazilian healthcare setting.

OBJECTIVE: Omalizumab add-on to standard-of-care therapy has proven to be efficacious in severe asthma patients for whom exacerbations cannot be controlled otherwise. Moreover, evidence from different healthcare settings suggests reduced healthcare resource utilization with omalizumab. Based on these findings, this study aimed to assess the cost-effectiveness of the addition of omalizumab to standard-of-care therapy in patients with uncontrolled severe allergic asthma in a Brazilian healthcare setting. METHODS: A previously published Markov model was adapted using Brazil-specific unit costs to compare the costs and outcomes of the addition of omalizumab to standard-of-care therapy vs standard-of-care therapy alone. Model inputs were largely based on the eXpeRience study. Costs and health outcomes were calculated for lifetime-years and were annually discounted at 5%. Both one-way and probabilistic sensitivity analyses were performed. RESULTS: An additional cost of R$280,400 for 5.20 additional quality-adjusted life-years was estimated with the addition of omalizumab to standard-of-care therapy, resulting in an incremental cost-effectiveness ratio of R$53,890. One-way sensitivity analysis indicated that discount rates, standard-of-care therapy exacerbation rates, and exacerbation-related mortality rates had the largest impact on incremental cost-effectiveness ratios. LIMITATIONS: Assumptions of lifetime treatment adherence and rate of future exacerbations, independent of previous events, might affect the findings. The lack of Brazilian patients in the eXpeRience study may affect the findings, although sample size and baseline characteristics suggest that the modeled population closely resembles Brazilian severe allergic asthma patients. CONCLUSION: Results indicate that omalizumab as an add-on therapy is more cost-effective than standard-of-care therapy alone for Brazilian patients with uncontrolled severe allergic asthma, based on the World Health Organization's cost-effectiveness threshold of up to 3-times the gross domestic product.

Longterm clinical outcomes of omalizumab therapy in severe allergic asthma: Study of efficacy and safety.

Omalizumab has been shown to be an effective add-on therapy for patients with uncontrolled severe persistent allergic asthma. There has been a steady accumulation of evidence on the long-term effectiveness of omalizumab; however, data on real-life outcomes beyond one year of treatment is limited. In this study, we report on long-term outcomes of omalizumab treatment. We collected data from our severe asthma registry on hospitalisations, exacerbations, corticosteroid sparing, asthma control, lung function, biomarkers and side effects, to determine if the benefit was sustained and treatment was safe on the long term. Forty-five patients [mean age 44.9 years (range 19-69), females 37/45 (82%), mean duration of omalizumab treatment = 60.7 ± 30.9 months (range 23-121) were included in the analysis. We observed a reduction in the annual acute asthma related hospital admissions for the total population from 207 at baseline to 40 on treatment (80.7% reduction), whilst the per patient annual hospitalisations were reduced from a mean of 4.8 to 0.89 post-omalizumab treatment (p < 0.00001). There was a 76.7% reduction in daily mean maintenance OCS dose (prednisolone equivalent) from 25.8 mg (n = 43) to 6.0 mg (p < 0.0001), associated with clinically significant improvement in asthma control questionnaire (ACQ) from mean score of 4.1 (range 3.7-4.7) to 2.27 (range 0.5-4.1) p < 0.0001. The mean % predicted FEV1 has improved from 59.2% at baseline to 75.7% on treatment (p = 0.001). There was a statistically non-significant reduction in median peripheral blood eosinophils (PBE) from 300 cells/µl (range 40-1050) at baseline to 175 cells/µl (range 0-1500) post-treatment (p = 0.068), and statistically significant reduction of median fraction exhaled nitric oxide (FeNO) level from 37 parts per billion (range 12-178) to 24 ppb (range 7-50) (p = 0.0067). The work/school missed days were reduced in 17/19 patients who were at employment or school. The overall safety profile of the treatment seemed acceptable and was consistent with published experience. In conclusion, results from this real-life study demonstrate that improved outcomes in patients with severe allergic asthma are sustained with longer-term omalizumab therapy.

Impact of omalizumab on medical cost of childhood asthma in Japan.

Omalizumab is effective in children with severe asthma, but its impact on medical cost in Japan is not clear. We evaluated the impact of omalizumab on medical cost by comparing the pre- vs post-omalizumab-initiation medical costs of 12 children with severe asthma who received omalizumab for 2 years, and calculating incremental cost-effectiveness ratio for omalizumab therapy. Health outcome was measured as hospital-free days (HFD). The median total medical costs and medication fee per patient increased significantly after omalizumab initiation because of the high cost of omalizumab. The median hospitalization fee per patient, however, decreased significantly after omalizumab initiation due to reduction in hospitalization. Omalizumab led to an estimated increase of 40.8 HFD per omalizumab responder patient per 2 years. The cost was JPY 20 868 per additional HFD. Omalizumab can therefore reduce hospitalization cost in children with severe asthma in Japan.

Cost Utility of Omalizumab Compared with Standard of Care for the Treatment of Chronic Spontaneous Urticaria.

BACKGROUND: Chronic spontaneous urticaria (CSU) negatively impacts patient quality of life and productivity and is associated with considerable indirect costs to society. OBJECTIVE: The aim of this study was to assess the cost utility of add-on omalizumab treatment compared with standard of care (SOC) in moderate or severe CSU patients with inadequate response to SOC, from the UK societal perspective. METHODS: A Markov model was developed, consisting of health states based on Urticaria Activity Score over 7 days (UAS7) and additional states for relapse, spontaneous remission and death. Model cycle length was 4 weeks, and total model time horizon was 20 years in the base case. The model considered early discontinuation of non-responders (response: UAS7 ≤6) and retreatment upon relapse (relapse: UAS7 ≥16) for responders. Clinical and cost inputs were derived from omalizumab trials and published sources, and cost utility was expressed as incremental cost-effectiveness ratios (ICERs). Scenario analyses included no early discontinuation of non-responders and an altered definition of response (UAS7 <16). RESULTS: With a deterministic ICER of £3183 in the base case, omalizumab was associated with increased costs and benefits relative to SOC. Probabilistic sensitivity analysis supported this result. Productivity inputs were key model drivers, and individual scenarios without early discontinuation of non-responders and adjusted response definitions had little impact on results. ICERs were generally robust to changes in key model parameters and inputs. CONCLUSIONS: In this, the first economic evaluation of omalizumab in CSU from a UK societal perspective, omalizumab consistently represented a treatment option with societal benefit for CSU in the UK across a range of scenarios.