XOLAIR (Omalizumab) Outcomes in Pediatric Allergic Asthma Patients in the United States

Study Overview
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Brief Summary
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A retrospective database pre-post cohort study, identifying asthmatic patients, aged 6- 11, with omalizumab use over 24 months

Detailed Description
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This was a retrospective database pre-post cohort study, identifying asthmatic patients with omalizumab use over 24 months. Omalizumab use was analyzed in two categories. First, initiators were defined as individuals with ≥ 1 omalizumab claims. Responders were defined as individuals with at least 4 omalizumab claims. Descriptive statistics are provided for both groups, but outcomes are reported only for responders. For this study, any persons from the Marketscan database with an asthma diagnosis and >= 1 omalizumab prescription/administration during the index period were identified. From this patient population the inclusion and exclusion criteria were applied, resulting in the final study cohort. Each person was assigned an index date based on their initial use of omalizumab. Study Period: 07/07/2015 - 12/31/2019 Index Period: 07/07/2016 - 12/31/2018 Index Date: The date of the first medical or pharmacy claim of omalizumab Baseline Period (pre-index): 12 months before index date Follow up Period (post-index): 12 months after index date The MarketScan® Claims Database (including commercial and Medicaid claims) was used. The claims database included data from 07/08/2015 - 12/31/2019. The MarketScan® Databases capture personspecific clinical utilization, expenditures, and enrollment across inpatient, outpatient, prescription drug, and carve-out services from a selection of large employers, health plans, and government and public organizations. The current study used the Commercial, Medicare Supplemental and Multi-State Medicaid Databases.

Official Title
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XOLAIR (Omalizumab) Outcomes in Pediatric Allergic Asthma Patients in the United States

Conditions
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Asthma

Intervention / Treatment
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* Drug: Omalizumab


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: We used all eligible Marketscan beneficiaries with an asthma diagnosis and omalizumab use between 07/07/2016 - 12/31/2018 (index period). Continuous enrollment in the Marketscan database was required to ensure the availability of claims data to capture study outcomes and covariates. Omalizumab cohort was defined as ≥1 prescription claims within the index period, with the date of first dispensing deemed the index date. Asthma was defined by ≥1 diagnosis code in any available diagnosis field on or prior to index date. ICD-9-CM: 493.xx OR ICD-10-CM: J45.x 6-11 years of age at the time of index ≥12-months pre-index and ≥12-months post-index continuous eligibility in medical and pharmacy benefits Enrollment gap of ≤30 days will be considered continuous enrollment Exclusion criteria: Patients were excluded from the study if they had one or more of the following: Bronchial Thermoplasty at any time during data capture. Current Procedural Terminology (CPT): 31660, 31661 Prior asthma-indicated biologic use during the 12 months pre or post-index Omalizumab: NDC: 50242004062; 50242004201 or HCPCS: J2357; S0107; C9217 Mepolizumab: NDC: 00173088101, 00173088185 or HCPCS: J2182 Reslizumab: NDC: 5931061031 or HCPCS: J2786 Benralizumab: NDC: 0310173030 or HCPCS: C9466 Dupilumab: NDC: 0024591400 or 0024591800

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Commercial Population<br>Individuals with at least one omalizumab prescription in the Marketscan commercial claims during the identification period (07/07/2016 - 12/31/2018) | Drug: Omalizumab<br>* Omalizumab use was analyzed in two categories. First, initiators were defined as individuals with ≥ 1 omalizumab prescription. Responders were defined as individuals with at least 4 administrations of omalizumab.<br>|
| Medicaid Population<br>Individuals with at least one omalizumab prescription in the Truven Medicaid claims during the identification period (07/07/2016 - 12/31/2018) | Drug: Omalizumab<br>* Omalizumab use was analyzed in two categories. First, initiators were defined as individuals with ≥ 1 omalizumab prescription. Responders were defined as individuals with at least 4 administrations of omalizumab.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients with uncontrolled asthma (impairment + risk criteria) post omalizumab treatment initiation | Uncontrolled asthma was based on either impairment or risk criteria in a 12- month consecutive period. Impairment was identified as ≥6 Short-acting Beta Agonist (SABA) prescriptions dispensed in 12 months. Each prescription should reflect one month days supply. Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. The date of uncontrolled asthma assessment was defined as the earliest date between the sixth SABA prescription (impairment criteria) or the first exacerbation outcome during a consecutive 12-months (risk criteria). | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Proportion of patients with excessive SABA use (≥6 SABA prescriptions dispensed in 12 months) (impairment criteria) | Proportion of patients with uncontrolled asthma (impairment criteria) were reported. Impairment was identified as ≥6 SABA prescriptions dispensed in 12 months. Each prescription should reflect one month days supply. - The date of uncontrolled asthma assessment was defined as the earliest date between the sixth SABA prescription (impairment criteria). | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Proportion of patients with exacerbations requiring IP visits (risk criteria) | Proportion of patients with uncontrolled asthma (risk criteria) were reported. Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. - The date of uncontrolled asthma assessment was defined as the first exacerbation outcome during a consecutive 12-months (risk criteria). | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Proportion of patients with exacerbations requiring ED visits (risk criteria) | Proportion of patients with uncontrolled asthma (risk criteria) were reported. Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. - The date of uncontrolled asthma assessment was defined as the first exacerbation outcome during a consecutive 12-months (risk criteria). | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Proportion of patients with exacerbations requiring Oral corticosteroids (OCS) prescriptions (risk criteria) | Proportion of patients with uncontrolled asthma (risk criteria) were reported. Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. - The date of uncontrolled asthma assessment was defined as the first exacerbation outcome during a consecutive 12-months (risk criteria). | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Mean daily dose of Inhaled Corticosteroids (ICS) use | Mean daily dose of Inhaled Corticosteroids (ICS) use was reported during the follow-up | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Mean cumulative dose of OCS use | Mean cumulative dose of Oral corticosteroids were reported during follow-up | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Mean number of prescriptions of OCS use | Mean number of prescriptions of OCS use were reported during follow-up | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 
| Mean days of supply of OCS use | Mean days of supply of OC were reported during follow-up | 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of uncontrolled asthma patients (total: risk and impairment criteria) | Uncontrolled asthma was based on either impairment or risk criteria in a 12- month consecutive period. Impairment was identified as ≥6 Short-acting Beta Agonist (SABA) prescriptions dispensed in 12 months. Each prescription should reflect one month days supply. Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. The date of uncontrolled asthma assessment was defined as the earliest date between the sixth SABA prescription (impairment criteria) or the first exacerbation outcome during a consecutive 12-months (risk criteria). | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Proportion of uncontrolled asthma patients (per impairment criteria) | Proportion of patients with uncontrolled asthma (impairment criteria) were reported. Impairment was identified as ≥6 SABA prescriptions dispensed in 12 months. Each prescription should reflect one month days supply. - The date of uncontrolled asthma assessment was defined as the earliest date between the sixth SABA prescription (impairment criteria). | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Proportion of uncontrolled asthma patients (per risk criteria) | Risk was defined as ≥1 unique exacerbation(s) in 12 months, including asthma-related ED visits or hospitalizations (emergency or hospital care with a diagnosis of asthma in the first position only [ICD-9 code 493.xx, ICD-10 code J45.xx]) or an OCS dispensing within 7 days AFTER an outpatient visit with an asthma diagnosis in ANY position. - The date of uncontrolled asthma assessment was defined as the first exacerbation outcome during a consecutive 12-months (risk criteria). | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Mean difference in the total number of asthma-related prescriptions | Mean difference in the total number of asthma-related prescriptions between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Mean difference in daily dose (of days supply) of ICS use | Mean difference in daily dose (of days supply) between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Proportion of patients with a reduction in daily dose of ICS use | Proportion of patients with a reduction in daily dose between the baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Mean difference in daily dose of OCS use | Mean difference in daily dose between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Mean difference in number of prescriptions of OCS use | Mean difference in number of prescriptions between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Mean difference in days of supply of OCS use | Mean difference in days of supply between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 
| Proportion of patients with a reduction in total prescriptions of OCS use | Proportion of patients with a reduction in total prescriptions for OCS use between baseline and follow-up periods were reported | through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Asthma,, Omalizumab (Xolair),, Asthma Control,, Corticosteroids,, Pediatric,, Children,, Adolescents,, Healthcare Expenditures