Table 4.

Outcomes in Phase 3 Trials for FDA-approved biologic agents

Primary Outcome	Frequency	Definition(s)	Trials (Time Frame)
Annualized Rate of Severe Exacerbation Events	1	A severe exacerbation was defined as a deterioration of asthma requiring: use of systemic corticosteroids for >=3 days; or hospitalization or emergency room visit because of asthma, requiring systemic corticosteroids. Annualized event rate was the total number of exacerbations that occurred during the treatment period divided by the total number of participant-years treated.	NCT02414854 (baseline to week 52)
Annual Asthma Exacerbation Rate	2	The annual exacerbation rate is based on unadjudicated annual exacerbation rate reported by the investigator in the eCRF.	NCT01914757 (baseline to week 56) NCT01928771 (baseline to week 48)
Frequency of Clinical Asthma Exacerbations (CAEs)	2	An exacerbation event was considered a CAE if the patient met either or both of the criteria listed below and this was corroborated with at least 1 other measurement to indicate the worsening of clinical signs and symptoms of asthma: Use of systemic, or an increase in the use of inhaled, corticosteroid treatment for 3 or more days; or an increased 2 or more fold for at least 3 or more days for patient's already on corticosteroids. asthma-related emergency treatment, such as an unscheduled visit to the physician's office or emergency room for nebulizer treatment or other urgent treatment to prevent worsening of asthma symptoms, or an asthma-related hospitalization.	NCT01285323 (baseline to month 12) NCT01287039 (baseline to week 52)
Number of Clinically Significant Exacerbations of Asthma Per Year	1	Clinically significant exacerbations of asthma are defined as worsening of asthma which required use of systemic corticosteroids (IV or oral steroid like prednisone, for at least 3 days or a single intramuscular (IM) corticosteroid (CS) dose is required. For maintenance of systemic corticosteroids, at least double the existing maintenance dose for at least 3 days was required) and/or hospitalization and/or emergency department (ED) visits.	NCT01691521 (baseline to week 32)
Frequency of Each of the Two Criteria for CAEs	2	An exacerbation event was considered a CAE if the patient met either or both of the criteria listed below and this was corroborated with at least 1 other measurement to indicate the worsening of clinical signs and symptoms of asthma: Use of systemic, or an increase in the use of inhaled, corticosteroid treatment for 3 or more days; or an increased 2 or more fold for at least 3 or more days for patient's already on corticosteroids. asthma-related emergency treatment, such as an unscheduled visit to the physician's office or emergency room for nebulizer treatment or other urgent treatment to prevent worsening of asthma symptoms, or an asthma-related hospitalization CAEs were adjudicated by committee to assure consistency.	NCT01285323 (baseline to month 12) NCT01287039 (baseline to week 52)
Absolute Change from Baseline in Pre-Bronchodilator FEV1	1	FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by spirometer.	NCT02414854 (baseline, week 12)
Change from Baseline in FEV1	2	FEV1 is a standard measurement of air movement in the lungs of patients with asthma. It is the volume of air expired in the first second of a forced expiration. Improvement in FEV1 is a measure in the reduction of bronchospasm, the reduction of airway inflammation, or both. FEV1 was measured using forced expiratory air spirometry.	NCT01270464 (baseline, week 4, 8, 12 and 16)
Percentage Reduction from Baseline in OCS Dose While Maintaining Asthma Control	1	Percentage reduction of OCS dose was calculated as (optimized OCS dose [mg/day] at baseline - final OCS dose at Week 24)/optimized OCS dose at baseline x 100.	NCT02528214 (baseline, week 24)
Percentage Reduction from Baseline in Final OCS Dose While Maintaining Asthma Control	1	Baseline OCS dose is the dose upon which the patient is stabilised at randomisation (Week 0). Final OCS dose is the dose at Week 28. The percentage reduction from baseline is defined as: {(Baseline dose-final dose)/baseline dose}*100%. If a patient discontinues from the study during a given dose reduction period, or the patient experiences an exacerbation between Weeks 24 and 28 or immediately before discontinuation, then the final OCS dose will be 1 dose level higher than that which directly preceded the event.	NCT02075255 (baseline, week 28)
Median Percentage Reduction from Baseline in OCS Dose While Maintaining Asthma Control	1	A supplementary presentation of the Primary Outcome Measure data; result is presented as median (inter-quartile range). Percentage reduction of OCS dose was calculated as (optimized OCS dose [mg/day] at baseline - final OCS dose at Week 24)/optimized OCS dose at baseline x 100.	NCT02528214 (baseline, week 24)
Number of Participants with the indicated Percent Reduction from Baseline in OCS Dose While Maintaining Asthma Control	1	Baseline (BL) dose was the prescribed optimized prednisone/prednisolone dose following the OCS Optimization Phase. Maintenance (MN) dose was the mean of all daily prednisone/prednisolone doses during the MN Phase (weeks 20 to 24). The percent reduction of OCS dose during weeks 20 to 24 compared to BL dose was calculated as: 100 x (BL dose minus MN dose)/BL dose. Asthma control between weeks 20 and 24 was defined as no clinically significant exacerbation (worsening of asthma that required use of systemic corticosteroids or hospitalization and/or emergency department visits) during this period. The percent reduction of OCS was categorized as: 90 to 100%; 75 to <90%; 50 to <75%; >0 to <50%; no decrease in prednisone dose, or lack of asthma control, or withdrawal (WD) from treatment.	NCT01691508 (baseline, week 20-24)