TABLE 4

Assessment of effectiveness in biologics: ranking of objective criteria

RankingPatients on maintenance OCSItem scorePatients not on maintenance OCSItem score
National Leads Survey (n=28)
1Reduction of exacerbation rate by 75% over 1 year3.4Reduction of exacerbation rate by 75% over 1 year2.5
2Reduction of chronic dose of OCS by 50%3.4Reduction of exacerbation rate by 50% over 1 year2.8
3Stopping chronic maintenance OCS3.5Reduction of exacerbation rate by 25% over 1 year4.8
4Reduction of exacerbation rate by 50% over 1 year4.1Reduction in ACQ by 1 at 4/6 months5
5Reduction of exacerbation rate by 25% over 1 year6Increase in AQLQ by 1 at 4/6 months5
6Increase in AQLQ by 1 at 4/6 months6.1Reduction in ACQ by 0.5 at 4/6 months5.5
7Reduction in ACQ by 1 at 4/6 months6.2Increase in AQLQ by 0.5 at 4/6 months5.7
8Reduction in ACQ by 0.5 at 4/6 months6.6Reduction of chronic dose of ICS by 50%6.2
9Increase in AQLQ by 0.5 at 4/6 months6.9Improvement of 5% predicted FEV17.6
10Improvement of 5% predicted FEV18.5
Experts Broad Survey (n=263)
1Reduction of exacerbation rate by 75% over 1 year2.6Reduction of exacerbation rate by 75% over 1 year1.9
2Reduction of exacerbation rate by 50% over 1 year3.1Reduction of exacerbation rate by 50% over 1 year3.1
3Stopping chronic maintenance OCS3.5Reduction of exacerbation rate by 25% over 1 year4.5
4Reduction of chronic dose of OCS by 50%4.1Reduction in ACQ by 1 at 4/6 months4.6
5Reduction in ACQ by 1 at 4/6 months4.8Reduction in ACQ by 0.5 at 4/6 months5.2
6Reduction of exacerbation rate by 25% over 1 year5.1Reduction of chronic dose of ICS by 50%5.6
7Reduction in ACQ by 0.5 at 4/6 months6.3Increase in AQLQ by 1 at 4/6 months6.3
8Increase in AQLQ by 1 at 4/6 months7.3Increase in AQLQ by 0.5 at 4/6 months6.8
9Increase in AQLQ by 0.5 at 4/6 months8.5Improvement of 5% predicted FEV17.8
10Improvement of 5% predicted FEV18.9

These items are clinical efficacy criteria used in most RCTs to assess effectiveness of biologics in severe asthma. The lower the mean score of the item is, the higher its importance as effectiveness criteria is, according to participants. OCS: oral corticosteroids; AQLQ: Asthma Quality of Life Questionnaire; ACQ: Asthma Control Questionnaire; ICS: inhaled corticosteroids; FEV1: forced expiratory volume in 1 s.