Exploring different composite definitions of responders and non-responders to biologic treatment for severe asthma (FULL BEAM)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Registry-based cohort study

Study drug and medical condition

• Medical condition to be studied:
  Asthma

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  4000

Study design details

• Main study objective:
  1)Define the study population To describe pre-biologic demographic, clinical and functional characteristics of patients initiating biologics, overall and by biologic class 2) Quantify levels of response and characterize patients by levels of response To operationally assess levels of response to biologics (from non-response to highest level of response) in individual domains
• Outcomes:
  Clinical remission and response to treatment. While clinical remission will be assessed in all patients irrespective of their status pre-biologic initiation, response can only be assessed in patients impaired when initiating the treatment. Impairment will be defined for each domain independently as: 1) exacerbations: ≥2 exacerbations in the year preceding biologic initiation, 2) long-term OCS use
• Data analysis plan:
  Objective 1 aims at describing the study population used in objectives 2 and 3. To this end, the pre-biologic asthma-related outcomes will first be described individually and in composite variables. For individual asthma-related outcomes, both continuous and categorical variables will be used. The distributions will be compared between initiated biologic class using t-tests, Kruskall Wallis tests, or Person’s Chi-squared tests as appropriate. The proportion of patients responding to biologics as defined in section 5 will be computed overall and by biologic classes, for each individual domain and composite definition of response. Patient characteristics will be described by levels of response using means, standard deviations, medians and interquartile ranges for continuous variables and numbers and percentages for categorical variables.