A 2-Year Observational Study to Describe the Characteristics and Progression of Patients Suffering from Idiopathic Pulmonary Fibrosis Treated with Esbriet® in the Conditions of Use

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  PIRFENIDONE
• Medical condition to be studied:
  Idiopathic pulmonary fibrosis

Population studied

• Short description of the study population:
  Patients suffering from idiopathic pulmonary fibrosis (IPF) treated with Esbriet.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with idiopathic pulmonary fibrosis (IPF)
• Estimated number of subjects:
  214

Study design details

• Main study objective:
  Describe the clinical progression over two years of patients suffering from IPF treated with Esbriet in the conditions of use.
• Outcomes:
  Over total treatment time (up to 2 years):• Change in % predicted Forced Vital Capacity (FVC)• Change in distance travelled during the 6MWT, 1.Modalities used to determine IPF2.Cases of IPF comorbidities, in particular acute exacerbation and PAH3.Progression-free survival, defined as time from initiation of Esbriet to the first occurrence of the following events:•An absolute decline in % predicted FVC ≥ 10% over the duration of the FU •An absolute decline in 6MWT distance ≥ 50m over the duration of the FU•Death from any cause
• Data analysis plan:
  The statistical analyses will be descriptive.Continuous variables, including changes from baseline for continuous variables, will be summarized with means, standard deviations, medians, minimums and maximums, and amount of missing data.Categorical variables, including changes from baseline categories, will be summarized with counts and percentage of patients, and the amount of missing data.Kaplan-Meier estimates will be used to summarize the data for time-to-event variables.The primary and secondary endpoints will be estimated by the mean with 95% confidence interval for continuous variables and the frequency (percentage) with 95% confidence interval for categorical variables.