Real-World Effects and Utilisation Patterns of Elexacaftor, Tezacaftor, and Ivacaftor Combination Therapy (ELX/TEZ/IVA) in Patients with Cystic Fibrosis (CF)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  KAFTRIO
• Medical condition to be studied:
  Cystic fibrosis

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  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)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  21000

Study design details

• Main study objective:
  To evaluate, among patients treated with ELX/TEZ/IVA in the real-world setting: 1. Safety outcomes 2. Effectiveness outcomes / CF disease progression 3. Safety and effectiveness outcomes/ CF disease progression in genotype subgroups 4. Frequency and outcome of pregnancy in female patients 5. Drug utilisation patterns and characterise potential off-label use outside of the labelled indication
• Outcomes:
  Safety analyses: death, organ transplant, hospitalisations, pulmonary exacerbations, CF complications, respiratory microbiology, liver function tests Disease progression analyses: Percent predicted FEV1, BMI Pregnancy analyses: pregnancy outcome, gestational age, congenital anomalies (data availability varies by registry) Drug utilization analyses: ELX/TEZ/IVA use outside of labeled indications
• Data analysis plan:
  Data will be analysed separately for each registry over the course of the 5 year study. Results of analyses will be presented in annual study reports. Each annual report will include patient data collected through the end of the previous calendar year. Descriptive statistics will be presented for all study outcomes. Continuous variables will be summarised using the following descriptive summary statistics where appropriate: the number of observations (n), mean, SD, SE, 95% CI, median, minimum value, maximum value, and 25th and 75th percentile values. Categorical variables will be summarised using counts, percentages, and 95% CIs as appropriate. All safety outcomes, effectiveness / CF disease progression outcomes, and pregnancy outcomes will be evaluated in the ELX/TEZ/IVA Cohorts in the US CFFPR and German CF registry. In addition to these 2 registries, the ECFSPR will be used to provide additional information for the evaluation of drug utilisation patterns in the European region.