DARWIN EU® - Drug utilisation study in individuals with cystic fibrosis in Europe

Study type

• Study topic:
  Herbal medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name, other:
  Ivacaftor; Ivacaftor and lumacaftor ; Ivacaftor and tezacaftor; Ivacaftor, tezacaftor and elexacaftor; Ursodeoxycholic acid; Multienzymes (lipase, protease etc.); Dornase alfa (desoxyribonuclease); Mannitol, acetylcysteine, ambroxol; Salbutamol; Tobramycin ; Proton pomp inhibitors;
• Medical condition to be studied:
  Cystic fibrosis

Population studied

• Short description of the study population:
  The study population (objectives 1 - 5) will include individuals with first recorded CFTR modulator treatment in the period between 1st of January 2015 and 31st of December 2024 (or latest date available) after CF diagnosis. Only individuals with first recorded CFTR modulator treatment at least 180 days prior to the end of data availability in each database will be included. Eligible individuals must have at least one year of data visibility prior to the first recorded CFTR modulator treatment and no use of CFTR modulator treatment before the index date. This requirement of one year of prior data history will not hold for children below 1 year of age.; For objectives 4 and 5, additional exclusion criteria will be applied on an event-specific basis. Individuals will be excluded from the analysis of a specific adverse event if they have a record of that condition within one year before the index date (objective 4). This includes:; • A SNOMED disease code for depression (for exclusion from the depression outcome), a SNOMED disease code for anxiety (for exclusion from the anxiety outcome), a SNOMED disease code for cataract (for exclusion from the cataract outcome), a SNOMED disease code for haemoptysis (for exclusion from the haemoptysis outcome).; Additionally, individuals with a SNOMED disease code of upper or lower respiratory tract infection, requiring treatment with antibiotics or antiviral medications within 30 days prior to the start of follow-up, will be excluded from the analysis of pulmonary exacerbation (objective 5).;
• Age groups:
  • In utero
  • 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)

Study design details

• Study design:
  A cohort study will be conducted using routinely collected health data from 7 data sources.
• Main study objective:
  1. To describe treatment patterns of CFTR modulators at the active ingredient level, from the first recorded CFTR modulator treatment after CF diagnosis until end of follow up, including the proportion of individuals switching between CFTR modulators, overall and stratified by paediatric and adult populations.; 2. To characterise individuals initiating CFTR modulator therapy, overall (any CFTR modulator) and by active ingredient, in terms of demographics, and use of other CF related therapies, overall and stratified by paediatric and adult populations. ; 3. To characterise CFTR modulator use, overall (any CFTR modulator) and by active ingredient, including treatment duration, cumulative dose, number of repeated prescriptions, overall and stratified by paediatric and adult populations.; 4. To estimate the background incidence rates of pre-specified adverse events of special interest (cataract, depression, anxiety, and haemoptysis) in the CFTR modulator treated individuals, overall (any CFTR modulator) and by active ingredient level, presented overall and stratified by paediatric and adult populations and by calendar year. ; 5. To measure the incidence of pulmonary exacerbation following CFTR modulator initiation, overall (any CFTR modulator) and by active ingredient, presented overall and stratified by paediatric and adult populations, and time since CFTR modulator initiation (one- and two-years post-initiation). ;