DARWIN EU® - RR Characterisation of individuals with cystic fibrosis in Europe

Study type

• Study topic:
  Human medicinal product
• Study topic, other:
  cystic fibrosis
• Study type:
  Non-interventional study
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  • Ivacaftor; • Ivacaftor and lumacaftor ; • Ivacaftor and tezacaftor; • Ivacaftor, tezacaftor and elexacaftor;

Population studied

• Short description of the study population:
  For patient-level characterisation (objective 1), incidence analyses of selected events of special interest and pulmonary exacerbation (objectives 3 and 4), the study population will include all individuals with a CF diagnosis recorded in the period between 1st January 2015 and 31st December 2024 (or latest date available). ; To ensure sufficient follow-up, only individuals with a CF diagnosis record no later than 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 record of CF diagnosis. The requirement of one year prior data availability will not hold for children below 1 year of age. ; Additionally, for the incidence analysis of pulmonary exacerbation (objective 4), individuals should not have experienced pulmonary exacerbation in the 60 days prior to study inclusion, defined as a SNOMED disease code of upper or lower respiratory tract infection, requiring treatment with antibiotics or antiviral medications.; For clinical characterisation (objective 2), the study population will include individuals with a 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 a 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 prior use of CFTR modulator treatment at active ingredient level. ; This requirement of 1 year of prior data history will not hold for children below 1 year of age.
• 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 6 data sources.
• Main study objective:
  1. To characterise all individuals with a CF diagnosis recorded during the study period in terms of demographics, prespecified comorbidities, Pseudomonas aeruginosa colonisation, CF screening, genotyping test, and CFTR modulator treatment use, overall and stratified by paediatric and adult populations. ; 2. To characterise timing and availability of key clinical measurements, including Forced Expiratory Volume (FEV), height, weight, Body Mass Index (BMI) measurements, sweat chloride levels, and genotyping tests in individuals who initiated any CFTR modulator treatment after CF diagnosis, overall and stratified by paediatric and adult populations.; 3. To estimate the background incidence rates of pre-specified events of special interest: cataract, depression, and anxiety in the CF population, overall and stratified by paediatric and adult populations and by calendar year.; 4. To measure the annual incidence of pulmonary exacerbation among all individuals with a CF diagnosis recorded during the study period, overall and stratified by paediatric and adult populations. ;