Drug utilisation study of Eliglustat for the treatment of Gaucher Disease Type 1 in Europe using electronic healthcare records (ELIGLC06913)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

Study drug and medical condition

• Name of medicine:
  CERDELGA
• Study drug International non-proprietary name (INN) or common name:
  ELIGLUSTAT
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX10) eliglustat

  eliglustat
• Medical condition to be studied:
  Gaucher's disease type I

Population studied

• 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)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The main objective of the DUS is to estimate the dose and duration of Eliglustat therapy as well as the proportion, type, and duration of past and concomitant medication use in GD1 patients treated with Eliglustat. ; The concomitant medications of interest are strong and moderate inhibitors of CYP2D6 and/or CYP3A inhibitors, strong CYP3A inducers, P-gp substrates and CYP2D6 substrates.
• Data analysis plan:
  Only descriptive analyses will be performed: ; - Descriptive analysis of Eliglustat therapy in terms of duration and dose. ; - Descriptive analysis of past (one year prior to Eliglustat initiation) and concomitant medication use in terms of proportion, type, and duration. The treatments of interest will be strong and moderate inhibitors of CYP2D6 and/or CYP3A, strong CYP3A inducers, P-gp substrates and CYP2D6 substrates. The proportions will be computed taking into account all treatments of interest and also by type of treatments and by duration. Proportions for the group CYP inhibitors will also be computed. ; - Descriptive analysis of the health care service pattern (i.e. prescriber’s specialty and types of patient visit) for the use of concomitant medications in patients treated with Eliglustat. All these analyses will be conducted by country. Stratification analyses based on sex and age will be performed if the number of GD1 patients taking Eliglustat allows it.