Drug Utilization Study of eliglustat in the US population using MarketScan® Database (ELIGLC06912)

Study type

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

  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

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

Population studied

• Short description of the study population:
  Patients with at least one prescription of eliglustat in the MarketScan™ database from September 18, 2014 (date of market availability) until September 30, 2017.; Also, to assess the proportion of patients who were genotyped for CYP2D6, US patients from the ICGG Gaucher Registry database will be used as an alternative data source for estimating the proportion of eliglustat treated patients who were genotyped for CYP2D6.
• Age groups:
  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)
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  Objectives:; 1) To estimate the proportion of patients in the U.S. who have been genotyped for CYP2D6 prior to the initiation of eliglustat therapy,; 2) To estimate the dose and duration of eliglustat therapy, as well as the prevalence, duration and type of past and concomitant medication use of strong and moderate inhibitors of CYP2D6 and CYP3A, strong CYP3A inducers, P-gp and CYP2D6 substrates, in patients treated with eliglustat in the U.S.;
• Data analysis plan:
  The following data analyses were performed:; • Descriptive analysis of patients who have a genotyping procedure for CYP2D6 prior to treatment initiation of eliglustat (i.e. N, proportion) in the claims records.; • Descriptive analysis of patient’s dose and duration on eliglustat therapy within the study period from index date until the end of continuous treatment (i.e. N, mean, standard deviation, median, min, max). ; • Descriptive analyses of the prior medication use of concomitant medication of interest (proportion, type, and duration) in the year prior to treatment initiation of eliglustat therapy.; • Descriptive analyses of the concomitant medication use of concomitant medication of interest (proportion, type, and duration) in patients while receiving eliglustat therapy.