EVALUATION OF THE EFFECTIVENESS OF RISK MINIMISATION MEASURES: A SURVEY AMONG HEALTH CARE PROFESSIONALS AND PATIENT/CAREGIVERS TO ASSESS THEIR KNOWLEDGE AND ATTITUDES ON PRESCRIBING AND HOME ADMINISTRATION CONDITIONS OF VELAGLUCERASE ALPHA (VPRIV®) IN 6 EUROPEAN COUNTRIES (VPRIV Home infusion)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB10) velaglucerase alfa

  velaglucerase alfa
• Medical condition to be studied:
  Gaucher's disease

Population studied

• Age groups:
  • Infants and toddlers (28 days – 23 months)
  • 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)
• Estimated number of subjects:
  96

Study design details

• Main study objective:
  to assess the proportion of prescribers treating patients with Gaucher Disease who receive, understand and implement the Educational Material messages.
• Data analysis plan:
  The statistical results of the six countries will be presented in the same report, overall, per country and per physician’s specialty group Continuous variables will be described by their number (of valid cases, of missing values), mean, standard deviation, and median, Q1, Q3, minimum and maximum.Categorical variables will be described as the total number and relative percentage per category. These will be the percentage per category.In case of multiple choice questions, the frequency of each option provided by the physicians will be reported in the statistical results. Different combinations of the answers provided will not be considered. Confidence intervals of 95% will be evaluated, when relevant.The proportions of correct and appropriate answers to selected questions asked in the questionnaire will be expressed among physicians with complete analysable web