Evaluation of referring HCPs’ and parents’/carers’ understanding of specific risks associated with Strimvelis™ treatment (STRIM-001)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

  Other
• Non-interventional study design, other:
  Survey

Study drug and medical condition

• Name of medicine:
  STRIMVELIS
• Medical condition to be studied:
  Adenosine deaminase decreased

Population studied

• Short description of the study population:
  A survey of healthcare professionals (HCPs) and parents/carers of patients enrolled within the registry or other patients outside of the registry receiving treatment with Strimvelis.; Inclusion criteria for HCPs ; • HCPs or HCPs’ close family members may not have been employees of Orchard Therapeutics, GlaxoSmithKline, Pharmaceutical Product Development LLC (PPD), the Food and Drug Administration (FDA), or the European Medicines Agency (EMA). ; • HCPs must be licensed. ; • An HCP must not have previously completed a survey regarding Strimvelis educational materials. ; • An HCP must have previously referred a patient for Strimvelis treatment. ; ; Inclusion criteria for Parent/Carer survey, parents or carers: ; • Parents/carers or parents’/carers’ close family members may not have been employees of Orchard Therapeutics, GlaxoSmithKline, Pharmaceutical Product Development LLC (PPD), the Food and Drug Administration (FDA), or the European Medicines Agency (EMA). ; • A parent/carer must not have previously completed a survey regarding Strimvelis educational materials. ; • A parent’s/carer’s child must have received treatment with Strimvelis approximately twelve months prior to participation.
• Age groups:
  • Paediatric Population (< 18 years)

    • Children (2 to < 12 years)
• Estimated number of subjects:
  20

Study design details

• Main study objective:
  To Evaluate the effectiveness of routine and additional risk minimization measures (e.g. Summary of Product Characteristics/Patient Information Leaflet, educational materials) by assessing the understanding of HCPs and parents/carers with regard to specific risks associated with Strimvelis.
• Outcomes:
  Safety Concerns (malignancy due to insertional oncogenesis), autoimmunity, unsuccessful response to gene therapy, pregnancy, Requirement for long-term monitoring, patient alert card
• Data analysis plan:
  Data from the survey respondents will be analysed and reported as descriptive statistics. A frequency distribution of responses to each question will be presented. Summary statistics will be prepared describing the proportion of referring HCPs and parents/carers for whom questionnaires were returned relative to total number of referring HCPs and parents/carers of patient eligible for enrolment into the study.