Healthcare Professional (HCP) Survey to Assess the Effectiveness of the Additional Risk Minimization Measures (aRMM) for Casgevy® (exagamglogene autotemcel)

Study type

• Study topic:
  Other
• Study topic, other:
  Study of effectiveness of additional risk minimization materials
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Medicinal product name:
  CASGEVY
• Study drug International non-proprietary name (INN) or common name:
  EXAGAMGLOGENE AUTOTEMCEL
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AX05) exagamglogene autotemcel

  exagamglogene autotemcel
• Additional medical condition(s):
  sickle cell disease; beta thalassemia

Population studied

• Short description of the study population:
  The target population will be HCPs at Authorized Treatment Centers (ATC) in EU countries who have counseled patients regarding Casgevy as a treatment option.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Adults (65 to < 75 years)
    • Adults (75 to < 85 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Health Care Professionals
• Estimated number of subjects:
  30

Study design details

• Study design:
  This study uses a multi-national, observational cross-sectional design. It is a web-based survey using close-ended multiple-choice questions.
• Main study objective:
  1. The HCPs’ understanding of the important safety information in the Guide for HCPs regarding the important identified risk of delayed platelet engraftment, the important potential risks of neutrophil engraftment failure and gene editing-related oncogenesis, and the missing information on long-term effects. ; 2. The HCPs’ awareness of the aRMM tools. ; 3. The HCPs’ utilization of the aRMM tools (behavior). ;
• Setting:
  Data collection via a web-based survey will be initiated in all countries in the EU where Casgevy is launched approximately 18 months after first drug availability has been achieved in any EU country.
• Comparators:
  N/A
• Outcomes:
  The survey will commence with screening questions and will collect responses to each question required to address the study objectives: ; ; • Response to questions about important safety information detailed in the Guide for HCPs. ; • HCPs’ awareness of the Guide for HCPs, Guide for Patients/Carers and Patient Card. ; • Whether or not the Guide for Patients/Carers and the Patient Card are being distributed to patients treated with Casgevy. ; ; In addition, demographic information and information on where the HCP sourced the safety information will be collected.;
• Data analysis plan:
  Data collected from the survey will be reported as descriptive statistics. Categorical variables will be summarized using counts and percentages. ; ; Eighteen knowledge questions will test the HCPs understanding of 4 KRMs. The response to each of the 18 questions will be categorized as correct or incorrect. The table below outlines the 4 KRMs and the individual questions evaluating each message. ; ; Using the Completed Surveys analysis set, the rate of correctness (percentage of correct answers) will be summarized for each of the 4 KRMs. The aRMM pertaining to each KRM will be considered successful if the correct rate for the specific KRM is at least 80%. ;