Evaluation of the Effectiveness of a Xospata Routine Risk Minimisation Measure (RMM) and an Additional Risk Minimisation Measure (aRMM): A Cross-sectional Survey Study among Healthcare Professionals to Assess Awareness and Knowledge

Study type

• Study topic:
  Other
• Study topic, other:
  Disease/Epidemiology study
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  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:
  Multi-national survey study

Population studied

• Short description of the study population:
  A survey of health care professional, specifically those who specialize in blood diseases including hematologists and hematooncologists prescribing Xospata in the participating Europe countries (Czech Republic, France, Germany, Italy, Netherlands, and Sweden).; Inclusion Criteria:; 1. HCPs who treat patients diagnosed with leukemias, including AML, who can prescribe Xospata in the participating European countries, and ; 2. HCPs who provide permission to share their responses in aggregate with the EMA and/or the NCAs; ; Exclusion Criteria:; 1. HCPs who participated in the cognitive pre-testing of the survey, or ; 2. HCPs who have themselves or have immediate family members who have worked for Astellas, ICON (coordinating investigator), EMA, or the NCAs of the participating member states within the past 5 years.
• 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:
  400

Study design details

• Main study objective:
  The main objective of the study is to describe HCPs' awareness and knowledge of the three selected safety concerns (PRES, QT prolongation, and DS) associated with the use of Xospata and to assess percentage of HCPs that recall receiving and/or reviewing Xospata routine RMM (SmPC) and the Xospata aRMM (HCP educational materials).
• Outcomes:
  The percentages of HCPs with correct responses to questions regarding knowledge of the three selected safety concerns (PRES, QT prolongation and DS) associated with the use of Xospata. The percentages of HCPs that recall receiving and/or reviewing the Xospata routine RMM (SmPC) and the Xospata aRMM (HCP educational materials).
• Data analysis plan:
  A statistical analysis plan (SAP) will be developed to describe all planned analyses in detail, along with shells for variable lists, tables and figures to be produced. The study population included in the data analysis will include HCPs who completed at least one of the questions in the survey associated with the primary endpoints. Descriptive data analyses will be conducted. Levels of receipt, use, and knowledge will be calculated with 95% two-sided Confidence Intervals (CIs). These descriptive results will be reported for the overall population of respondents and by country, and for sub-groups such as HCP specialty, time since dissemination of HCP materials in each country, and history of prescribing Xospata, depending on the final distribution of data across these sub-groups. Additionally, a descriptive analysis of the characteristics of non-responders will be completed.