A Study to Evaluate the Effectiveness of the Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) in the European Economic Area (EEA) (ACE-536-MDS-005)

Study type

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

  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:
  Survey

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B03XA06) luspatercept

  luspatercept

Population studied

• Short description of the study population:
  A survey of healthcare professionals who prescribed REBLOZYL for the treatment of haematological diseases, were a part of additional risk minimisation measures (aRMMs) dissemination list and practiced in any of the participating European economic area (EEA) countries. ; Inclusion criteria:;  HCPs experienced in the treatment of haematological diseases who may intend to prescribe REBLOZYL in the participating European countries and were on the target group for dissemination of the aRMMs;  The HCP provides permission to share their responses in aggregate with the European Medicines Agency (EMA) or NCA, if requested; ; Exclusion criteria:;  HCPs who previously participated in the cognitive pre-testing of the survey questionnaire to be used for the study;  HCPs who have been direct employees of the MAH, the EMA, or the study vendor within the past 5 years
• 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:
  200

Study design details

• Main study objective:
  The overall goal of this PASS is to assess the awareness and knowledge levels of HCPs regarding key messages included in the nationally implemented aRMMs for REBLOZYL. Awareness encompasses receipt of the educational material and any other way HCPs can learn about the risks associated with REBLOZYL.
• Outcomes:
  Knowledge of the key messages included in REBLOZYL HCP Checklist, assessed as the percentages of HCPs with correct responses to each question. Levels of providing WCBP with the REBLOZYL Patient Card, assessed as the percentage of HCPs who report providing REBLOZYL Patient Card “always”, Levels of knowledge for all 5 core questions. Awareness, reading, use of REBLOZYL HCP Checklist, assessed as percentages of HCPs who report awareness, reading, and use of the REBLOZYL HCP Checklist. Distribution of responses regarding the primary source(s) from which HCPs learned about the messages included in the REBLOZYL HCP Checklist, assessed as the percentages of HCPs who report using each of the possible sources as the primary source.
• Data analysis plan:
  The primary analysis population will include all HCPs who met the eligibility criteria and who have completed all items included in the core question set. Knowledge levels for all key messages in the REBLOZYL HCP Checklist will be calculated with 95% 2-sided CI. Knowledge levels will also be stratified by relevant subgroups. The core question set includes 5 questions. For questions included in the core question set, and for the question regarding providing WCBP with the REBLOZYL Patient Card, the percentage of HCPs who answer each core question correctly will be estimated and assessed against the 80% (± 95% CI) target. The success criteria threshold will only be applied when the number of HCPs who answered a question is at least 30.