Hemlibra Survey to Prescribers and Patients/Carers to Evaluate Awareness, Knowledge and Compliance to Additional Risk Minimization Measures

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
• 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:
  HEMLIBRA
• Study drug International non-proprietary name (INN) or common name:
  EMICIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BX06) emicizumab

  emicizumab
• Medical condition to be studied:
  Haemophilia A with anti factor VIII

  Haemophilia A without inhibitors

Population studied

• Short description of the study population:
  The study population is HCPs who have prescribed Hemlibra® and patients with haemophilia A who have been treated with Hemlibra® or their carers, all of whom must meet the following inclusion/exclusion criteria:; ; Inclusion criteria; HCPs;  HCPs who prescribed Hemlibra® at least once outside the context of a clinical trial to patients with haemophilia A with FVIII inhibitors or patients with severe; haemophilia A without FVIII inhibitors;  HCPs who are willing to participate in this survey; Patients/Carers;  Patients who received at least 4 doses of Hemlibra® within the last 6 months, including patients on ongoing therapy;  Patients who are willing to participate in this self-administered survey, or their carers (parent or legal guardian) if the patient is under the age of 18; ; Exclusion criteria; HCPs;  HCPs who may have conflicts of interest with the study (e.g., HCPs employed by Roche or IQVIA); Patients/Carers;  Patients/Carers who may have conflicts of interest with the study (e.g., patients/carers employed by Roche or IQVIA);  Patients who have participated in a Hemlibra® clinical trial;  Patients with non-severe (i.e., mild or moderate) haemophilia A without FVIII inhibitors
• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  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:
  110

Study design details

• Main study objective:
  The primary objective for this study is to evaluate the awareness (of risks of TMA and TE and the potential risk of life-threatening bleeding due to misinterpretation of standard coagulation tests), knowledge, and adherence of prescribers (HCPs) and patients/carers to the additional risk minimization measures (RMMs) for Hemlibra.
• Data analysis plan:
  Statistical analyses will be described and further detailed in the Statistical Analysis Plan. The following described analysis might be revised, and adjustments might occur. All the analyses will be descriptive and results will be presented separately for HCPs and patients/carers, overall, by region, and other. Continuous variables will be presented by their number (of valid cases, of missing data if applicable), mean, standard deviation, median, first quantile, third quantile, minimum, and maximum. No missing data will be imputed. Categorical variables will be presented as the total number and relative percentage per category. Confidence intervals of 95% will be evaluated, when relevant. The percentage of correctly answered survey questions will be calculated. Success on the educational materials for additional RMMs will be defined a priori as achieving overall scores of >/=75% for awareness and adherence and >/=60% for knowledge for HCPs and >/=60% in all areas for patients/carers.

Documents

Study results

Final_CSR,_Study_BO40853,_,_Published_Output-1_Redacted