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

  • Paediatric Population (< 18 years)
    • Adolescents (12 to < 18 years)
    • Children (2 to < 12 years)
    • Infants and toddlers (28 days – 23 months)
    • Neonate
      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
  • 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

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.