Periodic Knowledge, Attitudes, and Behavior (KAB) Survey of Patients and/or Caregivers to Assess Understanding of the Risks with the BKEMV™ Risk Evaluation and Mitigation Strategy (REMS) (20240214)

Study type

• Study topic:
  Other
• Study topic, other:
  US-based, observational, cross-sectional survey of patients who are at least 18 years of age and caregivers of patients of all ages who have been dispensed at least 1 dose of BKEMV.
• 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

• Name of medicine, other:
  Eculizumab-aeeb (BKEMV)
• Medical condition to be studied:
  Paroxysmal nocturnal haemoglobinuria

  Atypical haemolytic uraemic syndrome

  Myasthenia gravis

Population studied

• Short description of the study population:
  The study will include patients who are at least 18 years of age and caregivers of patients of all ages who have been dispensed at least 1 dose of BKEMV.
• Age groups:
  Adult and elderly population (≥18 years)
• Estimated number of subjects:
  153

Study design details

• Study design:
  This US-based, observational, cross-sectional survey involves patients and caregivers who received at least one dose of BKEMV. It is self-administered via secure internet/telephone using the validated United BioSource LLC (UBC) Pathways® Knowledge Survey System.
• Main study objective:
  The primary objectives of the study are:; ; To describe patient and/or caregiver knowledge of:; • Key Message Domain 1: Patients should understand that receiving treatment with BKEMV increases the chance of getting serious meningococcal infections, which may quickly become life-threatening and cause death if not recognized and treated early.; • Key Message Domain 2: Patients should understand the need to be vaccinated against meningococcal infection and receive antibacterial drug prophylaxis if needed.; • Key Message Domain 3: Patients should be able to recognize the signs and symptoms of meningococcal infection and the need for immediate medical evaluation.
• Setting:
  Online survey
• Comparators:
  N/A
• Outcomes:
  The primary endpoint of the study is:; • The number of respondents who score 80% or greater in each key message domain
• Data analysis plan:
  Statistical analyses will be descriptive in nature. Counts and percentages will be calculated for each question/item in the questionnaire. 95% confidence intervals (CIs) for the survey end points will be calculated to provide an estimate of precision; however no formal hypothesis will be tested. ; All CIs around the percentages will be exact binomial 2-sided 95% CIs calculated according to the method of Clopper-Pearson. Analyses will be performed at the respondent level; therefore, within-respondent variation is not relevant.