Periodic Assessment Survey of Patients/Caregivers to Assess Understanding of the Risks with the BKEMV® Risk Evaluation and Mitigation Strategy (REMS)

Study type

• Study topic:
  Human medicinal product

  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

• Medicinal product name, other:
  Eculizumab-aeeb (BKEMV)
• Study drug International non-proprietary name (INN) or common name:
  ECULIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AJ01) eculizumab

  eculizumab
• Medical condition to be studied:
  Paroxysmal nocturnal haemoglobinuria

  Atypical haemolytic uraemic syndrome

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:
  75

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 validated vendor survey platform.
• Main study objective:
  The primary objectives of the study are:; ; To describe patient and/or caregiver knowledge of:; • Survey Knowledge 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.; • Survey Knowledge Domain 2: Patients should understand the need to be vaccinated against meningococcal infection and receive antibacterial drug prophylaxis if needed.; • Survey Knowledge 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 survey knowledge domain.
• Data analysis plan:
  Statistical analyses will focus on descriptive summaries, with formal hypothesis testing used to determine whether pre-defined knowledge rate thresholds are met. The statistical analysis plan has been revised to replace the previously used Clopper-Pearson method for confidence interval (CI) calculation with the hypergeometric method. This change provides improved accuracy for the calculation of 95% CIs, particularly in small or finite populations, thereby strengthening the reliability of the knowledge rate estimates. The primary analytic criterion remains the lower bound of the 95% CI for each knowledge rate, which will be used to determine whether pre-defined thresholds are met.