Myozyme (alglucosidase alfa) Safety Information Packet effectiveness evaluation: a health care professional survey

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

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Medicinal product name:
  MYOZYME
• Study drug International non-proprietary name (INN) or common name:
  ALGLUCOSIDASE ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB07) alglucosidase alfa

  alglucosidase alfa
• Medical condition to be studied:
  Enzyme level decreased

Population studied

• Short description of the study population:
  A survey of health care professional prescribing and monitoring myozyme patients to evaluate awareness, readability, usage, usefulness, understanding, knowledge of the management of risks associated with myozyme and behavioral implementation of key safety information contained in the updated Myozyme Safety Information Packet (SIP) as compared to the previous version of the SIP. The survey was conducted in the France, Germany, Italy, Spain, UK and Poland.; ; Inclusion criteria: ; • Managed at least one patient in the preceding year on Myozyme for Pompe disease through prescribing, monitoring or administering Myozyme therapy.; ; Exclusion criteria:; • Current or ex-employee of Genzyme, a Sanofi Company or Sanofi.
• 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

• Study design:
  Two-wave, multinational, cross-sectional survey of healthcare professionals managing patients with Pompe disease receiving Myozyme. Surveys were conducted before and after implementation of the updated Safety Information Packet (SIP) to assess its effectiveness.
• Main study objective:
  The main objective of the study was to evaluate the effectiveness of the belatacept (Nulojix®) Patient Alert Card by assessing distribution, awareness, utilization, utility, knowledge, comprehension and behaviour among patients and healthcare professionals, and by evaluating the association between these measures and clinical outcomes in patients.
• Setting:
  The study was conducted in France, Germany, Sweden and Austria in hospital and physician renal units. Eligible patients were adult renal transplant recipients who had received belatacept within the previous 3 months. Eligible HCPs were nephrologists and nurses involved in prescribing, administering or managing belatacept-treated patients within the previous 6 months.
• Comparators:
  No formal comparator group was included. Analyses evaluated levels of awareness, utilization, knowledge, understanding and implementation of key safety messages and their association with clinical outcomes.
• Outcomes:
  Patient survey: awareness, distribution, utilization, utility, knowledge/comprehension, behaviour and overall understanding and implementation of PAC messages. ; ; HCP survey: awareness, distribution, utilization, utility, knowledge/comprehension, behaviour and overall understanding and implementation of PAC messages. ; ; Clinical outcomes study: serious infections and infections leading to hospitalization (primary outcomes); infections leading to discontinuation, time from infection symptom onset to medical attention or treatment, and time from transplantation to biopsy-confirmed graft rejection (secondary outcomes).
• Data analysis plan:
  Descriptive analyses estimated awareness, distribution, utilization, utility, knowledge, comprehension and behaviour outcomes. Univariate and multivariable regression analyses were used to identify determinants of understanding and implementation. Associations between understanding/implementation scores and clinical outcomes were evaluated using regression methods and Kaplan–Meier analyses where appropriate.