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

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  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

• Main study objective:
  The main objective of this survey is to assess 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 safety information packet (SIP) as compared to the pr
• Outcomes:
  The updated safety information packet (SIP) will be compared to the previous version of the SIP for differences in: awareness, usage of the SIP, levels of HCPs’ knowledge and understanding related to the key messages, behavior of HCPs around key safety messages and levels of readability, degree of usefulness, The updated safety information packet (SIP) will be compared to the previous version of the SIP for differences in proportion of HCPs who have received the SIP, determinants of response, opinion and qualitative reasons for lack of immunological testing.
• Data analysis plan:
  The primary analysis will assess, for each survey wave, awareness, readability, usage, usefulness, understanding, patient management and behavioural implementation by percentage and mean scores. Comparisons of these endpoints between the two waves will be made. For each survey, distribution and opinion will be assessed by percentage.

Documents

Study results

Summary of Study report_EUPAS10301_ALGMYC08432