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

  • 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

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 previous version of the SIP among HCPs.

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.