Fabrazyme (agalsidase beta) home infusion educational materials effectiveness evaluation: a survey for healthcare providers and patients/caregivers (Fabrazyme Home Infusion Ed Mat Survey)

Study type

• Study topic:
  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

• Name of medicine:
  FABRAZYME
• Study drug International non-proprietary name (INN) or common name:
  AGALSIDASE BETA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB04) agalsidase beta

  agalsidase beta

Population studied

• Short description of the study population:
  Online survey was conducted independently in two populations: 1) HCPs and 2) Patients or their caregivers ; HCPs include both physicians and nurses:; A) Physicians: A target list of physicians known to be prescribing Fabrazyme home infusion will be provided by the MAH and used for recruitment.; B) Nurses: To recruit nurses we will use a mix of both targeted recruitment and via physician referral; for targeted recruitment the MAH will provide a list of external home care provider companies with contact details per market. Potentially eligible nurses will be contacted via telephone or email with information about this study.; ; Inclusion/exclusion criteria:; • Involved in the treatment of at least 1 patient with Fabry disease in the last 12 months; • Have prescribed Fabrazyme for home infusion and/or monitor, oversee the management, and/or provide in person medical supervision of patients on Fabrazyme home infusion in the last 12 months; • Not a current or ex-employee of Sanofi; ; Patient/caregivers recruitment; All HCPs from the target lists provided by the MAH and who are contacted regarding the study will also be asked to refer all patients they are treating for Fabry disease for inclusion within the study.; Patient/Caregiver inclusion/exclusion criteria include; • Diagnosed with Fabry disease / caring for a patient diagnosed with Fabry disease; • Patient received Fabrazyme in the home infusion setting in the past 12 months; • Not a current or ex-employee of Sanofi
• Age groups:
  Adolescents (12 to < 18 years)

  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:
  52

Study design details

• Main study objective:
  The overall objective of this study was to assess the effectiveness of the home infusion educational materials from both the healthcare provider and the patient / caregiver perspectives. Effectiveness was measured through questions on knowledge and understanding of key content areas. Implementation of the logbook around the key content areas was also used to assess effectiveness of the materials.; The primary objectives were to determine:; - The level of knowledge and understanding regarding key content areas of the educational materials including: evaluation of home infusion (HCP) / elements to address when training the patient / caregiver / actions to be taken in case of an adverse event.; - Implementation of the logbook (owned by the patient but reviewed by the HCP).
• Outcomes:
  • The distribution and usage of the materials.; • The level of understanding regarding:; - Requirement and organisation for Fabrazyme home infusion,; - Preparation and administration of Fabrazyme home infusion,; - Safety reporting and actions to be taken in case of an adverse event.; • Opinions on how to improve the materials.; • Implementation of the logbook (owned by the patient but reviewed by the HCP).
• Data analysis plan:
  The analysis will be cross-sectional and descriptive. Categorical data will be summarized by counts and percentages. Continuous data will be summarized using number, median, minimum and maximum values. Missing data will be noted for each variable.

Documents

Study results

rdctEUPASS-AGALSC08994 Study Report Abstract-Final PDFA version-29APR2020