A long-term non-interventional study to assess the incidence of skin malignancies in patients with dystrophic and junctional epidermolysis bullosa receiving treatment with Filsuvez (FOSTER)

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  FILSUVEZ GEL
• Name of medicine, other:
  Oleogel-S10 ; Birch bark extract
• Anatomical Therapeutic Chemical (ATC) code:
  (D03AX13) Betulae cortex

  Betulae cortex
• Medical condition to be studied:
  Epidermolysis bullosa

Population studied

• Short description of the study population:
  Patients with either dystrophic epidermolysis bullosa (DEB) or junctional epidermolysis bullosa (JEB) who are considered at risk for developing skin malignancies.
• Age groups:
  All

  Paediatric Population (< 18 years)

  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  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:
  580

Study design details

• Study design:
  Post-authorization safety study (PASS) with a non-interventional cohort design, based on primary data collection from patients enrolled at participating dermatology sites and secondary use of registry data from patients included in EB registries.
• Main study objective:
  To estimate the incidence of first skin malignancy during follow-up in patients exposed to Filsuvez.
• Setting:
  Patients who are receiving standard of care from their physician or who are enrolled in EB registries will be followed for the occurrence of skin malignancies from the date of study enrollment to the date of discontinuation or end of the study. Follow-up will be for approximately 5 years.; ; Patients with a confirmed diagnosis of either DEB or JEB are eligible. There are no exclusion criteria. The incidence of skin malignancies will be assessed both in patients receiving Filsuvez and those not receiving it.
• Comparators:
  Patients will receive whatever treatment their doctor is prescribing for them, whether Filsuvez or something else. Out of the planned total of 580 patients, it is expected that approximately half will be using Filsuvez.
• Outcomes:
  The primary outcome measure is incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez. Patients will be followed for the occurrence of skin malignancies from the date of study enrolment until either the date of discontinuation or termination of the study. The main secondary outcome measures is the incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez over this same time period.
• Data analysis plan:
  This study is purely descriptive, and will not include any statistical testing of outcomes between Filsuvez-exposed and unexposed patients. Demographic, clinical characteristics, Filsuvez use patterns, and characteristics of skin malignancies will be included in the descriptive analyses of the Filsuvez-exposed and unexposed patients. Continuous variables will be described using mean, standard deviation, median, first and third quartiles, interquartile range, range, as appropriate. Categorical variables will be described with counts and percentages (relative to the number of non-missing observations for each variable).