A non-interventional, post-authorisation safety study of patients treated with efgartigimod alfa

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Other

  Safety study (incl. comparative)
• If ‘other’, further details on the scope of the study:
  Post-Authorization Safety Study (PASS)
• 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

• Medicinal product name:
  VYVGART
• Study drug International non-proprietary name (INN) or common name:
  EFGARTIGIMOD ALFA
• Medical condition to be studied:
  Myasthenia gravis

Population studied

• 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:
  680

Study design details

• Study design:
  This is a non-interventional, prospective, post authorisation safety study.
• Main study objective:
  A non-interventional 10-year Post-Authorization Safety Study (PASS) in a post-marketing setting comparing cohorts of patients with Generalised Myasthenia Gravis (gMG) treated with efgartigimod to those treated with other Myasthenia Gravis (MG) medication, in order to estimate the incidence rates of serious infection and other safety events.
• Setting:
  Patients with gMG who are expected to start treatment with efgartigimod at enrolment or are within their first cycle of efgartigimod at enrolment will be eligible to enrol into the efgartigimod cohort. Patients with gMG who have not been exposed to efgartigimod and for whom it is not planned to start treatment with efgartigimod at enrolment will be eligible to enrol into the non-efgartigimod cohort.
• Outcomes:
  To evaluate the overall long-term safety of efgartigimod including the occurrence of serious infections in gMG patients treated with efgartigimod compared to gMG patients not exposed to efgartigimod.
• Data analysis plan:
  Categorical endpoints will be summarised using counts and percent; continuous endpoints will be summarised using mean, standard deviation, median, minimum and maximum values. Statistical analysis will be mainly descriptive in nature using metrics such as incidence proportion, exposure adjusted incidence rate, confidence intervals, etc. Details of the analysis plan will be specified in the Statistical Analysis Plan (SAP).