Post-Authorisation Safety Study (PASS) to Evaluate the Risk of Malignancies in Patients with Myasthenia Gravis (MG) Treated with Efgartigimod

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Other
• 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
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AA58) efgartigimod alfa

  efgartigimod alfa
• Medical condition to be studied:
  Myasthenia gravis

Population studied

• Short description of the study population:
  The study will identify two cohorts from each selected database:; - Efgartigimod Cohort: Patients with an MG diagnosis and who have initiated efgartigimod.; - Reference Cohort: Patients with an MG diagnosis and who have not initiated efgartigimod but are on any other therapy for the treatment of MG. Immunoglobulins and plasmapheresis are not considered here, as they are usually used for acute management of MG crisis.
• 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:
  4074

Study design details

• Study design:
  This is a non-interventional, longitudinal database cohort post authorisation safety study.
• Main study objective:
  To evaluate the long-term risk of malignancies overall and by type in patients with MG treated with efgartigimod compared to MG patients on any other MG therapy and who do not have malignancy history in the lookback period, in a real-world setting.
• Setting:
  The observational study will identify two cohorts from each targeted database:; - Efgartigimod Cohort: Patients with an MG diagnosis and who have initiated efgartigimod.; - Reference Cohort: Patients with an MG diagnosis and who have not initiated efgartigimod but are on any other therapy for the treatment of MG. Immunoglobulins and plasmapheresis are not considered here, as they are usually used for acute management of MG crisis.
• Outcomes:
  - Long-term risk of malignancies overall and by type in patients with MG treated with efgartigimod compared to MG patients on any other MG therapy and who do not have malignancy history in the lookback period, in a real-world setting.; - Long-term risk of malignancies overall and by type in MG patients with malignancy history in the lookback period.; - Long-term risk of malignancies by duration of efgartigimod exposure.; - Long-term risk of malignancies in subpopulations with increased risk for malignancy: elderly (age 65 years and older); chronic use (i.e., ≥1 year) of corticosteroids.