A target trial emulation with an extension to subgroups: an example for relapsing-remitting multiple sclerosis

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Method development or testing
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  TECFIDERA
• Study drug International non-proprietary name (INN) or common name:
  DIMETHYL FUMARATE

  GLATIRAMER ACETATE
• Anatomical Therapeutic Chemical (ATC) code:
  (L03AX13) glatiramer acetate

  glatiramer acetate

  (L04AX07) dimethyl fumarate

  dimethyl fumarate
• Medical condition to be studied:
  Relapsing-remitting multiple sclerosis

  Secondary progressive multiple sclerosis

Population studied

• Short description of the study population:
  Swedish multiple sclerosis patients registered in SMSreg.
• 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:
  500

Study design details

• Study design:
  Target trial emulation following a strict and pragmatic scenario (Retrospective new user active comparator cohort study).
• Main study objective:
  To compare the effect estimates from the strict scenario with those obtained from the pragmatic scenario to identify the effect estimates of subpopulations neglected in the strict scenario and in the CONFIRM trial.
• Setting:
  Swedish multiple sclerosis patients registered in SMSreg initiating either dimethyl fumarate or glatiramer acetate.
• Comparators:
  Glatiramer acetate (active comparator).
• Outcomes:
  Time to first confirmed relapse.; Annualized relapse rate after two years.