ExPloring efficAcy, safeTy, and adHerence oF dIsease-modifyiNg antirheumatic Drugs through trajEctoRy model: the PATHFINDER study

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L04AA24) abatacept

  abatacept

  (L04AA29) tofacitinib

  tofacitinib

  (L04AA37) baricitinib

  baricitinib

  (L04AB) Tumor necrosis factor alpha (TNF-alpha) inhibitors

  Tumor necrosis factor alpha (TNF-alpha) inhibitors

  (L04AC07) tocilizumab

  tocilizumab

  (L04AC14) sarilumab

  sarilumab
• Medical condition to be studied:
  Rheumatoid arthritis

Population studied

• Age groups:
  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:
  3000

Study design details

• Main study objective:
  This research will identify patterns of utilization of rheumatoid arthritis treatments and their predictors, and it will explore the relationship of these trajectories with endpoints of effectiveness and safety.
• Outcomes:
  We will identify trajectories of utilization of bDMARD and tsDMARD over the time of follow-up (3 years). Trajectories will be identified by using trajectory model approach, patients will be classified according to their most likely pattern of adherence (i.e. the trajectory that the patient has the highest probability of belonging to). We will test several variables as predictors. a) Validate the selected RA patientsb) Investigate effectiveness over the "off treatment" and "on treatment" periodsc) Evaluate the occurrence of ED admission/hospitalization over the "off treatment" and "on treatment" periods d) Assess the occurrence of ED admission/hospitalization related to trajectories
• Data analysis plan:
  Validation analysis: sensitivity, specificity, positive and negative predictive value.Drug-utilization analyses: trajectory model approach.Effectiveness: descriptive analysis.Safety analyses: proportion and time free from events.