An Active Surveillance, Post-Authorization Study to Assess Tofacitinib Utilization Patterns and to Characterize the Safety of Tofacitinib Use in Patients with Moderately to Severely Active Ulcerative Colitis in the Real-World Setting Using Data from a US Administrative Healthcare Claims Database

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Active surveillance

Study drug and medical condition

• Name of medicine:
  XELJANZ
• Study drug International non-proprietary name (INN) or common name:
  TOFACITINIB CITRATE

  TOFACITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AF01) tofacitinib

  tofacitinib
• Medical condition to be studied:
  Colitis ulcerative

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  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:
  11857

Study design details

• Main study objective:
  The main objectives are to describe the utilization patterns of tofacitinib in the US with regard to on-label and off-label use, and to estimate the incidence rate of malignancy, excluding non-melanoma skin cancer (NMSC), among adult UC patients who initiate tofacitinib in the course of routine clinical care.
• Outcomes:
  1. Indication for tofacitinib use, classified as on-label, off-label, or unknown, based on medical events (diagnoses and/or medication use) identifiable around the time of the first ever tofacitinib prescription and,; 2. Malignancy, excluding non-melanoma skin cancer in adult UC patients exposed to tofacitinib in the course of routine clinical care, NMSC, Serious infections, Opportunistic infections (e.g. tuberculosis), Herpes zoster (HZ) reactivation, Major adverse cardiac events (MACE), Venous thromboembolic events (VTE, deep venous thrombosis DVT and pulmonary embolism PE), Hepatic events, Progressive multifocal leukoencephalopathy (PML), Gastrointestinal (GI) perforations, Interstitial lung disease (ILD), Surgery for UC, Death
• Data analysis plan:
  For the drug utilization study, proportions of patients with an on-label, off-label, or unknown tofacitinib indication will be estimated (and described) with corresponding 95% confidence intervals (CIs). For the safety endpoints of interest, descriptive statistics, counts and proportions, cumulative incidence proportions, and incidence rates (number of events per 100 person-years) and associated 2-sided 95% CIs will be calculated as appropriate. Patients with a baseline history of an outcome of interest will be excluded from the calculation of the incidence rate for that particular outcome of interest (e.g. patients with a baseline history of malignancy will be excluded from the calculation of the incidence rate for malignancy). This will be a time to first event analysis based on an index date defined for each cohort with appropriate censoring rules applied (based on therapy switches, end of study, etc.) for those who do not experience an event by end of follow-up period.