An Active Surveillance, Post-Authorization Study to Characterize the Safety of Tofacitinib in Patients With Moderately to Severely Active Ulcerative Colitis in the Real-World Setting Using Data From the United Registries for Clinical Assessment and Research (UR-CARE) in the European Union (EU)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  XELJANZ
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AF01) tofacitinib

  tofacitinib
• Medical condition to be studied:
  Colitis ulcerative

Population studied

• Short description of the study population:
  The study population will include adult UC patients aged ≥18 years enrolled in UR-CARE who are initiating treatment with tofacitinib (Cohort 1) from 01 July 2018 (date of approval of tofacitinib for UC patients in the EU) through 31 March 2025.
• 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:
  500

Study design details

• Study design:
  This is a 7-year active cohort study of adult UC patients aged ≥18 years treated with tofacitinib compared to patients receiving alternative treatment or no treatment. The study will use secondary data collected in the UR-CARE platform.
• Main study objective:
  What are the incidence rates of safety events of interest in adult ulcerative colitis (UC) patients aged ?18 years treated with tofacitinib in routine clinical care, as compared to the incidence rates in UC patients treated with other approved systemic agents, and UC patients naïve to biologics and immunomodulators/immunosuppressants (hereafter referred to as immunosuppressants)?
• Setting:
  The UR-CARE platform is set up for daily care use (i.e. individual sites may upload patient information directly onto the UR-CARE platform) and contains medical records, including identifying data, of patients from multiple EU and non-EU countries.
• Comparators:
  The study will also include the following comparator cohorts: Cohort 2: UC patients who; initiate biologics, with/without concurrent immunomodulators/immunosuppressants, stratified on TNFi/non-TNFi use and number of previous biologic treatments; Cohort 3: UC patients who initiate immunomodulators/immunosuppressants without concurrent biologics; Cohort 4: UC patients naïve to both biologics and immunomodulators/immunosuppressants.
• Outcomes:
  Estimate the incidence rates of malignancy and VTE (deep venous thrombosis DVT and pulmonary embolism PE) among adult UC patients years who initiate tofacitinib in the course of routine clinical care, as well as the incidence rates in UC patients treated with other approved systemic agents such as biologics and immunosuppressants, and in UC patients naïve to biologics and immunosuppressants, Estimate incidence rates of other safety events among adult UC patients who initiate tofacitinib in the course of routine clinical care, in UC patients treated with other approved systemic agents, and in comparator cohorts. Estimate incidence rates of primary and secondary safety events of interest stratified by tofacitinib dose.
• Data analysis plan:
  Baseline demographic and clinical characteristics for each cohort, including proportion of patients with ?1 VTE risk factors will be described. For all the safety events of interest, descriptive statistics, counts and proportions, unadjusted cumulative incidence proportions, and crude incidence rates (i.e. number of events per person-years) and age/sex standardized incidence rates with associated 2-sided 95% confidence intervals will be calculated as appropriate. The estimated incidence rates will be based on survival analysis of time to first event based on an index date defined for each cohort with appropriate censoring rules applied for those who do not experience an event by end of follow-up period.