An Active Safety Surveillance Study to Estimate Incidence Rates of Safety Events of Interest among Patients Treated with Tofacitinib for Polyarticular Course Juvenile Idiopathic Arthritis (pcJIA) within the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry in the United States

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  XELJANZ
• Additional medical condition(s):
  polyarticular course juvenile idiopathic arthritis

Population studied

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

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  1

Study design details

• Main study objective:
  To estimate the rates of safety events of interest among pcJIA patients treated with tofacitinib and among pcJIA patients treated with bDMARDs in the postapproval setting.
• Outcomes:
  The primary objective is to estimate the incidence rate of thrombosis,infections (including opportunistic infections and serious infections), all malignancies combined (excluding NMSC), NMSC, lymphoma, lung cancer, growth effects, and fractures among pcJIA patients who initiate tofacitinib post-approval as well as pcJIA patients who initiate approved bDMARDs. The secondary objective is to estimate the incidence rate of major adverse cardiovascular events (MACE) and vaccine preventable infections among pcJIA patients who initiate tofacitinib post-approval as well as pcJIA patients who initiate approved bDMARDs.
• Data analysis plan:
  The interim analysis will consist of descriptive comparisons of baseline characteristics, drug utilization data describing all tofacitinib treatment patterns, including switching patterns from tofacitinib to any other biologic agent including TNFi or nonbiologic agent used in the treatment of pcJIA and vice versa and duration of use before switch, and crude event rates by treatment cohorts. The final analysis of outcomes will provide treatment group-specific rates of events overall and in subgroups defined by baseline characteristics. Detailed methodology for summary and statistical analyses of data collected in this study will be documented in a statistical analysis plan (SAP), which will be dated, filed and maintained by the Sponsor. The SAP may modify the plans outlined here, however any major modifications of primary outcome definitions or their analyses will be reflected in a protocol amendment.