A Post-Authorisation Safety Study of the Utilisation and Prescribing Patterns of Xeljanz® (tofacitinib) Using an Administrative Healthcare Database in France

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

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

Population studied

• Short description of the study population:
  The study population will be sourced from the French Système National des Données de Santé (SNDS) database. ; SNDS contains healthcare utilisation data, including pharmacy data, with a coverage of 99% of the population in France. ; The study population will consist of patients of all ages who newly initiated tofacitinib in French routine clinical setting.
• Age groups:
  • Adult and elderly population (≥18 years)

Study design details

• Study design:
  This is a retrospective cohort study to provide real-world evidence on the utilisation of tofacitinib in France.
• Main study objective:
  1. Describe the characteristics of patients treated with tofacitinib and by indication. ; 2. Evaluate prescribers’ adherence to the tofacitinib aRMMs for treating patients with RA, PsA and UC, specifically:; • Adherence to the recommended posology per indication (average daily dose [ADD]) and duration of use;; • Adherence to recommendations for patient screening and laboratory monitoring prior to and during tofacitinib treatment; ; • Adherence to recommendations for limitations of use, including:; • Contraindicated use; ; • Use with medications not compatible with tofacitinib.; 3. Describe changes in the utilisation of tofacitinib following the updated recommendations and limitations for use implemented after the 2019 Article 20 referral and the 2021 signal evaluation procedure, specifically:; • Use in patients with risk factors for VTE;; • Use in patients aged 65 years and older;; • Use in patients with risk factors for CV;; • Use in patients with risk factors for malignancy.; 4. Describe changes in the utilisation of tofacitinib following the updated recommendations and limitations for use implemented after the JAKi 2022/2023 Article 20 referral, specifically:; • UC maintenance treatment dosage for patients with CV and malignancy risk factors, in addition to VTE risk factors.;
• Setting:
  The study population will be sourced from the French SNDS database. SNDS contains healthcare utilisation data, including pharmacy data, with a coverage of 99% of the population in France. ; The study population will consist of patients of all ages who newly initiated tofacitinib in French routine clinical setting.
• Outcomes:
  Detailed in protocol section 9.3.1 and Table 3
• Data analysis plan:
  Descriptive statistics will be provided for all results. ; Categorical variables will be reported using frequency distributions. ; Ordinal variables will be reported using frequency distributions, means, standard deviations (SDs), minimums, 25th percentiles, medians, 75th percentiles and maximums, unless otherwise specified. ; Continuous variables will be reported using means, SDs, minimums, 25th percentiles, medians, 75th percentiles and maximums, unless otherwise specified. ; When relevant, 95% confidence intervals (CIs) will be calculated for the study outcomes outlined in protocol Section 9.3.1 (Outcomes) and Table 3. ; All analyses will be stratified by the approved indication or indication groups (“RA or PsA” and “UC”), unless otherwise specified. ; In addition, for the objective of “Describe prescribing patterns over time”, comparative statistical analyses will be conducted to describe changes in the use of tofacitinib across reporting periods to assess the statistical significance of reductions in the use of tofacitinib among patients with risk factors. P-value <0.05 indicates statistical significance.; ; Missing values will be reported as missing and no imputation will be undertaken. ; Results will be summarised in tables and figures in Microsoft® Excel and/or Word format.; ; 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 in the protocol; any major modifications of primary endpoint definitions or their analyses would be reflected in a protocol amendment.;