An evaluation of early use patterns to assess the effectiveness of Xeljanz® (tofacitinib citrate) in rheumatoid arthritis: A retrospective non-interventional database study of observational data embedded within Optimising Patient outcome in Australian RheumatoLogy - Quality Use of Medicines Initiative (OPAL-QUMI) (OPal study of xeljanz Effectiveness in RA - OPERA)

Study type

• Study topic:
  Human medicinal product

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

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

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Retrospective study

Study drug and medical condition

• Name of medicine:
  XELJANZ
• Medical condition to be studied:
  Rheumatoid arthritis

Population studied

• Short description of the study population:
  Patients aged 18 years or older diagnosed with rheumatoid arthritis (RA), received treatment with tofacitinib or a bDMARD and have at least 1 year of follow-up identified from the OPAL registry.
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with rheumatoid arthritis
• Estimated number of subjects:
  3000

Study design details

• Main study objective:
  To understand the patterns of treatment, clinical effectiveness, patient-reported outcomes and treatment adherence among Australian adult patients with RA treated with tofacitinib. Similar data will also be collected for patients treated with bDMARDs to provide descriptive information about clinical management of RA in real-world Australian clinical practice.
• Outcomes:
  (1) To describe tofacitinib treatment patterns among Australian adult patients with RA. (2) To assess the clinical effectiveness of tofacitinib, as defined by disease severity markers and percentage of patients reaching targeted treatment goals, in Australia. (3) To assess patient reported outcomes and treatment adherence in Australian adult patients with RA who are prescribed tofacitinib. (1) To describe bDMARD treatment patterns among Australian adult patients with RA. (2) To assess patient reported outcomes and treatment adherence in Australian adult patients with RA who are prescribed bDMARDs. (3) To describe the safety profile of Australian adult patients with RA who have been prescribed tofacitinib.
• Data analysis plan:
  Patients meeting the inclusion and exclusion criteria described above will be categorised into one of two mutually exclusive drug cohorts, based on the type of DMARD received (tofacitinib or bDMARDs). All continuous variables will be summarised using n (non-missing sample size), mean, standard deviation, median, minimum and maximum. The frequency and percentages (based on the non-missing sample size) or observed levels will be reported for all categorical measures. Descriptive summaries will be produced for each data cut, providing there is sufficient data available, and again at the final analysis. All summaries are descriptive and there are no comparative analyses being undertaken, therefore, no adjustments for multiple data cuts and multiple endpoints are required. Patients who discontinue their index treatment (tofacitinib or bDMARD) will continue to be followed for a period of 1 year.

Documents

Study results

A3921292 Non-Interventional Study Report Abstract 23 August 2021_Redacted

Study report

A3921292 Non Interventional Study Report 07 June 2021_Redacted