Non-interventional post-authorization effectiveness study to assess long-term outcomes of Nintedanib treatment in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD) (Nintedanib long-term outcomes in patients with SSc)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  NINTEDANIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX09) nintedanib

  nintedanib

Population studied

• 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:
  2000

Study design details

• Main study objective:
  The main objective of this post-authorization effectiveness study is to assess the long-term effectiveness of Ofev® on a composite outcome of time to forced vital capacity (FVC) decline, lung transplantation (indicating end-stage ILD), or mortality, in addition, to other outcomes of interest in patients with SSc-ILD using data from the EUSTAR registry.
• Outcomes:
  To assess long-term treatment effectiveness of Ofev® based on a composite outcome of time to FVC decline, time to lung transplantation, or death. ; To assess long-term treatment effectiveness of Ofev® based on FVC decline, time to lung transplantation, time to death, QoL, patterns of disease progression based on changes in FVC decline, effects of concomittant or previous therapies, safety and effectiveness profile in patients with pulmonary hypertension, and incidence rate of major bleeding, gastrointestinal perforation, and thromboembolism.
• Data analysis plan:
  This ten-year study will include a two-year pilot analysis followed by analyses updated biennially. ; Descriptive statistics will be used to describe patient characteristics at baseline, primary and secondary outcomes. Univariate and bivariate distributions of exposures, outcomes, and relevant covariates will be summarized using frequencies and percentages for categorical variables and summary measures for continuous variables. Comparative analyses, if feasible, will be conducted using a Cox proportional hazards regression model with time-varying exposure to estimate HRs and 95% CIs. ; Ofev® treated patients will be propensity score matched to Ofev non-users to balance variables that are strongly prognostic for the outcomes. ; All primary and secondary outcomes will be analyzed, if feasible, using the aforementioned descriptive and comparative methods for the SSc-ILD sub-population with PH. Descriptive analyses of the safety outcomes will also be conducted by treatment group.