Characteristics of IPF patients initiating nintedanib, pirfenidone or no antifibrotic treatment in the US

Study type

• Study topic:
  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Idiopathic pulmonary fibrosis

Population studied

• Short description of the study population:
  US population receiving healthcare in the ambulatory setting.; Patients were included in one of three study cohorts based on prescription history from October 1, 2014 to April 30, 2018, allowing for a 12-month pre-index period to describe patient demographic, clinical and treatment characteristics.
• Age groups:
  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  13300

Study design details

• Main study objective:
  To understand characteristics of IPF patients treated with nintedanib and pirfenidone, as well as patients who do not receive a prescription for an antifibrotic treatment in the United States
• Outcomes:
  The primary outcome will be the ASD and p-value comparing baseline patient characteristics between patients treated with nintedanib vs. pirfenidone, nintedanib vs. untreated and pirfenidone vs. untreated, The secondary outcomes are the adjusted odds ratios of receiving one treatment over another (or probability of receiving antifibrotic treatment) between the following cohorts:• Nintedanib vs. pirfenidone• Treated patients (i.e. nintedanib or pirfenidone) vs. untreated
• Data analysis plan:
  For the primary objective, baseline patient characteristics will be described using descriptive analysis, i.e. described as counts and percentages for categorical variables and measures of central tendency (mean, median, SD, and min/max) for continuous variables. Differences between the cohorts will be assessed using ASD, where an ASD of at least 10% will be considered a meaningful difference. Patients with missing data will be reported as missing and excluded from the comparison for the missing variable. Bivariate statistical significance tests such as Chi-square test for categorical variables and Wilcoxon rank sum test or t-test for continuous variables (depending on distribution of data) will be performed to compare differences in baseline characteristics between the three cohorts. In addition to standardized differences, p-values will be reported. A multi-variable logistic regression model will be constructed for the secondary objective.

Documents

Study, other information

study_1199-0375_ct.gov_form