Responder definition

Background

• When a new patient-reported outcome (PRO) is developed, there is interest in deriving the following, to support interpretation of data collected using the new tool:
  • Minimal Clinically Important Difference (MCID) / Responder Definition (RD)
  • Meaningful Between-Group Difference
  • Meaningful Score Categories
• These are usually derived by using an existing, validated measure as the ‘gold standard’ to quantify e.g. disease status

Data

• Simulated dataset: 1000 subjects, 2 timepoints (Baseline, Week 16)
• Treatment allocation is not important and not provided
• At each time point, data is provided for two tools:
  • PGIS – the “gold standard” – graded on a 4-point scale (‘None’, ‘Mild’, ‘Moderate’, ‘Severe’)
  • ‘NEWTOOL’ – the new measure, scored on an 11-point numeric rating scale from 0 to 10

The data set can be found here.

Challenge

Use visualisations to support in the derivation in any or all of the following:

• MCID
• Meaningful between-group difference
• Meaningful score categories