1. Home
  2. Insights
  3. Therapeutics insights
  4. Rare and orphan diseases
  5. Leveraging historical data for use in rare disease trials

Leveraging historical data for use in rare disease trials

Understanding the Bayesian and Frequentist approaches

Whitepaper

There are two primary ways to analyse historical data - the Bayesian approach and the Frequentist approach. Please login or register to access all premium content and additional website features.

Register

Both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have shown a willingness to accept studies with historical controls. There are two primary ways to analyse historical data - the Bayesian approach and the Frequentist approach. This paper aims to:

  • Increase awareness among drug developers of these two approaches
  • Provide an overview on how to use Frequentist and Bayesian methods to incorporate historical data into the study design
  • Outline the differences between the two approaches
  • Review recent developments in the regulatory landscape
  • Demonstrate how this approach has been used by the US FDA and by ICON clients