Online Program

A Novel Statistical Approach for Leveraging Real-World Data in Regulatory Clinical Studies (306612)

Keywords: Real-world data, Propensity scores, Augmenting patient cohort

In medical product development, there has been an increased interest in utilizing real-world data which have become abundant owing to advances in biomedical science, information technology and engineering. High-quality real-world data may be utilized to generate real-world evidence for regulatory or healthcare decision-making. This presentation will introduce a propensity score-based approach for leveraging patients from a real-world data source for augmenting a single-arm clinical study. The proposed approach uses the propensity score methodology to identify real-world patients who are like those enrolled into the investigational clinical study in terms of baseline characteristics. Either frequentist or Bayesian method can then be applied to outcome data analysis, with the option of down-weighting information from real-world data source. An example based on pre-market regulatory review experience is provided to illustrate the implementation of the proposed approach.