Examining the Use of Real-World Evidence in the Regulatory Process

Clin Pharmacol Ther. 2020 Apr;107(4):843-852. doi: 10.1002/cpt.1658. Epub 2019 Nov 14.


The 21st Century Cures Act passed by the United States Congress mandates the US Food and Drug Administration to develop guidance to evaluate the use of real-world evidence (RWE) to support the regulatory process. RWE has generated important medical discoveries, especially in areas where traditional clinical trials would be unethical or infeasible. However, RWE suffers from several issues that hinder its ability to provide proof of treatment efficacy at a level comparable to randomized controlled trials. In this review article, we summarized the advantages and limitations of RWE, identified the key opportunities for RWE, and pointed the way forward to maximize the potential of RWE for regulatory purposes.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic / legislation & jurisprudence*
  • Clinical Trials as Topic / methods
  • Clinical Trials as Topic / statistics & numerical data
  • Decision Making
  • Evidence-Based Medicine / legislation & jurisprudence*
  • Evidence-Based Medicine / methods
  • Evidence-Based Medicine / statistics & numerical data
  • Humans
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence*