Examining the Use of Real‐World Evidence in the Regulatory Process. Issue 4 (14th November 2019)

• Record Type: Journal Article
• Authors: Beaulieu‐Jones, Brett K.; Finlayson, Samuel G.; Yuan, William; Altman, Russ B.; Kohane, Isaac S.; Prasad, Vinay; Yu, Kun‐Hsing
• Is Part Of: Clinical pharmacology & therapeutics. Volume 107:Issue 4(2020)
• Page Start: 843
• Page End: 852
• Publication Date: 2019-11-14
• Subjects: Pharmacology -- Periodicals; Therapeutics -- Periodicals; 615.5
• DOI: 10.1002/cpt.1658
• Languages: English
• ISSNs: 0009-9236

Abstract

Abstract : 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.