Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Mar;14(2):345-349.
doi: 10.1177/1932296819832661. Epub 2019 Mar 12.

The New FDA Real-World Evidence Program to Support Development of Drugs and Biologics

David C Klonoff  1
Affiliations

The New FDA Real-World Evidence Program to Support Development of Drugs and Biologics

David C Klonoff. J Diabetes Sci Technol. 2020 Mar.

Abstract

FDA has launched a Real World Evidence (RWE) Program for using real-world evidence (RWE) to help support new indications for already approved drugs or biologics and postapproval studies. The plan also includes stakeholder engagement efforts, demonstration projects, leadership activities, and development of guidance documents to assist developers interested in using real-world data (RWD) to develop RWE to support FDA regulatory decisions. This plan was mandated by the Cures Act passed in 2016. Over the 24-month period from passage of the law until FDA officially announced their program, FDA has gone to considerable efforts to educate the public about the benefits of RWE and encourage researchers to consider situations where RWE trials can generate useful information. Through a variety of stakeholder engagement projects, including publication of articles in medical journals, participation in public meetings, and development of initiatives, FDA has put more effort into preparing the medical community for its new emphasis on RWE than any other new policy that I can recall.

Keywords: FDA; observational; pragmatic; randomized controlled trial; real-world evidence.

PubMed Disclaimer

Conflict of interest statement

Declaration of Conflicting Interests: The author declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: DCK is a consultant to EOFlow, Lifecare, Merck, Novo, Roche, and Voluntis.

References

    1. US Food and Drug Administration. Food & Drug administration work plan and proposed funding allocations of FDA innovation account. Updated June 6, 2017. Available at: https://www.fda.gov/downloads/RegulatoryInformation/LawsEnforcedbyFDA/Si.... Accessed January 30, 2019.
    1. US Food and Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s new strategic framework to advance use of real-world evidence to support development of drugs and biologics. Updated December 6, 2018. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627760.htm. Accessed January 30, 2019.
    1. US Food and Drug Administration. Framework for FDA’s real world evidence program. Updated December 2018. Available at: https://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RealWorldEvi.... Accessed January 30, 2019.
    1. Blonde L, Khunti K, Harris SB, et al. Interpretation and impact of real-world clinical data for the practicing clinician. Adv Ther. 2018;35:1763-1774. - PMC - PubMed
    1. US Food and Drug Administration. Real world evidence. Updated December 19, 2018. January 4, 2019. Available at: https://www.fda.gov/scienceresearch/specialtopics/realworldevidence/defa.... Accessed January 30, 2019.

Publication types

MeSH terms

Substances