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 Oct;29(10):1336-1340.
doi: 10.1002/pds.5005. Epub 2020 Apr 16.

Assessing strength of evidence for regulatory decision making in licensing: What proof do we need for observational studies of effectiveness?

Jim Slattery  1 , Xavier Kurz  1
Affiliations

Assessing strength of evidence for regulatory decision making in licensing: What proof do we need for observational studies of effectiveness?

Jim Slattery et al. Pharmacoepidemiol Drug Saf. 2020 Oct.

Abstract

Before a medicine can be recommended for a marketing authorization research must be provided to regulators that convincingly supports the benefit-risk of the product in the claimed indication. The established criteria for such research are usually expressed in terms of evidence from randomized controlled trials (RCT). If studies in real-world data (RWD) are to be accepted as all or part of the package of evidence, it is necessary to understand the relationship between information from studies of RWD and that from RCTs. The aim of this review is to consider how the strength of such evidence can be quantified in a manner that relates to the decision-making process, what research is currently available to further this understanding and what additional information will be required.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest.

References

    1. Beaulieu‐Jones BK, Finlayson SG, Yuan W, et al. Examining the use of real‐world evidence in the regulatory process. Clin Pharmacol Ther. 2019;107:843‐852. 10.1002/cpt.1658. - DOI - PMC - PubMed
    1. Neyman J, Pearson E. On the problem of the most efficient tests of statistical hypotheses. Philos T R Soc A. 1933;231(694–706):289‐337.
    1. Wald A. Statistical Decision Functions. New York: Wiley; 1950.
    1. Accessdata.fda.gov . CFR—Code of Federal Regulations Title 21 [online]; 2020. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfCFR/CFRSearch.cfm?f.... Accessed February 24, 2020.
    1. Chow SC, Shao J, Wang H. Sample Size Calculation in Clinical Research. 2nd ed. New York: Chapman and Hall; 2008.

MeSH terms