Expanded access as a source of real-world data An overview of FDA and EMA approvals.

08:00 EDT 22nd March 2020 | BioPortfolio

Summary of "Expanded access as a source of real-world data An overview of FDA and EMA approvals."

To identify, characterize, and compare all FDA and EMA approvals that included real-world data on efficacy from expanded access programs
Cross-sectional study of FDA (1955-2018) and EMA (1995-2018) regulatory approval documentation. We automated searching for terms related to expanded access in 22,506 documents using machine learning techniques. We included all approvals where expanded access terms appeared in the regulatory documentation. Our main outcome was the inclusion of expanded access data as evidence of clinical efficacy. Characterization was based on approval date, disease area, orphan designation and whether the evidence was supportive or pivotal.


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This article was published in the following journal.

Name: British journal of clinical pharmacology
ISSN: 1365-2125


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Reproduction of data in a new location or other destination, leaving the source data unchanged, although the physical form of the result may differ from that of the source.

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