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

ABSTRACT:

Aims

To identify, characterize and compare all Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals that included real-world data on efficacy from expanded access (EA) programmes.

Methods

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

Results

EA terms appeared in 693 out of 22,506 (3.1%) documents, which

SUBMITTER: Polak TB 

PROVIDER: S-EPMC7444779 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature