Posted by Michael Wonder on 31 Mar 2020
Expanded access as a source of real‐world data: an overview of FDA and EMA approvals
22 March 2020 - The authors set out to identify, characterise, and compare all FDA and EMA approvals that included real‐world data on efficacy from expanded access programs.
They conducted a cross‐sectional study of FDA (1955‐2018) and EMA (1995‐2018) regulatory approval documentation. They automated searching for terms related to expanded access in 22,506 documents using machine learning techniques. They included all approvals where expanded access terms appeared in the regulatory documentation.
The main outcome was the inclusion of expanded access data as evidence of clinical efficacy. Characterisation was based on approval date, disease area, orphan designation and whether the evidence was supportive or pivotal.
