Many rare disease drugs rely on just one trial for approval, FDA authors find
New research published by FDA authors found that 67% of non-oncologic rare disease marketing applications approved between 2020 and 2023 used only one clinical trial plus confirmatory evidence to demonstrate effectiveness. The law defines "substantial ...
New research published by FDA authors found that 67% of non-oncologic rare disease marketing applications approved between 2020 and 2023 used only one clinical trial plus confirmatory evidence to demonstrate effectiveness.
The law defines "substantial ...
