Alexandre Mejat

Bridging the translational gap in drug repurposing for rare diseases: clinical success versus regulatory conversion.

Repurposing of drugs to treat rare diseases (RDs) is vital, but the translational gap between clinical evidence and regulatory approval remains unquantified. Analysis of 47 clinical trials showed that 66 % of completed studies met their primary or key secondary endpoints. Nevertheless, only 39 % achieved marketing auth...