Posted by Michael Wonder on 11 Apr 2018
Breakthrough-therapy designation — an FDA perspective
12 April 2018 - Recent medical advances, such as targeted therapies for specific tumour genomic defects, checkpoint inhibitors for cancers, and direct-acting antiviral agents for hepatitis C, have led to increasing numbers of potentially important candidate drugs for patients with serious or life-threatening diseases.
These advances challenge the FDA to prioritise and focus resources on the most promising products in the pipeline that target diseases for which no satisfactory treatment options exist. Fast-track and breakthrough-therapy designations are intended to do just that.
The added focus, along with greater interactions with FDA staff, may help accelerate development but does not alter the standard for approval — the requirement to provide substantial evidence of effectiveness.
