FDA Prepares Approval Pathway Plan for Rare Disease Treatments

The FDA plans to soon release a new approval pathway for rare disease treatments that could allow some drugmakers to bypass certain clinical trial requirements, the agency’s gene therapy chief, Vinay Prasad, said Wednesday.

“The commissioner and I will soon unveil what we call the plausible mechanism pathway,” Prasad said at a workshop on rare disease clinical trials, held by Duke-Margolis Institute for Health Policy. “This will be a novel pathway for drug developers who are truly pursuing bespoke therapy.”

A new pathway has the potential to open the door for more rare disease drug approvals. That has been a ...