FDA introduces new approval pathway for ultra-rare disease therapies
The FDA just rolled out a fresh approval pathway for ultra-rare disease therapies, called the "plausible mechanism" pathway.
Announced on Monday, it's designed for cases where traditional clinical trials aren't realistic because there are so few patients.
This is the first major update in this area in decades.
The new pathway allows for faster access to treatments
Instead of waiting for big trial results, this pathway lets scientists show their therapy targets the genetic or molecular cause of a disease—think CRISPR gene editing or RNA-based drugs.
If the science checks out and they can prove they've hit the right target, that could be enough to get these personalized treatments approved.
A revolutionary advance in regulatory science
Vinay Prasad from the FDA called this a "revolutionary advance in regulatory science."
For patients with no current treatments, this could mean real hope—and much faster access to life-changing medicines.