FDA Drowning in Orphan Drug Applications
Wednesday, October 19, 2016
The various incentives meant to encourage the development of drugs for rare diseases have created an avalanche of products that the US Food and Drug Administration (FDA) is struggling to keep up with, said agency officials.
This comes in tandem with more, and ever more sophisticated, overtures to the agency from patient advocates who have rare diseases, who have been encouraged both by law and by the FDA to take a greater role in the development of products for their conditions.
It is a double-edged sword, said FDA officials at the National Organization for Rare Disorders (NORD) Breakthrough Summit held here. Orphan drugs are being approved at a record level, but the agency is not equipped to handle the rising tide of submissions, they said.