FDA Designations For Rare Disease Products: A Complete Guide

By Marissa Berry, PhD, RAC

The US FDA offers sponsors a variety of special designation programs to incentivize them to develop and deliver therapies to treat unmet patient needs, such as Fast Track Designation, Breakthrough Therapy Designation, and Qualified Infectious Disease Product Designation. In this paper, we will highlight those designation programs available specifically for products with rare disease indications: their criteria, timelines, maintenance, and benefits.

We’ll answer questions like:

• How can sponsors determine if they qualify for ODD, RPDD, or HUD designation?
• What are the timelines for submission, approval, and maintenance of rare disease designations?
• What information does a request for one of these designations typically include?
• What incentives does the FDA offer to sponsors who receive a rare disease designation?