The Pediatric Rare Disease Voucher Program was just reinstated by an act of Congress. This gives us until September 2024 to gain Pediatric Rare Disease Designation (PRD). While this program is not as important to large drug development companies, who can simply purchase a voucher if they think it will improve their balance sheet, it is mission critical for smaller companies in the rare disease space.
Spur Investor Interest
A PRD, which is usually applied for in conjunction with an Orphan Drug Designation (ODD), spurs investor interest and is a key factor in gaining funding to support clinical trials. The time to apply for PRD/ODD is during the preclinical development stage.
Data from the therapeutic (or preventative) in an animal model of the exact disease is critical. The model should not be general, and should only apply to the indication sought. For example, a model of general cardiovascular disease would not be acceptable for a rare pediatric disease impacting the cardiovascular system. Further, the drug/biologic used in the study should be the exact same therapeutic we seek designation for. Data from drugs in the same class, even if they have similar mechanisms of action, are not sufficient.
Lastly, there are some important regulatory aspects to observe even after the PRD designation is obtained. PRD vouchers are only valid in new chemical entities (or biologics), so the first approved designation needs to include a pediatric population.
The experienced team at KLA Breakthrough Consulting is happy to extend a 30-minute educational analysis regarding your unique situation. Please give us a call at (951) 294-7739, or email me personally at kellyaustin@KLA_BC.com for more information on what designations you can qualify for!