Definition
Rare Pediatric Disease Designation is an FDA designation for drugs intended to prevent, diagnose, or treat rare diseases that primarily affect individuals aged 18 or younger. Sponsors receive a Priority Review Voucher (PRV) upon approval, which can be used or sold.
How It Works
Sponsors request designation from FDA before approval. Upon NDA/BLA approval for the rare pediatric indication, FDA awards a transferable Priority Review Voucher.
Eligibility Criteria
- Disease primarily affects children (majority under 18)
- Affects fewer than 200,000 Americans
- Serious or life-threatening condition
- Drug provides meaningful therapeutic benefit
Program Benefits
| Benefit | Value |
|---|---|
| Priority Review Voucher | Transferable, valued $100-350M |
| Orphan Drug Overlap | Can combine with Orphan designation |
| Fast Track Eligibility | May qualify for expedited development |
| Tax Benefits | Orphan drug tax credits if applicable |
Priority Review Voucher Sales
| Year | Sale Price Range |
|---|---|
| 2014-2016 | $125-350 million |
| 2017-2019 | $80-150 million |
| 2020-2025 | $100-200 million |
Why BD Teams Track Rare Pediatric Disease
For business development professionals, PRV eligibility adds significant value:
- Deal Implication: PRV can be worth $100M+ upon approval, substantially affecting deal economics
- Due Diligence Focus: Verify pediatric prevalence data and PRV eligibility confirmation from FDA
- Opportunity Signal: Companies developing rare pediatric products may monetize PRV separately