Time flies. In July 2019 the FDA released revised draft guidance describing their thinking on the Rare Pediatric Disease Priority Review Voucher program, originally set forth in Section 529 of the FD&C Act and revised by the Advancing Hope Act of 2016.
These revisions include the following considerations:
- Defining the pediatric population as from birth to 18 (rather than birth to 16)
- A revised interpretation of the definition of “rare pediatric disease” to read “the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years”
- Confirming the disease or condition must be one that affects less than 200,000 persons in the US.
- Incorporates a rare pediatric disease PRV program sunset clause
- 30 SEPTEMBER 2020 – deadline for RPD designation
- 30 SEPTEMBER 2022 – deadline for drug approval
- And lastly, outlines additional PRV eligibility requirements and process.
Let’s consider the fourth point above, the deadline.
The guidance states: “After 30 September 2020, FDA MAY only award a voucher if the drug has rare pediatric disease designation and that designation was granted by 30 September 2020. After September 30, 2022, FDA may not award any rare pediatric disease priority review vouchers”, to be safe, consider a sunset outlined in a guidance as a deadline.
It is now 04 March 2020. Depending on when you submit your RPD designation application the FDA review could take anywhere from 60 days when submitting alone (or with a Fast Track) or 90(+) days if submitted with your Orphan Drug Designation request. Let’s call the agency review time 100 days; working backwards from 30 September 2020, this means, if a RPD PRV is an objective of your program, you need to submit the RPD designation request NO LATER than MONDAY, 22 JUNE 2020.
Again, depending on how you plan to submit your designation (with an ODD or Fast Track, or thereafter), the process of preparing the application for submission typically takes 4-8 weeks, possibly longer depending on your resources and internal review cycles. If we are being conservative, let’s say 8 weeks. Finally, this means you should be initiating this entire process MONDAY, 27 APRIL 2020, if not earlier (which is NOW).
