TB, from my recall, someone asked if the Rare Paediatric Disease...

TB, from my recall, someone asked if the Rare Paediatric Disease Priority Review Voucher program was something that PYC might benefit from. After all, PYC is developing a therapy for rare inherited retinal disease and, typically, these diseases begin to impact in childhood. It was agreed that the program could be relevant to PYC and could be highly valuable.

The program is something that is also relevant to NEU, which I also hold, so I’ve patched together the following information from previous postings on that forum.

Under the FDA's Rare Paediatric Disease Priority Review Voucher program, which came into operation in its present form in 2014, a drug sponsor may request a priority review voucher from the FDA when submitting an NDA for a rare paediatric disease indication. If the FDA agrees to grant the request, the drug sponsor receives a priority review voucher if its drug receives marketing approval.

The priority review voucher can be redeemed by the drug sponsor to obtain priority review for a subsequent marketing application for a different drug. “Priority review” means that the FDA will review the drug within 6 months instead of the standard 10 months. Alternatively, the voucher can be sold to the highest bidder.

PRVs have been sold for as much as $350 million (4 years ago). Three PRVs have been sold during the past year, each of which has returned between $80 and $105 million.

Although rare paediatric PRVs don’t expire, the voucher program itself is due to expire in October 2020. After that date, only drugs already designated as rare paediatric treatments and which receive marketing approval by October 2022 can receive a voucher.

My thinking is that, unless the program is extended, PYC would be unlikely to make the cut-off date for the program.

https://www.rdmag.com/article/2019/02/understanding-fdas-priority-review-voucher-system