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Rare Diseases: Pediatric Priority Review Voucher Incentive Program

August 20, 2013

Special Note: This is the second of a series of Blog Posts on rare diseases and pediatrics. By request, a previous Blog Post is republished with updates.

The FDA Safety & Innovation Act (S. 3187), or FDASIA , is signed by President Obama in July 2012. The new legislation impacts the future of rare diseases and orphan drug development. Section 908 of FDASIA is directly intended to encourage innovation in pediatric rare disease: a new priority review voucher program to reward sponsors of pediatric rare diseases (Reference Section 908, pages 102 – 106, using the above link to FDASIA).

The FDA Priority Review Voucher (PRV) program, which became law in 2007, was intended as an incentive for companies to invest in new drugs and treatments for Neglected Tropical Diseases (NTD). The NTD PRV entitles the sponsor to a priority review for a product of their choice or can be sold to another developer. The NTD PRV speeds approval of the drug, giving patients quicker access. But by speeding up the drug review process for a NTD PRV, there is the possibility of slowing the review process for other drugs. The FDA aims to give a decision in 6 months.

What is interesting about the NTD PRV, is that there is a debate as to the number of times it can be transferred. According to Duke University’s website on PRVs, the law says that “the sponsor of a NTD product that receives a priority review PRV under this section may transfer (including by sale) the entitlement to such voucher.”

There is also controversy as to whether the NTD voucher program has been effective. According to a February 2013 article by Cato Research, “so far only one PRV has been awarded. It was issued with the approval of Novartis’ anti-malarial drug Coartem in 2009 ….. Novartis did not sell the PRV, instead they kept the voucher in-house and redeemed it for the review of Ilaris, a drug they were developing for gouty arthritis. The Agency determined that Llaris was not approvable for the indication Novartis was seeking. The review was completed in 6 months.”

FDASIA’s Section 908, “Rare Pediatric Disease Priority Review Voucher Incentive Program”, extends the existing NTD PRV program to Rare Pediatric Diseases (RPDs) on a trial basis – with 3 vouchers to be awarded for this program. After the third RPD PRV, the FDA will report on the program to Congress. The following are several of the changes to the existing NTD PRV program for RPD PRVs:

•   The pediatric treatment developer can ask the FDA in advance for an indication of whether the disease qualifies as a RPD
•   RPD PRV can be transferred an unlimited # of times, whereas with the NTD PVR, transferring is not clearly stated
•   RPD PRV user notifies the FDA 90 days prior to using the voucher, whereas with the NTD PVR, notification must be 1 year before
•   2013 rate for RPD PRV is $3.6 million; the 2012 fee was $5.6 million
•   RPD PRV user risks having the voucher revoked if the treatment is not marketed within a year
•   RPD PRV user has to report to the FDA about the use of the pediatric treatment within 5 years of approval.

According to Section 908 of FDASIA, a sponsoring company can apply for a RPD PRV :

•   If the drug is for the prevention or treatment of a RPD & contains no active ingredient that has been previously approved in any other application
•   FDA says drug is eligible for priority review
•   Drug’s clinical data are from studies with a pediatric population & drug dosages for that population
•   Doesn’t apply for approval for an adult indication in the original RPD application
•   Drug approved after date of FDASIA enactment.


Arnold & Porter LLP “A Reference Guide to FDASIA” (Reference pages 27 – 29 for Section 908.

Please Note: “Stick Figure Standing On Grass” by Weedwacker (Own work) [CC-BY-SA-3.0] | Wikimedia Commons

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