The Rare Pediatric Disease PRV, the Difference in the PRVs, and the Future of the Program
The Rare Pediatric Disease Priority Review Voucher
(If you haven’t read Part 1 yet, start there first!)
A new Priority Review Voucher (PRV) has been introduced that could be a game changer with respect to the program. Nancy Goodman of Kids v Cancer, a childhood cancer research advocacy group, was the brainchild of the “Creating Hope Act” which sought to incentivize research for Rare Pediatric Diseases (RPD) using the Neglected Tropical Disease (NTD) PRV blueprint. The Creating Hope act was co-opted into the Food and Drug Administration Safety and Innovation Act (FDASIA) which was signed in to law by President Obama on July 9th 2012. §908 of FDASIA created §529 in the Federal Food, Drug, and Cosmetic Act entitled “Priority Review to Encourage Treatments for Rare Pediatric Diseases”. The program is new enough that §529 is not written into the online copy of the FD&C Act that the Agency provides. There have been no RPD PRVs awarded and guidance for the voucher is currently being written. One thing is for sure, the pediatric PRV differs significantly from the tropical disease PRV.
The RPD PRV legislation was written with the criticisms and concerns surrounding the original PRV in mind. There are a number of substantive differences that were put in place designed to make the program more functional and attractive to both developers of drugs that qualify for the voucher and the eventual end user of the voucher. Those differences are bulleted below:
- The original PRV required a 1 year advanced notice to FDA for prior to redemption of the voucher. For the RPD PRV the notice time was shortened to 90 days.
- The User Fees were significantly reduced. In the case of Ilaris the additional user fee to redeem the voucher was $4.6M for priority review, the 2012 figure was $5.6M had someone used a voucher. The additional user fees for the RPD PRVs have been revised down substantially, the announced 2013 rate for the RPD PRV is $3.6M
- There is a debate with regards to the number of times that the original PRV can be transferred, some believe the resale is capped at one transaction. A clarification was made in the regulations for the RPD PRV, that an unlimited number of transfers are allowed.
- The RPD PRV has a revocation provision. §529(d) states that if the rare pediatric disease product is not marketed within 365 days of approval that the awarded voucher can be revoked
- The RPD PRV has been instituted on a trial basis. Three vouchers will be awarded for this program, after the issuance of the third voucher the program the Secretary will report on the program to congress.
An important point that detractors of the PRV program miss is that it has only been in effect since 2007. The structure of the program requires approval of a drug before a voucher can be issued. Given the timelines for drug development it is not surprising that there has only been one PRV issued to date. In fact, those who have looked at this suggest that, for the NTD PRV alone, there are about 24 drugs in development that are eligible for the PRV. Of those 24 there are five vaccines and three small molecules in Phase 3 development. Add to this the fact that a second, and in many ways more attractive PRV program has been initiated, it is a safe bet that several vouchers will be issued in coming years. The questions that remain unanswered are what free market value these vouchers will bring, if NTD and RPD vouchers will be valued differently, and what the ripple effect of multiple priority reviews, presumably for potential blockbuster drugs, will have on industry.