The President signing off on “Advancing Hope Act of 2016,” which modifies the FDA’s priority review voucher program for rare pediatric diseases means an extension of the program through the end of the year, for now.
Even more importantly, this means that between now and December 31st, Congress will examine and deliberate ways to amend the program and extend it past 2016.
The sign off also clarifies one of the more controversial aspects of the bill – the definition of “rare pediatric diseases.”
Previous definition: “The disease primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
New definition: “The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
More about the program:
This priority review voucher program which is meant to further incentivize the development of novel therapies to treat rare pediatric diseases has been a huge success and it has been our hope to see this extended.
To date there have been six vouchers awarded under this specific program.
Looking to obtain a PRV?
Well, you can either buy one from a company who has already been awarded one for $350M, OR you can develop a novel therapy for a rare pediatric disease. If the latter, you would apply for a Rare Pediatric Disease Designation which qualifies you for a PRV.
In order to apply, you can request the designation for a Rare Pediatric Disease Priority Review Voucher from the Office of Orphan Products Development (OOPD) and you can do so in your earlier development stages.
The criteria for this program are as follows:
1. Your therapy must show medical plausibility to treat a disease which ““The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
And,
2. Be a rare disease according to the ODA of 1983 where the prevalence of the disease falls under 200,000 persons or fewer.
Once the voucher is awarded (or purchased!) you can decide to use it to have one of your drugs reviewed under FDA’s priority review system or you can sell (or re-sell) it to an interested party for a LOT of money. The largest purchase price thus far was $350M (AbbVie in August 2015).
The incentive for small companies has really been the ability to sell the voucher for a tremendous amount of money, which can be used to continue developing novel orphan therapies. The motivation for big pharma to obtain or acquire these has been to use the benefit of a priority review to beat competitors to the market, gaining market share and name recognition (amongst other benefits, all equating to huge financial incentives).
Read more about the extension here.
