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Regulatory News | 03 October 2014 | By Alexander Gaffney, RAC
For the first time, the US Food and Drug Administration (FDA) is establishing the fees required for a company to use a Rare Pediatric Disease Priority Review Voucher, a new incentive intended to spur the development of products for rare pediatric diseases.
FDA currently maintains two priority review voucher systems: one known for tropical diseases and the other for pediatric rare diseases.
The tropical disease priority review voucher system was established under the FDA Amendment Act of 2007 (FDAAA). Under the system, companies that receive approval for a tropical disease treatment are eligible to receive a transferrable voucher that allows the bearer to receive priority review status for any future product. Products undergoing priority review are generally given an approval decision—positive or negative—within six months (instead of the usual 10 months) after the applicant's filing date.
But the tropical disease voucher system hasn't been used much, in part because it's somewhat restrictive. As FDA explains in its guidance document on the voucher, "The sponsor redeeming the voucher must notify FDA of their intent to submit a human drug application with a priority review voucher at least 365 days prior to submission of the human drug application for which a priority review voucher will be used to obtain a priority review." That advance review time makes it impractical for many companies, which may not be willing to purchase a voucher until they know their product will be able to be submitted to FDA.
And just as important, some drugs simply won't benefit from a faster review from FDA. For example, if a drug has outstanding questions that must be answered, the voucher might simply facilitate a quicker complete response letter (CRL).
And in addition to the purchase price of the voucher from another company, users of the vouchers must also pay FDA to use them. Because these reviews cost more than traditional drug reviews, the cost per application is more. However, under the voucher program, any company willing to pay the additional cost of the review may have its product reviewed by FDA. As of FY2015, each recipient of a tropical priority review voucher must pay $2,562,000 to use it.
FDA has also recently established a second priority review voucher system: the rare pediatric disease priority review voucher program.
While the vouchers are conceptually and relatively similar to their tropical counterparts, they have a key difference that could make them far more valuable for sponsors. Unlike the 365-day waiting period to use a tropical disease voucher, a pediatric rare disease voucher can be used just 90 days after notifying FDA of a company's intent to use it (FDASIA, Section 529).
The first rare pediatric disease voucher was given to the pharmaceutical company BioMarin in February 2014 after its rare disease drug Vimizim was granted FDA approval. While FDA is still in the process of writing a guidance on the pediatric voucher program, the program, which was established under the 2012 FDA Safety and Innovation Act (FDASIA), contains a notable improvement over the tropical disease voucher program.
The voucher appears to already be considerably more valuable than its tropical counterpart, which has yet to be used successfully. On 30 July 2014, BioMarin announced that it had sold its voucher to Sanofi and Regeneron for $67.5 million.
The "novel bet" made by Sanofi and Regeneron is that the faster review time for their prospective blockbuster PCSK9 cholesterol drug, alirocumab, will pay off in increased revenue through accelerated market access—assuming the drug is approved in six months. Whether it is will mean the difference between a "bet" that pays off and an expensive blunder that adds tens of millions to the cost of the drug's development.
Now Sanofi and Regeneron have a new piece of information: How much the voucher will cost to use.
In a 1 October 2014 Federal Register announcement, FDA said it had set the user fee for the rare pediatric disease priority review voucher at $2,562,000—exactly the same as the tropical disease priority review voucher.
The fee must be paid in addition to the standard new drug application (NDA) filing fee for drugs, which as of FY 2015 is $2,335,200 for an application with clinical data.
FDA Federal Register Notice