Rare Pediatric Disease Priority Voucher – What Will You Pay for Speed?

Today, the FDA announced the 2015 fee for the use of a Rare Pediatric Disease Voucher in a Federal Register (FR) notice (here). Created by the FDA Safety and Innovation Act (FDASIA), this program provides a voucher to a sponsor that gains approval for an NDA for a product used to treat an indication that has been classified as a rare pediatric disease.  That sponsor may use the voucher to gain priority (6 month) review of any NDA that it subsequently submits, or the sponsor may transfer the voucher (including by means of sale) to another sponsor to use.  There is a price for use of the voucher and it ain’t cheap-the voucher fee for 2015 is fixed at $2,562,000.

The voucher fee (which, by the way, is in addition to the usual PDUFA fee) is calculated by determining the difference in the average cost of a standard NDA and the average cost associated with a priority review NDA.  The FR notice cited to above provides some interesting insight into those costs.  FDA estimates that the cost for a standard NDA review (10 month review time) is $5,122,000 which is calculated from data that Agency has gathered for 2013 and its past experience (note the data for 2014 is not yet available because FY 2014 is not closed out, but FDA must publish the FY 2015 fee data prior to October 1, 2014).  It should be noted that this estimate assumes the inclusion of clinical data in the application.  FDA admits that it does not collect cost data on priority review applications but, based on the time difference, the FDA puts a premium on those costs by taking a ratio of the 10 month review time of a standard NDA to the 6 month review time for a priority review NDA (10/6=1.67) and then multiplies that by the standard review costs to generate the priority review costs. When factoring in Biologic Licensing Application (BLA) review costs (which are slightly lower) and using the multiplier, the cost for a priority review application was found to be $6.256, 000.  The difference is then a simple subtraction of the priority review costs and the standard review costs, which is $2,510,000.  That difference is then subject to an adjustment based on the increased costs incurred by FDA over the last three years (inflation, salary and benefit/cost increases, etc.) and that brings us to the $2,562,000 figure.

When one adds the cost of the PDUFA fee for FY 2015 with clinical studies ($2,335,200) to the Pediatric Rare Disease Voucher cost, you get a bill for a new NDA for priority review of $4,897,200.  And that is if you own the voucher; if you buy the voucher it could be considerable more costly (one recently sold for $67.5 million (see here). This figure represents the cost of speeding your drug onto the market!  Is it worth 4 months?  Only you can decide!