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Regulatory News | 25 September 2015 | By Zachary Brennan
The US Food and Drug Administration (FDA) on Friday announced that it would increase the rare pediatric disease priority review voucher fee rate for FY 2016 by about $200,000.
The new rate -- $2,727,000 – is effective on 1 October and will remain in effect for the next year. FDA previously set the fee rates at $2,562,000 for FY 2015 and $2,325,000 for FY 2014. The new rate is based on FDA's estimate that the cost of a standard review for new molecular entity (NME) new drug applications (NDAs) and biologic license applications (BLAs) will be about $3,989,000, while the cost of a priority review for NME NDAs and BLAs is about $6,662,000.
The setting of the new rate seems to be an indication that FDA does not expect the rare pediatric disease priority review program to end in March 2016.
Currently, the Food and Drug Administration Safety and Innovation Act (FDASIA) mandates that FDA can only award three rare pediatric disease priority vouchers each year, and the third was awarded to Asklepion Pharmaceuticals upon the approval of its rare disease drug Cholbam.
Accordingly, FDA's rare pediatric disease priority review voucher program will formally end on 17 March 2016 unless Congress takes additional action.
One of the ways the program will continue is if new legislation, known as the Advancing Hope Act of 2015, introduced on 23 March 2015 is signed into law.
That bill would make permanent the rare pediatric disease priority review voucher program in the hopes of maintaining an incentive for pediatric drug development. In addition, the bill would also modify the definition of "rare pediatric disease"—now any disease primarily affecting fewer than 200,000 children—to specifically include "any form of sickle cell disease" and "any pediatric cancers."
The rare pediatric voucher system is closely modeled off the tropical disease voucher system, both of which create incentives intended to attract the development of drugs to treat rare diseases.
The incentive is a voucher which allows its owner to have any experimental drug the company owns reviewed by FDA under its "priority review" pathway. The pathway allows for FDA to review a drug in just six months instead of the standard 10 months. FDA has committed to reviewing and acting on 90 percent of the applications granted priority review status within this expedited timeframe.
But the vouchers may also be sold to other companies. Here's a list of the status of the priority review vouchers already on the market:
Status of Existing Priority Review Vouchers
Status of Voucher
Unsuccessfully used by Novartis to accelerate the review of its Biologics Licensing Application (BLA) for Ilaris (canakinumab).
Rare Pediatric Disease Unused
Sold to Sanofi and Regeneron for $67 million. Used successfully to speed the approval of Praluent.
Sold to Gilead Sciences for $125 million. Gilead is using the voucher in support of its NDA filing for a new HIV drug.
Sold to AbbVie for $350 million in August 2015. AbbVie has not disclosed how it plans to use the voucher.
Transfered to Retrophin under an existing agreement. Sold to Sanofi for $245 million in May 2015.
Tags: priority review vouchers, rare pediatric disease, review voucher program, Advancing Hope Act of 2015, Cholbam