FDA: Drug Companies Must Pay $2.5M To Use Pediatric Disease Priority Review Voucher

The Regulatory Affairs Professionals Society (RAPS) reports that the FDA has set the fee drug companies must pay to use a priority review voucher at $2.5 million. This fee will be charged over and above the usual new drug application fee of $2.3 million and will reduce the turn-around time for an FDA decision from 10 months to 6 months.

The voucher program, implemented in 2012 as part of the FDA Safety and Innovation Act (FDASIA), provides incentive for drug companies researching rare and currently untreatable pediatric diseases. The function of the program is similar to another voucher system established in 2007 to accelerate research of tropical disease, with one key difference.

Any company which submits and application and receives approval for a tropical disease treatment outlined by the 2007 incentive receives a transferable voucher, which they may use to expedite a future product. If the company chooses to use the voucher, according to the guidance, they must provide a year’s notice to the FDA and pay an additional application fee.

Raps explains that this time constraint makes the voucher difficult to use, as most companies cannot predict which compounds will be ready for review that far in advance.

The pediatric priority review voucher (PPRV), however, only requires 90 days notice, making it a far more attractive option to drug developers, especially because the vouchers can be sold. The company which receives the voucher may choose to sell it to another company for a profit.

BioMarin was the first drug company to receive a PPRV for the approval of their drug Vimizim, a biological product indicated for the treatment of Mucopolysaccharidosis type IVA, also known as “Morquio A syndrome.”

This disease meets the criteria outlined by the FDA because it is diagnosed in a population fewer than 200,000 and is most prevalent in children under the age of eighteen. Treatment research for these diseases does not have a market large enough to recoup the company’s investment funding.   The FDA incentive is meant to counter-balance that loss.

BioMarin announced in July its decision to sell its voucher to Regeneron for $67.5 million.

Jean-Jacques Bienaime, CEO of BioMarin, said that the decision was in the best interests of BioMarin’s goals. He said, “Leveraging the sale of the Priority Review Voucher to reinvest in products to treat rare and ultra-rare diseases makes the most sense for BioMarin.”

A few days after the deal was struck, Regeneron announced its plans to use the PRV for their new cholesterol-reducing biologic, Alirocumab.

In order for the move to be fiscally worthwhile, Regeneron must  recoup the $67 million investment plus the nearly $5 million FDA application fee in the four months following approval, which the FDA may or may not grant.

According to Ned Braunstein, VP of regulatory affairs at Regeneron, his company’s investment is proof that the program works. “Our decision to acquire and leverage the voucher is clear evidence that it is a valuable incentive for biopharmaceutical companies.”