Drug Development

As politicians debate the priority review voucher program, the FDA is rooting for its demise

Sarepta’s regulatory win on eteplirsen this week came with a big bonus. The approval delivered a rare pediatric priority review voucher that the company now plans to sell — potentially for hundreds of millions of dollars. And if the FDA’s internal war over an OK had gone on longer, the biotech may have missed that big score.

Congress will likely have until the end of the year now to decide the fate of the priority review voucher program at the FDA, moving past a September 30 deadline if a compromise extension built into a continuing resolution bill on government funding is passed, as RAPS’ Regulatory Focus Managing Editor Zachary Brennan explains in a wonderfully detailed review of the situation. Congress, though, has been wrangling over what to do. And agency officials are crossing their fingers and hoping to see the whole thing disappear.

The program has been very effective for the companies landing these PRVs, and the biopharma outfits which have been buying them for as much as $350 million. Armed with a PRV, the big companies can hotwire the drug review process, forcing a quicker decision on drugs that they’re anxious to get out onto the market.

The FDA, though, has hated it. In a GAO review out last spring, agency officials made it abundantly clear that there was no evidence that the PRV program does what it’s designed to do: Ramp up more development efforts for rare pediatric diseases. And the program ties their hands, forcing them to give an application top priority, even if it doesn’t warrant urgent attention.

From the GAO report:

“They expressed concern that the program adversely affects the agency’s ability to set its public health priorities by requiring FDA to provide priority reviews of new drug applications that would not otherwise qualify if they do not treat a serious condition or provide a significant improvement in safety or effectiveness. Additionally, FDA officials said that the additional workload from the program strains the agency’s resources.”

Whether now or later, the FDA will still want to see this program killed off.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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