Another Chance to Make Priority Review Vouchers Work
This article was originally published in RPM Report
One of the “innovation” pieces of the FDA Safety & Innovation Act is a priority review voucher program for drugs to treat pediatric rare diseases. The new PRV is designed to avoid some of the glitches that have complicated the value of the program in tropical diseases—but it can’t avoid the core problem inherent in trying to capture the value of a priority review by legislative fiat.
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FDA is subject to Congressional oversight from many different committees in the House and Senate. But Homeland Security isn’t usually one of them. Maybe that’s why some of the testimony felt about 15 years out of date.
FDA says that Morquio A syndrome qualifies as a rare pediatric disease because most patients with a severe form of the disease will not survive into adulthood, paving the way for BioMarin to receive the first rare pediatric disease priority review voucher.
Regeneron and its partner Sanofi have become the first biopharma companies to buy a Priority Review Voucher. The $67 million price is a nice bonus for the seller, BioMarin, but probably not the start of a big new market for PRVs.