Uncertainty around the pediatric priority review voucher (PRV) program

The Rare Pediatric Disease Priority Review Voucher (PRV) program has been a lightning rod within the orphan drug sector, and pending Congressional action, the program is set (at presstime) to sunset on October 1. Designed to spur innovation in the pursuit of drugs for “rare pediatric diseases,” the PRV program rewards a drug company that gains regulatory approval for such a drug with a voucher. The voucher entitles its owner to get a priority review of a subsequent drug—thereby shortening the FDA-review cycle from 10 months to 6 months. In FY 2015, FDA’s OOPD granted 21 of 31 requests for the “rare pediatric disease” designation (not all such designated drugs are eligible for the PRV program).

Since the program was created in 2012, six PRVs have been granted. Per the program rules, the drug company awarded the voucher can monetize it in one of two ways—it can either exercise the voucher itself to expedite the review of any future product (orphan or mass market) in its own pipeline, or, it can sell it to another drug developer (thereby generating what could be a huge cash influx for itself), allowing that buyer to speed the review for a new drug filing in its pipeline, orphan or otherwise. To date, sales of these vouchers have fetched purchase prices ranging from $67 million to $350 million, clearly demonstrating their perceived value in the marketplace.

Last March, the Government Accountability Office (GAO) published an assessment of the voucher program to date, and suggested that the jury’s still out on whether or not the program itself could take credit for spurring innovation in drug development for rare pediatric diseases. Within that GAO report, some FDA officials have been very outspoken against the voucher program, even calling for it to end (http://www.gao.gov/assets/680/675544.pdf). In essence, the program takes some of the discretion on how clinical reviews are prioritized at FDA out of its hands.

Not surprisingly, the National Organization of Rare Disorders (NORD) is a supporter of PRV, says Peter Saltonstall, CEO. “It’s been very effective in terms of spurring development, and has by no means been overused. And in fact, the first few vouchers granted have demonstrated every possible scenario that the program designers had envisioned.”

Robin Huff, PhD, global regulatory strategy leader at Quintiles and a member of the company’s Pediatric and Diabetes Centers of Excellence (and who spent seven years at FDA prior to joining Quintiles) agrees with Saltonstall. The first few vouchers that have been granted and sold in the open market “have demonstrated their financial value in terms of providing an economic incentive,” she says. “It’s a really interesting way to use private capital, in amounts that greatly exceed any direct government support possible, to drive biomedical innovation and focus efforts on an area of particularly high unmet need.”

“While we were disappointed that the US Senate would not vote on its Cures legislation (also known as the Senate Innovations for Healthier Americans Initiatives) before it broke for summer recess, we feel confident that the Pediatric Rare Diseases Priority Review Voucher program will be reauthorized in September, as part of the 21st Century Cures Act—it’s just a matter of what the specific language will look like,” says Saltonstall of NORD. “We’ve asked for it to be permanently reauthorized, but if Congress decides to set another sunset date, we support the Senate language that reauthorizes the program through 2022, and allows vouchers to be awarded through 2027 that had been designated before 2022.”