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The Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PPRV) Program was top of mind during BIO’s March Patient Advocacy Coffee Chat: Show Your Stripes! The conversation brought together patient advocacy leaders and illustrated both the urgent need for incentives to support rare disease drug development and the power of patient advocacy. As BIO’s Karin Hoelzer, Senior Director of Patient Advocacy and moderator for the discussion, explained, this is a key priority for BIO and the patient advocacy community, and they have a long history of working collaboratively on the issue.
“Rare diseases, by statute, are those impacting fewer than 200,000 Americans,” explained Jamie Sullivan, Vice President of Policy, EveryLife Foundation for Rare Diseases at the top of the discussion. “However, the vast majority of rare diseases are affecting far fewer than 200,000. There are likely more than 10,000 identified rare diseases and that number is growing every day. And one of the biggest challenges we work to solve is the fact that 95% of rare diseases have no FDA approved treatment.”
The creation of the PPRV program in 2012 incentivized drug developers to focus their attention and resources on rare pediatric disease treatments, which in many respects are particularly challenging to develop. The program was vastly successful—and according to the Congressional Budget Office does not cost tax payers any money. But despite broad bipartisan support, the PPRV program’s authorization sunset last December. Rare disease advocates are working feverishly to get Congress to reauthorize the program as soon as possible so patients don’t get left out in the cold.
How the voucher program got started
Dr. David Ridley, Faculty Director for Health Sector Management at Duke University’s Fuqua School of Business and one of the lead authors of a seminal paper that paved the way for enactment of the PPRV program, explained how the program got started.
“A bunch of academics came up with the idea for the voucher program,” Dr. Ridley began. “And BIO actually played an important role in getting the word out about vouchers and how it works.”
He went on to explain how the PPRV program works, noting that there is a fair amount of confusion. “There’s two drugs for every voucher,” he said, “one drug wins the voucher, and the other drug uses the voucher. Both drugs get priority review. While one drug, for a rare pediatric disease, gets priority review because it’s a really important drug that addresses an urgent unmet need, the second drug gets priority review thanks to the voucher.”
The system, Dr. Ridley explains, allows more commercially viable drugs to work in tandem with drugs for rarer diseases so that they can both quickly reach a market that needs them.
“Vouchers have been used for diabetes, HIV, and other diseases that are economically viable and also do a lot of good,” Dr. Ridley explains. “Getting a rare disease drug to market also helps people. But the main incentive is trying to create an opportunity to get resources for these drugs to move them forward.”
Why the program matters
As both Ronald J. Bartek, Co-Founder & President, Friedreich’s Ataxia Research Alliance (FARA) and Brigid Garelik, The Chief Medical Officer of the Children’s Tumor Foundation (CTF), emphasized, developing drugs for rare pediatric diseases is in many respects particularly challenging, and incentives like the PPRV program are key. As Bartek explained, the PPRV program was instrumental in bringing the first drug for Friedreich’s ataxia to market, and in making a robust pipeline of additional treatments and potential cures economically feasible. Similarly, as Garelik explained, the CTF community now has two approved drugs which benefitted from the PPRV voucher.
And the data clearly shows how successful the PPRV program has been—as many as 60 drugs have earned a voucher to date—the vast majority for rare pediatric diseases that had absolutely no treatment options before the program’s inception.
Common misconceptions about the program debunked
“We all know that there’s initially been some pushback over the years over the vouchers and other rare orphan product incentives, thinking that maybe we’ve gone too far and over incentivized,” said Ronald J. Bartek, Co-Founder & President, Friedreich’s Ataxia Research Alliance. “Well believe me, we haven’t.” Initially, too, the FDA seemed to have some reservations about the agency’s ability to provide the expedited reviews guaranteed by the vouchers. The FDA, however, is now strongly supportive of the voucher program, seeing it as an important incentive in the concerted effort to develop safe, effective treatments for children with rare diseases as early as possible. And indeed, the program now has strong bipartisan support in Congress and from FDA.
As Victoria Gemme, Director of Policy and Regulatory Affairs, National Organization for Rare Disorder (NORD), explained there was concern about what types of drugs the vouchers were redeemed for, but when NORD researched how the PPRV program was being used, they found something surprising.
“We actually found in our study that only 3 drugs for which vouchers were redeemed ranked among the top 50 by Medicare drug spend in 2022,” she said. “Moreover, only 23 of the 53 rare disease pediatric priority review vouchers awarded by April 30, 2024 have been redeemed so far. So it’s also not quite amassing a level of burden that might have been concern about with FDA, staff time and resources.”
Jamie Sullivan, Vice President of Policy, EveryLife Foundation for Rare Diseases, echoed this sentiment. She pointed out that the cornerstone of much of the critique was a result of an outdated Government Accountability Office (GAO) study that claimed that the PPRV programs had little or no effect on drug development—a claim that Sullivan deemed way too premature.
“As with any incentive for therapy development, it takes time to work,” she said. “It takes time to influence decisions made 10 years before you would see a drug approval. And so the importance of keeping this data up to date and continuing to tell the story that evolves every year can’t be overstated. People seized on that 2019 GAO study to say, Look, the program is not really having the intended effect, but it was way too early to say.”
An urgent argument for renewal
“Where are we at now?” Sullivan asked. “Unfortunately, we’re not listening to the data. We have an expired program, so companies would have had to get their rare pediatric disease designation by December 20th 2024 to get their approval by 9/30/2026 to still earn the voucher.“
Sullivan lamented the fact that despite the PPRV program’s potential, and strong engagement from the patient community, it may ultimately die on the vine. “We got so close,” she said. But her frustration is not an indication of defeat—far from it. In fact, advocates are more motivated than ever to see the renewal of this program in the new legislature.
And time is ticking. “It might seem like there’s really no harm in waiting,” said Ridley, “but there is harm because it’s creating uncertainty. And this means that people aren’t getting resources, and then it means that children are going to have to wait longer to get important medicines.”
This frustration is compounded by the fact that great headway had been made last year to build support for the program, only to fizzle out and put advocates back at square one.
“Last year, the House passed unanimously a bill called the Give Kids a Chance Act, which packaged up a number of pediatric rare disease therapies as well as the RARE Act and a couple other issues, and it included a five-year reauthorization of the priority review voucher program that passed unanimously in the house,” explained Sullivan. “Unfortunately, in the Senate, they didn’t get to put that package through regular order and through a hearing in the health committee. But there was clearly bipartisan support, and it was present in the bipartisan health care policy package that was set originally to be passed along with the end of year funding legislation on December 20.”
That package, unfortunately, was dropped at the end of last year, meaning that the voucher program expired, and advocates had to start over with the new Congress.
Regardless, advocates are continuing to pound the drum of rare disease drug development in hopes that incentive programs like the PPRV program will be protected. And they understand that to be most successful, they have to work together.
“One thing that I’ve learned over the years is having everybody at the table early on in development is key,” said Garelik. As Sullivan explained, the EveryLife Foundation published a toolkit with extensive advocacy materials. And Gemme shared the link to the NORD action alert that allows advocates to reach out directly to their elected official to express support for the health package that includes PPRV reauthorization.
“We’ve got a long way to go but we’re confident in our path. We’ll get there together,” concluded Bartek.
