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Patient advocates retold emotional stories of lives saved, changed—or at risk—as they discussed one of the most important factors enabling breakthroughs in fighting rare diseases, the Pediatric Priority Review Voucher (PPRV), during the December Patient Advocacy Coffee Chat of the Biotechnology Innovation Organization (BIO).
“Rare disease advocates are working feverishly to get Congress to reauthorize the PPRV program before the end of the year so patients don’t get left out in the cold,” explained Karin Hoelzer, Senior Director of Patient Advocacy at BIO as she began the Coffee Chat dedicated exclusively to the PPRV program, a federal program for incentivizing rare disease research that currently cannot take new applicants until Congress reauthorizes it.
“What’s at stake is a bunch of kids’ lives, including the lives of my daughters Belle and Abby Andrews,” said Pam Crowley Andrews, Executive Director and Co-Founder of the Firefly Fund. “Without the vouchers, it’s not realistic or viable for these drugs to make their way through the regulatory process. These vouchers provide real support that is critical for drug development for the ultra rare disease population.”
The PPRV program’s authorization lapsed a year ago, but advocates hope Congress will pass reauthorization before year’s end.
“We are at an exciting inflection point, where hopefully we can see this incredibly impactful and important program reauthorized, possibly in the next couple of days,” said Karin Hoelzer, BIO Senior Director, Patient Advocacy, and moderator of the Coffee Chat, a regular online gathering for patient advocates.
As Jamie Sullivan, Vice President of Policy at the Everylife Foundation for Rare Diseases, explained during the Coffee Chat, the Food and Drug Administration (FDA) PPRV program provides vouchers to companies that gain approval for drugs that treat rare diseases primarily impacting children. The company can use the voucher to expedite the review of their next drug candidate, or they can sell the voucher to another company. The program costs the taxpayers nothing, but it has proven to be a huge incentive for bringing out new drugs for children with life-limiting rare diseases and few if any current treatment options.
According to a new study from the National Organization for Rare Disorders (NORD), since the program’s inception in 2012, there have been 63 vouchers awarded for drugs targeting 47 different rare pediatric diseases, including many conditions that lead to death before the children reach adulthood. Before the PPRV program, only four of these 47 rare pediatric diseases had any FDA-approved treatments.
The program has been reauthorized every few years since it started in 2012, but it was allowed to expire in 2024. The House on Dec. 1 passed the Mikaela Naylon Give Kids a Chance Act, which includes reauthorization of PPRV, and advocates hope the Senate will pass its own version before the year’s end, putting the program back in action.
Similar to the patient advocacy community, BIO has made PPRV a priority, advocating for swift reauthorization with lawmakers on Capitol Hill, and producing a campaign with videos from the children who need the program to help them with their rare conditions. Through this Coffee Chat, BIO also provided a platform for patient groups to discuss strategy on urging reauthorization.
Why PPRV means hope for children with rare diseases and their families
The advocates joining the Coffee Chat represent just a few of the many rare disease communities that have seen life-changing treatments developed thanks to the program. They passionately explained what the PPRV program has done so far, and what is at stake.
Andrews created the Firefly Fund, which supports research and education into rare, neurodegenerative, genetic diseases after her daughters were diagnosed with Niemann-Pick Type C. She explained Niemann-Pick Type C is a genetic disease that affects the body’s ability to transport lipids and can affect different organs, including the liver, spleen, and brain.
“In terms of treatments, there are now two therapies FDA approved and available to our patient community and we are anxiously awaiting regulatory approval of two additional compounds” said Crowley Andrews, adding that at least two of these therapies received vouchers from the PPRV program. “Vouchers provide a lot of assistance for further research as well as the commercialization portion of drug development.”
Ron Bartek, president of the Friedreich’s Ataxia Research Alliance, said he co-founded the organization after his stepson Keith was diagnosed with Friedreich’s Ataxia (FA). He told of a meeting a few years ago with the CEO of a company developing a treatment for FA. He said the PPRV program was essential in enabling the company to include children in the initial clinical trial—because the devastating, irreversible muscle damage caused by FA occurs at that age.
“I told him about the rare pediatric disease priority review vouchers that could bring his small biotech that was struggling for cash a couple hundred million dollars if it worked out. That convinced him and ultimately he made the decision in the protocol to include children as young as 16-year-olds.”
As Bartek explained, it can be impossible for companies to begin a drug development program to address rare pediatric diseases without the PPRV. “It’s all about the incentive that’s needed. There aren’t other powerful incentives,” and with the limited number of potential patients, it’s hard to attract investment in rare disease treatments, he said.
The Muscular Dystrophy Association serves people with rare neuromuscular diseases, which are genetic and progressive and therefore often cause the most damage during childhood. Once the muscle is lost, it cannot be regained, so time is of the essence. The PPRV program is extremely important to the muscular dystrophy community, explained Paul Melmeyer, Executive Vice President for Public Policy and Advocacy, at the Muscular Dystrophy Association.
“Thankfully this program has been extremely effective in neuromuscular diseases,” he said. He noted there have been seven vouchers awarded for treatments for Duchenne Muscular Dystrophy, three for treatment of spinal muscular atrophy, one for treatments for Friedrich’s Ataxia, and one for TK2D (Thymidine Kinase 2 Deficiency), a rare mitochondrial disease causing progressive myopathy.
“Prior to the program’s existence, there were zero approved therapies for any of these conditions,” Melmeyer said.
The small patient populations for rare disease therapies mean it is difficult for companies to invest in developing new drugs or using existing drugs as new treatments for these conditions without the PPRV, he added.
“There are a lot of companies that had to potentially press pause on considering pursuing that additional pediatric indication or considering going into that other neuromuscular pediatric disease,” Melmeyer said.
Sullivan of the EveryLife Foundation for Rare Diseases, recalled meeting with several small and mid-size companies at various stages of their drug development journey and said that all of them urgently need the PPRV program.
“The way the voucher matters to them is a little different in every case, but the fact remains that it is the single most important thing making it possible for them to continue those programs,” she said. “We asked each person what happens to this program if Congress doesn’t act in time, and nearly without exception, they said those programs will be abandoned.”
Pushing Senate action
Panelists discussed advocacy for the PPRV reauthorization legislation, which has passed the House and is currently under consideration in the Senate.
As Sullivan explained, there is a chance that the bill could be put to a full Senate vote before the holiday break. She said the most likely scenario is a “hotline” vote, a fast-track legislative process allowing Senators to pass legislation by unanimous consent—as long as every senator agrees. For this reason, she urged patient advocates to reach out to Senate leaders, HELP Committee leaders, and bill sponsors.
“I think by and large, our message remains one of urgency and getting this across the finish line,” Sullivan said. “Really just encouraging leaders to make it happen, to step up and take action and make this possible before the end of the year.”
