Thousands of children’s lives have been saved by a cost-free government initiative that expired last December. Congress needs to renew this program quickly because too many babies are dying needlessly.
Housed at the Food and Drug Administration and fully funded by pharmaceutical companies at no cost to taxpayers, the rare pediatric disease priority review voucher program offers a carrot to companies developing treatments for children with rare and life-threatening diseases. Since so few kids suffer from any single illness, these drugs rarely make enough money to fund the incredibly expensive research underpinning them. So instead of expensive taxpayer-funded grants or subsidies, the government has another way to make up the shortfall. Companies that successfully bring a treatment for a rare pediatric disease to market get a voucher for a speedy FDA review that can be used for a different, unrelated drug. These vouchers can then be sold to other companies.
Since some medicines earn billions of dollars annually, these certificates can be enormously valuable to the giant pharmaceutical companies that develop blockbuster drugs to treat adult-onset illnesses. A priority review voucher recently sold for $150 million — enough to fund years of research. The vouchers are a kind of Robin Hood program in which huge conglomerates give money to tiny, cash-strapped biotechnology firms to save the world’s most vulnerable patients.
In 2015, I founded BridgeBio, one of the firms that leverages vouchers to fund rare and ultra-rare drug development. So of course I’m eager to see this program reauthorized by Congress. But here’s the thing: The benefits of the voucher program are so obvious that just about everyone supports it.
Previous congressional reauthorizations have been noncontroversial. But Washington is juggling competing priorities right now, so that even widely hailed programs with bipartisan support are falling by the wayside. I’m hoping that if a few more people write a few more letters to a few more members of Congress, the voucher program will finally get unstuck.
In 2021, BridgeBio got FDA approval for Nulibry, a drug to treat an ultra-rare genetic disease called MoCD Type A. Untreated, kids with this disease suffer seizures and brain damage soon after birth. They quickly become quadriplegic and usually die before the age of 4, an inexorable and horrifying process that parents must watch helplessly.
Only about 100 children suffer this disease worldwide — not enough to make Nulibry profitable in the usual ways. Indeed, Nulibry had been sitting undeveloped on the shelves of another company for years because executives couldn’t justify the enormous expense of the usual development and FDA review processes.
We were able to make Nulibry in large measure because we built our business model to include some programs that the PRV pushes into becoming financially viable. Since the inception of the Priority Review Voucher program in 2012, the FDA has granted 53 coupons for treatments addressing 39 different rare pediatric diseases. These medicines have collectively helped more than 200,000 children.
There are over 10,000 rare diseases affecting more than 30 million Americans with more than half being children, many of whom will not live to see their fifth birthday. Despite this crushing need, only around 5% of those 10,000+ diseases have an FDA-approved treatment largely because rare disease drug development is seen as too financially risky. The drug developer incurs tens or hundreds of millions of dollars in expenses and faces the same risks as any other drug development program. In the case of rare diseases, however, there is little opportunity within the health care system to recoup the money spent because patient populations are so small.
Even with the voucher program, only 13 out of the 201 drugs approved from 2020 through 2023 were “first-in-indication” treatments, meaning they were drugs for illnesses that were previously devoid of medicines. Such novel treatments are rare because investors would rather stick to the well-trod, predictable pathway of developing drugs for diseases that already have treatments.
The FDA’s voucher program offers exactly that kind of predictability for medicines that treat newly identified illnesses or those that affect tiny patient populations, including the kind of rare genetic conditions that kill so many babies.
Unfortunately, this vital voucher program was allowed to expire in December 2024.
Hope is forward-looking, though. The Give Kids a Chance Act of 2025 has been reintroduced in the House and the Senate, and its passage would reauthorize these coupons for another five years.
There are children alive today because of the treatments that the FDA’s priority review vouchers have made possible. But there are so many more kids out there who are looking to Congress and the biotechnology industry for some hope. There is more beautiful science out there, and more companies that want to move some of it forward.
And so, we are asking our members of Congress: Please help us to help those children. Please give those kids a chance.