OP / ED
In labs across America, scientists and researchers are making breakthroughs that could transform the treatment of rare diseases.
These projects give much-needed hope to rare disease patients around the world. Yet they may have a harder time attracting the investment they need if lawmakers let a little- known — but critical — government program expire at the end of this year, December 20, 2024.
The Rare Pediatric Disease Priority Review Voucher program isn’t headline news. It doesn’t grab the attention of cable pundits or trend on social media. But for millions of children living with rare diseases — and for the scientists who dedicate their careers to helping them — it is life-changing.
As the cofounder of HC Bioscience, I understand the impact of this program firsthand. My company is building a platform that helps cells read mutated genes and produce healthy, functional proteins without the need to alter the genome. This approach may allow us to treat a broad spectrum of rare diseases using the same therapeutic molecule.
The PPRV program helps make cutting-edge scientific research possible. But its significance goes far beyond individual research projects. It helps address the economics of rare-disease research, a consistent obstacle to progress.
The fundamental problem is that developing a single new drug can cost billions of dollars, but the market for treating any single rare disease is tiny. While collectively, more than 10,000 rare diseases affect more than 30 million Americans, each one, by definition, affects fewer than 200,000.
This imbalance creates a natural disincentive to invest in rare-disease treatments. The PPRV program addresses this issue by offering drug developers a valuable “carrot” in the form of a voucher that can be used to expedite FDA review of any future drug application. This voucher can be redeemed by the original drug developer to accelerate the approval and commercialization process or sold to another company. Both options extend a financial lifeline to developers, ensuring that beneficial rare disease research remains economically viable.
Companies of HC Bioscience’s size and stage — which conduct some of the most groundbreaking rare disease research — would find it harder to attract private funding without the PPRV program in place. Absent PPRV, many investors might reasonably conclude that the risks are simply too great.
Some might argue that our potential profits should be incentive enough. But this ignores the harsh realities of biotech research. Most would-be treatments fail in clinical trials, and for rare diseases, the risks are even higher because the patient populations are so small. The PPRV program helps mitigate these perils, allowing us to pursue vital research and clinical trials.
The program’s track record gives an idea of its impact. Since launching in 2012, it has awarded 53 vouchers for medicines to treat 39 rare pediatric diseases, benefiting more than 200,000 patients. For 36 of these diseases, there were no previously approved therapies. Each approval represents children and families whose lives have been transformed.
The program’s impact reverberates far beyond rare diseases, fostering innovation that advances all of medicine. Rare disease research often unlocks new ways to treat more common conditions, creating a ripple effect that benefits millions. A single rare disease treatment might lead to an entirely new class of therapies with applications for many patient populations.
Lawmakers’ end-of-year decision on the PPRV program will undoubtedly impact our ability to keep pursuing groundbreaking treatments that meet unmet medical needs. Just as importantly, it will influence whether we, as a society, can offer every child with a rare condition a fighting chance.
Leslie Williams is the president and chief executive officer of HC Bioscience. This originally ran in the Boston Business Journal.
