Rare disease research incentives - Understanding the issues
Research dollars for small populations are lacking for obvious reasons - insufficient financial rewards, regulatory obstacles, etc. - so regulators have enacted alternative incentives to drive this still-important research.
By Mike Dobbyn
Research dollars for small populations are lacking for obvious reasons - insufficient financial rewards, regulatory obstacles, etc. - so regulators have enacted alternative incentives to drive this still-important research.
By Mike Dobbyn
What Is the PPRV—and Why Should We Care?
The Pediatric Priority Review Voucher (PPRV) is an FDA program created in 2012 that rewards companies for developing treatments for rare pediatric diseases. When a company succeeds, they get a voucher that can speed up FDA review of another drug—or be sold for millions of dollars.
Why does this matter? Because it creates a real financial incentive for companies to take on diseases like Sanfilippo Syndrome, where the patient population is tiny and the return on investment is uncertain. Without this kind of motivation, many therapies simply wouldn’t be pursued.
This represents the critical issue in the rare disease world; and it's why the Congress approved the "orphan drug" designation for drug application process.
The Role of Orphan Drug Designation
The Orphan Drug Act was created and signed into law in the United States in 1983. It was designed to encourage the development of drugs for rare diseases (those affecting fewer than 200,000 people in the U.S.). Most experimental treatments for rare diseases receive Orphan Drug designation, which offers:
Combined with the PPRV, these incentives help small biotech companies move forward with research that would otherwise be too risky or expensive to pursue. However, with the expiration of PPRVs, drug developers now lack one of their few remaining tools to make pediatric rare disease treatments financially viable.
But the Give Kids a Chance Act of 2025 aims to change and reverse that.
What Is the Give Kids a Chance Act?
The Give Kids a Chance Act of 2025 is bipartisan legislation that aims to preserve/revive and strengthen the PPRV program, which expired at the end of 2024. Its aim is to ensure that the program continues to support rare pediatric drug development—without adding new costs to taxpayers. As of July 2025, the bill is pending in Congress, not yet law—though its passage is considered critical by rare disease advocates. It’s a small policy with a massive impact for families like ours who are waiting for clinical trials and treatments to move forward.
Unfortunately many of those trials do not move forward for any number of reasons...
A Wake-Up Call: The Recent FDA Delay for Sanfilippo Type A
Just recently, families were devastated to learn that a long-awaited clinical trial for Sanfilippo Syndrome Type A was delayed by at least six months. The FDA cited the need to reinspect the manufacturer’s facilities. For families living with a degenerative condition, every month lost is a month of irreversible decline. This isn’t just about policy—it’s personal. It reminds us that time, transparency, and urgency matter. And it underscores why we need well-structured, well-supported programs like the PPRV to keep the pipeline moving.
What Comes Next: Post-BBB Outlook
Following the passage of the Big Beautiful Bill (BBB), there’s renewed focus on FDA reforms, drug pricing, and accountability. While it’s early, here’s what we expect:
In the coming months, as BBB provisions take shape, advocates will be pushing to ensure rare pediatric diseases remain a priority—not an afterthought.
What You Can Do
Final Thoughts
For our children, the disease they face is not rare; we live with it every day. But we cannot expect others to fund research into these rare diseases purely out of a sense of human charity. Business doesn’t work that way - every cost must be associated with an opportunity. Incentive programs like the PPRV and Give Kids A Chance Act have many flaws, but they also generate an outsized benefit for disease communities like ours.
We cannot and will not be complacent about policy changes that affect our families. At The Sanfilippo Project, we’ll continue exploring these urgent issues and challenges —speaking with experts, families, and advocates who are on the front lines of rare disease policy and drug development. We certainly don’t have all the answers, but we will continue to do whatever we can to help families like ours make sense of this rapidly changing landscape. As policies shift and pressure builds, we hope to be a trusted voice connecting the dots and sharing what matters most—for our children, for our future, and for the fight we all share.
Helpful Resources
The Pediatric Priority Review Voucher (PPRV) is an FDA program created in 2012 that rewards companies for developing treatments for rare pediatric diseases. When a company succeeds, they get a voucher that can speed up FDA review of another drug—or be sold for millions of dollars.
Why does this matter? Because it creates a real financial incentive for companies to take on diseases like Sanfilippo Syndrome, where the patient population is tiny and the return on investment is uncertain. Without this kind of motivation, many therapies simply wouldn’t be pursued.
This represents the critical issue in the rare disease world; and it's why the Congress approved the "orphan drug" designation for drug application process.
The Role of Orphan Drug Designation
The Orphan Drug Act was created and signed into law in the United States in 1983. It was designed to encourage the development of drugs for rare diseases (those affecting fewer than 200,000 people in the U.S.). Most experimental treatments for rare diseases receive Orphan Drug designation, which offers:
- Tax credits for clinical trial costs
- Seven years of market exclusivity after FDA approval
- Waived FDA fees
Combined with the PPRV, these incentives help small biotech companies move forward with research that would otherwise be too risky or expensive to pursue. However, with the expiration of PPRVs, drug developers now lack one of their few remaining tools to make pediatric rare disease treatments financially viable.
But the Give Kids a Chance Act of 2025 aims to change and reverse that.
What Is the Give Kids a Chance Act?
The Give Kids a Chance Act of 2025 is bipartisan legislation that aims to preserve/revive and strengthen the PPRV program, which expired at the end of 2024. Its aim is to ensure that the program continues to support rare pediatric drug development—without adding new costs to taxpayers. As of July 2025, the bill is pending in Congress, not yet law—though its passage is considered critical by rare disease advocates. It’s a small policy with a massive impact for families like ours who are waiting for clinical trials and treatments to move forward.
Unfortunately many of those trials do not move forward for any number of reasons...
A Wake-Up Call: The Recent FDA Delay for Sanfilippo Type A
Just recently, families were devastated to learn that a long-awaited clinical trial for Sanfilippo Syndrome Type A was delayed by at least six months. The FDA cited the need to reinspect the manufacturer’s facilities. For families living with a degenerative condition, every month lost is a month of irreversible decline. This isn’t just about policy—it’s personal. It reminds us that time, transparency, and urgency matter. And it underscores why we need well-structured, well-supported programs like the PPRV to keep the pipeline moving.
What Comes Next: Post-BBB Outlook
Following the passage of the Big Beautiful Bill (BBB), there’s renewed focus on FDA reforms, drug pricing, and accountability. While it’s early, here’s what we expect:
- Closer review of programs like PPRV to ensure they’re delivering real benefits
- Potential new rules around how voucher sales are reported and used
- A chance to strengthen protections for diseases that might otherwise be ignored
In the coming months, as BBB provisions take shape, advocates will be pushing to ensure rare pediatric diseases remain a priority—not an afterthought.
What You Can Do
- Contact Congress and ask for support of the Give Kids a Chance Act (GKaCA)
- Tell your story—especially if you’ve experienced delays or barriers to treatment
- Support foundations funding Sanfilippo research
- Stay connected—we are stronger together, and our voices matter
Final Thoughts
For our children, the disease they face is not rare; we live with it every day. But we cannot expect others to fund research into these rare diseases purely out of a sense of human charity. Business doesn’t work that way - every cost must be associated with an opportunity. Incentive programs like the PPRV and Give Kids A Chance Act have many flaws, but they also generate an outsized benefit for disease communities like ours.
We cannot and will not be complacent about policy changes that affect our families. At The Sanfilippo Project, we’ll continue exploring these urgent issues and challenges —speaking with experts, families, and advocates who are on the front lines of rare disease policy and drug development. We certainly don’t have all the answers, but we will continue to do whatever we can to help families like ours make sense of this rapidly changing landscape. As policies shift and pressure builds, we hope to be a trusted voice connecting the dots and sharing what matters most—for our children, for our future, and for the fight we all share.
Helpful Resources
- EveryLife Foundation: https://everylifefoundation.org
- Cure Sanfilippo Foundation: https://www.curesanfilippofoundation.org
- National MPS Society: https://mpssociety.org
- Congress.gov – Give Kids a Chance Act: https://www.congress.gov
|
Mike Dobbyn founded the Sanfilippo Project to support the children and families impacted by Sanfilippo Syndrome. By finding the courage to share our individual stories we hope to help others.
#SanfilippoProject #Learn #Share #PayItForward |
