Legislative Advocacy

Legislative Advocacy                                

Bold research needs bold policy. At the Foundation to Fight H-ABC/TUBB4A, we are committed to advocating for legislation that advances science, improves access, and protects the futures of children living with rare diseases like H-ABC (Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum).

Our work on Capitol Hill ensures that rare voices are heard and that families like ours are represented in decisions that shape access to research, treatment, and care.

🧬 Renewal of the Rare Pediatric Disease Priority Review Voucher (PRV) Program

The PRV Program incentivizes pharmaceutical companies to develop treatments for rare pediatric diseases by awarding vouchers for priority FDA review. These vouchers can be used or sold, making them a powerful driver of innovation in rare disease drug development.

Why it matters:
Without renewal, the PRV program will expire—removing one of the few incentives that exist for drug companies to invest in ultra-rare diseases like H-ABC.

We support bipartisan efforts to permanently extend and strengthen the PRV program.

👉 LINK Rare Disease Congressional Caucus

🏥 Accelerated Kids Access to Care Act (AKAC)

This bipartisan bill ensures that children with complex medical needs—especially those on Medicaid—can receive care across state lines without bureaucratic delays.

Why it matters:
Many H-ABC families must travel to out-of-state specialists. But current Medicaid rules make this difficult. The AKAC Act removes unnecessary red tape and helps families get the care they need, where they need it.

👉 LINK Treating Children Without Delays

⚖️ Give Kids a Chance Act

Too often, children are excluded from clinical trials—especially combination therapy trials that are already available to adults.

The Give Kids a Chance Act would give the FDA authority to require companies to study combinations of cancer and rare disease drugs in children when adult data already exists.

Why it matters:
This legislation opens the door for pediatric patients with life-threatening illnesses to access advanced therapies faster, ethically, and based on medical need—not market size.

👉 LINK Priority Review Voucher Program

👶 Expanding Newborn Screening for Rare Disorders

Early diagnosis of leukodystrophies—including H-ABC—can be life-changing. But most rare diseases are not currently part of standard newborn screening panels.

We support initiatives to:

  • Expand the Recommended Uniform Screening Panel (RUSP)

  • Fund newborn screening infrastructure and technology

  • Encourage pilot programs for emerging rare disease tests

Why it matters:
Early detection can lead to earlier intervention, improved outcomes, and participation in clinical trials before symptoms progress.

🧪 NIH, FDA, and CDC Funding for Rare Disease Research

We advocate for strong federal funding of agencies that play a crucial role in rare disease research and drug development:

  • NIH: Funding for fundamental and translational research into conditions like H-ABC

  • FDA: Resources to speed regulatory review and modernize rare disease pathways

  • CDC: Surveillance and data collection to better understand rare conditions

Why it matters:
Public research funding fills the gap where private industry won’t. Every dollar invested in these agencies brings us closer to diagnostics, treatments, and cures.

How You Can Help

🗣️ Raise your voice
Contact your legislators. Share your family’s story. Ask them to co-sponsor these vital bills.

📧 Join our Advocacy Network
Receive action alerts and template emails so you can make your voice heard quickly and easily.

đź’Ş Be a changemaker
Every conversation with a policymaker brings us closer to progress.