When the Feds Step Back, the States Must Step Up: Why I Drafted a State Bill for Galactosemia
By Gillian Sapia
The ground beneath rare disease families is shifting—and fast. As federal protections shrink and agencies like the FDA and CMS face restructuring, one thing is clear: we can no longer count on Washington alone. While we continue to fight for federal action—funding for NIH, FDA approval of treatments, and equitable CMS policies—we must also build a parallel path forward.
That’s why I started drafting state legislation. Because waiting on the unpredictable is not a strategy. Planning for it is.
A New Political Reality
We are witnessing a deliberate rebalancing of responsibilities—federal agencies are being downsized, programs like PASE (FDA’s Professional Affairs and Stakeholder Engagement) are quietly dismantled, and rare disease infrastructure is becoming collateral damage in a larger political agenda. Whether this shift is framed as “efficiency” or “decentralization,” the effect is the same: the most vulnerable are left behind.
In this climate, rare disease advocates must adapt. If the federal government won’t act fast enough to secure treatments, diagnostics, and coverage—we will at the state level.
Why I Wrote the Florida Galactosemia Coverage Equity Act
Florida is home to an estimated 105 individuals with classic galactosemia, an ultra-rare metabolic disorder with no FDA-approved treatment. These patients face a $7.23 million lifetime burden from preventable costs including disability services, lost earnings, therapy, special education, and uncovered medical needs.
The Florida Galactosemia Coverage Equity Act is my solution—a state bill that ensures:
Medicaid and state-regulated insurance must cover metabolic formulas (even for orally-fed children).
Rare disease therapies are covered whether or not they are FDA-approved for the specific condition, provided they are supported by clinical guidelines.
Off-label use of FDA-approved drugs is honored when prescribed for medically accepted rare disease use.
Out-of-network access is guaranteed when no in-state specialist exists.
Adult services are included, recognizing that rare diseases do not end at 18.
This bill doesn’t just serve galactosemia patients. It creates a legislative model that can be copied and scaled to other rare diseases—many of which are also neglected due to small patient numbers or complex care needs.
The Real Message: Demonstration, Not Isolation
Writing a bill like this isn’t just about one disease or one state. It’s a demonstration project. It proves that state governments can and should step in where the federal safety net is fraying. It’s a declaration that ultra-rare doesn’t mean unworthy. And it shows lawmakers—both state and federal—that patients and caregivers are organized, informed, and unwilling to wait for permission to survive.
Key Takeaways for Other Advocates
Don’t wait for federal salvation. Build local solutions now.
Your bill doesn’t need to be perfect to start—just urgent and people-centered.
Every state has the authority to fill federal gaps in coverage, care, and access.
Rare disease communities are stronger when we act across levels—local, state, and national.
What you build for your community can become a framework for others.
The Bottom Line
We are in a new era of rare disease advocacy. The federal system is no longer guaranteed to hold, and pretending it will only delays solutions. By writing and pushing state legislation, we’re not giving up on federal support—we’re refusing to be paralyzed by its absence.
This is what it means to prepare for the unpredictable. This is what it means to lead.
