New Year, Same Fight: Why 2025 Demands More Than Hope
Each January, we enter the new year clinging to hope—hope for change, for breakthroughs, for relief. But for the rare disease community, hope alone is not enough. We are stepping into 2025 carrying the weight of unresolved battles, broken systems, and an exhausting truth:
We are still being left behind.
We’ve seen promises from policymakers that haven’t materialized. We’ve watched budgets get slashed while lives hang in the balance. We’ve sat through meetings with regulators who ask us to wait—again—while our children grow, decline, or die without answers.
Families impacted by ultra-rare and rare diseases don’t get the luxury of waiting. Time isn’t theoretical to us—it’s measured in missed milestones, hospital visits, and the increasing urgency of what might never come.
What We’re Facing in 2025:
Research Funding Cuts: Ongoing threats to NIH and rare disease research grants mean fewer studies, fewer clinical trials, and delayed innovation.
Regulatory Gridlock: The FDA continues to silence or sideline patient voices, treating us like checkboxes instead of collaborators. Have you tried the FDA rare disease hub? Well I have and have yet to get a response. It the same thing dressed up as a “new.”
Coverage Gaps: CMS and insurers still deny medically necessary treatments, devices, and therapies—especially for those with complex or “non-standard” needs.
Newborn Screening Failures: Too many states still don’t screen for treatable rare diseases, allowing preventable damage to continue unchecked.
Where We Go From Here:
This year, I refuse to sugarcoat the challenges ahead—but I also refuse to give up. 2025 will be the year we organize louder, show up stronger, and demand more.
I’m working to:
Expand national awareness and action through coalition-building across the rare disease space.
Fight for newborn screening policy reform that saves lives instead of letting them slip through cracks.
Protect but ensure FDA, CMS, and federal partners are held accountable for how their decisions impact our real lives—not just what looks good on paper. Even though there are funding cuts, we still have the same problems just a new administration.
Push forward legislation with teeth—not just words.
These are not new problems—but they are now compounded by exhaustion. Many of us are not just advocates—we’re caregivers, nurses, researchers, teachers, therapists, and navigators of systems that were never built for us. We are burned out, under-resourced, and often invisible.
And yet, we rise.
To every family reading this: I see you.
To every policymaker reading this: We’re done whispering.
To the system that keeps trying to overlook us: We’re not going anywhere.
This isn’t just another year. This is the year we stop asking and start insisting.
Rare is not an excuse. It’s a rallying cry.
Let’s get to work.
— Gillian Sapia
