Through the Noise: Rare Disease Still Deserves Equity
This week has been loud.
Between headlines about Trump’s renewed tariff war and waves of political hysteria on every channel, it’s easy to lose your footing—to question where to focus, what matters most, or whether your voice still carries in the roar.
But here's the thing: the same ask remains.
And it’s not partisan. It’s not flashy. It’s not new.
It’s this: Rare disease patients deserve equitable access to care.
That’s not a trending topic. That’s a human rights issue.
And no amount of election-year chaos should bury it.
Distraction Is the Oldest Trick in the Book
If you’re feeling stretched thin this year, you’re not alone.
The distractions haven’t just been annoying—they’ve been weaponized.
In 2025 alone, rare disease advocates have had to fight:
NIH budget threats targeting genetic research
FDA inaction on ultra-rare trial flexibility
CMS silence on coverage gaps for life-saving formulas
Disability benefits denials that ignore real-world impairment
All while fielding a dozen new political battles that grab oxygen, airtime, and urgency.
Meanwhile, patient families still face $7.8 million in lifetime costs for classic galactosemia . Children still lose access to therapies based on flawed metrics. And families still fall through the cracks because the system is not built for people like them.
The Ask Hasn’t Changed—But the Burden Has Grown
Let’s say it clearly:
Rare Americans are being harmed by budget cuts and policy stagnation.
And as media swerves between outrage cycles, it’s up to us to hold the line.
This year, our advocacy has expanded. It had to.
We’re not just pushing for diagnostic equity or newborn screening anymore.
We’re demanding accountability when the FDA dismisses ultra-rare therapies due to sample sizes they know are unachievable.
We’re naming the cost of delayed treatment—not just in dollars, but in irreversible brain and organ damage.
We’re showing how outdated CMS rules deny coverage to those who are medically disabled but not yet visibly broken.
Focus Is a Form of Resistance
In a time of chaos, clarity becomes radical.
So if you’re tired this week, if your head is spinning from the news, take a breath. Then come back to this:
1 in 10 Americans lives with a rare disease
Most are misdiagnosed, dismissed, or denied care
The burden is real, the data is clear, and the solutions exist
We are not going away
And no matter who yells louder on the news this week, rare families will still wake up tomorrow needing help—needing truth—needing a system that sees them.
So we keep going. Eyes forward.
Because our clarity?
It just might be the loudest thing in the room.
The Budget Is a Moral Document
If you want to know what a country values, look at where it puts its money.
And this year’s proposed budgets tell a grim story for rare disease families.
NIH: Starving the Pipeline Before It Starts
The National Institutes of Health (NIH) remain chronically underfunded relative to the scale of need. While flashy headlines tout “record-breaking” totals, inflation-adjusted funding has not kept pace with scientific opportunity or rare disease burden.
In 2025, over 95% of rare diseases still lack an FDA-approved treatment. The NIH remains one of the only federal mechanisms for initiating basic research and natural history studies in these conditions. But rare disease trials are longer, more expensive, and often depend on patient registries or academic centers that are now seeing funding cuts.
Slashing NIH grants means fewer pilot programs. Fewer translational studies. And ultimately, fewer chances at a cure for families who don’t have time to wait.
CMS: Cutting Coverage While Denying Disability
The Centers for Medicare & Medicaid Services (CMS) quietly shapes the daily lives of rare disease patients—especially those with lifelong needs and no approved treatments.
When CMS rules classify critical formula, medical foods, or durable equipment as “non-essential,” coverage evaporates. In states where Medicaid budgets are being squeezed, families are being told to “reapply” for benefits they’ve relied on for years.
At the same time, SSI and SSDI approval rates for rare conditions remain abysmally low, because the system still hinges on outdated diagnostic categories and visible disabilities. For conditions like classic galactosemia, where the disability is cognitive, neurological, and metabolic, many are told they are not “sick enough”—until the damage is permanent.
FDA: Paralysis by Policy
The Food and Drug Administration is perhaps the most misunderstood—and infuriating—player in the system. Despite a mandate to protect public health, the FDA continues to apply traditional, large-population statistical thresholds to ultra-rare diseases with populations under 500 people.
When gene therapies or metabolic stabilizers are submitted for approval, they’re often rejected for lacking placebo groups or sufficient power—even when the data show biologically plausible benefit and safety.
The agency’s slow progress on adaptive trial design, patient-centered endpoints, and post-market surveillance models is holding back treatment pipelines that are ready now.
And when a drug is rejected? That burden doesn’t fall on the agency.
It falls on the child whose brain development won’t wait for bureaucracy.
