“I Shouldn’t Be Telling You This”: The Secret Dismantling of Rare Disease Advocacy at the FDA

Afraid to Speak. Afraid Not To.

“I’m even scared to share this.”

That’s the thought that keeps looping in my head. After five years of advocating for rare disease patients at the FDA, what I’m witnessing now is so disturbing, so far beyond bureaucratic dysfunction, it feels dangerous to even name it.

I’ve heard from FDA employees—first in cautious whispers, now in urgent warnings. They tell me my name is likely on lists. My emails and calls, they say, are being logged. The fear is palpable: “They could easily pull that log and fire anyone who’s helped you.”

This is what it feels like to be a rare disease advocate in 2025. Not empowered. Not included.

And it’s happening right under our noses.

The Collapse Is Quiet. And Intentional.

The FDA’s Professional Affairs and Stakeholder Engagement program—PASE—is gone. No press release. No notice to Congress. Just vanished.

The Rare Disease Innovation Hub? It’s been silent since April 1. My emails go unanswered. The phone number doesn’t work. I’m told to “just send an email.” I do. Nothing comes back.

I’ve called every contact, every alternate line, every office I know. What I get is confusion. Departments that no longer exist. Phone trees that lead nowhere. Staff who whisper warnings and beg to stay anonymous.

“There’s another round of cuts coming in June.” “I shouldn’t be telling you this.”

These aren’t random voices. These are seasoned FDA staffers. And they’re terrified.

A System on Fire—and No Fire Alarm

For years, I fought to amplify the patient voice, representing the entire rare disease community, including families battling galactosemia and other conditions. I built trust. I gave the FDA every benefit of the doubt. But now?

What I’m experiencing is unlike anything I’ve seen before.

Most FDA staff who once championed collaboration are gone—resigned, reassigned, or silenced. Those left describe an unstable, chaotic agency, reshaped under the Trump administration into something that feels politically weaponized. The infrastructure that supported patients—especially those with rare diseases—has been dismantled piece by piece.

And there’s no public acknowledgment of any of it.

NIH and CDC Hit Just as Hard

It’s not just the FDA.

The NIH’s budget has been slashed 40%, from $47 billion to $27 billion. For rare diseases like galactosemia, where sometimes only four researchers nationwide are working on a cure, this is a death sentence for progress.

CDC programs like EHDI for early hearing detection, PRAMS for maternal health data, and the Advisory Committee on Heritable Disorders in Newborns and Children—are shut down.

I’ve learned 18 states are facing likely shutdowns of newborn screening and hearing

What You Can Do — Right Now

📢 Tell Congress What’s Happening
Use Democratic Leader Hakeem Jeffries’ portal to share your story:
👉 https://democraticleader.house.gov/shareyourstory

📞 Call your Representatives and Senators
Demand emergency hearings on rare disease cuts across the FDA, NIH, and CDC.

💥 Use your platform
Tag your posts with #ProtectRarePatients and #RareDisease #NIH #FDA #CMS

We Are the Fire Alarm

The agencies are failing. The phones are quiet. The programs are gone.

But our voices aren’t.

This post may be a risk. But silence is a bigger one.