Learning the The Language of FDA Patient Engagement

Rare & Relentless

Two weeks ago, I had my first meeting with the FDA Rare Disease Innovation Hub. Today was supposed to be our follow-up. I took detailed notes, prepared my questions, and came ready with organized thoughts.

But just now, I received an email: the meeting was canceled.

The sacrifices I make to be at these meetings are not small. I rearranged my life to show up. And just yesterday, I found out my daughter needs a second brain surgery. That news is still sinking in—and now this.

Maybe this is the moment I finally say it out loud: my time is valuable too.

This isn’t the first time I’ve had an FDA meeting canceled. It’s happened before—an Advisory Committee (ADCOM) meeting earlier this year, and several more during the administration transition. Most recently, the FDA even canceled its Rare Disease Day event.

It’s taken a tremendous amount of advocacy, time, and emotional labor to get here—to even begin to understand how the FDA operates. So yes, I’m frustrated and discouraged. These meetings matter. They require preparation, clarity, and heart.

And still—I’m grateful. Because I know how hard it is to be seen and heard at the FDA. That’s why I stay.

To make any progress here, I’ve had to practice saint-like patience. Where others walk away, I say, “Thank you. I understand,” and keep going. I know FDA staff are under strain—their offices turned upside down—and rescheduling is sometimes unavoidable.

So I wait. Because this work is long-term. It’s a marathon, not a race.
And the only way forward is to be politely relentless.

I never meant to learn a new language when I became a rare disease advocate. But here I am, years later, navigating a full glossary of acronyms—just to make sure our stories count. Not just as emotional background, but as data that drives decisions.

After my meeting, I read the FDA guidance that the Hub (off the record) recommended. Here’s what I learned:

When “Patient Engagement” Doesn’t Mean What You Think It Means

What does patient engagement at the FDA really look like?
Is it a conversation—or just a formality?
Is it storytelling—or a story rewritten to fit their forms?
Is it a seat at the table—or a folding chair in the hallway?

Managing Expectations: A View From the Trenches

Over the last five years, moms of children with ultra-rare galactosemia have emailed the FDA again and again, expecting a response. That effort matters. But eventually, reality sets in:

At the FDA, “patient engagement” is a defined term.
It’s not a conversation. Not an emailed story. Not even showing up in good faith.

While it’s not yet a legal definition (though it should be), FDA-recognized patient engagement is a formally structured process—governed by policy, protocols, and gatekeeping. And most of it happens through pharmaceutical companies, not patients.

When caregivers write in, FDA often responds with citations:
21 CFR 312.130 and 21 USC § 331(d)—rules designed to prevent insider trading. These laws prohibit the agency from discussing investigational drugs, even when patients are desperate for information.

Because in the past, people exploited FDA access for financial gain.
Now, the system protects companies—not conversations.

And while we’re not insider traders—we’re just trying to survive—it still makes the open dialogue we need nearly impossible.

When Patient Engagement Turns into a Checklist

This is what "engagement" too often becomes:

• A 65-page PDF that buries lived experience in red tape

• A story rewritten by pharma, with your voice softened or stripped away

• A checkbox ticked to satisfy a requirement, not drive real change

• A comment portal that feels like screaming into a void

• A meeting where your heartbreak is called “anecdotal”
  —unless you’ve already translated it into spreadsheets

We come with everything—grief, hope, expertise.
And too often, we leave feeling like no one really listened.

We expect to be collaborators.
Instead, we're treated like an afterthought—seen as desperate, uninformed, or non-expert.

Let me be clear: I know galactosemia inside and out.
If called to testify, I could stand in court as an expert witness.
What I struggle with isn’t knowledge—it’s the dismissive gaze from professionals who see pity, not insight.

If you gave me real time—a real moment—you’d see just how much I carry.
And how much I can offer.

So What Does Real Engagement Look Like?

At the FDA, official guidance isn't designed for direct patient dialogue.
It’s designed for industry and advocacy groups to engage—through legal, structured channels.

Laws like 21 CFR 312.130 and 21 USC § 331(d) restrict the FDA from discussing unapproved therapies. So patients rely on pharma to bring our voices forward—packaged to match their investment pitch.

But who’s protecting patients?

Too often, it's just you—standing beside pharma, hoping your story isn’t just a PR checkbox.
That’s a conflict of interest from the start.

Especially if you’re new, you may not know when you’re being used. Or that the company representing you doesn’t understand how FDA engagement really works. That can lead you down the wrong path.

So please—trust your gut.
If something feels off, it probably is.
Be true to your story. Always.

I’ve read the guidance. I’ve gotten lots of bad advice.
I’ve learned through rejection, persistence, and asking uncomfortable questions.
I even helped pass legislation to make patient engagement easier.
But it’s still not happening the way it should.

We—foundations, advocates, caregivers—are building from the ground up.

So if you’re preparing to show up at the FDA table, ask yourself:
Do I speak their language?
Because if not, they’ll nod politely—and move on.

And that’s why we learn it.
So they can’t look away.

Learn the FDA's Language of Patient Engagement: A snap shot of terms

(A real-world glossary, made for patients—not policy experts)

PPI – Patient and Public Involvement

What FDA means: Patients help design trials, shape guidance, and define outcomes.

Real-world example: A galactosemia caregiver co-authors a burden of disease report used in trial design.

RWE – Real-World Evidence

What FDA means: Data from outside trials—registries, EHRs, caregiver tracking—used to support decisions.

Real-world example: School absences, mobility logs, and fatigue data support drug expansion for Duchenne.

PRO – Patient-Reported Outcome

What FDA means: How you say you feel—no doctors involved.

Example: “I used to dance. Now I can’t climb stairs.”
Used in cystic fibrosis and pain approvals.

PROM – Patient-Reported Outcome Measure

What FDA means: A structured tool (like a survey) that quantifies your experience.

Problem: Most don’t fit rare diseases. They miss nuance. That’s why qualitative data—like interviews—matters.

COA – Clinical Outcome Assessment

What FDA means: Includes PROs, ClinROs (by doctors), ObsROs (by caregivers), and PerfOs (tests).

Example: Timed walk test in SMA. Seizure log from parents. Swallowing scores from speech therapists.

Why It All Matters

These aren’t just acronyms.
They’re how your pain gets converted into the kind of data the FDA accepts.

When you speak their language, they have to listen.
And when the system still doesn’t work for you?
We challenge it. We rebuild it. Together.

Where Do We Go From Here?

We keep showing up.
We keep emailing.
We keep learning—not to be polite, but to be powerful.

We translate our pain into terms the system understands—and then we push it to understand more.

We’re not here to fill out more forms.
We’re here because the stakes are life and death.

Patient engagement isn’t a PDF.
It’s a relationship.

And if systems like the FDA want our trust—
they need to meet us halfway.

FDA Contact List for Patients & Advocates

1. Rare Disease Engagement
OD_RareDisease@fda.hhs.gov
(For meetings, burden of disease data, and treatment development input.)

2. General Patient Affairs
PatientAffairs@fda.hhs.gov
(For questions about engagement pathways and how to formally participate.)

3. Expanded Access / Compassionate Use
CDER-EAP@fda.hhs.gov
(For help navigating access to investigational drugs.)

4. Drug-Specific Input
DRUGINFO@fda.hhs.gov
(For questions about FDA processes or where to direct concerns.)

5. Public Comments on Guidance or Policy
Submit at https://www.regulations.gov
(Search for docket numbers to submit formal input.)