When the Door Finally Opens, It’s Paper-Thin
Rare and Relentless
It took me five years to get here—my first official meeting with the FDA’s Rare Disease Division. Five years of calls, advocacy, writing, and knocking on every bureaucratic door. When it finally opened, it felt less like a triumph and more like a window cracked just wide enough to pass a stack of paperwork through. After years of advocacy from Everyife foundation the FDA created the Rare Disease Innovation HUB in 2025, we will see in time how this program fills in gaps for patient engagment.
They recommended a document—“guidance,” they called it. Five-point font, industry jargon, and a structure built not for patients but for partnerships with industry. Every sentence read like a legal agreement between the FDA and pharmaceutical companies. What forms the the FDA uses for Real World Evidience and Patient Experience. Patients? We were a footnote at best.
So now, I sit at my desk with my glasses on, reading five pages a day, mining for anything—anything—that can be used to help patients navigate this system. But the truth becomes harder to ignore: patients do not control the narrative here. Pharma does.
And I think that’s ridiculous.
For decades, patients have walked into these agencies believing they were entering safe spaces. We believed there were protections. That if we shared our voice, someone would be listening. But the street knowledge starts to seep in—stories of clinical trials gone wrong, consent forms that meant something different than what was explained, and a system that feels more like a machine than a partner.
I recently listened to a lecture on the placebo effect. One study showed that the very language used in consent forms can alter a patient’s placebo response. Imagine that—just the way something is written can change a body’s physical reaction. Some conditions, especially those involving pain, show higher placebo responses because of dopamine and serotonin receptor activity. It blew my mind. But what really struck me was how early the placebo effect starts: at the moment of belief.
And right now, that belief is being manufactured by industry relationships—not by true partnership with patients.
My meeting with the FDA’s Rare Disease Innovation Hub was meant to be about introductions. I came prepared, armed with a long list of questions:
What can you help me with?
Where does the patient voice fit in?
How do we build a meaningful, lasting relationship?
It wasn’t confrontational. It was hopeful. But that hope is hard-earned and cautious.
Years ago, I had one other FDA meeting. It was filled with the phrases repeated like a wall: “We cannot comment on investigational products.” At the time, I was told this stance was due to the risk of violating federal rules. Some people, even within advocacy circles, believe there’s a blanket five-year jail sentence for FDA staff or others who disclose investigational drug information.
So let me set the record straight.
I listened to a lecture from Dr Robert Calliff early last year. He stated the FDA “is the only government office that has a 5 year sentence for leaking information.” I have not found the law that automatically imposes a five-year prison sentence for talking about investigational products. However the FDA does strictly enforce confidentiality during the drug development process, especially under its Investigational New Drug (IND) regulations. In some serious cases—like insider trading using non-public FDA data—people have received five-year sentences. But that’s not about patients asking questions. It’s about criminal misuse of confidential information for personal gain.
So while the “no comment” policy isn’t criminal law per se, it functions as a structural wall. It allows pharmaceutical companies to control all investigational information flow to patients—not the FDA. That’s a huge conflict of interest, and patients deserve to understand that.
This blog series will share my experiences as I continue to engage with the FDA, NIH, CMS, and other federal systems. I’ll expose the cracks in the safety net and shine light on the places where patients are treated as spectators instead of partners.
This was my start.
I’ll keep updating you on the journey.
Because five years for a first meeting is unacceptable.
