I just returned from Rare Disease Week in Washington, DC, where patients, caregivers, and advocates come together to push for change in the healthcare system. In this episode, I share what federal advocacy actually looks like—from packed briefing rooms to meetings on Capitol Hill—and why these conversations matter so much for people living with rare diseases. I talk about what surprised me, what I learned, and why patient voices are essential in shaping healthcare policy.

High Liver Enzymes on Interferon: Why I Had to Stop Treatment – Ep. 33 – The Fine, But Not Fine Podcast

When you treat a cancer that can't be cured, you don't just manage the disease, you manage what the treatment does to the rest of you. My liver numbers climbed high enough that I had to come off my medication entirely. This episode is the cascade that followed: the referral, months of waiting, vials of blood, a FibroScan, and the relief of an answer.

1. High Liver Enzymes on Interferon: Why I Had to Stop Treatment – Ep. 33 13:25
2. 150 Appointments: What Rare Disease Treatment Actually Costs – Ep. 32 10:01

Transcript

You look fine, but you’re not fine, and that’s exactly what we’re here to talk about. Welcome to Fine, But Not Fine, the podcast about navigating rare disease, health care battles, and the messy reality of chronic illness. I’m Kelly Paul, and I’ve been living with Mycosis Fungoides since 2015. This is a space for real talk, real experiences and practical advice, because surviving is one thing, but figuring out how to actually live, that’s the hard part.

I walked into a Senate meeting room that was way too small for the number of people who showed up, patients, parents, advocates, all. Everyone was there for the same reason, because something about the health care system isn’t working fast enough for people who don’t have time to wait. And I remember thinking, this isn’t dramatic.

There’s no spotlight, just structured conversations inside complex buildings. This episode isn’t about policy. It’s about what advocacy at the federal level looks like. Because I think most people imagine something very different.

So the last week of February, I was in Washington, DC for Rare Disease Week. I attended as a board member of the Cutaneous Lymphoma Foundation and as someone who’s been living with cutaneous lymphoma for well over a decade. And this week’s activities were coordinated by the EveryLife Foundation for Fare Diseases.

There were about 1,000 people. All of us descended on Capitol Hill for Rare Disease Week, and together, we had more than 300 appointments with elected officials. And I want to kind of share with everyone what it was actually like to advocate at the federal level.

Now you usually don’t meet with the senator or congressman or woman that you have registered to meet with. You typically meet with their staffers, and that does matter. Staffers are the ones who are evaluating proposals. They’re drafting memos. They’re advising on votes. They’re highly informed, sharp individuals who ask real questions.

Now the meetings are short, 15 to 30 minutes, and you are not alone in these you’re in a group parents, patients and other carers and concerned family and friends. So, more people with senators than with representatives, because there are only a couple representing your state, right? And it’s important to note all of us, you me, my next door neighbor, we all have different communication styles, and we all have different comfort levels with policy.

So before we walked into each meeting, we as a group — and the groups differed for each meeting — had to align on who’s opening the conversation, who was going to talk about which issue? How are we going to connect personal experience to specific legislative asks? And I wouldn’t call it scripted, per se, but it’s certainly structured. And it needs to be, because if you just tell a story without connecting it to action, nothing is going to move.

The staffers we met with really did ask thoughtful questions. So a few examples of that. How is this proposed legislation going to be funded? What would prevent abuse of what it is you’re asking for. How does this impact current patients, and what are the potential unintended consequences of this legislative ask? And it is really worth noting, this is not opposition, it’s governance.

I think sometimes people imagine advocacy is super emotional storytelling, and yes, emotion is present, believe me, but emotion alone, that doesn’t change the law or the regulations. You have to connect experience to feasibility, and you have to be prepared for pushback and to say, I don’t know, but I’ll find out and get back to you.

And there’s an important lesson here. The meeting is not the win. The meeting is the beginning. You leave. You need to send a follow up email. You need to thank someone for the time they spent with you. You need to provide any information they requested or that you said you would follow up with and you need to stay engaged.

You need to have a plan for the next several months and next year on how you’re going to kind of build a relationship with these individuals and send them pertinent information related to your experience and story, and the legislative ask. And you need to keep in mind that mind that advocacy is relational, not transactional. And yeah, the cynic in me is like, well, is this mostly true, right? Or at least it should be.

Another thing I want to talk about is the people. Many of the rare disease patients in those rooms, are also professionals. These are executives, nonprofit leaders, parents and patients who manage impossible schedules and still show up, prepared and committed. You know, rare doesn’t mean fragile. It means navigating complexity every single day.

And there is something grounding about being in a space where people are sharing deeply personal stories and then immediately shifting into here’s the policy solution. It requires composure, it requires discipline, and it requires humility. And I am telling you, listening to some of the parents share their experiences and how the government’s action or inaction blocked the extension of life for their children. It was absolutely heartbreaking.

And listening in a separate hearing in the Senate to Dr. Schmahmann, a doctor at Massachusetts General Hospital, Ataxia Center, when testifying before the Senate Special Committee on Aging, he shared his opinion of how the FDA approached treatment for those he cares for. It was absolutely chilling. He said, It’s as if the FDA demonstrates they are and I’m quoting “lacking charity, mercy or kindness.” And from his testimony and the testimony of others, I completely agreed.

As rare disease patients and carers for those with rare disease, we have to get the word out to people about what is happening to rare disease patients, and we need to drive effective change, and we need to do it fast.

So a few honest observations about the week. It is overwhelming. I mean really overwhelming. The buildings are imposing, but everyone, and I mean everyone in the hallways sees you, and you’ve got that look like you don’t know where the heck you’re going or how to get there, and they help you get where you want to go.

Some offices can feel political in ways that can intimidate people and those highly charged decorations outside the doors. Well, you know, there’s one thing about that. You know, it’s interesting. People have their opinions. People have what they support. But I’ll tell you something. I’m not sure if the elected officials and their staffers realize or care about. They preclude a lot of their constituents from taking photos to share about their meetings, and I would think they would want people from both sides of the aisle to be able to do that comfortably.

And not everyone realizes, you know, this is ongoing work. It is not a one and done trip. What it is is spark inducing. You see how change actually moves slowly, procedurally through relationships, and you realize your role isn’t to storm the system, it’s to understand it well enough to work within it.

If you take nothing else from this episode, take this: advocacy is not dramatic. It is disciplined. It is structured. It is the start of conversations, not the end.

Thanks for listening to Fine, But Not Fine. If this episode resonated with you, subscribe so you don’t miss what’s next. And if you’ve got a story, question, or just need to vent, reach out. I’d love to hear from you. Until next time, take care and keep on going