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4 Stories of Advocating for Rare in DC
At the end of February, through the cold, and amidst all the change happening in Washington D.C., several members of our histio community ventured out to participate in Rare Disease Week activities. Today we will be highlighting four of our ambassadors who each had their own interactions with congressional and senate representatives for their states and worked with the Everylife Foundation in advocacy training as part of the Rare Disease Legislative Advocates.
First, let’s here from our newest Ambassador, Molly from Pennsylvania. She wonderfully broke down the details of the event, which helps inform details the other three ambassadors who wrote up their thoughts in this post allude to.
Molly
My daughter, Juliet, was diagnosed in March 2024 with HLH, so this was my first Rare Disease Week experience. The EveryLife Foundation provided one full day of legislative updates and training on Day 1. I found it to be incredibly interesting and informative. There were representatives from the rare disease space and from D.C. consulting firms. They each had slightly different perspectives, which was very helpful.
As someone in the “learning phase”, I deeply appreciated the time and effort the presenters put into their sessions. Members of the EveryLife Foundation laid out their “legislative asks” – these are the priority areas that the foundation asked us to focus on in our meetings. The four main asks were:
- Support Steady and Robust Leadership, Federal Biomedical Research Funding, and Public Health Agencies
- Support the Reauthorization of the Rare Pediatric Disease Priority Review Voucher Program
- Ask your Member of Congress to Join the Rare Disease Congressional Caucus
- Include the Accelerating Kids’ Access to Care Act (AKACA)
I am a Pennsylvania resident, so in the afternoon of Day 1, I met with other PA residents who I would be on the Hill with the following day. We discussed who would ask for which priority in our planning meeting. I chose to focus on the reauthorization of the Rare Pediatric Disease Priority Review Voucher Program (PRV) because one of the medications my daughter received prior to her bone marrow transplant that stabilized her (Gamifant) was developed under the PRV.
Day 2 was a full day of meetings on the Hill, all scheduled by EveryLife Foundation and their advocacy arm, Rare Disease Legislative Advocates (RDLA). Senator Dave McCormick was having a “coffee with constituents” event, which unfortunately, served as our “meeting” with him. There were many constituents in the meeting with competing agendas, but we were able to ask him a few questions and I did get to quickly tell him that Juliet’s life was saved by a drug developed under the PRV and that the program has zero cost to taxpayers. He seemed to be interested in it; however, attempts to connect with him since then, through staff contacts, have been unsuccessful. There has been a bill introduced in the Senate since we met with him and I would love to talk with him about it, but his team has been unresponsive.
Our next meetings were with staffers for two US Representatives – Chrissy Houlahan and Mary Gay Scanlon. Both sets of staffers were very interested in our stories and we had more time to tell them. We sat in their offices and each had a few minutes to speak. I was able to tell Juliet’s story and explain how grateful we were that she had a treatment available – but that so many in the rare disease community do not, and that is why the PRV is so important. The staffers understood the urgency; however, they repeatedly expressed frustration with the current environment in DC and that they did not have a lot of leverage.
Overall, my first Rare Disease week was inspiring and positive. I met so many wonderful people and I have kept in touch with a few of them. This has allowed me to learn of more opportunities for getting involved with advocacy. I am, however, frustrated that so little progress has been made with these initiatives since our time on Capitol Hill and I am also frustrated with the lack of response from my Senator’s team. It is truly an uphill battle. I met families with children who have drugs in clinical trials right now through the PRV, but unless it gets reauthorized, these trials will be canceled. The impact is truly devastating. This motivates me to keep going and I look forward to more advocacy opportunities in the future.
Next, Hank from Illinois had a meeting with two congressional representatives from Illinois and had pre-scheduled a meeting with his senators and representative before the EveryLife Foundation sessions that week.
Hank
It was a great opportunity to meet and collaborate with patients and caregivers at this forum. It was also an opportunity to take advocacy to another level by meeting with congressional members or their designees and share issues, concerns, and proposals.
The meets I participated in with the EveryLife Foundation had their downsides, however. Since they were prescheduled, participants were grouped together with 8 or more individuals. Because of this large number, the chance to provide an “ask” for individual legislative ideas was not often available. Other than sharing one’s story, there was little time left to actively engage the congressional member because only a 15–30-minute window was available. At one meeting, I noticed the designee doodling on a piece of paper while advocates were sharing their story. Despite not getting to that point in our meetings, it was great that the EveryLife Foundation already provided the “ask” that individuals were to request if time was permitted.
Fortunately, I took the initiative prior to the caucus to set up an individual meeting with my two congressional senators and my representative. I had 30-45 minutes to express my issues and concerns and request their assistance to champion rare cancer initiatives. I provided each a draft piece of legislation I was working on in addition to a few other items of interest that required no legislative action.
These too, had their downsides. Two senate meetings were very unproductive and both designees spent the majority of the time blaming, bashing, and finger pointing the other political party. I politely informed them of their responsibility to the voter in addition, I expressed my disappointment with their rhetoric.
My congressional representative experience was positive one. After that engagement, I have provided my representative four legislative proposals; two directly relate to cancer and two are indirect proposals. Another one-on-one meeting with them is being scheduled soon.
Many nonprofit organizations, foundations, or associations promote advocacy as individuals’ groups. There is tremendous opportunity to organize these groups with the same initiatives and become a meaningful delegation to empower patients and caregivers to hold legislators accountable and responsible to meet and exceed the rare cancer community expectations.
Our Youth Ambassador Nate really enjoyed his experience, him and his dad worked through the process alongside him.
Nate S.
This year was my first time participating in Rare Disease Week, and it was an incredibly rewarding experience. I appreciated the opportunity to advocate not only for myself, but also for others in the rare disease community, while connecting with people from diverse backgrounds across the country. Many participants had traveled thousands of miles to meet with legislators—an inspiring reminder of the power of shared purpose.
Since my hometown of Washington, DC doesn’t have a voting representative in Congress (something we really need to change!), I spent most of my time on Capitol Hill talking with staffers from Virginia and with staff from DC’s fantastic non-voting delegate, Eleanor Holmes Norton.
Our advocacy groups were pretty small, which meant everyone had plenty of opportunities to share their personal stories and clearly communicate our key policy requests in every meeting. We focused on a couple of important legislative issues:
- Making sure that crucial public health agencies like NIH, FDA, and CDC continue receiving strong funding and support. These agencies are essential for research and medical breakthroughs that greatly help the Histio community, and it’s crucial that we protect them from budget cuts.
- Passing the “Give Kids a Chance Act” (H.R. 1262), which would restore the Rare Pediatric Disease Priority Review Voucher Program—a no-cost, life-saving program that encourages innovation and brings hope to families facing rare diseases.
Most of the staff we spoke with were supportive and empathetic, though some Democratic staffers felt limited by the current political climate.
While it’s always great to speak with people who agree with you, I found it especially valuable to talk with people who didn’t already share our viewpoints. One meeting I won’t forget was with the office of a Virginia representative known to hold different opinions—not just on rare disease issues but overall. It stood out because it was a genuine chance to bridge differences and maybe even shift perspectives. Even if it doesn’t immediately lead to legislative change, the conversation itself was really meaningful because it challenged us to find common ground and communicate our message effectively.
Rare Disease Week deepened my understanding of advocacy. Not only offering an incredible opportunity to build connections and gain insight, but also powerfully illustrating how sharing personal stories, as a rare disease survivor, can drive meaningful change.
Meanwhile, Nate M. was going on his own advocacy adventure in D.C. Here are his thoughts on it:
Nate M.
“The week was about community, advocacy, and action. I had the privilege of standing alongside so many incredible advocates, each bringing their own powerful stories and perspectives to the table. Together, we took on Capitol Hill as one unified voice for the rare disease community.
One of the highlights of my Hill meetings was lobbying for the reauthorization of the Priority Review Voucher (PRV) Program—a key incentive that drives innovation in rare disease treatments. Ensuring continued support for this program is critical for fostering research and development for rare conditions that often lack effective treatment options.
Moments like these remind me why I do this work. Every conversation, every story shared, and every legislative ask matters. Change happens when we show up, speak up, and push for the policies that impact our community.
To everyone I had the privilege of connecting with—thank you for your passion and dedication. Let’s keep the momentum going. See you on the Hill next year!”
He also spoke this past week at the Access USA Rare Disease Summit in Philadelphia. Click here to view and to read more about that from his respective LinkedIn posts.
I am so proud of all of these Ambassadors and so excited to see what they do next in advocacy!
