by Lisa Van Allen, Chair of RSDSA’s Advocacy Committee
I wish I could put into words exactly how and why Rare Disease Week was so empowering and transformative. Maybe it had something to do with my excitement over the freedom I felt, traveling on my own for the first time in years due to improved health. Maybe it was being in the US Capitol and having access to people in power during such an important time in history. Maybe it was the camaraderie and kinship I felt in meeting so many people living with the challenges that come with a rare disease.
The EveryLife Foundation did an amazing job in putting this important event together. They helped us find common ground in the isolation we often feel, the struggles we have in getting an accurate diagnosis, and the yearning we all have for an effective treatment and cure. They put a lot of thought into the comfort and needs of people in pain and/or with mobility challenges. The lanyards were colored red, yellow or green, indicating your preference for social distancing. The food was labeled with potential allergens or if it was gluten or nut free. There were plenty of breaks, as sitting still is hard even for the able-bodied. And people were incredibly kind and willing to help in any way needed. I go into all this because I don’t want anything to stop YOU from going with me next year!
Congressional Briefing
The meat of the conference was on Tuesday, which was packed with briefings from legislative advocates and healthcare lobbyists. We were given a deep dive on key policies selected specifically for their impact on rare diseases. This training was to prepare us for our meetings with legislators scheduled for Wednesday.
Ask #1: Ensuring Steady and Robust Federal Biomedical Research Funding & Public Health Agency
The current political climate has increased tensions with staffing cuts and potential budget cuts on the horizon. Dr. Amy Bower (a medical researcher and rare mom from Creighton University) made a strong case on how decreased funding impacts not just individual lives, but entire communities, states, and the nation. The three key federal agencies that support the rare disease community are the NIH (NCATS, the National Center for Advancing Translational Services, is considered the home for rare disease research at NIH), the FDA (which partners with rare disease researchers and device developers to ensure safe, effective treatments), and the CDC (which provides information on disease and protects from threats).
Ask #2: Reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program at the FDA
The FDA approves new treatments for rare pediatric diseases and assigns a Prioritized Review Voucher to the company conducting the research. In spite of bipartisan support, the program expired in December 2024. After September 30, 2026, the FDA will no longer be able to award PRVs.
The Give Kids a Chance Act of 2025 (HR 1262) includes a reauthorization of the PRV program. The Senate is expected to introduce companion legislation soon.
Ask #3: Include Accelerating Kids’ Access to Care Act (AKACA) in the First Legislative Package
Many children do not have access to specialized care for their rare diseases in their home states. Bureaucratic rules currently require children accessing care through Medicaid or Children’s Health Insurance Plans to treatment in their home state.
On September 17, 2024, the House unanimously passed the Kids’ Access to Care Act, but unfortunately, healthcare policies were dropped from the end-of-year budget package and AKACA did not pass.
Ask #4: Ask Your Members of Congress to join the Rare Disease Caucus
The Rare Disease Caucus is a bipartisan, bicameral caucus that raises awareness of rare diseases. To see whether your congressional representatives are part of the Rare Disease Caucus, click here.
Ask #5: Support for HB 6094, the “PROTECT Rare Act” (Providing Realistic Opportunity to Equal and Comparable Treatments for Rare)
You have seen me talk about this bill in previous blog posts and on social media. This ask has the potential of improving the lives of people with rare diseases (like CRPS/RSD) by ensuring access and insurance coverage for ‘off-label’ medications (like IV Ketamine for Chronic pain, as the FDA has not yet approved it’s use for chronic pain). There is a precedent for this access, as cancer patients were given access to off-label medications and treatments in the 1980’s and people’s lives were improved and saved. The Rare Disease Community is asking for parity in access to these potentially life-changing medications.
The conference day ended with opportunities for individuals new to advocacy to meet with other advocates from their state and to work on their “ask”. The group from Iowa (where I live) was small, with only four of us attending. This was actually a good thing, as it gave opportunity for each of us to briefly tell our story and make a specific “ask”.
Congressional Meetings
The group from Iowa met outside Senator Grassley’s office at 9 AM and were pleased to discover the Senator was attending the meeting with his healthcare staff Nic Pottebaum. This is a special honor as Sen. Grassley is a man with heavy influence and is President Pro Tempore, making him 3rd in the line of Presidential succession. I’ve known Senator Grassley from my past work in Community Health Improvement Planning for Eastern Iowa. He also knows my husband as a fellow UNI grad and Panther football and basketball supporter. But I have not ever shared my CRPS story with the Senator, and I appreciated his listening with empathy and interest in my ask – that he support HB 6094, the Protect Rare Act. I shared a bit about how IV ketamine has improved my life, even making my trip possible. I shared that there is only ONE outpatient infusion center offering ketamine for chronic pain in the entire state and that I drive 4 hrs. out of state for my infusions. Senator Grassley and Nic agreed to research this bill and that the access to care it promises is needed for people living with rare diseases.
We had a little extra time after our stories and asks were made, and the Senator asked if we had any other questions. I asked about his assessment on cuts anticipated from Medicare/Medicaid, as well as from the FDA and NIH and the potential impact it would have on the rare disease community. He spoke briefly about the current turbulence in Washington, and how things are changing at a pace that hasn’t been seen before. But, he gave a brief civics lesson on how changes to policy and to the budget requires that the Senate and the House come to some agreement. The House and Senate are currently very divided on where budgets ought to be made, and so this process will be slowed until consensus is found. I shared that I found this reassuring in that this gives us time to appeal to our representatives in Congress and to remind them of what we want – consistent funding for rare disease research and access to care. The Senator agreed that he works for Iowans… as every legislator works for their constituents, and needs reminded of this.
The rest of our meetings were with legislative staffers for Representatives Miller-Meeks (who already supports HB 6094), Feenstra and Hinson and Senator Ernst. We each told our stories and made our asks, and I felt each staffer listened and cared about our concerns. Everyone we talked to was very supportive of the pediatric asks, and understood our concerns about funding for research. I got commitments from each staffer that they would share the literature I left on CRPS and on HB 6094 with their legislator. I made particular mention of the frequent contact I have had with veterans with CRPS to Sarah Melone, Senator Ernst’s healthcare policy assistant, as the senator is very active in veteran’s affairs. She was moved by the story I shared about one man who called me, and how his life has been transformed since getting help because of information he found here on the RSDSA blog.
Rare Artist Reception
Every year the EveryLife Foundation selects several ‘Rare Artists’ who have submitted written (poetry) or visual art. The Rare Artists are honored at a reception and are invited to participate in additional training to be able to speak out and share their stories at congressional meetings. You can see the 2024 artists works and hear a bit of their stories here.
I spent a little time during a break with two of the artists, Dana Simmons and Eugenia Hsu. (I learned Eugenia and I both worked with the Disability Rights Education Defense Fund – DREDF in past lives, and attended the same grad school at about the same time. Small world!). Both women talked about the challenges of diagnosis, the isolation of being misunderstood, and the power art has given them to express their emotions. As an artist myself, I was impressed with the power to raise awareness with our work and help others understand a little more about what we face every day. I KNOW I’m not the only artist who uses painting or drawing or sculpture or poetry to deal and distract from my pain. We need to have someone with CRPS/RSD represented next year! I plan on submitting a piece – who else will take on this challenge?
NIH Scientific Presentations
Originally, my plan was to be in DC for the entire week, but I learned a couple weeks before leaving that the Thursday and Friday sessions at the NIH had been cancelled. Honestly a part of me was thankful my trip was shortened – I was nearing my pain tolerance limit by Wednesday night. Even so, I was looking forward to being at the NIH as I’ve been told their presentations last year were the highlight of the conference. NIH staffing cuts caused the cancellation of the scientific presentations. It is a strange feeling when national news items hit you personally. DOGE staffing cuts and impending budget cuts were on everyone’s minds and part of nearly every conversation. This is not a political issue – it is a healthcare issue. Regardless of where you land on the political spectrum, your voice is needed. We each need to contact our representatives are ask them to protect funding for research and access to care for rare diseases.
Here’s Your Call to Action:
1. Write your legislators about your concerns about the coming budget cuts. I encourage you to focus on:
- Ask #1: Ensuring Steady and Robust Federal Biomedical Research Funding & Public Health Agency Support, and
- Ask #5: Support for HB 6094, the “PROTECT Rare Act” (Providing Realistic Opportunity to Equal and Comparable Treatments for Rare)
You can find your legislator’s contact information here under the “Contact Your Member” section.
2. Put Rare Disease Week 2026 on your calendar. It’s the last week of February – and you don’t want to miss it!
3. Consider entering a piece of your work if you are an artist in the Rare Artist contest.
Let me know when you’ve done these things! And don’t hesitate to ask any questions – if I don’t have the answer I will find someone who does!
