A collective coverage of my shared experience at Rare Disease Week with the EveryLife Foundation.
Who am I?
My name is Zach Newbill, I am twenty-three, and I live with type two Hereditary Angioedema. I was one of the youngest diagnosed for my time at the early age of one and a half. Since that fateful morning when my mother woke up to my foot being the size of a football, I’ve grown up my entire life with aggressive HAE attacks, ER visits, and adversity within my day-to-day life. It’s not all doom and gloom, though, at the age of ten I attended my first HAEA Summit in Las Vegas, Nevada. At this event, I acquainted myself with people I could finally relate to, made lifelong friends, and met people who would go on to battle for my health and safety for years. I’ve found support and family in people like Drew, Noah, Ally, and many more amazing humans I’m lucky enough to call friends.
The HAEA is a second family for me and one I am happy to call home. I’ve been advocating with HAEA as a part of the Youth Leadership Council and Youth Advocacy Achievement League for some time now. I always want to show folks the power of the rare disease community!
Outside of the HAEA, I am a full-time nerd! I am the Social Media and Community Manager and a credited writer for Kobold Press, a fantasy tabletop gaming company. I spend my days with the Kobolds knee-deep in spreadsheets, traveling to conventions, and marketing our books on your favorite social media platforms.
Why was I in DC with EveryLife and the HAEA?
Once a year, on the last day of February, we celebrate Rare Disease Day! This is what Rare Disease Week was about. When I arrived at the heart of our nation in Washington, DC, I was filled with excitement and tenacity that only the following events would be able to quell. Over these next few days, we honed in on specific and pertinent policies vital for the rare disease community, not just for HAE but for all thirty million Americans in the United States diagnosed with a rare illness. With that policy in hand and heart, we took to the capitol, speaking with our representatives and political leaders and urging them to be leaders in making a better and healthier America.
What kind of policy were we honing in on? There’s a plethora of policies we tackled at Everylife’s Rare Disease Week conference, but I especially wanted to speak to politicians about the Rare Disease Caucus. A quick briefing for folks who don’t know what that is, the Rare Disease Caucus is essentially a committee of elected officials who meet to discuss legislation and policy directly relevant to rare diseases. I wanted to bring this up because despite the Rare Disease Caucus being rather large, and as a constituent of Tennessee, the Volunteer State, you would think we Tennesseans would be loud and proud in the Caucus representation, but that’s unfortunately not the case. Tennessee has 11 possible bodies that could be put on that council, yet Senator Cohen is the only TN representative on the Rare Disease Caucus. This is even more disheartening when you remember TN is a Biotech Mecca of the United States, between Vanderbilt, Revance, and other large Biotech and big Pharma companies, we’re a powerhouse for producing rare disease treatments, so it was paramount to me to bring up the lack of representation within the Caucus.
We also specifically pushed for a few bills, including the
Accelerating Kids Access to Care Act (S. 752/ H.R. 1509), a policy allowing young people on Medicaid to cross state lines to see the doctors they need. This bill would help the many young people without a local rare disease specialist who have to pay high out-of-pocket costs to get their life-saving treatments. It comes across as a common-sense bill, but I’ve been fighting for this policy for over a year, and the rare disease community at large has been pushing for something like this for even longer. If you want to learn more about this legislation, you can read more about the
Accelerating Kids' Access to Care Act (S. 752) here.
A pertinent piece of legislation is the
Safe Step Act, a bill that adds safeguards against step therapy policies on treatments for severe and potentially fatal conditions. Often, insurance companies will ask you to take a cheaper treatment before they will give you the treatment your doctor prescribed, or what we call a “fail first” policy, in hopes of saving their profit margin. This is even done sometimes without proper research or care of a medication's side effects. The
Safe Step Act assures that the only people who have a say on what medication you take are your doctor and you. This specific ask is widely known and had broad bi-partisan support in the last Congress. We are waiting for the
Safe Step Act to be reintroduced in the 119th Congress.
Another important piece of legislation that might be a bit more familiar to my fellow HAE Advocates is the
HELP Copays Act. This has been on the agenda for HAEA Capitol Hill days, as well as many of the initiatives and activations we’ve done at previous HAEA Summits. A quick refresh in case we’re newer in our advocacy journey though, the
HELP Copays Act is a very important bill as all of us with a rare disease know the financial burden it can place on us. This bill would require health insurance plans to include third-party co-payment assistance towards your medical bills to count towards your maximum out-of-pocket or deductible. As a young adult, who is quite familiar with their ER, max out-of-pocket is my arch nemesis when it comes to financial freedom and I know I am not alone in that fact. If you want to learn more about this legislation, you can look at the
HELP Copays bill (S. 864) here. Lastly, I’ll bring up a final piece of legislation related to orphan drug development that is a massive issue for the rare disease community at large: the renewal of the
Priority Review Vouchers (PRV) Program. The easy way to think about PRVs is like a golden ticket to the chocolate factory. A biotech company with a PRV can fast-track a novel drug through the approval process by ensuring a shorter wait time for FDA approval. A big note for PRVs is that they are transferrable, meaning that the voucher can act as a safety net for companies when a drug trial doesn’t successfully end in a new product on the market. This means a biotech company can look at rare diseases as a way to get their feet off the ground and accelerate their timeline. Per Pharmaceutical Technology, a leading reporting and research group in the medical industry spoke about these transferable PRV tickets having a market value of around one hundred million dollars. This money allows these companies to look at rare disease treatment as a viable avenue for building a sustainable business, despite how rare patients for these conditions are. If you want to learn more about PRVs, you can check out the
Give the Kids a Chance Act (H.R. 1262) here.
What did I learn from Rare Disease Week?
I learned many things. I learned about so many rare conditions and disorders, including cystic fibrosis, MSP1, and Sanfilippo syndrome, which were just a few of the conditions my fellow constituents were advocating on behalf of. I highly encourage reading up on these rare diseases, the stories of those who live with these conditions, and how intensive the care can be. I also learned about the existence of RDACs, which I took a particular interest in. These are state-based political groups known as Rare Disease Advisory Councils (RDACs). I even found out TN has an RDAC and one of the constituents in attendance was a frequent communicator with Tennessee’s RDAC! I’m coming off Rare Disease Week, and I'm excited to speak with her more and possibly become involved in some fashion with the RDAC.
I also learned about Young Adult Rare Representatives (YARR) through one of the panels hosted by the EveryLife Foundation, specifically targeted at young adults. I don’t mean to toot my horn when I say I’ve met some incredible people in my lifetime, but these four panelists were something else.
Camryn gave an impassioned speech about her struggle through her diagnosis journey of Fibrous Dysplasia, which also occurred amid the pandemic, how the four walls of her ER room were the spark for her flame of intense advocacy, and about her time as an advocate for her condition and the adversity she has faced. Two of the other panelists were these wonderful advocates out of Indiana, Amelia, and Anneliese, who also happened to be twins! They not only are founding their states’ RDAC, but they are also superheroes in their ways.
How did my advocacy meetings go?
In one word, amazing! Originally, I only had four meetings on my schedule, but my fellow Tennesseans, in true volunteer fashion, empowered me to join them for the three more Tennessee-based meetings. We were deep in the Capitol atmosphere all day with a full docket. From 10 am to 5 pm, I met with TN representatives in both the House and Senate, and I was lucky enough to snag photos with Congressman Chuck Fleischmann and Senator Marsha Blackburn! They were both swamped as they were in session that day, so I’d like to make a space here to thank them personally.
While I will continue to meet with you and your staff to push for better medical rights, better policy, and more outstanding advocacy for rare disease patients, I will say that you are both some of the hardest-working people in the country. I sincerely appreciate your staff's kindness, knowledge, and engaging conversation, brought to our sessions. Thank you for reminding us why we love Tennessee so much. Go Rocky Top!
What action am I taking after Rare Disease Week?
Well, firstly, there's this blog! On the Sunday after returning from DC, I cozied up to a mug of some hibiscus and lemon tea and wrote out all of my follow-up emails to my representatives. I’ve also emailed Tennessee’s RDAC inquiring about any virtual meetings, or volunteer work I can do. I know that is a lot for just a week or two after the event, but there’s no time like the present! We live in an age of uncertainty, and we can only be sure of one thing—our tenacity. As long as we remain persistent and ambitious, the struggles of those with rare diseases are numbered!
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