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Rare Disease Week: Shannon Reedy

Advocacy Stipend Attendee Summary by Shannon Reedy

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I was fortunate enough to be able to take some vacation time and attend Everylife Foundation’s Rare Disease week on Capitol Hill. I know how hard it is to care for well family, sick family, a full time job, a home and yourself and still take on advocacy issues. We can’t always attend these critical events and I feel very fortunate that I’ve been able to get away and go a few times now. It is phenomenal that the National Niemann Pick Disease Foundation was able to send delegates this year.   

And… this year was the biggest yet! Nearly 900 advocates were present and actively working to explain our needs in the House and Senate, we converged on Thursday and met with multiple representatives in DC from our home states to compel them to back legislation that would greatly benefit our community. Whether it was with staffers or the actual elected officials we were received with rapt attention and concern. They legitimately wanted to hear from us and asked insightful follow up questions.  

There were various “asks” we as rare disease legislative advocates were tasked with in each meeting; below is a copy of the email I drafted and sent after our RI visits to give you an idea of the legislation that would benefit our community. I encourage you to send your own emails to your own representatives, feel free to use this is a template! 

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We understand time management may prohibit the Senators membership in the Rare Disease Caucus. As mentioned you may have an even more impactful way to assist; lending your signatures and votes to the issues we discussed on Thursday. They improve our daily lives at a fundamental level and help speed up research and eventual cures.

If you have a question, ask, we appreciate them. Per our discussion we are looking for you to back:

*Creation of The Rare Disease Center of Excellence at the FDA
Please sign the letter of support that will be circulating soon.  The FDA already has the authority to create this center under the 21st Century Cures Act. An oncology center has been successfully enacted and functioning, a model exists. This center will allow for efficiency and consistency in our Rare Disease Space, beginning to solve one of the biggest challenges in rare diseases.

*Enact the Newborn Screening Save Lives Reauthorization Act, H.R. 2507/ S 2158
Presently stalled in the Senate this Act expired on 9/30/19.  It would reauthorize critical newborn screening that saves lives and resources that could be devoted to research and disease management.  

*BENEFIT Act: Better Empowerment Now to Enhance Framework and Improve Treatments
What will presumably trickle down from this legislation’s enactment will change the course of treatment for many rare patients. Patient experience data is not routinely included in the regulatory review process at the FDA.  In small patient populations every single experience is critical.  Exclusion of the patient voice in critical analysis is one of the obstacles for getting access to supportive care in rare diseases. Valuing and lending credibility to the patient experience helps patients advocate for themselves for supportive care as well. 

*Co-Sponsor the Medical Nutrition Equity Act, H.R. 2501 (Senate: Bob Casey/ House: Jim Mcgovern & Jaime Herrera-Beutler)
This Act makes sense for everyone, not just rare disease patients. There are inherent metabolic and digestive disorders that lead to death or devastating disability that can be completely avoided by accessing specialized nutrition. There are hurdles to access for many people who desperately need medical foods, this legislation will help remove them.  

*Support Increased Funding for the FDA Orphan Products Clinical Trial Grants & Natural History Grants Programs
Clinical Trials are expensive. Rising costs mean that the FDA is only able to grant a fraction of the applications they receive. If discretionary funding to the FDA is increased via the 2021 Agricultural Appropriations Act more of our families will have their hopes funded. 

Your Support is Everything.

Warmest Regards,
Your Constituents

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There Rare Disease Week events not only connect us with decision makers in our government they also connect us with other rare disease advocates, the people in the trenches that know exactly what we are up against. Connections made at these types of meetings lead to collaboration and cures. They are also a way to recharge yourself. While we are strong as a Niemann Pick Disease group it can feel like we have a small world sometimes. We don’t, the other seven thousand rare diseases go through a lot of the same things we go through. Some of them are starting to win too. It’s easy to feel disheartened when NPD is part of your life, we haven’t won a ton of battles. Yet. The stories of triumph are out there. The director of the Everylife Foundation Mark Dant got the win. His son Ryan has MPS1 and they found a treatment in time. Mark sticks around though because there is so much work left to be done in the rare disease space. I wholeheartedly implore you to get involved with RDLA at your local level whenever you can because the rules for Rare Disease desperately need to change. 

Will we see you next year for Irish coffee at Kramer’s Books and Afterwords Cafe with Dr. Rami Ballout and the incredible NPC advocate Havi Martinez? Perhaps catch up with Dan & Dee Reynolds too?! Havi made beautiful buttons with pictures of our kids on them that really made it clear why we do what we do, thank you to the many special people who have been on this journey right along with me; in person and spirit. 

Shannon Reedy