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Narcolepsy Advocates Attended Rare Disease Week

March 9th, 2020

Narcolepsy Network sent 20 advocates to Washington, DC to be a voice for the narcolepsy community. Every year, the Rare Disease Legislative Advocates (RDLA), in coordination with the Everylife Foundation for Rare Diseases, hosts a free week-long event that brings together rare disease members from across the country to be educated on legislative issues, meet other advocates, and share perspectives with legislators.


The week kicked off on Monday, February 24 with a public meeting at the FDA, followed by a Congressional Caucus briefing the following day. The next day was a cocktail reception and documentary screening of Tin Soldiers, a film about the rare disease fibrodysplasia ossificans progressive. Then on Wednesday, February 26, the advocates received an entire day of training. Thy learned about specific “asks” that are important to the rare disease community.


These “asks” included:

Encouraging the FDA to Establish a Center of Excellence for Rare Diseases
Establishing a Center of Excellence for Rare Diseases would improve treatments and health outcomes for those suffering from these conditions. Developing therapies for small patient populations presents unique challenges and a Center of Excellence for Rare Diseases could help to improve treatments for some of the patients who need them most.


Supporting Funding for Orphan Products Clinical Trial Grants Program and Natural History Grants Program
The Orphan Products Clinical Trial Grants Program supports new and continuing research projects that test the safety and efficacy of promising new drugs, biologics, devices, and medical foods through human clinical trials in extremely vulnerable populations often with life-threatening conditions. Over 700 new clinical trials have been funded through this program to date. Orphan Products Clinical Trial Grants have supported the marketing approval of more than 60 orphan products for serious or life-threatening orphan indications.

Natural History Grants Program supports studies that advance rare disease therapy development through characterization of the natural history of rare diseases and development and/or validation of clinical outcome measures.

Reauthorization of the Newborn Screening Saves Lives Act
Diagnosis through newborn screening saves lives, improves healthcare outcomes, and reduces long term healthcare costs by allowing for detection and intervention at the earliest moment possible. Newborn Screening is the practice of testing every newborn for certain genetic, metabolic, hormonal, and functional conditions that are not otherwise apparent at birth. Federal newborn screening programs expired on September 30, 2019.


Rare Disease Congressional Caucus
This Caucus helps bring togther public and Congressional awareness to the needs of the rare disease community and creates opportunities to address roadblocks in the development of and access to crucial treatments. The Caucus gives a permanent voice to the rare disease community on Capitol Hill. The advocates were tasked with urging their legislators to join if they were not already members. To see if your legislator is a member of this caucus go to https://rareadvocates.org/rarecaucus/.


BENEFIT Act
The Better Empowerment Now to Enhance Framework and Improve (BENEFIT) Act would ensure that patient experience, Patient-focused drug development (PFDD), and related data, be considered as part of the benefit-risk assessment. This would send an important signal to all stakeholders that patient experience will be incorporated into the review process.



Medical Nutrition Equity Act
Medical nutrition intervention is a must for patient management who have Inherited Metabolic Disorders. The intervention must begin shortly after birth to prevent death, intellectual disability, and other adverse health outcomes. This act will provide coverage of medical formula and low-protein modified foods under federal health programs and private insurance.

Each advocate was asked to bring one of these topics to their member of Congress. To learn more about these topics, visit this page by RDLA.

After a day of training, the advocates were armed with knowledge on how to present their case to their legislators. They then spent the day talking with legisaltors and their staff to inform them on important issues that affect the rare diease community.

Rare Disease Week on Capitol Hill will take place next year on March 1-4, 2021. If you want to get involved, be sure to watch your email or follow us on our social media. Find more pictures of the week on our FacebookTwitter, or Instagram.

Special thanks to Brandi Hill (@brandihillcom) for photographing the advocates during Rare Disease Week. 

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Date Created: March 9th, 2020
Last Updated: March 9th, 2020

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