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Rare Disease Week 2018

April 6th, 2018
Narcolepsy advocates standing in front of the White House.

Heidi Hartmann-Rothe, Shannon Burkoth, Sharon O’Shaughnessy, and Keith Harper in front of the White House.

On February 25th, seven Narcolepsy Network advocates and board members joined 750 other advocates in Washington, DC to participate in Rare Disease Week. The events, organized by the Everylife Foundation, are an opportunity for patients, family, and rare disease advocates to connect with each other and meet with members of Congress.

What is a rare disease?

Narcolepsy is one of more than 7,000 rare diseases, which are classified as having an incidence of less than 200,000 cases per year in the United States. About 80% of rare diseases are genetic in nature. Even though 1 out of every 10 people have a rare disease, funding and research remain scarce. Rare Disease Week activities aim to change this equation for patients and their families, by enabling advocates to share personal stories and deliver specific asks to lawmakers in Congress.

Why did we participate?

An important part of Narcolepsy Network’s mission is to advocate for both our members and the community of people with narcolepsy at large. We do this in a few different ways, with the most visible event being Rare Disease Week. Meeting with lawmakers and their staff gives us the opportunity to influence legislators and their staff at both the state and national level. Our advocates share their personal stories, experiences, and facts about narcolepsy. Each group of advocates (we break into states and districts, based on where each advocate lives) make specific asks to each Congressperson. It is truly democracy in action!

What happened during Rare Disease Week?

On Sunday, advocates got together to network over cocktails and watch a screening of The Ataxian, a film about Freidreich Ataxia patients that bike across the United States in the Race Across America. Every year a different rare disease documentary is featured. Senators and Representatives often stop by to speak and welcome everyone to Washington.

On Monday, we gathered at the Legislative Conference to learn how to be effective advocates. Inspirational speakers like Michael Manganiello (a founder of ACT UP, which was created in 1987 to find a cure for AIDS/HIV) told us about what they did to get the government’s attention during the 1980s and ’90s. Dr. William Gaul spoke about the important work being carried out at the NIH (National Institutes of Health), and how they need advocates to continue pushing for federal funding. Dr. Debra Lewis (from the FDA) spoke about how important our efforts are, especially since advocates must go through Congress to reach the FDA.

In the afternoon we attended breakout sessions on the OPEN Act and other specific legislative asks. We attended a mock meeting with lawmakers, to prepare for any curveballs that might come our way. (Some pro tips: be prepared to give your pitch with very limited time, keep your stories and conversation non-partisan, and always finish with a specific ask). We practiced and refined the “pitches” that we would give the next day.

On Tuesday, we got up early and listened to Joel White (from Horizon Government Affairs) deliver some final advice on meeting with members of Congress. In a serendipitous turn of events, we even met a fellow advocate who has cataplexy without narcolepsy (which is incredibly rare!), and helped him when he had a cataplexy attack during breakfast.

Then, we went off to Capitol Hill with our state- and district-specific groups, to meet our Senators and Representatives. Each group shared their stories, basic facts about each rare disease, and together as a group we discussed the details of our specific asks.

On Wednesday, some of our advocates attended the Rare Disease Congressional Caucus Briefing.

Narcolepsy Network board members and staff, including our Executive Director Dr. Eveline Honig, traveled to the FDA to discuss the seriousness of narcolepsy, and the need for expanded treatments with senior staff. You can read more about our meeting with the Food & Drug Administration here.

On Thursday, advocates traveled to the NIH. I’ll let Heidi Hartmann-Rothe, one of our advocates and a co-organizer of the New York Narcoleptics support group, tell you more:

On March 1, 2018, for the first time, I attended Rare Disease Day at the National Institutes of Health (NIH) as a person with narcolepsy. The global theme was research (continued forward from 2017), and this year’s slogan was: patients are not only subjects but also proactive actors in research.

The event was open to the public, and was well attended by researchers, scientists, stakeholders, policymakers, patient advocates, and people/caregivers affected by rare disease alike.

The NIH Clinical Center studies more rare diseases than anywhere else.

One resounding theme came across loud and clear: collaboration! This means cooperation, communication and organization within and between the rare disease community, and across researchers, funders and policymakers. Imagine a future where we move toward making collaboration the norm across health care and across disease conditions, in an atmosphere of hope, love, and non-competition. All the while, with a focus on “meaningful engagement” of patients, families, communities, advocates, and researchers; not just as a tool but with a greater scope in mind. Who takes ownership/accountability of meeting patient/caregiver needs, and how do we help people move “from despair to hope and action? A strong, unified rare disease community that provides timely and appropriate communication and education; support and resources; advocacy and encouragement of involvement; and commitment to balanced leverage of funding.

Remember: each connection we make as a person with a rare disease can drive this process forward, every patient counts.

What were our asks?

Co-Sponsor the OPEN Act
The OPEN Act would incentivize pharmaceutical companies to develop more treatment options for rare disease patients. It would also help to  generate massive amounts of clinical data, which is needed to for research into transitioning more off-label therapies into on-label therapies. This is very important for patients, and the availability of on-label treatments is key for insurance purposes. Additionally, when patients use an off-label treatment, they don’t benefit from all of the research into quality, safety, efficacy, etc that comes with on-label treatments.

Support the Advancing Access to Precision Medicine Act
This legislation would reduce barriers to genetic testing and whole genomic sequencing important science that matters greatly  for children with rare diseases.

Join your state’s  Rare Disease Congressional Caucus
These groups meet frequently throughout the year with lawmakers and serve as a direct conduit to the patients in each state.

Increase funding for the NIH and FDA
The NIH is a world leader when it comes to funding research and supporting the development of treatments for people with rare diseases. Both the FDA and NIH are vitally important to people with narcolepsy, and all other rare diseases.

What were the results?

  • The OPEN Act is awaiting passage by the Senate—it was passed by the House in July of 2015, and has been reintroduced several times since with amendments. Senator Orrin Hatch (R-UT) is the primary sponsor of this bill.
  • 5 Senators and 9 Representatives joined their states’ respective  Rare Disease Congressional Caucus
  • Congress approved an increase of $3B in funding for the NIH, and the FDA received an increase of $130M.

How can I get involved?

Narcolepsy Network is always looking for volunteer advocates. Participating in events such as this are fantastic opportunities to get involved and directly impact the lives of people with narcolepsy in the United States. We plan to expand access to these opportunities in the next year. Please sign up for our newsletter to stay updated, or join the network to support our advocacy efforts.

Written by Keith Harper & Heidi Hartmann-Rothe

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Date Created: April 6th, 2018
Last Updated: December 8th, 2020