Rare Disease Week 2020: Working Towards a Better Future

This year, the entire Patient Worthy team traveled up to Washington, DC for Rare Disease Week 2020. This event is always a big one for the rare disease patient community. Hosted and organized by the EveryLife Foundation for Rare Diseases, the event consists of critical opportunities for advocacy, networking, and education. Rare Disease Week was held from February 25th to the 28th this year.

The week started out on Tuesday with a briefing from the Rare Disease Congressional Caucus. This bicameral and bipartisan caucus consists of legislators that work to spread awareness about rare diseases. The rare disease community is actively seeking to get more lawmakers to join. Later that evening there was a screening of a very powerful documentary called Tin Soldiers. This film was focused on the challenges faced by patients with an extremely rare disease called fibrodysplasia ossificans progressiva (FOP), an unusual and tragic illness that causes body tissue to turn to into bone, eventually robbing patients of movement. The documentary interviewed patients with the disease from around the world, and explored how they cope with the diagnosis, the struggle to find an effective treatment, and the undiagnosed patients that are still out there in need of help.

Wednesday consisted of a day long Legislative Conference. The day was focused on the state of legislation that was relevant to the rare disease community and preparing attendees for Hill Day, when we would meet with our representatives and senators to advocate for legislation that would benefit rare disease patients. There were a variety of sessions and speakers that conveyed content that gave more in-depth information on the policy initiatives that we wanted to see become law. Other sessions focused on tips for lobbying, such as how to approach the conversation and display professional and courteous conduct. We also attended a meetup in the evening of YARR (Young Adult Representatives of RDLA) where we got to engage with several younger patients (RDLA = Rare Disease Legislative Advocates).

The next day was of course “Hill Day” which consisted almost entirely of meeting and speaking to staffers from the offices of our representatives and senators. Attendees were divided up by state to determine who they would meet with.

There were a variety of “asks” that we presented in the meetings. These asks included:

  • Supporting the creation of a Rare Disease Center of Excellence at the FDA, which would help focus expertise on rare conditions at the agency and help improve the development of rare disease treatments
  • Vote in favor of the Newborn Screening Saves Lives Reauthorization Act, which is currently bogged down in the Senate (until passage, Federal screening programs remain expired)
  • Support increasing funding for the FDA’s Orphan Products Clinical Trial Grants and Natural History Grants Programs
  • Joining the Rare Disease Congressional Caucus
  • Support the BENEFIT Act, which will allow for the incorporation of patient-focused drug development and patient experience data to be incorporated in the benefit-risk framework currently followed by the FDA
  • Sponsor the Medical Nutrition Equity Act, which would improve access to medical foods that are an essential part of treatment for certain rare disease patients.

That evening we also attended a Rare Artist Reception that featured many impressive artworks created by rare disease patients. Many of the artists spoke about the art and how their experiences as patients had inspired them to create.

The final day was Rare Disease Day, which was held at the National Institutes of Health (NIH). A diverse array of advocacy groups had display tables set up and there were presentations throughout the day featuring some of the latest achievements of NIH researchers and powerful stories from rare disease patients.

Overall, we had a great time networking, covering the event on social media, interviewing patients, and getting involved in advocacy.

To learn more about the activities of the EveryLife Foundation for Rare Diseases, click here.

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