The last day of February is designated internationally as World Rare Disease Day and organizations representing patients and advocates dealing with rare diseases come together to raise awareness through local events and activities. Additionally, each year in the United States, hundreds of rare disease advocates convene in Washington, D.C. to meet with their members of Congress to continue to advocate for rare disease research and funding for vital agencies such as the National Institutes for Health (NIH) and the Food and Drug Administration (FDA).
This year, the non-profit organization that I helped create in honor of my youngest daughter, Hannah, who was born with and passed away from a rare disease, held its first 5K charity event called Vegas Cares About Rare 5K/1M on February 25th in Las Vegas to celebrate World Rare Disease Day. With over 500 people in attendance, this was by far one of our most successful events. While most of the attendees participated as a result of knowing a child with a rare disease, many did not know that 1 in 10 Americans are affected by rare disease or that there are over 7,000 rare diseases in existence. Events like these serve as an opportunity to help educate the general public and raise awareness about rare disease issues.
The day after our successful 5K event, my wife Carrie and I traveled to D.C. to participate in a week full of events recognizing World Rare Disease Day. On Monday, we visited the NIH to meet other advocates and learn about the latest innovations in research and development. On Tuesday, I had the privilege to moderate the Legislative Advocacy Conference organized by the Rare Disease Legislative Advocates (RDLA) group who supports the advocacy of all rare disease patients and organizations. This was my 4th year in attendance and I was happy to share my experiences with other advocates in attendance.
Wednesday was the day on the Hill. Grouped by state, advocates met with members of Congress and/or their staff to stress the importance of research and funding and to develop and maintain a member-constituent relationship to enhance communication for the upcoming year. Our Nevada group met with the staff of Senator Heller, Congresswoman Rosen and Congressman Kihuen. During our last meeting, we were lucky to meet with our newest Senator, Catherine Cortez Masto, who indicated her support of our issues.
The best part of these meetings is to connect with other advocates, representing hundreds of other rare diseases. Individually, our voices are quite soft as our numbers are not as large as more common disease groups such as cancer, HIV or autism. When we all get together, our voice becomes louder. Research and funding affects all of us and can even benefit people who are not suffering from a rare disease or condition.
On the actual World Rare Disease Day, President Trump gave his address to Congress. Our community was thrilled that he not only mentioned World Rare Disease Day, but he also highlighted the strength of one of our own, Megan Crowley, affected by Pompe Disease, who was in attendance. The President acknowledged some of the issues that many rare disease patients have experienced, particularly concerning the burdensome approval process at the FDA. This was the first time that a sitting President mentioned World Rare Disease Day in a Congressional address, thereby raising much needed awareness.
The work does not stop at the end of World Rare Disease Day. Advocates must continue to communication with their members of Congress and continue to raise awareness through local events across the country to keep this issue on the top of the minds of those in power. Those interested in keeping up-to-date on the issues affecting the rare disease community are recommended to visit the RDLA website and join their mailing list.
In the meantime, planning is already starting for our Vegas Cares About Rare event for 2018. Learn more through our website – vegascaresaboutrare.org
Robert Ostrea is the President of the Little Miss Hannah Foundation, a 501(c)3, non-profit organization which was created to honor his 3-year-old daughter, Hannah, who lost her battle to Gaucher Type 2/3, a rare metabolic genetic disorder. The organization’s mission is to help enhance the quality of life for young children diagnosed with life-limiting rare diseases, undiagnosed complex medical needs, and children who have been placed in hospice or palliative care throughout Southern Nevada. You can learn more about the Little Miss Hannah Foundation through their website – www.littlemisshannah.org