Watch the video to get a full recap, and see how many times you can see Wayne!
Video: RDW 2025 | Stronger Together
A Message From Wayne
Over the last week of February, I was privileged to represent HDSA with over 1,000 other advocates from a large list of rare diseases across the US sharing their stories with our Members of Congress during Rare Disease Week. This was the 14th annual event hosted by EveryLife Foundation for Rare Diseases. Their mission and purpose is to improve health outcomes by driving change through evidence-based policy, leading science-driven policy and regulatory research, activating the community to advocate for their rights and needs and strengthening the rare disease community.
In the days leading up to the lobbying efforts, we were educated on a variety of legislative and policy proposals impacting the entire rare disease community.
Some facts about the Rare Disease Community:
- a rare or orphan disease has been defined as having fewer than 200,000 diagnosed/affected Americans (1 in 2,000 Europeans)
- included are tropical or neglected diseases and many pediatric diseases,
- there are an estimated 10,000 identified rare/orphan diseasesir
- over 30 million Americans are currently affected, almost 10% of our total population
- 72% are genetically sourced
- more than 95% do not have an FDA approved treatment
- patients wait an average diagnostic odyssey time (time it takes to be diagnosed) of 6+ yrs and involves 17 medical providers
- average cost of diagnostic odyssey ranges between $86-517,000
- in 2019, Economic Impact was over $1 Trillion
- over 70% have pediatric onset
- 39% of patients travel more than 60 minutes for care, many having to travel outside their state
- more than 50% of all American children rely on Medicaid for their healthcare needs
- it takes an average of 15 yrs for a drug to be developed and approved by the FDA
- delays in receiving diagnosis result in an average of $220,000 of avoidable costs
On the last day my fellow Illinois advocates and I visited the offices of Rep Sean Casten (D-6th Dist), Rep Robin Kelly (D-2nd Dist), and my own Rep Danny Davis (D-7th Dist). In the afternoon we sat with representatives from Sen Dick Durbin (D) and Sen Tammy Duckworth (D).
During these meetings we shared our personal stories while also asking for support. Our legislative "asks" included:
#1 Ensuring the Continuing, Steady and Robust Federal Biomedical Research Funding & Public Health Agency Support
Among the federal agencies that support our community are:
- National Institutes of Health (NIH)
- Food & Drug Admin (FDA)
- Centers for Disease Control and Prevention (CDC)
#2 Reauthorizing the Rare Pediatric Disease Priority Review Voucher (PRV) Program at the FDA
Developing drugs for rare pediatric diseases is challenging due to the small populations affected, difficulties associated with
conducting clinical trials for children, delays in diagnosis, and more.
The Creating Hope Act expanded Priority Review Voucher Program to include drugs that treat rare pediatric diseases in 2012.
The Rare Pediatric Disease PRV Program was last reauthorized in 2020 for a 4 yr period.
The Give Kids a Chance Act of 2025 (H.R. 1262) includes a 5 yr reauthorization.
#3 Include Accelerating Kids Access to Care Act (AKACA) in the first available legislative package.
Will ensure better, faster healthcare for kids who need it most by reducing the burden on the doctors who treat them.
#4 Ask our Members of Congress to join the Rare Disease Congressional Caucus (RDCC)
The RDCC is a bipartisan, bicameral caucus that works to raise awareness of rare diseases and gives a permanent voice to the rare disease community.
In a follow-up, the Illinois General Assembly declared Wed, March 5, to be Rare Disease Day in the State of Illinois.
In summary: ...in order to raise awareness of rare diseases, support individuals and families who struggle with rare diseases, bring attention to the need for research and funding to support the discovery and development of therapies designed to treat and potentially cure rare diseases, and support the continued work of the Illinois Rare Disease Commission.
We missed '25 Illinois Rare Disease Day activities but will be involved in '26.
Finally, over 15 contacts from various other rare disease communities within Illinois will be working together with HDSA on collaborative efforts in support of legislation, cross-supportive education, outreach programs, and advocacy work within our state.
Hard work, creativity and collaboration lead to hope.... and hope for a cure is at the very core of our mission.
Wayne Galasek
HDSA Illinois Chapter