United for Change: PWSA | USA Advocates Make a Powerful Impact During Rare Disease Week 2025

Rare Disease Week on Capitol Hill 2025 was a transformative experience for the Prader-Willi syndrome (PWS) community and the broader rare disease advocacy movement. From February 24–26th, 36 advocates from PWSA | USA, including 10 individuals living with PWS and members of the Advocacy Ambassador program, traveled to Washington, D.C., to share their stories, educate policymakers, and push for legislative action. 

This year’s event, organized by Rare Disease Legislative Advocates (RDLA) with additional events supported by Patients Rising, was the largest yet—bringing together nearly 1000 advocates from across the country, united by one goal: ensuring rare disease patients receive the care, treatments, and policy support they deserve. 

A Week of Powerful Advocacy on Capitol Hill 

PWSA | USA advocates participated in a series of high-impact events, engaging with legislators, industry leaders, and fellow advocates. 

Day 1: Building Connections and Raising Awareness 

1. *Rare Reels Screening & Reception – A powerful evening event featured documentary films that highlight the real-life struggles and triumphs of individuals living with rare diseases. Advocates, including members of the PWS community, participated in discussions on how storytelling can drive policy change. 
2.  
3. *Networking with Advocates – PWSA representatives connected with hundreds of rare disease advocates from all 50 states, strengthening collaborative efforts to push for legislative progress. 

Day 2: Legislative Conference – Preparing for Capitol Hill 

1. *Deep Dive Policy Training: Experts guided advocates through key legislative issues, including: 
2.      -The urgent need to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV)                 Program, which incentivizes rare disease drug development. 
3.     -The Accelerating Kids’ Access to Care Act (AKACA), aimed at reducing red tape so children              with rare diseases can access out-of-state specialty care without delays. 
4.    -The importance of sustained federal funding for biomedical research and public health                     agencies, ensuring continued progress in rare disease treatments. 
5.  
6. *Advocacy Strategy Workshops: Attendees refined their storytelling and legislative “asks” in preparation for meetings with members of Congress the following day. 

Day 3: Capitol Hill Meetings – Making Our Voices Heard 

On February 26, PWSA | USA advocates met face-to-face with Members of Congress and their staff, ensuring that PWS and other rare diseases remained a priority for the new Congress. Their advocacy focused on: 

1. *Expanding newborn screening programs to detect rare diseases earlier. 
2.  
3. *The urgent need to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, which incentivizes rare disease drug development. 
4.  
5. *Increasing funding for biomedical research that supports rare disease treatment development. 
6.  
7. *Strengthening the Rare Disease Congressional Caucus, encouraging more legislators to join in support of rare disease-focused initiatives. 

A Pivotal Moment for Rare Disease Research in Florida 

While advocates took to Capitol Hill, another major development was unfolding in Tallahassee, FL. Dorothea Lantz, PWSA | USA’s Director of Community Engagement, and her son Hunter, age 7 living with PWS, attended the official opening of the FSU Institute for Pediatric Rare Diseases (IPRD)—a groundbreaking new center dedicated to advancing research and treatment for pediatric rare diseases. 

During the event, Florida Representative Adam Anderson unveiled House Bill 907 – the Sunshine Genetics Act, a bold legislative initiative aimed at: 

1. *Providing free, opt-in newborn genetic screening for Florida families, setting a national model for early diagnosis and intervention. 
2.  
3. *Establishing a research consortium uniting Florida universities, children’s hospitals, and industry leaders to accelerate discoveries and treatment development for rare diseases. 

Rep. Anderson’s leadership on HB 907 reflects a growing commitment at the state level to improving outcomes for families affected by rare diseases. His remarks reinforced that Florida aims to become a leader in rare disease research and early intervention, aligning with the goals of national advocacy efforts. 

The Lasting Impact of Rare Disease Week 2025 

The convergence of advocacy on Capitol Hill and legislative advancements in Florida marked a turning point for the PWS and rare disease communities. 

Why This Matters: 

1. *Policymakers gained firsthand insight into the daily challenges of living with PWS, reinforcing the urgency of legislative action. 
2.  
3. *The FSU Institute for Pediatric Rare Diseases is now a reality, giving hope to families seeking better research and treatment options. 
4.  
5. *The Sunshine Genetics Act is poised to drive historic change in newborn screening and research collaboration. 

PWSA | USA remains committed to pushing these efforts forward. Advocacy doesn’t end when Rare Disease Week is over—it continues in every conversation, every policy push, and every effort to ensure that rare disease patients are never forgotten. 

Join Us in the Fight for Change 

To stay engaged and support ongoing advocacy efforts: 

1. *Contact your legislators and urge them to support rare disease policies. Find your representative HERE. 
2.  
3. *Follow PWSA | USA on social media for advocacy updates. 
4.  
5. *Share your story to help build momentum for change. 

The week was a testament to the power of collective action, proving that when rare disease advocates unite, real change happens. Together, we are making history. 

Learn more about participating in Rare Disease Week advocacy at Join Rare Disease Week Advocacy from Home – Prader-Willi Syndrome Association | USA