In a powerful week of advocacy, Dorothea Lantz, Director of Community Engagement at PWSA | USA, represented Prader-Willi syndrome (PWS) on Capitol Hill and at the 2024 NORD Breakthrough Summit in Washington, D.C., from October 20–22. The Summit gathered leaders across the rare disease community, including patient advocates, healthcare providers, researchers, and policymakers, all united to tackle issues in rare disease advocacy, research, and healthcare policy. Key discussions covered equitable healthcare access, regulatory challenges, and the need for more inclusive healthcare solutions for rare disease patients.
At the Summit, a major focus was placed on creating streamlined drug development processes and advocating for policies that support rare disease treatment innovations, essential for conditions like PWS, given the small and diverse patient populations. Panels explored patient-centered care models and the critical need for robust policies to support orphan drug development. The event underscored the importance of cross-sector partnerships in driving meaningful change for the rare disease community.
Building on this momentum, Lantz joined forces with KPM Group, a D.C.-based public affairs firm, and Jill Yersak, Senior Director of Patient Advocacy and Government Affairs at Soleno Therapeutics, for advocacy meetings with congressional offices. These meetings aimed to elevate awareness of PWS and sought support for key legislation, including:
- Regulatory Support
Addressing the unique challenges of rare disease drug development, the team advocated for S.4583, the Creating Hope Reauthorization Act, which aims to extend the Rare Disease Pediatric Priority Review Voucher Program through 2030, incentivizing pediatric rare disease treatments.
- Improving Access to Care
They urged support for S.2372, the Accelerating Kids’ Access to Care Act, a bill to streamline out-of-state Medicaid provider enrollment, making it easier for children with rare diseases to access specialized care across state lines.
The group held discussions with representatives from the offices of Sen. Bob Casey (D-PA), Sen. Chris Coons (D-DE), Sen. Tim Scott (R-SC), Sen. Markwayne Mullin (R-OK), Rep. Doris Matsui (D-CA-07), Rep. Kevin Mullin (D-CA-15), Rep. Kat Cammack (R-FL-03), and Sen. Dick Durbin (D-IL). A key highlight came from a meeting with Kelly Kim, Legislative Correspondent for Sen. Chris Coons, who showed strong support for including PWS as a designated topic area within the Peer Reviewed Medical Research Program (PRMRP), an initiative that could significantly advance PWS research and funding opportunities.
Additional advocacy asks included support in identifying state-level contacts to recognize PWS as an intellectual and developmental disability and insights into each office’s legislative priorities for the remainder of the session.
This impactful week of meetings and collaborations further cemented PWSA | USA’s commitment to driving forward policies that ensure healthcare access, advance research, and foster innovation for the PWS community and the broader rare disease space. Through these efforts, PWSA | USA is working to secure a brighter future for those living with PWS and all rare diseases, championing policies that prioritize their unique needs and challenges.
Jennifer Bolander has been serving as a Special Education Specialist for PWSA (USA) since October of 2015. She is a graduate of John Carroll University and lives in Ohio with her husband Brad and daughters Kate (17), and Sophia (13) who was born with PWS.
Perry A. Zirkel has written more than 1,500 publications on various aspects of school law, with an emphasis on legal issues in special education. He writes a regular column for NAESP’s Principal magazine and NASP’s Communiqué newsletter, and he did so previously for Phi Delta Kappan and Teaching Exceptional Children.
Evan has worked with the Prader-Willi Syndrome Association (USA) since 2007 primarily as a Crisis Intervention and Family Support Counselor. Evans works with parents and schools to foster strong collaborative relationships and appropriate educational environments for students with PWS.
Dr. Amy McTighe is the PWS Program Manager and Inpatient Teacher at the Center for Prader-Willi Syndrome at the Children’s Institute of Pittsburgh. She graduated from Duquesne University receiving her Bachelor’s and Master’s degree in Education with a focus on elementary education, special education, and language arts.
Staci Zimmerman works for Prader-Willi Syndrome Association of Colorado as an Individualized Education Program (IEP) consultant. Staci collaborates with the PWS multi-disciplinary clinic at the Children’s Hospital in Denver supporting families and school districts around the United States with their child’s Individual Educational Plan.
Founded in 2001, SDLC is a non-profit legal services organization dedicated to protecting and advancing the legal rights of people with disabilities throughout the South. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act. Recently in November 2014, Jim retired.