Contributed by Dorothea Lantz, PWSA | USA Director of Community Engagement
We’re celebrating a monumental victory for the rare disease community! On December 1, 2025, the U.S. House of Representatives passed the Give Kids a Chance Act — a landmark step toward restoring critical pathways for treatments that children with rare diseases and pediatric cancers so desperately need.
Why This Matters
- – The Give Kids a Chance Act re-authorizes the Rare Pediatric Disease Priority Review Voucher Program (PRV program), which expired at the end of 2024.
- – It revives incentives for drug developers to create therapies addressing rare pediatric conditions — including rare genetic syndromes and childhood cancers — markets that are too small for typical drug-development economics.
- – It levels the playing field: children deserve the same chance at breakthroughs that adults receive. The law expands authority for the U.S. Food and Drug Administration (FDA) to require testing of novel drug combinations in children — an opportunity that could open the door to more effective, combination-based treatments.
- – It restores confidence that rare disease communities will not be left behind simply because their conditions affect fewer individuals. For families like ours — raising a child with a rare condition such as Prader-Willi syndrome (PWS) — the revived PRV program and renewed regulatory incentives are a lifeline.
What This Means for the PWS Community & Other Rare Disease Families
For too long, many rare and pediatric diseases have lacked sufficient research investment — not because of a lack of scientific promise, but because of limited financial incentive for developers. The Give Kids a Chance Act re-establishes those incentives.
- – It increases the likelihood that companies will pursue therapies tailored to rare pediatric conditions.
- – It helps ensure that future innovations — gene therapies, small molecules, or new drug combinations — remain viable even when the affected population is small.
- – It brings hope to families who have often felt they were running out of options, reminding us that advocacy works and that lawmakers are listening.
What’s Next — And Why We Must Keep Pushing
While today’s House vote is a profound win, we still need the Senate to act. We encourage all members of the PWSA community — families, advocates, friends — to raise their voices: contact your Senators, share your stories, remind them of the children waiting for hope. Rare Coalition+1
Let’s use this moment of victory as fuel for renewed advocacy. Let’s amplify the message: Rare is not hopeless. Rare is urgent. Rare deserves action.
With Gratitude & Determination
To the sponsors, cosponsors, coalition partners, advocates, and every family who amplified their story — thank you. Today we celebrate together. And tomorrow, we keep fighting — because our children deserve the cures that are within reach.
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.
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.
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.
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.
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.