On June 17, 2021, representatives from PWSA | USA, FPWR and experts in the field of Prader-Willi syndrome research met with the FDA’s Center for Drug Evaluation and Research to discuss issues related to PWS clinical trials.
The purpose of this meeting was to promote dialogue between PWSA | USA, FPWR and members of the PWS patient community to ensure that the FDA has a full understanding of the unique challenges of PWS. PWSA | USA and FPWR shared information on the dire unmet medical need, the community’s tolerance for risk and uncertainty of benefit as it relates to new treatments, and the impact of the COVID-19 pandemic on PWS clinical trials. We also discussed the PWS community’s perception as to what constitutes meaningful changes in addressing hyperphagia and other behavioral aspects of PWS, the unique challenges of performing clinical trials in the PWS population, and considering those concerns, discussed how to efficiently advance new medical products that may be safe and effective for PWS.
After an opening statement from FDA, PWSA | USA, and FPWR led the discussion, sharing the perspectives and concerns of the PWS community, with time for questions and discussion with FDA staff on specific topics of interest.
Following the Patient Listening Session, representatives from PWSA | USA and FPWR compiled notes that were taken into one organized and informative document. This summary provides key information shared at the meeting and reactions from the FDA.
Please click here to read the full summary of PWSA | USA and FPWR’s Patient Listening Session meeting with the FDA.
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.