Bringing Together the PWS Patient Voice!
Prader-Willi Syndrome’s First Externally Led Patient-Focused Drug Development Meeting
Thank you to everyone who shared their voice in-person and virtually at the FDA Externally-Led Patient-Focused Drug Development Meeting. Your time and commitment to our loved ones living with PWS is truly incredible.
A recording of the EL-PFDD meeting is now available to view below. As for next steps, we filed the "Voice of the Patient Report" with the FDA, which includes the testimonials shared both during the EL-PFDD, and those collected on our website. You can view this report by scrolling below.
Voice of the Patient Report
On May 1st, the beginning of PWS Awareness Month, we’re thrilled to announce the release of the Voice of the Patient Report. This timely filing marks a significant moment for the Prader-Willi syndrome community! The report gives crucial insights that amplify the perspective of individuals and families affected by PWS and sheds light on aspects of PWS that may not be fully captured in drug trials alone. Integrating the patient’s voice throughout the drug approval process for PWS treatments is essential for the U.S. Food and Drug Administration’s (FDA) comprehensive understanding and impactful decision-making.
The Voice of the Patient Report was created following the June 2023 PWS Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting and is compiled of testimonials shared during the EL-PFDD meeting, as well as those submitted virtually.
PWSA | USA, FPWR, and IPWSO extend heartfelt gratitude to the entire PWS community for contributing to the success of this project. To truly amplify the patient’s voice, our entire community must commit to ongoing advocacy efforts. This entails incorporating patient perspectives into all facets of processes and systems, ensuring genuine and enduring change. It’s not solely about isolated advocacy initiatives—it’s about steadfast dedication to embracing and elevating the voices of those directly affected by Prader-Willi syndrome.
You can read the Voice of the Patient Report by clicking the button below.
FAQ’s
This meeting provides an opportunity for patients, caregivers, and advocates to share their perspectives and experiences with the FDA and drug developers. By attending this meeting, you can make your voice heard and help shape the development of new treatments and therapies for Prader-Willi syndrome. Together, we can work towards improving the lives of those affected by this rare and complex genetic disorder.
You can attend the meeting either in-person or virtually via Zoom. The in-person meeting will take place during PWSA | USA’s 37th National Convention on Thursday, June 22, 2023 from 12:00 – 4:30 p.m. EST at the Hilton Orlando Buena Vista Palace in Orlando, FL. The virtual meeting will take place over Zoom and will be a “live look” into the EL-PFDD meeting for those who are not able to attend in-person.
A recording will be available on PWSA | USA’s website following the meeting.
No, this meeting is free to attend and is open to the public. For those planning to join us in-person, a free lunch will also be provided.
You can register for the EL-PFDD meeting when you register for PWSA | USA’s 37th National Convention at https://pwsausaevents.cventevents.com/37thPraderWilliConvention. You will see on the 2nd page of our registration process the option to select how many people in your group will be attending the in-person EL-PFDD meeting.
If you have questions or experience any issues during the registration process, contact us at info@pwsausa.org or (941) 312-0400.
Register for the virtual EL-PFDD meeting at https://us02web.zoom.us/meeting/register/tZcqfuuupz8iGN2NmiL9qGDkGJ2Kt67uXP1v. Once you have registered, you will receive a Zoom meeting link and calendar invitation to attend. Make sure to log in to the Zoom meeting link on June 22, 2023.
Those who attend virtually via ZOOM will have the option to have their cameras on or off and default microphone setting will be mute. You will have the opportunity to pose questions to the panelists via the chat during the facilitated discussion and engage in live audience polling.
The agenda will consist of speakers and panelists who will educate the FDA about living with PWS. There will be two panels consisting of PWS parents/caregivers who will provide the FDA with a glimpse of the everyday challenges. This meeting also includes facilitated discussions to provide additional perspectives from people in the audience with PWS and their caregivers.
Everyone! We want to see as many members of our community as possible to attend either in-person or virtually.
Yes! All are welcome to attend. We do want to make sure our parents are aware there may be information discussed that could be sensitive and/or uncomfortable for your child to hear. We will also have a childcare option available during the EL-PFDD meeting time if you prefer to not have your loved one attend. If you are interested in the childcare opportunity, please email us at info@pwsausa.org with the child’s name and age.
Yes! We will have a free boxed lunch available to all EL-PFDD in-person attendees from 11:30am-12:00pm ET.
The meeting will take place in English. Closed captioning will be available for those who attend virtually via ZOOM in ~30 languages. American Sign Language (ASL) interpretation will be available for those attending in-person and virtually.
The EL-PFDD meeting will be recorded and available online. By attending the meeting you consent to all recordings.
This EL-PFDD meeting is designed to engage people living with PWS and their caregivers to elicit their real-life experiences including impact on quality of life and gaps in the current standard of care. With this information, the goal is to enable the FDA and other stakeholders to knowledgeably review and approve future therapies that addresses the needs of the PWS community.
We have two requests! First, please avoid mentioning any specific drugs or trials. You can talk about treatment generally or about your trial experience but avoid mentioning any companies or brand names. Second, we ask that you keep your comments short. We anticipate having close to 500 people participating either in person or virtually, and we want to hear from as many people as possible.
PWSA is collecting testimonials from the community online before, during and after the EL-PFDD meeting: https://www.pwsausa.org/el-pfdd-testimonial/. The testimonials may be included in the Voice of the Patient Report, which becomes a published public online report.
The images and quotes you provide may be included in the Externally-Led Patient-Focused Drug Development meeting, in the Voice of the Patient Report, or in other associated documents.
We will write the Voice of the Patient Report. This will be sent to the FDA and will be used to help in their decisions to approval potential treatments for PWS. The Voice of the Patient Report will be publicly posted on PWSA | USA’s website
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.