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FPWR and PWSA | USA Engage FDA in Patient Listening Session

More than 60 FDA representatives attended the 1-hour meeting to learn more about PWS and the needs of our community.

On June 17, 2021, FPWR and PWSA | USA engaged the FDA in a Patient-Led Listening Session to share our community’s experiences related to Prader-Willi syndrome (PWS). The purpose of this meeting was to promote dialogue between the FDA and members of the PWS patient community to ensure the FDA has a full understanding of the unique challenges of PWS and issues related to clinical trials.

More than 60 FDA representatives attended the 1-hour meeting along with representatives from FPWR, PWSA | USA and advocates from the PWS research field:

  • FDA PLS SUMMARY DOWNLOADTheresa Strong*, Director Research Programs, FPWR, parent of an adult with PWS
  • Rob Lutz*, Member Board of Directors, PWSA | USA, parent of an adult with PWS
  • Elizabeth Dykens*, Professor, Vanderbilt University
  • Paige Rivard, CEO, PWSA | USA, parent of a child with PWS
  • John Walter, CEO, FPWR
  • Susan Hedstrom, Executive Director, FPWR, parent of a child with PWS
  • Lauren Schwartz-Roth, Clinical Psychologist, FPWR, parent of an adult with PWS
  • Elizabeth Roof, Senior Research Specialist, Vanderbilt University
  • Caroline Vrana-Diaz, Research Project Coordinator, FPWR

What is a Patient Listening Session?

Patient Listening Sessions allow patients, caregivers, and their advocates to share their experiences and perspectives by talking directly with FDA staff. The sessions can play an important role in drug development and safety as they help the FDA better understand what is most important to our specific community as medical products are being developed. Listening sessions cannot be used to discuss specific medical products but are an opportunity to share disease-related experiences and perspectives.

What Was Covered During the PWS Listening Session?

Throughout the hour-long Listening Session, we provided insight and perspective on the unique challenges of PWS, with particular focus on issues related to clinical trials. We discussed how moderate changes in PWS-associated symptoms could lead to meaningful improvements in daily living and the ability of the person with PWS to achieve more independence. We also shared our community’s tolerance for risk as it relates to new treatments, and the impact of the COVID-19 pandemic on PWS clinical trials. We provided valuable information about the unmet medical needs of our community to the lead review division and FDA colleagues.

During the meeting, we provided information and resources on the following areas of awareness:

  • Review of patient experience data from the PWS community including: the severity of PWS, unmet medical need, treatment preferences, and risk tolerance
  • Summary of the perspective of individuals with PWS
  • Impact and meaningfulness of modest improvements in PWS-associated behaviors
  • Impact of COVID-19 on PWS families and implications for clinical trials
  • Discussion of how PWS patient experience informs clinical trial conduct and interpretation (benefit: risk profile); feasibility challenges for future PWS trials

A full summary of the listening session is available here >>





Topics: News

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.