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.