With your support, FPWR and PWSA | USA have been granted a meeting with the FDA, which will take place via teleconference later this month. This small, non-public meeting will allow FPWR and PWSA | USA to connect firsthand with the FDA staff and share important information that should be considered during the review of new drugs for PWS. While the meeting will not be about a specific drug, it will allow us to share our rare communities’ perspective and ensure the FDA has a full understanding of the unique challenges of PWS, with a particular focus on issues related to clinical trials. During the meeting we will:
- Review PWS patient experience data that documents unmet medical need, severity of PWS, and treatment preferences and priorities
- Share the impact of the COVID-19 pandemic on PWS families and its implications for clinical trials
- Share the community’s tolerance for risk for even modest improvements in PWS-associated behaviors
- Discuss the Agency’s perspective on how regulatory flexibility can be applied to efficiently advance new medical products that are safe and effective for PWS
- Invite the FDA to ask further questions that will help them better understand our treatment needs and preferences moving forward
We thank our community of supporters for taking action and supporting our joint advocacy efforts. We look forward to sharing a post-meeting summary with you soon!