Share your voice with the FDA and Other Key Stakeholders.
Please complete by November 22, 2024
Angelman Syndrome EL-PFDD Meeting January 2025
Landmark event to raise the voice of the Angelman syndrome community with drug developers and the Food and Drug Administration (FDA).
August, 1, 2024 – We are excited to announce that on January 29, 2025, the Angelman syndrome community will have a unique opportunity to share our experiences on living with the condition, including our perspectives on treatments for our loved ones, with drug developers, clinicians, and FDA staff at a virtual externally-led patient-focused drug development (EL-PFDD) meeting.
Co-hosted by the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation, Hope in Action: an EL-PFDD Meeting on Angelman Syndrome, is a platform for our community to share insights and priorities directly with the FDA and other stakeholders, ultimately shaping the future of therapy development.
Goals of the Half-Day Meeting
Spotlight
Spotlight
Spotlight
To spotlight what matters most to caregivers on behalf of individuals living with Angelman syndrome.
Improve
Spotlight
Spotlight
To improve understanding of the challenges of living with Angelman syndrome and aspects of the condition the community would most like to address with new treatments.
Inform
Spotlight
Inform
To inform drug development programs and related FDA reviews of potential Angelman syndrome treatments.
FAST | ASF • EL-PFDD Community Webinar Recording
Learn more about the importance of the EL-PFDD Meeting on January 29, 2025 and how you can get involved.
Frequently Asked Questions
Please email ryan.fischer@cureangelman.org if you cannot find an answer to your question.
The Externally Led Patient-Focused Drug Development (EL-PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. The EL-PFDD initiative started in 2012 as part of FDA’s commitments under the Prescription Drug User Fee Act (PDUFA) V. After conducting FDA-led PFDD meetings, FDA recognized there are many more diseases/conditions that can be addressed beyond those that were planned and conducted by FDA.
To help expand the benefits of FDA’s EL-PFDD initiative, in 2015, FDA announced the opportunity for externally-led (EL-PFDD) meetings.
EL-PFDD meetings are planned and hosted by patient organizations, with the input of FDA staff, and use the process established by FDA-led EL-PFDD meetings as a model.
It's the singular opportunity for perspectives and insights to be heard for the first time by the FDA by the parents and families of Angelman syndrome individuals. As parents and caregivers, you're truly the ones who are boots on the grounds, the experts, on what is pleasant and what is not.
Yes! If both primary caregivers wish to complete the survey, please do so but do not share your answers with each other.
We view this as a launching pad for a much broader array of initiatives. The EL-PFDD Meeting itself will not repeat but things will come out of the meeting itself - surveys, additional focus groups, etc, which will continue our interaction with the FDA. We need to keep an ongoing dialogue with the FDA.
We haven't heard a number that is "good" but that quality of information is important. In terms of the survey, we need a really good representative sample. The higher numbers of surveys, the more we can do to do sub-analysis and understand. We encourage everyone to participate - several hundred surveys would be greatly appreciated!
You can to participate in the survey as long as you can speak English and answer the questions. For the EL-PFDD Meeting itself, you can attend the meeting and participate.