Thank you to everyone who participated in the Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting for Angelman Syndrome on April 7, 2025.

The Patient-Focused Drug Development (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 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 (PFDD) meetings.

PFDD meetings are planned and hosted by patient organizations, with the input of FDA staff, and use the process established by FDA-led PFDD meetings as a model. 

Caregivers and family members can submit comments in response to the discussion questions. We encourage 1 family member from each household participate in the live polling discussions, and we ask that only parents / primary caregivers call in live.

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.