National Fragile X Foundation Leading Patient-Focused Drug Development Meeting

The National Fragile X Foundation (NFXF) will host a virtual patient-focused drug development (PFDD) meeting for fragile X syndrome on March 3.

To be held from 10 a.m. to 2 p.m. EST, the meeting provides an opportunity for members of the fragile X community to help make sure their experiences, perspectives, needs, and priorities are meaningfully incorporated into the development and review of future therapies.

In particular, the NFXF is encouraging patients, siblings, parents, caregivers, and other advocates to attend the meeting. Registration is open, and those interested must register ahead of time.

The U.S. Food and Drug Administration (FDA) started PFDD meetings as a way for the agency and key stakeholders, such as medical product developers, healthcare providers, and federal partners, to incorporate patient perspectives into treatment development and regulatory review.

In these meetings, stakeholders have an opportunity to hear about the symptoms that matter most to patients, how the disorders affects daily life, and personal experiences with available treatments.

Externally-led PFDD meetings, such as this one, follow the same format as those hosted by the FDA but have a different host. Noting that the community has experienced a number of failed studies, the NFXF applied to host this meeting to further amplify patient voices in fragile X research.

During the meeting, experts will discuss past and ongoing research into fragile X, and self-advocates will tell their stories and answer questions related, but not limited, to how their condition has affected their lives and what they are looking for in an ideal treatment.

Although attendees who are not patients, their family members, or other advocates will be in listen-only mode, there will be an open forum for comments after the meeting.

A video recording of the meeting will be made available, along with a transcript and a final report detailing what has been learned in the meeting here. Researchers launching fragile X clinical trials will receive a copy of the report, and the FDA may host it on its website.

The video will remain available for viewing and open for comments for 30 days after the meeting.

NFXF believes that because community members are the final users of any new therapy, their input should inform all fragile X treatment development.