The NFXF applied to host an externally led patient-focused drug development (EL-PFDD) meeting for Fragile X syndrome because we felt there was a need for patient input in developing treatments for FXS. This community has experienced several failed trials, and the opportunity to share your experience to guide future drug development is powerful. The purpose of PFDD meetings is to hear directly from patients, their families, caregivers, and patient advocates. The final report is available for download below. View the agenda (PDF).
Download the Voice of the Patient Report
Voice of the Patient Report: A Patient-Focused Drug Development Meeting for Fragile X Syndrome
Enter your information in the form below to download a copy of the report. You will also receive an email with a link to the PDF that you can access at any time.
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Watch the PFDD Meeting for Fragile X Syndrome
Watch the Full Recording
Check out the full agenda (available as a PDF), including presenters, panelists, and discussion questions. Please note: This video is 4 hours long.
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Learn More About the Externally-Led PFDD Meeting
What is Patient-Focused Drug Development?
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.
Why did the FDA start this initiative?
The patient perspective is critical in helping the Food and Drug Administration understand the context in which regulatory decisions are made for new drugs.
PFDD meetings give the FDA and other key stakeholders, including medical product developers, health care providers, and federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during FDA’s review of a marketing application.
What is an Externally-led PFDD meeting?
An externally-led patient-focused drug development (EL-PFDD) meeting uses the process established by FDA-led PFDD meetings, the host is just different.
The FDA has conducted over 25 disease-specific meetings since 2012.
The FDA now encourages externally-led PFDD meetings, which means the planning and hosting of the meeting is taken on by an outside group. Many patient advocacy organizations, like NFXF, take on the role of planning and hosting the meeting.
Watch the PFDD Meeting Community Forum
With Drs. Randi Hagerman, Liz Berry-Kravis, and Craig Erickson
JAN 20, 2021 — Hilary Rosselot hosted a community forum to share more about what to expect during and after the PFDD meeting, including details on how to participate and prepare, and what outcomes we might expect from the meeting.
Drs. Randi Hagerman, Liz Berry-Kravis, and Craig Erickson shared more on why this meeting will make a difference for Fragile X, and why it’s important for Fragile X parents, caregivers, and self-advocates to share what is most important to them in relation to drugs and treatments.