NFXF-Led Patient-Focused Drug Development Meeting

Share Your Voice!

Wednesday, March 3, 2021
10:00 a.m. – 3:00 p.m. ET

NFXF has secured an Externally-led Patient-Focused Drug Development (PFDD) meeting for fragile X syndrome. Your feedback will help inform drug development in FXS.

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.

NFXF believes all research should be patient-centric. There should be no treatments developed for fragile X without the community having a say — after all, you are the ones who will be using it! Each disease can only host one PFDD meeting, so this is our opportunity to bring the patient perspective to the table.

why we do what we do
Young boy wearing a red t-shirt and a medal around his neck standing on an indoor basketball court

Brayden

Male toddler wearing a blue and white striped t-shirt and baseball cap on backwards

Harrison

Young boy standing outside a large conference center

Ayden

A middle-aged brunette male sitting at a table wearing a white t-shirt with a cake and gifts on the table before him

Dougie

Young thin male riding an amusement park car wearing a t-shirt and shorts and smiling

Brayden

How the Meeting Will Work

The NFXF-led PFDD meeting will be a half day meeting held on Wednesday, March 3, 2021, from 10:00 a.m. to 3:00 p.m. ET.

Experts will share about fragile X syndrome and the work that has been done thus far. Self-advocates and family members will share their stories and answer questions like, “What symptoms of fragile X syndrome have the most impact on your life?” and “What specific things would you look for in the ideal treatment for yourself/your loved one with FXS?”

The meeting will be live streamed and we will provide an open docket for comment after the meeting. The video of the live stream, meeting transcript, and final meeting report detailing learnings from the NFXF-led PFDD meeting will all live here on this web page.

COMPLIANCE NOTE: There are very strict communication requirements that NFXF must adhere to when sharing about the EL-PFDD meeting in FXS. If you have government officials or industry contacts you would like to invite to this meeting, please contact hilary@fragilex.org so we can adhere to these requirements.

Thank you for helping us stay compliant!

Additional Details

Please check out the FAQs below for more details. NFXF will continue to share details closer to the date — watch this page, or to make sure you don’t miss anything, subscribe now. If you have questions about the PFDD meeting, please email hilary@fragilex.org.

Frequently Asked Questions

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 patient perspective is critical in helping FDA understand the context in which regulatory decisions are made for new drugs. PFDD meetings give 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.

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 EL-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.

NFXF applied to host an EL-PFDD meeting for Fragile X syndrome because we feel there is a need for patient input in the development of treatments for FXS. This community has experienced several failed trials, and the opportunity to share your experience to guide future drug development is powerful. NFXF has built programming like the NFXF Research Readiness Program to support the patient and expert voice in research and hosting a PFDD meeting amplifies your voice even more. The purpose of PFDD meetings is to hear directly from patients, their families, caregivers, and patient advocates; all other attendees will be in listening-mode.

You will not need to travel in order to participate. NFXF will send out more details closer to the EL-PFDD date.

NFXF will post the video of the meeting and transcript for comment on our website. You will be able to view and comment on the meeting for 30 days after the meeting concludes. NFXF will write up a final report of the meeting and share it with the community. This report will be given to any researcher that initiates a drug trial in FXS. FDA may host our final report on their website.

Questions?

If you have questions about anything research-related, we’d love to hear from you! You can reach out to Hilary Rosselot directly, or submit your question or comment through our contact form.

Hilary Rosselot

Hilary Rosselot,
Director of Research Facilitation
research@fragilex.org
(202) 747-6207

Last Updated: 11/12/2020