You’re Invited!

NFXF has secured an Externally-led Patient-Focused Drug Development (PFDD) meeting for Fragile X syndrome. This is an opportunity to share YOUR voice! Your feedback will help inform drug development in FXS. Each disease can only host one PFDD meeting, so this is our shot to bring the patient perspective to the table so drug developers can listen.

The NFXF-led PFDD meeting in Fragile X syndrome is scheduled for Wednesday, March 3, 2021. NFXF will continue to share details closer to the date. Check out the FAQs below for more details.

If you have questions about the PFDD meeting, please email hilary@fragilex.org.

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!

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.

The NFXF-led PFDD meeting will be a half day meeting. 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 EL-PFDD meeting will be live streamed and we will provide an open docket for comment after the meeting. There will be a section on the NFXF website dedicated to the EL-PFDD meeting. The video of the live stream, meeting transcript, and final meeting report detailing learnings from the EL-PFDD meeting will all live on that webpage.

The EL-PFDD meeting on Fragile X syndrome is scheduled for Wednesday, March 3, 2021, from 8:00 a.m.–1:00 p.m. ET. This meeting will occur after Advocacy Day 2021.

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 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.

Your voice matters. NFXF believes there should be no treatments developed for Fragile X without the community having say — after all, you are the ones who will be using it! The PFDD meeting structure gives us a platform to talk about FXS to regulators and industry who are working in drug development in Fragile X. All research should be patient-centric, this gives you the opportunity to be heard now to help build a brighter future for families just like yours.

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!

Tino
Jace Wheatley
A selfie of Cole Lake with his grandmother Carol Schreibeis
Jodie and her mentor Dayna Clark
Olivia Butler with her brother Liam
Paige & Mae Otterson in the kiddie pool with their Fragile X superhero brother
Paige & Mae Otterson in the kiddie pool with their Fragile X superhero brother