SYNGAP1 - PATIENT & CAREGIVER VOICES

Externally Led - Patient Focused Drug Development Meeting

SYNGAP1 - Awareness, Advocacy, Education & Research

SYNGAP1: Patient & Caregiver Voices
Externally Led - Patient Focused Drug Development

MEETING HUB PAGE

Miss the Meeting?
Your Voice Can Still Be Heard!

We Encourage You To Submit Your Comments for Inclusion in
the SYNGAP1: Voice of the Patient Meeting Report

Scroll Down for the
ONLINE WRITTEN COMMENT FORM

THE PUBLIC COMMENT PERIOD IS OPEN UNTIL -
DECEMBER 20, 2020
Suggested Comment Topics From the SYNGAP1 Patient & Caregiver Voices Meeting Include:

Living with SYNGAP1: Symptoms & Health Effects, Impact on Daily Life, Worries & Fears about the Future

SYNGAP1 Treatments: Treatment Successes, Treatment Downsides or Treatment Failure, What Type of Treatment Would You Most Like to See Developed & What Are Your Hopes for the Future
Complete the COMMENT FORM below & then Click on the Submit Button.

*Please note - There is a 30 day period after the meeting to submit public comments.
The
Online Written Comment Form will be available until, December 20, 2020.
MEETING AGENDA
THE POWER OF COMMUNITY

In Grateful Recognition of the Strength, Persistence and Contributions of
SYNGAP1 Families, Groups and Organizations from Around the World.
Externally Led - Patient Focused Drug Development

MEETING SPONSORS

EL - PFDD Meeting: Background Information

CALL TO ACTION

In November of 2020, Bridge the Gap – SYNGAP Education and Research Foundation will be hosting a SYNGAP1 Externally Led - Patient Focused Drug Development (EL-PFDD) Meeting.

This meeting provides the perfect opportunity for SYNGAP1 patients, caregivers and families to share their experience of living with SYNGAP1, how it impacts their daily life and what their priorities are for therapeutic treatment development.

THE BIG PICTURE -
Moving Closer to Therapeutic Drug Development for SYNGAP1

  • Why is this meeting so important for the SYNGAP1 Community?

    This meeting is, provides an amazing opportunity for SYNGAP1 patients, families and caregivers to share their life experiences, challenges, concerns about the future and what their hopes for the future are, in relation to living with SYNGAP1 or caring for someone with SYNGAP1.

    We want SYNGAP1 Voices to be heard loud and clear!

  • Please note: The FDA does not endorse specific patient organizations related to EL-PFDD Meetings. However, the FDA does provide staff to help guide and provide insight to patient organizations, as they plan and organize their EL-PFDD Meeting.
  • What is the format/structure of the meeting?

    This meeting will be hosted by Bridge the Gap and provides an opportunity for SYNGAP1 patients, family members and caregivers to share their experiences, symptoms and information about the impact SYNGAP1 has on their daily quality of life.

    Information about currently available approaches to treatment and the need for new, more effective therapeutic treatments will also be shared.

    The target audience for the meeting will be SYNGAP1 community members, regulatory/other federal agency leaders and staff, medical product developers, researchers and healthcare professionals. The meeting will be led by a facilitator, streamed as a live, interactive webcast and includes time for questions, online polling, and group discussion.

  • What are the expected deliverables from Bridge the Gap's SYNGAP1 Externally Led - Patient Focused Drug Development Meeting?

    Meeting deliverables will include a recording of the meeting webcast, a written transcript of the meeting and an extensive summary report.  The web recording, transcript and final summary report will be available, free of charge to the public on Bridge the Gap's website and the FDA’s website section for EL-PFDD Meetings. We anticipate all deliverables will be completed and available within 6 months of the meeting date.
  • What are the expected  benefits and outcome of the El-PFDD Meeting for the Global SYNGAP1 Community?

    The meeting provides an opportunity for SYNGAP1 patients, family members and caregivers, from around the world, to directly share their journey with a global audience of meeting attendees and the general public.

    The meeting provides a format in which SYNGAP1 clinical and research experts can provide information about SYNGAP1-related symptoms, characteristics, prevalence, demographics, current treatment guidelines and research insights.

    The meeting process ensures that SYNGAP1 patient, family and caregiver experiences, perspectives, needs and priorities are well-documented.  

    The meeting documentation will provide assistance to help ensure SYNGAP1 patient, family and caregiver voices, are incorporated in a meaningful manner into future SYNGAP1 clinical trial design, drug development and evaluation of therapeutic treatments.

    The meeting provides a rare and unique forum, where the voices of SYNGAP1 patients, families and caregivers, can truly be heard by those in positions that are most likely to guide future therapeutic development.