SYNGAP1 - Awareness, Advocacy, Education & Research
BRIDGE THE GAP: EXTERNALLY LED -
PATIENT FOCUSED DRUG DEVELOPMENT MEETING
November 19, 2020
12:30 PM - 5:00 PM (EST)
- Patient Focused Drug Development Meeting Hub
- Register for the Meeting
- Join the Live Meeting
SYNGAP1 - PATIENT FOCUSED DRUG DEVELOPMENT (PFDD) MEETING HUB
Welcome to BTG's Virtual
Patient Focused Drug Development Meeting
SYNGAP1 - Patient Voices!
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LIVE STREAM ACCESS
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PFDD MEETING SPONSORS
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PFDD Meeting: Background Information
CALL TO ACTION
This is a once-in-a-lifetime event for the SYNGAP1 Community!
Bridge the Gap – SYNGAP Education and Research Foundation has been invited by the FDA to host an
Externally Led - SYNGAP1 Patient Focused Drug Development Meeting (EL-PFDD)
Bridge the Gap's SYNGAP1 EL-PFDD 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 related to therapeutic treatment development.
THE BIG PICTURE -
Moving Closer to Therapeutic Drug Development for SYNGAP1
- Why is this meeting so important for the SYNGAP1 Community?
Bridge the Gap –SYNGAP Education and Research Foundation is 1 of only 30+ previous organizations - ever, (out of the countless number of organizations, representing over 7,000+ rare diseases,) to be invited by the FDA to host an Externally Led - Patient Focused Drug Development Meeting.
This meeting is, absolutely a once-in-a-lifetime 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!
- How was Bridge the Gap - SYNGAP Education and Research Foundation chosen to host an Externally Led - Patient Focused Drug Development Meeting for the SYNGAP1 Community?
The FDA has a formal process and committee designated for EL-PFDD meetings. Letters of Intent (LOI's) must be submitted to the FDA from patient organizations during designated time periods.
Each LOI must meet specific criteria outlined by the FDA including a detailed explanation about the need for an EL-PFDD meeting, how the organization plans to support the meeting and describe the intended benefit/outcome of the meeting for the organization's patient community.
The FDA EL-PFDD Meeting Committee convenes several times each year to review submitted Letters of Intent (LOI's) and then makes a decision on which organizations/diseases will be invited to host a meeting.
*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. FDA staff may attend an Externally Led-Patient Focused Drug Development Meeting, but in more of "listening/learning" capacity and not as a direct, active participant.
- 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, FDA 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 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 SYNGAP1 Community?
The meeting provides an opportunity for SYNGAP1 patients, family members and caregivers 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 SYMGAP1 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.