Sharing the Voice of The STXBP1 Community with the FDA!
On October 20, 2023 at 10:45AM and 1PM ET, the STXBP1 community will get the opportunity to make our voices heard during a live meeting with the FDA. The STXBP1 Foundation is bringing the STXBP1 patient community together for a first ever Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD), to inform the FDA and other stakeholders about the patient perspective of living with STXBP1-related disorders.
It is critical that parents and caregivers participate in this meeting, as it will focus on patients' experiences and insights for consideration in drug development and evaluation. The goal is to hear from families and caregivers on what it's like to live with the condition, so that we the FDA can better understand the patient and family experience.
What is needed from you
We need your voice! We need you to join the virtual PFDD meeting on October 20, 2023 at 10:45AM and/or 1PM ET to share your experiences with the FDA. Here are the sessions we would recommend you attend:
Session 1 on Living with STXBP1 Symptoms and Daily Impact:
1. Listen to our 5 panelists from 10:45am-11:10am ET
2. Share your thoughts however is easiest for you by calling in or writing in or voting in the polls from 11:10 am -12:30pm ET during our Audience polling & moderated discussion
Session 2 on Current and Future Treatments for STXBP1:
1. Listen to our 5 panelists from 1pm-1:25pm ET
2. Share your thoughts however is easiest for you by calling in or writing in or voting in the polls from 1:25pm -2:40pm ET during our Audience polling & moderated discussion
Here is the full agenda for the event.
Please be prepared to share your personal story, and the struggles your family and loved ones living with STXBP1-related disorders face. For an idea of what to expect, you can listen to Larry Bauer from Hyman, Phelps & McNamara in this PFDD webinar hosted recently by the STXBP1 Foundation.
What you need to do
Register for the virtual meeting Here
Provide a comment Here
Login on October 20th and attend as much of the meeting as possible
Call in or email with questions or comments during the meeting
Your Voice Matters!!! The information shared during this PFDD meeting can help the FDA to make informed decisions on approvals of potential medicines for STXBP1-related disorders, and pharmaceutical companies to design clinical trials that are meaningful for patients. These things can help accelerate FDA approvals, so it is critical that we tell our story.
Please join us to tell our collective story with the FDA. If you have questions, please contact us at info@stxbp1disorders.org.
