Your Voice. Their Blueprint.
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.
Externally - Led PFDD meetings (EL-PFDD) are one such approach. These meetings are a powerful way for patients, their families, caregivers and patient advocates impacted by a disease to directly tell regulatory decision-makers, treatment developers, academic researchers, and healthcare professionals what living what that condition is really like and what meaningful treatment look likes from the patient and caregiver perspective.
This is your chance to shape future treatment options for Alexander Disease with your voice.
Read on below or download our info sheet here.
Did you miss the ULF Family Conference?
Want to catch the special update about the EL-PFDD Call To Action?
Check it out here ⬇️
The Alexander Disease community will come together for a virtual EL-PFDD meeting, hosted by End AxD on December 5th, 2025 from 10:00 AM - 3:00 PM EST.
This is a rare and powerful opportunity for our community to speak directly to regulatory decision-makers, treatment developers, academic researchers, and healthcare professionals: sharing the real, lived experience of Alexander Disease and the urgent need for treatments.
This meeting is about US. And only US.
What Drug Development Stakeholders Want to Learn About:
The day-to-day effects of Alexander Disease (currently and how they change over time)
Your approaches to managing and treating Alexander Disease
Your priorities and preferences regarding new treatment options
Your feedback will help shape innovative treatment strategies for Alexander Disease. This meeting will help:
Identify unmet needs in current treatment approaches
Provide researchers and regulators with insights into the most significant challenges faced by those living with Alexander Disease
We encourage participation from all individuals living with Alexander Disease and their caregivers. The survey findings and meeting outcomes will be documented and shared in a Voice of the Patient Report.
We Have a Date!
Mark your calendars
December 5, 2025
10:00 AM - 3:00 PM EST
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The Goal
To host a virtual EL-PFDD meeting for Alexander Disease and uncover the real-life impacts, unmet needs, and hopes for the future from our community. FDA, treatment developers, academic researchers, and healthcare professionals will be attending in a listening role.
Uncover the real-life impact, unmet needs, and hopes for the future.
Result in a formal “Voice of the Patient” report that becomes part of the public record and a resource for FDA staff when assessing products under review and by advising drug sponsors on their development programs.
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Who should participate?
Patients living with Alexander Disease - newly diagnosed, early onset, adolescent, young adult, adult
Caregivers and family members of all ages
Siblings, loved ones, and advocates
Bereaved families whose voices still deeply matter
Anyone with lived experience, regardless of diagnosis timeline or disease stage
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What happens next?
August 2025:
Confirmation of panelists and conversation starters who have expressed interest will conclude this month
September - November 2025:
Kick-off meeting with panelists and launch of EL-PFDD survey to the community
Organize panels, survey the community, and prep materials
December 5th from 10-3pm EST
We share our stories as one united community
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How you can help?
Share your story
Join our contact registry
Add your name to the Patient Advocacy support list by emailing: info@endaxd.org
Help spread the word about the EL-PFDD meeting to fellow Alexander Disease families
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Key takeaways
This meeting is looking into the lives of those affected by Alexander Disease.
It’s hosted by the Alexander Disease community, not regulators
Sharing your story increases our chances for the Alexander Disease Community to be heard and for our input to inform FDA’s decisions and oversight both during drug development and during review of a marketing application for future treatments for Alexander Disease.
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FAQs
What is an EL-PFDD meeting?
An Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting is a unique opportunity for patients, their families, and other direct caregivers to share their experiences directly with the FDA, medical product developers, clinicians and academic researchers. The goal is to help those involved in developing treatments better understand the real-life impact of living with Wilson disease—and what matters most to patients and families.
Why is this meeting important for the Alexander Disease community?
Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings provide the FDA and other key stakeholders—including medical product developers, healthcare providers, and federal partners—with a valuable opportunity to hear directly from patients, their families, caregivers, and advocates. These meetings focus on the symptoms that matter most to patients, the impact of the disease on daily life, and experiences with current treatments. This input can help inform the FDA’s decisions and oversight throughout drug development and the review of marketing applications.
Who can participate in the meeting?
We’re looking for a wide range of voices across the Alexander Disease community - including patients, parents, caregivers, and at different stages of diagnosis. The most immediate need is for us to identify those who want to participate via video. We are currently looking for individuals to participate by:
Recording a 5-minute video (we’ll help guide you)
Joining the meeting live via Zoom as a “conversation starter”
If you’d like to share your perspective via video or live via Zoom, please reach out to: info@endaxd.org
How can I prepare to share my story?
If you are selected to participate in the meeting via video, we’ll provide:
Prompts and coaching for developing your statements and video submissions
Orientation and guidance for those speaking live
Guidance to help you focus on what’s most important: your experience, your needs, and your hopes for better treatments
If you are not selected to participate in the meeting via video or live via Zoom, DON’T WORRY! the entire community is invited to attend and share virtually during the live meeting. Your voice will be heard. Stay tuned for the details of how to participate in the live EL-PFDD meeting.
Will the FDA attend and listen?
Yes. The FDA will attend virtually, as well as review the meeting materials, listen to the stories, and use this information to understand what patients truly need and value in new treatments. While the FDA does not endorse or lead this meeting, it recognizes the importance of these voices in the regulatory process.
Want to see what a virtual EL-PFDD meeting looks like?
If you’re curious about what to expect, we encourage you to explore recordings and materials from other rare disease organizations that have hosted virtual EL-PFDD meetings. These examples offer a glimpse into how patient stories are shared, how the conversation flows, and the powerful impact these meetings can have.
https://swallowingdisorderfoundation.com/dysphagia-meeting/
https://ntsad.org/events/gm2pfdd/
Who can attend the EL-PFDD meeting?
Everyone is welcome. This virtual meeting is open to the entire Alexander disease community, including:
- Patients and caregivers
- Family members and advocates
- Clinicians and researchers
- Industry representatives
- Regulators and policy makers
- Anyone interested in learning more about Alexander disease
Whether you’re sharing your story or simply attending to listen and learn, your presence helps strengthen our collective voice.
Can non-US residents participate?
Absolutely! We welcome participation from the entire Alexander Disease community. Submit written comments that may be included in the official Voice of the Patient report shared with the FDA.
Will there be translation services available?
At this time, we are not able to provide live translation services during the meeting. However, recordings will include captions, and written submissions may be translated and incorporated into the official record. Families who would like live support are welcome to arrange for their own translator to join them. We recognize the importance of accessibility and are committed to making sure every voice is heard through the options available.
How can I participate virtually on the day of the event?
A virtual webcast link will be provided, making it easy for people from around the world to join and take part.
Closer to the event, information will be shared on End AxD social media, in the newsletter, and on our website. We encourage all to join!
What happens after the meeting?
A formal “Voice of the Patient” report will become part of the public record and a resource for FDA staff when assessing products under review and by advising drug sponsors on their development programs.
How can I stay involved to learn more?
Save the date: December 5, 2025 from 10-3pm EST
Sign up to receive our emails by clicking the “Stay Informed” button at the top of our website page: www.endaxd.org.
Contact us with questions or ideas by emailing info@endaxd.org
Your experience can help guide the future of Alexander disease care and bring hope to others in the community.