Your Voice. Their Blueprint.
Watch the recording and submit your comments until January 5, 2026.
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.
Submitting comments and filling out the “Voice of the Patient” survey is your chance to shape future treatment options for Alexander Disease.
Read on below or download our info sheet here.
Submit comments for the next 30 days (January 5, 2026)
Submit a comment below on the topics that were discussed at the EL-PFDD meeting. Comments should be submitted individually.
The comments collected may be used in the final Voice of the Patient report (with identifying information removed).
Topic 1 - Alexander Disease Symptoms & Daily Impact
Of all the health effects of Alexander Disease, which 1-3 symptoms have the most significant impact on you or your loved one’s life?
How does Alexander Disease affect you or your loved one on best and on worst days?
How have your or your loved one’s symptoms changed over time? How has the ability to cope with the symptoms changed over time?
Are there specific activities that are important to you or your loved one that you/they cannot do at all or as fully because of Alexander Disease?
What do you fear the most as you or your loved one gets older? What worries you most about you or your loved one’s condition?
Topic 2 - Current & Future & Alexander Disease Treatments
What are you currently doing to manage your or your loved one’s Alexander Disease?
How well do these treatments treat the most significant symptoms and health effects of Alexander Disease?What are the most significant downsides to your or your loved one’s current treatments and how do they affect daily life?
Short of a complete cure, what specific things would you or your loved one look for in an ideal treatment for Alexander Disease?
What factors would be important in deciding whether to participate in a new research trial?
The Alexander Disease community has come together for a virtual EL-PFDD meeting, hosted by End AxD on December 5th, 2025.
This was 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 was 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
The survey findings and meeting outcomes will be documented and shared in a Voice of the Patient Report.