Thank you to our Endorsers
Thank you to our Sponsors
COPPER
GOLD
SILVER
BRONZE
Carol and Sparky Terry
Community
In Honor of Joe Scanlan
Thank you to the Wilson disease community for a very successful Wilson Disease Externally Led-Patient Focused Drug Development (EL-PFDD) Meeting held on January 29, 2026 for the FDA, clinicians, researchers and drug developers. You can watch the full recording here:
Watch Now:
Thank you to all the Wilson disease patients and caregivers who submitted comments for inclusion in our Voice of the Patient Report. It will be completed mid-2026 and will be available here.
What Is an EL-PFDD Meeting?
An EL-PFDD meeting is a unique opportunity that puts patients, their families, and other direct caregivers at the center of drug development conversations. Through powerful firsthand stories, the FDA, product developers, clinicians, and academic researchers gain insight into:
- The daily realities of living with Wilson disease
- The greatest unmet needs
- Personal treatment goals and priorities
Your input can help the FDA, medical product developers, clinicians and academic researchers make better informed decisions during drug development and during review of marketing applications for potential treatments—shaping decisions that affect the entire Wilson disease community.
Frequently Asked Questions: Externally-Led Patient-Focused Drug Development Meeting
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.
We’re looking for a wide range of voices across the Wilson disease spectrum—including patients, parents, caregivers, and those with different disease manifestations and at different stages of diagnosis or treatment. 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 complete this Participation Interest Survey.
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 announcement of a Community Webinar where we will share more about participating in the live EL-PFDD meeting.
Wilson disease meets the goals for an EL-PFDD meeting:
- It’s chronic and life-altering
- Symptoms and impact vary widely and are not always captured in clinical trials
- There are limited treatment options and many come with serious side effects
- The disease affects both children and adults and can severely impact daily life
These factors make patient and caregiver insights especially valuable in shaping drug development.
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.
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/
Everyone is welcome. This virtual meeting is open to the entire Wilson 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 Wilson disease
Because this is a global community, we’re exploring options to provide live translation services to ensure broader access and participation. We want everyone—regardless of location or language—to have the opportunity to take part, listen, and be heard.
Whether you’re sharing your story or simply attending to listen and learn, your presence helps strengthen our collective voice.
Absolutely! We welcome participation from the entire global Wilson disease community. If you speak English, you can take part in the video participation survey, which helps us capture a wide range of patient and caregiver experiences.
Closer to the event, you’ll also have the opportunity to submit written comments that may be included in the official Voice of the Patient report shared with the FDA. And of course, you’re invited to attend and participate virtually in the Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting—no matter where you live!
The Wilson disease EL-PFDD meeting will be held virtually via live webcast on Thursday, January 29, 2026, from 10 a.m. to 3 p.m. ET making it easy for people from around the world to join and take part.
Closer to the event, we’ll open a free registration process so participants can sign up and receive the live webcast link and event details. Whether you’re tuning in to listen, sharing your story live via the call-in phoneline or as written comments, or participating in polls, you’ll be able to fully engage from anywhere with an internet connection.
We’re committed to making the meeting accessible to our global community and are exploring options to provide live translation services to support participation in multiple languages.
Stay tuned—we’ll share more details about how to register and participate as the date approaches.
We’ll create several deliverables, including:
- A video recording of the meeting that will be on the WDA website and YouTube channel
- A written transcript of the meeting that will be on the WDA website
- A Voice of the Patient (VOP) Report that summarizes the meeting, as well as written comments received from the WD community
These materials will be shared with the FDA and made publicly available here to support future research and drug development.
- Save the date: January 29, 2026, 10 a.m. to 3 p.m. ET
- Sign up to receive our emails by clicking the “Join our Email List” button at the top of our Home page: https://wilsondisease.org/.
- Contact us with questions or ideas by emailing events@wilsonsdisease.org
Your experience can help guide the future of Wilson disease care—and bring hope to others in the community.
The Wilson Disease EL-PFDD Meeting is a global event and to help our Spanish-speaking community participate our friends with the Asociación Española de Familiares y Enfermos de Wilson have provided this Spanish language translation.
Thank you to our Endorsers
Thank you to our Sponsors
COPPER
GOLD
SILVER
BRONZE
Carol and Sparky Terry
Community
In Honor of Joe Scanlan
Donations
Why Your Donation Matters
Your donation to the Wilson Disease Association does more than keep the lights on—it powers education, awareness and action. With your support, we’re able to maintain vital resources like this website, produce trusted educational materials, fund critical research and host events that bring together patients, caregivers and medical professionals. Most importantly, your generosity helps raise awareness so more people are diagnosed earlier and treated effectively—giving them the best chance at a full, healthy life.
When you give, you’re not just supporting an organization—you’re joining a powerful movement to change the future of Wilson disease. You’ll stay informed about the latest in research and treatment, gain access to exclusive webinars and educational resources, and remain connected to a global community that understands. For more than 40 years, our shared commitment has fueled advances in care, deepened understanding, and amplified the voices of those affected by Wilson disease. Together, we’re building a stronger, more informed future.
