You can help the FDA understand life with Usher syndrome

On April 8, 2027, people with Usher syndrome and their families can tell the U.S. Food and Drug Administration (FDA) what life with Usher syndrome is really like. This online meeting is called a Patient-Focused Drug Development (PFDD) meeting. This guide explains what it is, why it matters, and how you can take part.

The Foundation Fighting Blindness is leading this online meeting, with a planning group of the following Usher syndrome organizations: Cure 2 See, The Fairfield Fund, Save Sight Now, Usher 1F Collaborative, Usher 2020 Foundation, Usher III Initiative, Usher Syndrome Coalition, Usher Syndrome Society, and USH1D United.

As part of this group, the Usher Syndrome Coalition is helping make sure people with every type of Usher syndrome, who use all methods of communication, know about this opportunity and have the support they need to participate.

What is a PFDD?

At a PFDD meeting, people living with a condition tell the FDA what daily life is really like. They share which symptoms matter most, what treatments they have tried, and what they hope a treatment could do. This kind of meeting is sometimes called an EL-PFDD, which stands for Externally-Led PFDD. That means it is organized by patient groups instead of by the FDA.

This meeting is about all types of Usher syndrome.

Why does this matter for the Usher community?

There are no approved treatments for the vision loss in Usher syndrome. Companies are working on possible treatments, and the FDA will one day decide whether to approve them.

To make those decisions, the FDA needs to understand our lives. For example, is it a real benefit if a treatment keeps the vision you have now, even if it does not bring vision back? People with Usher syndrome can answer that question better than anyone else.

After the meeting, everything shared will go into a public report called the Voice of the Patient report. The FDA and drug companies use this report when they plan research studies, clinical trials, and review new treatments.

What would panelists do?

About 20 people across all Usher subtypes will be chosen to be panelists. Some will record a short video, about 5 minutes long, sharing their story. Others will speak live during the virtual meeting to help start the discussion.

Panelists will:

• Get coaching and practice starting in January 2027
• Take part in a practice session on April 7, 2027, and the live meeting on April 8, 2027
• Take part online, so no travel is needed

Communication access will be provided to panelists at no cost. 

Who can apply?

Adolescents and adults living with any type of Usher syndrome and caregivers or family members can apply. Children and their caregivers are also welcome to volunteer to co-record a joint statement. You do not need to live in the United States to serve as a panelist. 

The planning group hopes to hear from many kinds of people. This includes people who are newly diagnosed or were diagnosed long ago, people who have a range of diagnoses and symptom severity, people of different ages and backgrounds, and people who communicate in different ways, including spoken and signed languages.

How do I apply?

Fill out the interest form here: Usher Syndrome Patient Focused Drug Development Meeting

The deadline is November 20, 2026. If you need help with the form, or want to apply by phone or mail, email PFDD@FightingBlindness.org. 

Everyone who volunteered to be a panelist will be notified if they have been selected in December 2026.

What if I’m not chosen, or don’t want to be a panelist?

Your experience still counts. Everyone can watch the meeting online for free and participate in the live discussion. 

For a few months before and after the PFDD, written comments can also be submitted via the USH PFDD website or mailed to: 

Foundation Fighting Blindness
ATTN: PFDD
6925 Oakland Mills Road #701
Columbia, MD 21045

Spoken comments can be left as a voicemail at: 610-871-3704 

Comments shared before or after the meeting will also be incorporated in the Voice of the Patient report.

Learn more

A free online information session is planned for February 11, 2027 at 4:00 pm Eastern. Registration for the information session will be available on fightingblindness.org/usher-pfdd in early 2027.

Questions? Contact PFDD@FightingBlindness.org.