Usher Syndrome Patient Focused Drug Development Meeting

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About the EL-PFDD Meeting

The Foundation Fighting Blindness is conducting a virtual externally-led patient focused drug development (EL-PFDD) meeting on Usher syndrome on April 8, 2027.

The purpose of this meeting is to share with key FDA officials and other stakeholders the journey of Usher patients and their caregivers, prognosis, unmet needs, and current standards of care. We invite people living with all subtypes of Usher syndrome and their caregivers to join a live discussion with drug developers, researchers, and regulators, as well as healthcare policymakers and payers, to inform clinical trial design and benefit-risk decision-making for evaluating and approving treatments for this disease.

Because there are currently no approved therapies for Usher syndrome, stakeholders must understand how patients and their caregivers are affected by the condition and consider their unique perspectives for future development and therapeutic review. The Foundation Fighting Blindness is committed to ensuring that the data, infrastructure, and tools needed to support and drive drug development for patients with Usher are available.

This EL-PFDD meeting is a key component of realizing this objective, by capturing patient and caregiver insights that could set the context for FDA benefit-risk considerations. It will enable a comprehensive understanding of this disabling condition for key reviewers in all offices of the FDA.

Call for Panelists!

Your experience matters. Help ensure the voices and experiences of people living with Usher syndrome are part of the conversation about future treatments.

We’re looking for people living with all subtypes of Usher syndrome and their caregivers to share their experiences as part of this important conversation. You can self-nominate to be considered as a panelist or share this opportunity with someone in your community whose perspective should be heard.

Complete Form

A Conversation That Reflects the Usher Syndrome Community

The PFDD meeting will be structured to capture shared and unique experiences across subtypes, ensuring the final report reflects patient diversity. Following the meeting, a Voice of the Patient report will be developed and submitted to the U.S. Food and Drug Administration (FDA) and made publicly available. The report will capture the perspectives and experiences shared by the community during the meeting and can help inform future treatment development and regulatory review.

Join the Conversation

Whether you are living with Usher syndrome, caring for someone with Usher syndrome, or connected to the community in another way, your perspective can help inform the conversation about what comes next.

Consider nominating yourself—or share with someone whose experience should be represented.

For questions or additional information about the EL-PFDD meeting, contact PFDD@FightingBlindness.org.