Patient-Focused Drug Development Initiative

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Principal Agency or Investigator: Foundation Fighting Blindness

Focus Area: Critical Research & Treatments

Grant Amount: $50,000

Grant Time: February 2026 - February 2028

Grant Details

Grant Overview

The Foundation Fighting Blindness is leading a collaborative effort to bring multiple patient experiences to the FDA in the form of a formal Patient-Focused Drug Development (PFDD) Initiative. This work is essential to ensure that the lived experience of patients with Usher Syndrome inform every stage of research and development, from trial design to regulatory engagement. 

A Patient-Focused Drug Development (PFDD) with the FDA is a structured forum where patients and caregivers share their lived experiences directly with regulators so that patient priorities meaningfully inform how new treatments are evaluated and developed. They are important because they ensure that the FDA and drug developers understand what truly matters to patients — so treatments are designed, evaluated, and approved based on real-world needs, not just clinical measurements.

Impact on Usher Syndrome 1B Patients

Patient-Focused Drug Development initiatives ensure that new therapies are designed around what matters to patients, not just what is easy to measure or has been historically used for measuring success. Currently, there is no FDA approved endpoint that can be used to measure a successful treatment for Usher Syndrome broadly (or Usher Syndrome 1B more specifically). These PFDDs help offer the FDA an important lens into what matters to patients with Usher Syndrome 1B, which will ultimately be a factor in determining which treatments become FDA approved and which do not.