This report summarizes the Externally- Led Patient-Focused Drug Development (EL-PFDD) meeting that we held on November 15th, 2022. The EL- PFDD meeting was designed to communicate to FDA the impacts of hypophosphatasia on individuals daily lives, what types of treatment benefits make the most impact on peoples lives, and individuals and caregivers perspectives on how well available therapies are working.
This landmark report includes written statements and transcripts which are summarized in the report and shared in full in the appendices.
This report is dedicated to the courageous patients, caregivers, and families impacted by HPP.