The Galactosemia Foundation is collaborating with the National Organization for Rare Disorders (NORD) to host a virtual Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD). Patient stories of living with galactosemia are powerful and our perspectives are critical to the decisions made by drug developers, including the U.S. Food and Drug Administration (FDA) as well as researchers, health care providers, and drug manufacturers.
Through this EL-PFDD, FDA and other key stakeholders will hear directly from patients and their families about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application.
The meeting will be held on Thursday, September 1, 2022. If you would like to be considered as a patient or caregiver speaker for this virtual meeting, please fill out this questionnaire and share your story with us. If you have multiple family members with Galactosemia, please submit one form per patient. Speaker questionnaires will be accepted through Friday, May 6th, 2022 and we will be notifying all individuals whether they were selected or not after careful review of all submissions. Regardless of whether you were selected to speak, all are invited to participate during the meeting.
We expect this to be a truly historic meeting with FDA as we are inviting the entire global community of families and patients living with galactosemia to participate. We will email you in the coming weeks with details on how the general public and the community can register and participate. Thank you for your interest in participating!