Watch the GM1 EL-PFDD Recording
Voice of the Patient Report
Download and read the Voice of the Patient Report which summarizes the findings from the EL-PFDD meeting with FDA. This report was published on the FDA website and should be referred to by biotech companies and researchers working on GM1.
About the GM1 Gangliosidosis EL-PFDD
The GM1 Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) was held on Friday, October 14 as part of our 2022 virtual conference. This meeting’s aim was to shed light on the experiences and perspectives of GM1 gangliosidosis patients and those who care for them. The information gathered will influence the development and evaluation of drugs to treat GM1. The Voice of the Patient Report will be published in 2023.
The public and all interested patient advocacy groups were encouraged to attend, participate in, and support the EL-PFDD. GM1 families had an opportunity to share their experiences and perspectives on GM1 directly with the U.S. Food and Drug Administration (FDA), medical product developers, clinicians, and academic researchers.
“An EL-PFDD initiative aims to more systematically obtain the patient perspective on specific diseases and their treatments. The patient perspective is critical to help provide context when FDA makes regulatory decisions for new drugs.”
The US Food and Drug Administration
To learn more about what a PFDD is, visit: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development
View the recorded webinar, What is the EL-PFDD?
In this webinar you’ll learn what an EL-PFDD is, what happens during an EL-PFDD meeting, and why attending matters. This webinar was conducted by FDA regulatory consultants.
View the EL-PFDD Agenda
EL-PFDD FAQs
What is an Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting?
An EL-PFDD informs the FDA of the side effects and risks patients may be willing to accept to gain a certain level of symptom relief or a slowing of their disease progression. Patients also share needs regarding new drugs and their preferences for clinical trials.
Drug manufacturers also gain insight into patient concerns, like which symptoms or treatment side effects are or are not tolerable. This information helps them design clinical trials and develop treatments which matter to patients and which are aligned with their needs and preferences.
Why is Cure GM1 hosting an EL-PFDD?
At Cure GM1, we see an unmet need for treatments of GM1 gangliosidosis. Our goal is to educate the FDA on what it is like to live with and care for those with GM1: what symptoms patients experience, how their lives are impacted, what kinds of treatments they need, and what kinds of risks they are willing to accept to see improvements.
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