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.
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.