May 28, 2024 Externally-Led Patient-Focused Drug Development Meeting

The centerpiece of the 2024 GFPD Family and Scientific Conference is an Externally Led – Patient Focused Drug Development (EL-PFDD) meeting. Since 2015, the Food and Drug Administration (FDA) has given 176 patient advocacy groups an opportunity to plan and host an  EL-PFDD meeting. The GFPD is one of only seven groups hosting an EL-PFDD meeting in 2024. 

Peroxisomal disorders are an ideal candidate for an EL-PFDD meeting because they are chronic and affect the functioning and activities of daily living. The majority of an EL-PFDD meeting is dedicated to listening to patient and caregiver perspectives on the condition, especially the symptoms and daily impacts of the disease, as well as the treatments that would most improve their quality of life. Decision-makers at the FDA and other government agencies, medical product developers, academic researchers, clinicians, and healthcare professionals will listen to and learn from experts in the field- the patients and their caregivers.

Melissa Bryce, co-founder and executive director of the foundation, calls the EL-PFDD meeting during the 2024 conference a “transformative event” bringing together patients and caregivers to share their perspectives on living with a peroxisomal disorder.

“It is paramount that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation, and that is the goal behind this EL-PFDD meeting.”  

“I am so proud that just thirteen years after our founding, we are hosting an EL-PFDD meeting. This is an opportunity I never would have dreamed of when my daughter, Ginny, was diagnosed with a peroxisomal disorder in 2010. This conference and the EL-PFDD meeting will have monumental effects felt for years to come as we push drug development forward for peroxisomal disorders.  We have been waiting for this.” 

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