Debra of America and PC Project Lead Patient-Focused Drug Development Meeting With FDA

Meeting to take place on Friday, April 6, 2018.

The Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America) and the Pachyonychia Congenita Project (PC Project) are pleased to announce a joint Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting with the FDA on our respective rare diseases, pachyonychia congenita (PC) and epidermolysis bullosa (EB). 

This historic meeting will be held on Friday, April 6, 2018, at the College Park Marriott Hotel in Hyattsville, MD, near the U.S. Food and Drug Administration’s (FDA) headquarters. The purpose of this meeting is for individuals affected by PC and EB, as well as their caregivers, to provide unique perspectives to the FDA on living with their condition and what defines a meaningful treatment. The EL-PFDD meeting is divided into two sessions. The morning session will focus on input from PC patients and caregivers. The afternoon session will focus on EB patients and caregivers. Anna Bruckner, M.D., Children’s Hospital Colorado, will provide a clinical overview of each condition. 

The EL-PFDD meeting is designed to communicate to FDA the impacts of PC and EB on individuals’ daily lives, what types of treatment benefits make the most impact on peoples’ lives, and perspectives on how well available therapies are working.

The EL-PFDD meeting will be an in-person meeting with a live webcast for remote viewing, including the ability to respond to polling questions in real time. PC Project and Debra of America will identify and invite a small number of affected individuals and caregivers to participate in panel discussions during the meeting. A former FDA official will facilitate panel discussions, polling questions, and comments from the audience. The meeting is open to the public, and anyone is welcome to register to attend either in person or via the webcast.

The data and information from this meeting will be incorporated within the FDA’s risk-benefit analysis of potential new products or therapies intended to treat the symptoms and relieve the burdens of PC and EB. Additionally, the data and information may be relevant not solely to FDA, but for academic researchers, and the regulated drug industry designing clinical trials.

Pachyonychia Congenita (PC) is an ultra-rare genetic autosomal dominant skin disorder. PC is caused by a mutation in one of five keratin genes: KRT6A, KRT6B, KRT6C, KRT16, or KRT17. PC is a chronically debilitating disease due to impaired ambulation associated with plantar keratoderma, blistering, and pain that make walking difficult or impossible. Other standard features of PC may include palmar keratoderma, nail dystrophy, and cysts, all which can be extremely painful.

Epidermolysis Bullosa (EB) is a rare connective tissue disorder. There are many genetic and symptomatic variations of EB, but all share the prominent symptom of extremely fragile skin that blisters and tears from minor friction or trauma. Internal organs and bodily systems can also be severely affected by the disease. The list of manifestations and secondary illnesses can be long requiring multiple interventions from a range of medical specialists. EB is always painful, often pervasive and debilitating, and in the more severe cases lethal before the age of 30.

Patient engagement has become a priority for the FDA. Historically, the patient voice has been absent from the drug development process unless a clinical trial was failing or a drug was being considered for approval by the FDA. Today, the FDA wants to hear the patient voice throughout the process to ensure the most efficient and potentially the most efficacious drug development process.

If you are interested in attending this meeting either in person or via the live webcast, please contact PC Project at or Debra of America at

Media Contact:
Brett Kopelan
Phone: 917-566-6547

Source: debra of America

Categories: Medical Research

Tags: EB, Epidermolysis Bullosa, FDA, Pachyonychia Congenita, rare disease

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