FDA To Meet with Pair of Rare Advocacy Groups

In recent months, the U.S. Food and Drug Administration (FDA) has stressed its commitment by to make patient engagement a priority in the clinical development of drugs for the rare disease community, and an announcement made yesterday confirms its stance.

A historic meeting will be held on Friday, April 6, 2018, when the FDA will host a joint Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting with the Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America) and the Pachyonychia Congenita Project (PC Project).

The summit is being conducted with the intentions of the FDA learning from individuals affected by either epidermolysis bullosa (EB) or pachyonychia congenita (PC), and the regulatory authority hopes to receive unique perspectives on how patients live with and manage their conditions, and how they would define “meaningful” treatment. It will be divided into two sessions, the first of which will focus on input from PC patients and caregivers. The later session will focus on EB, and both sessions will feature a clinical overview from Anna Bruckner, M.D., from Children’s Hospital Colorado.

The EL-PFDD meeting will be an in-person meeting with a live webcast for remote viewing. 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, and those viewing via webcast will be granted opportunities to respond to polling questions in real time.

Fibrocell Science is currently conducting a Phase 2 for its potential EB therapy FCX-007. In an exclusive interview with Rare Disease Report at the New Jersey Rare Disease Day event on March 5, CEO John Maslowski stressed the need for patient voice in the drug development process: “All companies need to start communicating earlier, and these conversations need to happen before a Phase 1; not as your about to enter a Phase 3 or when you’re going into clinical trials. If you wait, you can learn the hard way that none of your endpoints matter, because none of what you’re evaluating matters to the patient.”

The data and information collected from the meeting will be incorporated within the FDA’s risk-benefit analysis of potential new products or therapies in development to treat the symptoms and relive the burdens of EB and PC. Beyond the FDA, what is learned could additionally provide insight for academic researchers and the those within the regulated drug industry who are responsible for designing clinical trials.

Current standard of care for EB includes medications and surgeries to alleviate pain and itching, but the condition typically progresses despite treatment. For PC, there aren’t any known treatments, however, therapy is generally directed toward improving palmoplantar keratoderma and its associated pain.

The meeting is open to the public, and hopes to open pathways of communication between the FDA and people impacted by these rare diseases. People looking to attend, either in person or via webcast, can contact the PC Project or Debra of America directly.

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