Debra of America (Dystrophic Epidermolysis Bullosa Research Association of America) and the Pachyonychia Congenita Project (PC Project) will soon meet with federal regulators to discuss patient-focused drug development in both rare skin diseases: epidermolysis bullosa (EB) and pachyonychia congenita (PC).
PC is a rare genetic skin disorder caused by a mutation in one of five keratin genes (KRT6A, KRT6B, KRT6C, KRT16, or KRT17). The two genetic conditions share some characteristics, such as skin blistering and pain.
The meeting is a first sign of the U.S. Food and Drug Administration’s commitment to make patient engagement a priority. It will take place April 6, 2018, at the College Park Marriott Hotel in Hyattsville, Maryland, near FDA headquarters.
According to a press release, Debra of America and the PC Project will invite a select number of EB and PC patients to join the meeting, so the FDA can hear directly from them about what it’s like to live with the conditions and which treatment options could be most meaningful.
The meeting will take up an entire day. A morning session will be dedicated to the PC community and the afternoon session will focus on EB.
The FDA will give the floor to patients and caregivers to talk about their daily lives, what types of treatments make the most impact, and their thoughts and perspectives on how well the available therapies work for them.
Anna Bruckner, MD, from Children’s Hospital Colorado, will provide a clinical overview of each condition.
The FDA will receive both patient communities in-person and via a live webcast, to include the possibility of responding to questions in real-time. Besides guest patients who will participate in panel discussions during the meeting, the public is invited to attend the event and participate as audience members.
A former FDA official will facilitate panel discussions and manage questions and comments from the audience.
All data and information gathered at this meeting will be included in the FDA’s risk-benefit assessment of potential new products or therapies intended to treat the symptoms and relieve the burden of the two rare diseases.
This information might not just be relevant for regulatory purposes, but also for academic and scientific research, as well as for the pharmaceutical industry.
Historically, patients’ voices have been absent from the drug development process — except in cases where a clinical trial was failing or a drug was being considered for approval.
Now, the FDA wants to hear patients’ own experiences and thoughts about their disease and care.
This initiative sends a positive message to the rare disease patient community, which has long waited to have their voices heard in the drug development process. Having patients directly communicating with the agency may help ensure more efficient and effective outcomes.
The meeting is open to the public and anyone is welcome to attend. Those who are interested in attending, either in person or via the live webcast, please email Debra of America at [email protected], or PC Project at [email protected].