PC Project sponsored an Externally-led Patient-Focused Drug Development Meeting (EL-PFDD) in 2018 to convey the severity of the disease to FDA officials.
The patient voice had historically been absent from the drug development process until a drug was approved by the Food and Drug Administration or when a clinical trial was failing. At that point, the FDA wanted input from actual patients to understand the problem. Now, the FDA wants to hear patient voices at the beginning and throughout the process. Patient engagement is now a priority for the FDA and PC Project was ready to tell our patients’ experiences.
PC patients were honored to be part of this historic opportunity to tell FDA officials about living with PC, managing PC, and what meaningful treatments will look like for patients. You can view the meeting below or at https://youtu.be/2rJmM4kQBwk.
This FDA meeting took place the morning of April 6, 2018, at the College Park Marriott Hotel in Hyattsville, MD, near FDA headquarters. Approximately 50-75 Pachyonychia Congenita (PC) patients and caregivers shared their voices through panel presentations, electronic polling and moderated audience discussions. (After a shared luncheon with PC patients, EB/EBS patients had a similar meeting in the afternoon.)
Click here for a pdf of the printed program.
Click here for a transcript of the meeting.
Click here for comments from PC patients received after the PC EL-PFDD meeting (appendix 1 topic 1).
Click here for comments from PC patients received after the PC EL-PFDD meeting (appendix 1 topic 2).
Click here to see Pachyonychia Congenita EL-PFDD listing on FDA website.
Watch the PC portion of the EL-PFDD meeting: