Externally-led Patient-Focused Drug Development Meeting with FDA for Pachyonychia Congenita

Click here to read the Voice of the Patient: Report from the Pachyonychia Congenita Project Externally-led Patient-Focused Drug Development (EL-PFDD) Meeting.

Pachyonychia Congenita Project co-organized an Externally-led Patient-Focused Drug Development Meeting (EL-PFDD) with debra of America for FDA officials.

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:

The patient voice has historically been absent from the drug development process until a drug was approved by the FDA or when a clinical trial was failing and the FDA wanted input from actual patients to understand the problem. Now, the FDA wants to hear patient voices at the beginning of the process and throughout. Patient engagement is now a priority for the FDA.

PC patients are 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 above or at www.youtube.com/c/Pachyonychia/live

Click here for instructions on downloading the meeting app that has the schedule and live polling questions.

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