Do you have a connection to CEP?
Whether in-person or online, we want to include your voice in this meeting! Read on to learn more. 

In an important milestone for the CEP community and rare disease advocacy, United Porphyrias Association will host an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting on Congenital Erythropoietic Porphyria (CEP)! 

What is the goal of an Externally-Led Patient-Focused Drug Development Meeting? 

The goal of this meeting is to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, and academic researchers an opportunity to hear perspectives from individuals with CEP on the health effects and daily impacts of their CEP, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to FDA’s PFDD initiative to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions. 

Following the meeting a final Voice of the Patient report summarizing the experiences and priorities identified during the meeting will be submitted to the FDA and made publicly available to inform regulatory decisions and future drug development. 

We are honored to serve as the host organization and grateful for the FDA’s attendance. 

What to Expect 

The main focus of the meeting is two CEP Patient Panels of five patients and/or caregivers. After each panellist shares some initial remarks, there will be a town hall style discussion where all CEP members in attendance will be able to contribute. 

• Panel 1: The health and daily impacts of CEP 

• Panel 2: Current approaches to treaments and priorities for treaments 

See full agenda.

The Details 

• Date: Tuesday, October 14, 2025 

• Time: 10:00 AM to 2:30 PM ET (find your local time here) 

• Location: Hilton Garden Inn, Bethesda, MD or online via Zoom 

Interested in participating? 

If you have a connection to CEP, we’d love to hear from you. Please contact us through the form below, we’d love to discuss how you can participate.