Congenital Erythropoietic Porphyria Voice of the Patient Report for the FDA

Ensure your CEP experiences and insights are reflected in a special report to the FDA.

Recording of the October 14, 2025 meeting

In an important milestone for the CEP community and rare disease advocacy, United Porphyrias Association hosted an Externally-Led Patient-Focused Drug Development (EL-PFDD) with the goal of providing the U.S. Food and Drug Administration (FDA) and other stakeholders an opportunity to hear perspectives from individuals with CEP on the health effects and daily impacts of their CEP and treatment goals.

The United Porphyrias Association is preparing a Voice of the Patient Report summarizing the issues and needs of CEP patients and families at the meeting. This report will be submitted to the FDA, researchers, and biopharmaceutical partners to improve understanding, care, and treatment for people living with CEP.

If you or a loved one is impacted by CEP, anywhere in the world, we want to include your voice in this report.

Whether you attended the meeting or not, your input matters.

Please provide your response in the form below. You may answer as many or as few questions as you wish. 

Please use your own words — there are no right or wrong answers.