About the Patient-Focused Drug Development Initiative

In 2013, the U.S. Food and Drug Administration (FDA) began holding a series of meetings to hear directly from patients, caregivers, advocates, and other key community stakeholders about what it’s like to live with various medical conditions and how well available treatments are meeting patients’ needs. This meeting series, called the “Patient-Focused Drug Development” initiative or PFDD, has now been expanded to enable patient organizations to host PFDD meetings in collaboration with FDA.


The CTX community was approved to hold a meeting by the FDA with the United Leukodystrophy Foundation as the host. The purpose of PFDD meetings in general and the Unlock CTX PFDD meeting is for FDA staff and researchers and developers of therapies to understand more fully the burdens of living with a particular medical condition and what patients most value in terms of treatment benefits, as well as the risks and side effects they may be willing to tolerate as a tradeoff for the benefits that matter most.

An example of how PFDD can benefit people living with CTX is to deepen understanding about all the ways in which CTX impacts people’s lives. You can help us tell the story fully of how it affects an individual’s physical, intellectual, emotional well-being and more.

You’re invited to participate virtually on September 14. RSVP today to be included. There is no cost to attend. Please share this information with others who may be interested in taking part!