PFDD Meeting for CDKL5 Community

On November 1, 2019, IFCR and the LouLou foundation will co-sponsor a Patient-Focused Drug Development (PFDD) meeting for CDKL5 Deficiency Disorder. This meeting is a very rare opportunity to convene patients, regulators, researchers, developers, and health-care professionals.

PFDD meetings are a once-in-a-lifetime opportunity. Find out how you can participate here.

What is a PFDD Initiative?

PFDD meetings are part of a greater initiative launched by the Food and Drug Administration (FDA) in 2012. These meetings are designed to help families affected by CDKL5 Deficiency Disorder gain a platform to voice their perspectives, experiences, needs, and priorities regarding current and future CDD treatments.

Patient voices have historically been disregarded. In PFDD meetings, however, caregivers are given the mic to share the daily impact of CDD on their children. Caregivers will tell stories to highlight the unmet needs of their children and describe the shortcomings and advantages of current therapies.

WHAT TO EXPECT from a PFDD meeting:

• We will target symptom areas where there is an identified need for CDD patient input on topics related to drug development
• We will get an opportunity to discuss these topics with the FDA and drug development companies: (1) Symptoms and daily impacts that matter most to patients and (2) current approaches to treatment
• We will participate in facilitator-led large group discussion, interactive webcast, discussion aids (e.g., polling tools)
• We will receive a Web recording, transcript, and summary report that will be available to anyone that is developing CDD specific treatments

Why is this important?

This PFDD meeting will strengthen understanding of CDD and treatment burden for FDA, drug developers, and additional stakeholders. All information will be summarized as a “Voice of the Patient” report. This report will be used to inform future CDD-focused treatment development and trial approvals.

Patient input from these meetings helps the FDA understand the daily needs of the CDKL5 community. These meetings also provide the possibility of gathering data to treat unmet needs, such as symptoms beyond seizures. They can also provide a framework for creating tools to assess potential therapies.

By raising awareness and engaging our patient community, the FDA and drug developers will have a better road-map to bring treatments and cures to the CDKL5 community.

How do I get involved?

You can join us in person at Hyattsville, Maryland, or via live-streaming, on November 1st. We will announce more details as they become available – please let us know if you are interested in participation by providing us with your contact information at this link. Numbers matter! We hope to see strong community representation in attendance at this groundbreaking meeting.