TruePKU EL-PFDD

Externally-led Patient-Focused Drug Development meetings give FDA and other key stakeholders, including medical product developers, and healthcare providers, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during therapy and device development and during their review of a marketing application.

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. Click here to learn more about Externally-led Patient-Focused Drug Development meetings.

In the early spring of 2025, NPKUA will hold a webinar to share more details about the importance of EL-PFDD meetings and ways you can be involved in the TruePKU meeting.

The TruePKU meeting will take place on May 8, 2025, 11:00-4:00 PM ET. It will be a virtual meeting that anyone can join, whether you are directly affected by PKU or working to improve the management and outcomes of PKU. The webcast will be available here, on this webpage, on May 8. There is no cost to participate or attend. The recording will also be available soon after the meeting for on-demand viewing.

Anyone impacted by PKU can join the meeting webcast on May 8. You can now submit a comment online that may be read during the meeting. Comments will answer questions posed by FDA on topics related to the burdens of living with and managing PKU. There will also be a chance to call in during the meeting to share responses to specific questions posed during the meeting.

PKU patients and caregivers will speak throughout the meeting, representing the many ways in which PKU and its management affect children, teens, and adults over the course of their lives.

On April 8, NPKUA held a webinar that gave an overview of PFDD, the impact of EL-PFDD meetings, and ways you can be involved in the TruePKU meeting. Watch the recording on NPKUA’s YouTube page.

Some people not impacted by PKU believe that individuals in the PKU community already have everything they want and need to live well. This meeting is our chance to clarify that the PKU community still has unmet needs regarding new and different treatment and management options. Many companies are working on PKU treatments and new approaches to monitoring PKU. The TruePKU meeting will help them better understand what is most important to the community for medicines, therapies, medical nutrition products, and medical devices. TruePKU will help companies understand the best approaches for studying and developing these treatments.

The goal of the TruePKU initiative 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 PKU on the health effects and daily impacts of PKU, treatment goals, and decision factors considered when seeking out or selecting a treatment. TruePKU will be the very first EL-PFDD meeting that brings together four centers from the FDA – Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH), and the Human Foods Program (formerly known as the Center For Food Safety and Applied Nutrition).

At the TruePKU meeting, patients and caregivers will answer specific questions provided by the FDA. These questions will focus on understanding:

• The symptoms you experience because of PKU
• Your PKU management regimen (dietary restrictions, medical nutrition/foods/formula and medications, other lifestyle adjustments, etc.) and how well it addresses your PKU
• The impact PKU has on daily life and longer-range life goals
• How you track and monitor protein intake and phenylalanine levels
• How PKU and its management may have changed over time
• Meaningful changes that future PKU treatments might address
• Attitudes toward participating in PKU clinical trials and barriers you might face

In keeping with the FDA’s mission and areas of responsibility, this meeting will not be an appropriate time to cover topics that we know are very important to members of the PKU community. This includes topics that the FDA does not have control over, such as insurance coverage, cost of medications, and proposed legislation such as the Medical Nutrition Equity Act (MNEA).

The TruePKU meeting webcast will be recorded, and the recording will be posted on this webpage immediately following the meeting.

Information shared as part of the initiative will be summarized in the TruePKU “Voice of the Patient” report. NPKUA will publish this report, submit it to FDA for posting on the agency’s website, and share it broadly. It will be an enduring resource for the PKU community to use beyond the TruePKU meeting to help with advocacy campaigns, to share the PKU lived experience with the general public, or to introduce new researchers to the needs of the PKU community.