Living with a rare disease often means limited treatment options and few opportunities to share the personal challenges of managing the condition. What symptoms persist despite treatment? Does the available treatment ensure good quality of life? The patient experience often goes unheard by the Food and Drug Administration (FDA) – yet it is crucial to shaping better solutions that meet patients’ needs.
To bridge this gap, the FDA established the Patient-Focused Drug Development (PFDD) program in 2012, later expanding it in 2015 to allow external organizations to host meetings.
These Externally Led Patient-Focused Drug Development (EL-PFDD) meetings give patients and caregivers a platform to share their experiences and priorities for care. By highlighting unmet needs, these discussions guide research and fuel development of better treatments for rare diseases, including inherited conditions of protein metabolism.
When patient and caregiver voices are brought to the forefront, they uncover critical gaps in care and inspire more targeted treatment development. This article explores how Patient-Focused Drug Development meetings work, their relevance to the flok community, and how you can get involved.
What is an EL-PFDD Meeting?
PFDD meetings led by patient advocacy organizations are known as Externally Led Patient-Focused Drug Development (EL-PFDD) meetings. To host one, a patient organization submits a letter of intent to the FDA explaining why the meeting is vital to those affected by the condition. After FDA review and approval, the organization funds, plans, and moderates the event, which can be held virtually or in-person.
Stakeholders including the FDA, drug developers, health care providers, and researchers attend to hear directly from patients and caregivers.
PFDDs Relevant to the flok Community
Upcoming
EL-PFDD Meeting hosted by National PKU Alliance (NPKUA) on May 8, 2025
Past
EL-PFDD Meeting for Classical HCU hosted by HCU Network America on October 27, 2023. VOP report pending.
FDA-led PFDD Meeting for Inborn Errors of Metabolism on June 10, 2014
Individuals directly affected by the condition share their experience with symptoms, quality-of-life challenges, and the limitations of existing treatment, including side effects and risks. These testimonials help the FDA and sponsors better understand patient needs, shaping future research, drug evaluation, and regulatory decisions.
What Happens During an EL-PFDD?
EL-PFDD meetings follow a town hall format with presenter panels, moderators, and open discussions. Depending on the disease of focus, one to four of the centers of the FDA will attend – 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.
- Opening Remarks: The FDA begins with introductory remarks, followed by a clinical overview of the condition, often presented by a medical expert.
- Panel Discussions:
- The first panel covers symptoms and daily life with the condition.
- The second panel focuses on existing treatments, their limitations, and perspectives on clinical trial participation.
- Audience Engagement: Moderators facilitate live discussions with the audience, encouraging patients and caregivers to discuss unmet needs, treatment gaps, and hopes for future care options. Participants can also engage through live polls or call-ins.
Danae Bartke, Executive Director of HCU Network America, hosted an EL-PFDD for Classical Homocystinuria in 2023 and emphasizes the value of honesty during these discussions:
“We so often focus on the positives of treatment, and this was an opportunity to unmask and share the raw truth of living with Homocystinuria. When we share our honest experience, people are inspired to develop new and better treatments for our community.” What Happens After the EL-PFDD Meeting?
After the panels conclude, the hosting patient organization creates a summary titled Voice of the Patient (VOP) report, which is archived by the FDA. The VOP serves as a public resource across the four centers of the FDA. The summary provides a comprehensive patient perspective on the needs and priorities to guide researchers in developing better treatment options and clinical trials.
Why are EL-PFDD Meetings important for our flok Community?
EL-PFDD meetings create opportunities for support, networking, and treatment advancement for the flok community.
- Validation and Connection: These meetings unite individuals in shared experience and reduce isolation often felt by those with a rare metabolic condition.
- Advocacy: The meetings strengthen relationships between patient organizations and drug developers, paving the way for more targeted treatments.
- Innovation: The needs and priorities expressed during EL-PFDD meetings unearth new research questions and spark innovation in treatment approaches.
Danae encourages flok community members to participate, whether as presenters or audience members:
“There are many opportunities to engage with the FDA, but there are so few when we can have our voices heard at this level of detail to change the course of drug development for our entire community." Stay informed about upcoming EL-PFDDs meetings relevant to the flok community planned by visiting our Events page.
