The Fanconi Cancer Foundation is excited to host an Externally Led Patient-Focused Drug Development (EL-PFDD) meeting to center the lived experiences of people affected by Fanconi anemia.
This meeting creates space for individuals with FA and caregivers to share what daily life is really like, including experiences with cancer, long-term health effects, and care decisions. These perspectives help inform future research and treatment development by highlighting what matters most to the FA community.
The EL-PFDD meeting will take place on October 3, 2026, in Phoenix, Arizona, the same weekend as the Scientific Symposium and Retreat for Adults with FA. This meeting is part of the Adult Retreat agenda, meaning, if you are attending the Adult Retreat, you will attend the EL-PFDD meeting as a part of the program.
An EL-PFDD meeting is a public meeting designed to gather input directly from people affected by a condition and share that input with the U.S. Food and Drug Administration (FDA) and other stakeholders. Rather than focusing on scientific data alone, these meetings focus on lived experience, including daily challenges, treatment experiences, and quality of life.
Fanconi anemia is complex and lifelong. Clinical data are essential, but they cannot fully capture the day-to-day realities of living with FA, including cancer risk, long-term health effects, and the impact of quality of life on individuals and families.
By centering lived experience, this meeting aims to help ensure future research and treatments reflect what meaningful progress looks like to the FA community.
EL-PFDD meetings are a recognized part of patient-centered drug development in rare diseases.
What is shared during the meeting is documented in a Voice of the Patient report. This report, along with a link to the meeting recording, can be made publicly available on the FDA’s website and can be referenced by researchers, clinicians, regulators, and medical product developers.
While participation does not guarantee specific outcomes, community input can help shape research priorities and how potential benefits and risks are considered.
This meeting is for people affected by Fanconi anemia, as well as those who want to listen and learn:
There are multiple ways to participate, such as participating as a panelist during the meeting, participating in the large-group facilitated discussion in-person or virtually, or submitting written input. Individuals who participate as panelists will receive preparation guidance and support.
Although the meeting is designed to inform the U.S. Food and Drug Administration and other key stakeholders, international voices are welcome and important. Perspectives from around the world help ensure the full lived experience of FA is represented.
We are working to make the meeting accessible to non-English speakers. More information soon.
The EL-PFDD meeting will take place Saturday, October 3 as part of the Adult Retreat programming.
All FA families are welcome and encouraged to attend. If you cannot make it live, you are invited to attend virtually.
An Externally Led Patient-Focused Drug Development (EL-PFDD) meeting is a public meeting designed to gather input directly from people affected by a condition and share that input with the U.S. Food and Drug Administration (FDA) and other stakeholders.
These meetings focus on lived experience, including daily challenges, long-term health impacts, experiences with available treatments, and what meaningful progress looks like from the community’s perspective.
“Externally led” means the meeting is organized and led by the Fanconi Cancer Foundation, not by the FDA.
While FDA staff may listen and learn, FCF is responsible for planning the meeting, shaping the discussion topics, and gathering community input.
FCF is committed to advancing patient-centered research and ensuring that the voices of people affected by Fanconi anemia are reflected in decisions that shape future treatments.
Because FA is complex and lifelong, lived experience provides essential context that cannot be fully captured through clinical data alone. This meeting is one way FCF is working to elevate those perspectives in a structured and meaningful way.
This meeting is for people affected by Fanconi anemia, including adults with FA, caregivers of adults and children with FA, and family members. Researchers, clinicians, regulators, and medical product developers may also attend as listeners to better understand community experiences and priorities.
No. This meeting is centered on lived experience, not scientific expertise.
Participants are invited to share their own perspectives, experiences, and priorities in their own words. There are no right or wrong answers.
There will be multiple ways to participate, including: speaking or serving on a panel during the meeting, attending in person or virtually and participating in general discussion, or submitting written input to be considered for the Voice of the Patient report. Individuals who participate as speakers will receive preparation guidance and support.
No formal preparation is required to attend the meeting.
For speakers, FCF will provide guidance and support in advance to help participants feel comfortable and confident. Attendees are encouraged, but not required, to review discussion topics and questions ahead of time once they are available.
The EL-PFDD meeting will take place on October 3, 2026, in Phoenix, Arizona.
The meeting is scheduled for the same weekend as the Scientific Symposium and Retreat for Adults with FA. Opportunities to participate virtually will also be available.
Yes. While the meeting is designed to inform the U.S. Food and Drug Administration, international voices are welcome and important.
Input from individuals and families around the world helps ensure the meeting reflects the full lived experience of Fanconi anemia.
Yes. The meeting will be recorded and shared publicly after the event.
Experiences shared during the meeting will be summarized in a document called a Voice of the Patient report. FCF expects to publish the Voice of the Patient report within several months following the meeting. Updates and timelines will be shared with the community as the process moves forward.
This report, which contains a link to the meeting recording, can be made publicly available on the FDA’s website. It can be referenced by researchers, clinicians, regulators, and medical product developers to help inform future research and treatment development.
