Rare Disease Listening Sessions
FDA’s Patient Affairs Staff (PAS) in partnership with the National Organization for Rare Disorders (NORD) is excited to lead a Rare Disease Listening Sessions Pilot. Patient Listening Sessions are intended to be a resource for the medical product Centers to expeditiously engage with patients or their advocates. Listening Sessions can either be FDA-requested (in cases where FDA has a specific set of questions to ask of a particular patient sub-population) or patient-led (when a patient community wants to share some perspectives with the FDA), and are generally focused on conditions of interest to staff across the medical product Centers. At this time, these meetings are not for industry stakeholders.
For all Listening Sessions (both FDA-requested and patient-led) PAS expects that the Listening Sessions could serve any of the following objectives:
- Inform regulatory decision-making
- Provide a starting point to inform early stage research & development
- Educate review staff about rare diseases or specific segments of non-rare diseases
- Help patients and their advocates understand the FDA’s mission and work
At present, PAS is focusing Listening Session efforts according to the following criteria:
- Rare disease area, defined as a condition affecting fewer than 200,000 patients in the U.S.
- Of interest to medical products staff in multiple Centers/programs
- No prior interactions and no currently planned interactions with the FDA on the same set of agenda topics
Following a Listening Session, PAS will post high level meeting summaries on this website. Check back often as we continue to add information about this important initiative.
FDA-Requested Listening Session Summaries
- May 13, 2019 - Sanfilippo Syndrome (summary coming soon)
- December 4, 2018 – Fabry Disease (PDF - 171KB)
- October 23, 2018 – Gene Therapy as a Treatment Modality for Hemophilia (PDF- 205KB)
Patient-Led Listening Session Summaries
- June 13th, 2019 - Children’s Tumor Foundation, Neurofibromatosis (NF) (summary coming soon)
- May 29th, 2019 - International Fibrodysplasia Ossificans Progressiva Association (IFOPA) (summary coming soon)
- January 16, 2019 - ALS Association (summary unavailable)
- November 5, 2018 - American Liver Foundation
Non-Rare Disease Listening Session Summaries
- February 20, 2019 - Celiac Disease (PFD-200KB)
Request a Rare Disease Listening Session
FDA is continuing to develop programs to incorporate patient perspectives in FDA’s activities. The rare disease listening session program is just one of several patient engagement initiatives across the medical product centers.
To request a Rare Disease Listening Session, email PatientAffairs@fda.gov. FDA recommends that requestors consider a listening session that represents their entire disease community.
Discussions in FDA Rare Disease Listening Sessions are informal. All opinions, recommendations, and proposals are unofficial and nonbinding on FDA and all other participants.
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