Summary Report from NF Patients’ Testimony to FDA about living with cutaneous neurofibromas

This past spring, a group of six patient representatives of the NF1 community met virtually with the FDA to share their perspectives on living with NF1 and cutaneous neurofibromas (cNFs) as part of thePatient Listening Sessions(PLS) FDA program.

This is the second time the Children's Tumor Foundation coordinated a Patient Listening Session. The first, in 2019, highlighted the experience of NF patients broadly, with a particular focus on plexiform neurofibromas, This meeting, on May 21, 2024, aimed to shed light on the lived experiences of patients with cNFs, an often under-appreciated manifestation that is present in 95% of NF1 patients. Whereas cNFs have been historically considered a "cosmetic" issue, recent studies have brought to light the fact that cNFs strongly impact quality of life (causing pain, itching, and significant social distress) and remain a top priority for the NF1 community.

CLICK HERE TO READ THE FDA PATIENT LISTENING SESSION REPORT

Dr. Annette Bakker, PhD, CEO of the Children's Tumor Foundation, detailed CTF's role in advancing treatments for NF. Dr. Jaishri Blakeley, MD, of Johns Hopkins University, then gave a brief overview of the disease state, recounting for the FDA the clinical characteristics of cNF in NF1 and what we know about the natural history of the condition.

We then moved to the reason we were all gathered for this session: the patient and care advocate testimonies. Each patient representative shared their story of living with NF1 and how cNFs impact their and/or their child's physical and emotional health. As one speaker, Wendy S., bravely shared, "Cutaneous neurofibromas can significantly impact an individual's physical and emotional well-being, and it's offensive to dismiss them as merely cosmetic concerns. Dismissing these concerns fails to recognize the profound impact it has on individuals." This frustration with the medical community for failing to understand the perspectives of patients living with NF1 and cNFs is exactly why centering the voice of the NF community is essential.

Patient listening sessions are an opportunity for the FDA to hear from the NF community directly.The FDA staff - who are experts in their fields and among the best science has to offer - listened attentively to the shared experiences of these brave NF patients, experiences that are not captured in medical charts or scans. These snapshots of living with cNF are powerful glimpses into the realities of NF that often go unheard because of lack of knowledge, or fear, or both.The next time an NF treatment comes to the FDA, they will have the voice of actual NF patients in mind as they evaluate it.

CTF's participation was initiated and organized by CTF's Patient Engagement team, who worked closely with the FDA staff to bring this opportunity to the NF community and prepare the patient representatives for their roles. If you or anyone you know is interested in learning more about CTF's Patient Engagement Program, please click here.