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Patients Help FDA Understand Life with NF

On June 13, a group of six NF patient advocates addressed the FDA to share their experiences of living with neurofibromatosis (read our first announcement here). Pam Knight, Director of Clinical Programs, was one of several Children’s Tumor Foundation staff members to join the group. Here, she talks about the experience and the impact this group had:  

Five young people with NF1 and one with NF2 traveled to Bethesda, MD with Children’s Tumor Foundation (CTF) staff on June 13, 2019 to offer vivid, unforgettable personal accounts of living with NF to officials from the Food and Drug Administration (FDA). The format, Voice of the Patient, is an informal discussion designed to help those involved in the drug approval process understand the obstacles and unmet needs of those who might benefit from emerging new drug treatments.

Following an emotional session in which representatives ranging in age from 14 to 26 and their parents described their medical journeys and hopes for the future, Andrea-Furia Helms, Director of the Patient Affairs Staff, expressed gratitude on behalf of the agency, saying, “You are the experts in NF. We need to hear from you. Your stories are critical for us to understand.”

Also present at the session were FDA officials from the Center for Biologics Evaluation and Research (CBER), Center for Drug Evaluation and Research (CDER), Oncology Center of Excellence (OCE), Office of Orphan Products Development (OOPD), and Office of Clinical Policy and Programs (OCPP). The session ran well past the one hour allotted as the FDA asked the youngsters for more details about the burden of NF, access to clinical trials, and ways to change the process to serve patients better, for example, with de-centralized clinical trials that would reduce the amount of time away from work, school, and family.

The patients, Aidan Frasier, Jack Burke, Jesse Sorman, Brianna Worden, Maureen Hussey (speaking for her daughter, Maggie), and McKinnon Galloway, were eloquent and impressive in both their knowledge of NF and the skill with which they communicated it.

“…I am actually one of the lucky people with NF, I am fortunate to have access to great doctors and the best care I could hope for. At the same time, I have been bullied, ignored and disregarded because of the way I look.When I walk down the street people stare at me. I understand because no one has seen someone who looks like me, but it is very, very hard,” Aidan Fraser shared.

Answering questions from the clearly engaged and concerned FDA audience members about how FDA could improve the clinical trial experience, the patient reps highlighted difficulties in finding opportunities and deciding about clinical trial participation, getting different specialists to communicate with one another, and the lost potential of talented young people who spend too much of their time with doctors and in hospitals. Unspoken, but strongly implied, was the idea that efficient FDA approval by FDA of safe, effective drugs now in development could revolutionize lives and free up much of this potential.

FDA was also interested in hearing about CTF-sponsored efforts to standardize clinical trial endpoints by supporting Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS), which REiNS and CTF Patient Representative Maureen Hussey explained in detail.

While the combination of long-distance travel and re-living physical and emotional challenges took a toll, all agreed the day was a great success. As CTF President Annette Bakker noted, the interaction between FDA and patients representatives demonstrated an amazing evolution toward empowered patients. Ms. Helms of the FDA expressed similar feelings, and noted that the day’s meeting would not be the last opportunity for patient reps to engage with FDA.

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

About the Rare Disease Listening Sessions: This initiative is a joint pilot of the FDA Patient Affairs Staff (PAS) and the  National Organization for Rare Disorders (NORD). The goal of the initiative is for the listening session to serve as a resource for the FDA medical product centers to engage with patients and patient advocates. This face-to-face meeting of NF patients with representatives from multiple centers at the FDA will facilitate the sharing of NF patient perspectives on disease burden, treatment burden, impact on daily activities, quality of life, and priorities to consider in medical product development programs. The objectives are to inform regulatory decision-making, educate review staff about NF, help patients understand the FDA’s mission and work, and provide a starting point to inform research & development.

Click here to view more photos.