On September 28, 2018, the Hereditary Neuropathy Foundation (HNF) successfully hosted an Externally-led Patient-Focused Drug Development (PFDD) meeting for the U.S. Food and Drug Administration (FDA). The meeting was one of 14 such externally-led events conducted to that date.

Its purpose was to accelerate therapy development for Charcot-Marie-Tooth disease (CMT) and other Inherited Neuropathies (IN), and to give the FDA and other stakeholders a clearer understanding of what matters most to patients and families. A key focus was helping regulators understand the benefit-risk tradeoffs patients are willing to consider when evaluating biologics and gene therapies.

Meeting highlights

  • 698 total attendees: 155 in person and 543 via webcast
  • 24 FDA officials attended, exceeding expectations
  • 14 patients and family members shared personal accounts of living with CMT; five were children or adolescents
  • HNF debuted its mini-documentary, “The Warren Family,” capturing the physical and emotional impact of daily life with CMT
  • HNF used Voice Activation Technology (VAT) to capture 27 additional patient testimonies in participants’ own voices for submission to the FDA
  • Leading CMT specialists provided the FDA and stakeholders with an overview of diagnosis, genetics, treatment options, the therapy pipeline, and clinical trials

Sponsors

The following organizations sponsored HNF to host this event: Pharnext, Acceleron Pharma, Cydan, FlexPharma, Athena Diagnostics, Ionis, Ceres, Champlain Valley Dispensary, Cresco Labs, Everylife Foundation for Rare Diseases, CMTA, and MDA.

Voices from the meeting

“Although the day was emotional and at times hard to listen to the heart-wrenching testimonies, the empathy and love shared throughout the room came through.”Allison Moore, CEO and Founder, HNF
“What I was impressed with from the beginning is that this is a very organized community. I’d like to recognize the fact that your board has been so forward-thinking about how they’ve been approaching positioning this community for drug development and therapeutic development in general. It sounds like you’ve been very diligent about developing a very good understanding of the natural history of the illness. You have characterized the variability and then done all the hard basic science research of characterizing the genetics that help understand all that variability, so that when it comes time for therapeutics to come into your community, it’s going to be very targeted and very easy to do. It’s those sort of things that will come out of your analysis of the survey work that you did today that will really make a difference for helping drug developers, or device developers or biologics or gene therapy developers understand what they should be measuring and how to address this population, meet you where you are for what you want in the future. So, at least from the FDA, I appreciate this meeting. I think it was very successful. You should all be very proud of the work you did today.”Lucas Kempf, MD, Acting Associate Director, Rare Disease Program, FDA Office of New Drugs, CDER

HNF looks forward to continuing to work closely with the FDA and all stakeholders on the accelerated path of therapy development for the CMT and inherited neuropathy community.