pfddmeeting fda

HNF’s Vision Comes to Fruition on September 28

HNF successfully executed the Externally-led PFDD Meeting for the FDA, one of 14 to-date. Its purpose was to accelerate therapy development, support our industry sponsors – Pharnext and Acceleron Pharma – and others to help facilitate a better understanding by the FDA and stakeholders of what matters most to patients and families living with Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN). Also important was developing an understanding of the benefit-risk that patients are willing to consider or tolerate when thinking about a biologic and/or gene therapy. HNF is an innovative patient advocacy and research organization with a history of success, but there is still much work to do, as HNF leads the way to bring new treatments and therapies to our patient community. Your donations are imperative to fund our accelerated strategy as HNF continues to build partnerships with industry leaders and stakeholders in the community through the Therapeutic Research In Accelerated Discovery (TRIAD) Program.

  • There were 698 in attendance (155 in person and 543 on the webcast)  
  • FDA officials attendance exceeded our expectations with 24 participants  
  • 14 patients and their families showed bravery as they provided snapshots of their lives with CMT, five of them were kids and adolescents  
  • Stakeholders stepped up and sponsored HNF to host this groundbreaking event. Thank you to our sponsors: Pharnext, Acceleron Pharma, Cydan, FlexPharma, Athena Diagnostics, Ionis, Ceres, Champlain Valley Dispensary, Cresco Labs, Everylife Foundation for Rare Diseases, CMTA, MDA  
  • The debut of the HNF mini-documentary, “The Warren Family” captured the daily physical struggles and the emotional impact of living with CMT  
  • HNF used innovative Voice Activation Technology (VAT) to capture 27 additional testimonies for the FDA in the patient’s own voice
  • Leading CMT experts gave an overview on the importance of diagnosis, genetics, treatment options, therapy pipeline and clinical trials to educate the FDA and stakeholders on various aspects of CMT

“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/Founder HNF

FEEDBACK FROM THE FDA INCLUDED: “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, PLLC, Acting Associate Director, Rare Disease Program at FDA, Office of New Drugs, CDER, FD

Thank you FDA and all who participated in this groundbreaking meeting! HNF looks forward to continuing to work closely with you on the accelerated path of therapy development for our patient community.

Stay-tuned for more news in early 2019!

YOUR VOICES WERE HEARD! WATCH THE PFDD MEETING

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