On Friday, September 28, 2018 HNF hosted an Externally-led Patient Focused Drug Development (PFDD) Meeting. In attendance were patients, caregivers, Government Officials, Healthcare Providers, Industry Representatives, and Payors. The PFDD meeting was an opportunity for patients and families to inform the FDA, drug developers and other key stakeholders, on the true burdens of living with CMT and how patients view the benefits and risks of treatments for CMT. This groundbreaking meeting included facilitated panel discussions designed to provide the FDA with perspectives from people with hereditary neuropathies, advocates and caregivers. The day focused primarily on a range of patient viewpoints on CMT, covering the symptoms and impacts to daily life that are most important to patients and patients’ perspectives on existing and future treatments.
We thank the countless individuals that participated in-person and the hundreds from the webcast.
Your Voices were HEARD.
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Welcome and Opening Remarks
Overview: What is CMT? – Michael Shy, MD
Overview: What is CMT? – Dr. Stephan Züchner, MD, PhD
Goals & Objectives for the Meeting, Participant Polling of Demographic Questions
Session 1: Symptoms and Daily Challenges for Pediatrics & Young Adults
Session 3: Current and Future Approaches to Treatments
Pipeline Overview Therapies in Development – Florian Thomas, MD, MA, PhD, MS
Summary from FDA & Closing Remarks