Patient-Focused Drug Development Meeting, September 28, 2018

On September 28, 2018, the CMT community gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA. Susan Dentzer, editor of Health Affairs Today, has described patient engagement as the “blockbuster drug of the 21st century.” At this meeting, patient voices were heard to give the FDA and other stakeholders a deeper understanding of the experience of living with CMT and other Inherited Neuropathies. By integrating patient perspectives directly, the meeting actively involved patients in shaping their healthcare.

Why Are PFDD Meetings Important?

Decision-makers are increasingly recognizing that the patient voice is a critical element in developing and approving therapies that provide meaningful benefit to those living with a particular disease. Without patient insights, the FDA’s ability to assess the benefits and risks of a therapy, and to ensure it provides real benefit to patients, is significantly limited.

The purpose of this meeting was to provide the FDA with insights from CMT patients, families, and caregivers on the impact CMT has on their daily lives, including the symptoms they consider most important. The FDA also sought to understand how patients treat and manage their CMT symptoms and what factors influence their treatment choices. This feedback informed the FDA on the severity of CMT, the benefit-risk balance of treatment options, and unmet medical needs. Patient voices from panelists, attendees, and webcast participants all helped shape the assessment of future therapeutics for CMT.

Patient-Centered CMT Behavioral Health Summit, September 29, 2018

Through the Hereditary Neuropathy Foundation Patient-Focused Research Initiative, HNF also held the first-ever Patient-Centered Behavioral CMT Health Summit on Saturday, September 29, 2018, at the College Park Marriott Hotel and Conference Center in Hyattsville, MD. The summit addressed the emotional and behavioral health needs of the CMT community.

Estela Lugo, HNF Medical Outreach Manager, described the vision for the event:

“We often hear so much in regards to the physical aspects of disease and disability, but very little on how it impacts our mental well-being, which for many can be more devastating. Our goal with this summit is to provide a curated ‘CMT Toolbox’ that attendees can apply to their daily lives for increased fulfillment and wellness across all avenues.”

The day opened with an interactive empowerment session developed for the CMT community by Lisa McCarthy, co-founder of The Fast Forward Group. Attendees also heard from orthopedic surgeon Glenn B. Pfeffer, MD, on how surgery outcomes can affect behavioral health. Two breakout workshops, moderated by Lainie Ishbia, founder of Trend-Able, and Alana Kessler, Registered Dietitian and Holistic Health Coach, explored lifestyle, nutrition, confidence, and social interactions.