The Hereditary Neuropathy Foundation Shares Voice of the Patient (VoP) Report

NEW YORK, Sept. 26, 2019 /PRNewswire/ -- The Hereditary Neuropathy Foundation (HNF) is proud to share the Voice of the Patient (VoP) Report which was recently published on the FDA website. The report highlights the results of the Externally-led Patient-Focused Drug Development (PFDD) Meeting. This is a tremendous milestone for the CMT community - the US Food and Drug Administration (FDA) and others are now aware of the burden of our disease and the unmet need for treatment. 

On September 28, 2018 hundreds of Charcot-Marie-Tooth/Inherited Neuropathies (CMT/IN) Patients, Caregivers, Government Officials, Healthcare Providers, Researchers, Industry, Patient Advocates and Payors came together for an Externally-led PFDD meeting for CMT/IN to have their voices heard, and to prepare for a brighter future. We heard from the  community through stirring testimonies, community polling, and open discussions addressing the critical issues that patients and their families are dealing with throughout their lifetimes. Our mission was to improve inclusion and empathy, reduce stigma, and address the unmet medical needs of our community today. We want to thank the FDA for honoring HNFs request to hold this very important meeting, and for all the industry and advocacy sponsors who made this possible. Special thanks go to our honorable FDA speaker, Lucas Kempf, MD, PLLC, Acting Associate Director, Rare Disease Program at FDA, Office of New Drugs, CDER, FDA, for his time and support of this meeting, and to all the senior FDA leaders who attended the meeting. A very special thanks for the ever-present expertise and meeting planning guidance received from Meghana Chalasani, Decision Support and Analysis Team, FDA.

We would like to thank our sponsors: Pharnext, Acceleron Pharma, Cydan, FlexPharma, Athena Diagnostics, Ionis Patient Advocacy, Ceres, Champlain Valley Dispensary, Crescolabs, and Everylife Foundation for Rare Diseases. In addition, the Charcot-Marie-Tooth Association and the Muscular Dystrophy Association for their sponsorship of the meeting and for their support in the development of this VoP report on behalf of the entire CMT/IN community. This meeting simply would not have happened if not for the amazing collaboration with James Valentine, JD/MHS, who served not only as an excellent moderator, but also as a critical advisor as we prepared for the meeting. This VoP report marks the beginning of a brighter future for people living with all types of CMT/IN throughout the world. We especially want to recognize the panelists who generously gave their time and who were so brave in sharing the intimate stories of their lives and experiences living with CMT/IN. We are grateful to all the patients and their caregivers, and to all the people who selflessly participated live and on the webcast www.cmt-pfdd.org in this landmark event. Thank you all for sharing your hopes, expectations and desires for future treatments. Your voices will impact the future of CMT/IN and without your contributions; this meeting would not have been possible! 

Read the report here: https://www.fda.gov/drugs/development-approval-process-drugs/external-resources-or-information-related-patients-experience

About Hereditary Neuropathy Foundation (HNF)
HNF, a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of CMT and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures. HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. Currently, TRIAD involves many groups that span the drug discovery, drug development and diagnostics continuum. 

The Global Registry for Inherited Neuropathies (GRIN), HNFs natural history study on CMT/IN was relaunched in 2018. Anyone diagnosed with CMT or other Inherited Neuropathies can join GRIN. The information is always kept confidential: only approved research investigators and industry partners can see the de-identified information. If you are already a participant, we still need you! It's important to update your profile and participate in many of our Patient-Reported Outcomes Research Studies. With all of the current positive research momentum, there is no better time than right now to join GRIN. Become a part of the effort to find treatments and cures for all Inherited Neuropathies!

Contact: Allison Moore
T:  1-855-HELPCMT (435-7268)
E: [email protected]

SOURCE Hereditary Neuropathy Foundation