Recently, the Hereditary Neuropathy Foundation (HNF) announced they received the prestigious Eugene Washington Engagement Award from Patient-Centered Outcomes Research Institute (PCORI) to help fund the Patient-Centered Charcot-Marie-Tooth Summit that will be held in New York City, October 6, 2016. In an exclusive interview with Rare Disease Report, Allison Moore, founder and CEO of HNF describes the summit and the value of the PCORI award in making the summit a ‘game changer’ for Charcot-Marie-Tooth research.
What is the purpose of the HNF Patient-Centered Charcot-Marie-Tooth Summit?
To identify gaps of Patient-Centered Outcomes Research and Comparative Effectiveness Research (PCOR/CER) for Charcot-Marie-Tooth (CMT) and Inherited Neuropathy (IPN) Community. The goal is to identify patient-centered measurement tools that are fundamental to research and clinical practice. Patients will have the chance to hear the latest in research development, upcoming clinical studies and trials, and participate in panel discussions with clinicians, and voice their needs and priorities in regards to CMT and treatments.
Why is such a summit necessary?
A summit in real time will help to bring patients, caregivers, clinicians and researchers together to discuss Charcot-Marie-Tooth research and the issues surrounding the lack of awareness and understanding of a disease that affects hundreds and thousands of people that are suffering in silence. By uniting one strong voice, HNF sees this as a “game changer” research opportunity to increase research and engage industry and other stakeholders to develop therapies for CMT.
What is the agenda and/or some of the speakers scheduled?
The agenda is chock full of interesting topics ranging from Embracing Differences; engagement to empower one voice among all stakeholders, The Caregivers Voice Matters; the role of the caregiver in patient driven research, and Participating in a Clinical Trial: The Good, the bad and the benefits! Speakers will be announced throughout the spring to excite all CMT patients and stakeholders to attend what is surely to be a landmark event!
Is funding available to help families attend the summit?
We are working on pricing now, and will be sure to keep the costs reasonable for patients and their families. The summit will include breakfast, lunch and a cocktail party with hors d’oeuvres afterwards so patients can mingle with clinicians, researchers, and industry sponsors that are dedicated to developing therapies for CMT.
What is the Eugene Washington PCORI Engagement Award?
PCORI is an independent, non-profit organization authorized by Congress in 2010 to fund comparative effectiveness research that will provide patients, their caregivers, and clinicians with the evidence needed to make better-informed health care decisions.
How can the PCORI concepts help rare disease communities?
HNF is determined to bring the unique voice and experiences of patients and family members and advocates to play a central role in the Summit. The earlier we engage patients, caregivers and stakeholders in the PCORI research process from topic discussions to dissemination and implementation of results, the more likely this will show direct benefit for patients to move research toward a more patient-centric approach. Not only is HNF dedicated to curing CMT, but it is an advocate for all rare diseases and the concept can be applied to any rare disease to accelerate therapy development.
Courtesy of Jim Radke, Rare Disease report