Dear Friends:
As we embark on the New Year, I am humbled by the support of our stakeholder community and the dedication to accelerate therapies to you and your families in the next few years. We are so close to the first treatment for CMT and we are doing our best to accelerate more treatments in the next few years. 2018 will be “the year” for our community as we prepare for the externally-led Patient-Focused Drug Development Meeting.
This meeting will take place in Washington, DC, on Friday, September 28. Our annual Patient-Centered Summit will follow on Saturday, September 29.
FOR MORE INFORMATION: WWW.CMT-PFDD.ORG
HNF is inviting all advocates and stakeholders in the CMT community to join this meeting to advocate for the needs of the CMT/IN patient community. Patient voices are critical in understanding how to develop and approve therapies that provide clinical and meaningful benefit to those living with CMT. Bringing the voices of patients, caregivers, and advocates to the conversation will produce the data and documentation needed to publish a summary of the day’s proceedings in the form of the “Voice of the Patient” report that can be used by FDA leadership as well as other stakeholders in the drug development process. This input can help inform the FDA’s decisions and oversight during drug development and the review of marketing applications for new drugs.
It is important for us, as patients to understand the years it can take to reach commercialization of a therapy approval by the FDA. The typical timeline from “bench to bedside”, which in layman’s terms means from disease models to successfully treating the disease, takes many years. The process of testing possible therapies in assays (cell and animal models), Biomarker Development is 12-15 years minimum. That means from the time of Basic Research (1-5 years) and Discovery to PreClinical (5-10 years) to Clinical to Distribution and Access (2-3 years). For people like us living with CMT, this is way too long!
HNF Stands for Acceleration of Therapies to Patients TODAY!
What’s next? HNF will be relaunching the new and improved Patient Registry — Global Registry of Inherited Neuropathies (GRIN) — and now, more than ever, we need your help! These patient reported outcome (PRO) research studies will be analyzed by CMT experts in the field and presented at the PFDD meeting. These patient outcomes will enhance the therapy development of our industry partners, inform all CMT scientists that have had break-throughs that may lead to treatments and most importantly, give us more insight into what matters most to you as CMT patients.
It is our time to let our voices be heard by FDA officials, legislators, the National Institute of Health and all stakeholders who have committed to joining us on this monumental day, September 28, 2018.
I am getting my third CMT related operation next week. I will be off my feet 6 weeks. They will fuse my big toe (stands up straight). Correct my other toes. They say next I will need total reconstructive surgery to move my heel and ankle, so they align. I thought AFOs helped with that.