Providing real-world data to the FDA
HNF has taken a strong stance by participating and supporting the role of an advocacy group in providing real-world data to the FDA and other stakeholders to help facilitate a better knowledge of the experience patients have living with CMT.
For FDA and other regulatory agencies to endorse drugs or biologics, FDA has to understand the impact of CMT on a patient’s life and determine the best outcomes and what matters most to patients when thinking about a potential treatment. Decisions are made based on minimizing any health risks, life-threatening side effects, and ideal endpoints to improve outcomes to determine if a treatment benefits patients.
In accordance with the FDA’s patient-focused drug development guidance for enhancing the incorporation of the patient’s voice in medical product development and regulatory decision making, HNF has developed a regulatory strategy to engage with industry early on to assist with collecting and submitting robust and meaningful patient experience data, and other relevant information from patients and caregivers, such as identifying what is important to patients.
Resources & Tools
Repository of Patient Information
- Global Registry for Inherited Neuropathies (GRIN)
- Voice of Patient Report
- Electronic Health records (EHRs)
- CPIM Voice Activation Survey
- PFFD Meeting
- CMT Mobile App
PFDD Meeting, September 28, 2018
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.
September 28, 2018, PFDD Meeting
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. Click here for details.
“It’s those sort of things that will come out of your analysis of the survey work that you did today that will really make a difference for helping drug developers, or device developers or biologics or gene therapy developers understand what they should be measuring and how to address this population, meet you where you are for what you want in the future.
So, at least from the FDA, I appreciate this meeting. I think it was very successful. You should all be very proud of the work you did today.”
Landmark “Voice of the Patient” PFDD CMT Meeting Needs All Hands on Deck!
Help support this ground-breaking PFDD CMT Meeting being held on September 28, 2018 in Washington, D.C.
Hope on the Horizon: CMT & FDA Patient-Focused Drug Development Meeting for Charcot-Marie-Tooth to be Held in 2018
HNF will be holding a landmark externally-led Patient-Focused Drug Development (PFDD) Meeting CMT & INs in Washington, D.C. on September 28, 2018. The meeting will be held in conjunction with September National CMT Awareness Month.
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
Are You a Patient With CMT 1A? New Clinical Trial Opportunity Available For You!
For the first time investigators are looking for patients to participate in a pivotal Phase 3 clinical trial of Pharnext’s lead investigational pleodrug, PXT-3003 for the potential treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Pharnext Opens First U.S. Study Site For Its PLEO-CMT International Pivotal Phase 3 Trial In Charcot-Marie-Tooth Disease Type 1A
French pharmaceutical company Pharnext announced the opening of the first U.S. trial site for its PLEO-CMT pivotal Phase 3 clinical trial of its lead pleodrug PXT-3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!
The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.
Positioning HNF to Translate Treatments to Clinical Trials
In June 2013, HNF made the important decision to refine the organization’s mission. We now place more importance on new initiatives that take our research discoveries and translate them to the next phase in the drug discovery process.
We divide our research initiatives into 3 areas: