FDA Patient-Focused Drug Development
In September 28, 2018, HNF became the first patient organization to convene the FDA for an externally-led Patient-Focused Drug Development (PFDD) meeting for Charcot-Marie-Tooth disease, so the FDA could hear, directly from people living with CMT, what the condition really takes and what they need from a treatment. It was a groundbreaking day, and its findings still shape how CMT therapies are developed and reviewed.
What the meeting was
A Patient-Focused Drug Development meeting is an FDA initiative to bring the patient’s voice into medical product development and regulatory decision-making. HNF’s meeting was externally-led, meaning HNF convened it on the community’s behalf. It was an opportunity for patients and families to inform the FDA, drug developers, and other stakeholders about the real burdens of living with CMT, and about how patients weigh the benefits and risks of treatment.
The day centered on a range of patient viewpoints: the symptoms and daily-life impacts that matter most, and patients’ perspectives on existing and future treatments. HNF thanks the countless people who took part in person and the hundreds more who joined by webcast.
Who was in the room
The meeting brought every part of the drug-development conversation into one room, with patients at the center.
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.
From the meeting
Watch the FDA testimony
What did the FDA have to say about HNF’s approach to preparing for CMT clinical trials with natural history studies? In a short testimony from the meeting, Dr. Kempf put it plainly: “Doing those natural history studies provides all the information about what you need to measure, how often you can measure it, what is tolerated by your community.”
Watch the meeting sessions
The full recordings from the September 28, 2018 Patient-Focused Drug Development meeting, from the opening remarks through the patient sessions to the FDA's closing summary.
What the meeting produced
- 1 The patient voice, on the record Facilitated panels let people with hereditary neuropathies, advocates, and caregivers describe the symptoms and daily-life impacts that matter most to them, and their views on existing and future treatments.
- 2 The Voice of the Patient report The meeting produced a Voice of the Patient report, a structured account of patient experience and treatment priorities that HNF still shares with the FDA, biotech, and pharma.
- 3 A reference for trial design Developers use the findings to understand what to measure, how often, and how to design trials around the CMT population, meeting patients where they are.
A lasting resource
The Voice of the Patient report
The report distilled what was heard at the meeting into a document the FDA, drug developers, and biologics and gene therapy teams can use. For the FDA to endorse a drug or biologic, it has to understand the impact of CMT on a patient’s life and what matters most to patients in a treatment. The Voice of the Patient report is HNF’s way of putting that understanding on the record, and HNF continues to share it as therapies move toward trials.
From the meeting
The meeting program book
The program book from the September 28, 2018 meeting lays out the day the CMT community brought its experience to the FDA: the agenda, the panels, and the patient and caregiver voices that shaped the Voice of the Patient report.
Frequently asked questions
What is a Patient-Focused Drug Development (PFDD) meeting?
A Patient-Focused Drug Development (PFDD) meeting is an FDA initiative to bring the patient voice into medical product development and regulatory decision-making. An externally-led PFDD meeting is convened by a patient organization on the community’s behalf, so patients and families can tell the FDA and drug developers directly what living with a condition is really like.
When did HNF hold its PFDD meeting for CMT?
HNF convened its externally-led PFDD meeting for Charcot-Marie-Tooth disease in September 28, 2018. HNF was the first patient organization to convene the FDA for a PFDD meeting for CMT.
What is the Voice of the Patient report?
The Voice of the Patient report is the document produced from the meeting. It distills what patients said about their symptoms, daily-life impacts, and treatment priorities into a structured account that HNF still shares with the FDA, drug developers, and biologics and gene therapy teams.
How can I add my voice to the patient record?
The patient voice is strongest when it is backed by data. Joining GRIN, the Global Registry for Inherited Neuropathies, and completing its natural-history survey adds real-world evidence the FDA and developers can use to evaluate future CMT treatments.
Help the FDA hear more
The patient voice is only as strong as the data behind it. By joining GRIN and completing the natural history survey, you add real-world evidence the FDA can use to evaluate future CMT treatments.
Working on a CMT therapy? See how to bring patient-experience data into your program on our FDA & regulatory page, or read about patient-reported outcomes →