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At the end of the day, if there were a drug that stopped FSHD in its tracks, would you want it, even if it did not restore what you have already lost? What if a drug made you stronger temporarily, even if it did not stop the disease? Only those who are directly affected by FSHD—the patients, family members, caregivers—can say what is a meaningful benefit and acceptable risk of a therapy. The “voice of the patient” in these matters carries great weight with the Food and Drug Administration (FDA) when it considers an application by a drug company to put a new therapy on the market.
The FSHD Society’s Patient Focused Drug Development (PFDD) meeting, scheduled for April 21, 2020, is our forum to present testimony by patients and family caregivers to the FDA. This is your chance to educate the FDA on how FSHD has impacted your life or the life of your loved ones. It also provides an opportunity for you to describe what is most important and how a successful therapy could improve your quality of life. The resulting Voice of the Patient report will be published and submitted to the FDA’s Division of Neurology Products, Office of New Drugs, for inclusion in the framework used to evaluate future FSHD therapies. The FDA will use that information as it evaluates the potential benefits and risks of a therapy to the FSHD patient community.
Our PFDD is a milestone event to gather and summarize input from patients across the country, so we encourage everyone, at every stage of disease progression, to get involved, whether through in-person attendance at the meeting, on-line attendance and voting, or through participation in surveys we will send out to gather data prior to the meeting.
The Patient-Focused Drug Development is a key initiative of our 2020 FSHD Therapeutics Accelerator, and another way the FSHD Society is working to get treatments to you faster.
I am a late onset symptomatic FSHD person, will be turning 71 soon. At the least I would like this to be stopped in it’s progression. Reversal would be great, probably not in my lifetime though. This condition has taken away a good deal of independence that I was used to up until 2010. I saw my Mother progress to being wheelchair bound, I’m fighting that. She lived to be 90, and I live in fear that that will be my fate as well. She hated it, not really knowing what it was taking away her ability to just use the bathroom by herself.