Patient Focus Drug Development Meeting Posted on March 19, 2022 by melelllan
On March 11, the global Rett Community had an extraordinary opportunity- to educate the FDA about the severe consequences of having a child with Rett syndrome- for the child, the family, the community and society. As we listened to the brave parents who shared their stories, it was draining, emotional, painful and exacted a myriad of other feelings.
I ached for each and every parent. As a parent of an older child, I felt each of Jack’s words like they came from my own heart. As Jenna described the life of her son, Madden, I sat and cried for her, for him, for his family. I felt AJ’s frustration at having waited so long for a treatment; every year that has passed since Katelin participated in the NNZ-2566 (Trofinetide) trial is just one more year of knowing what could have been. I feel like I’m on a deserted island and counting the days with lines chiseled into a granite rock, so the reminder never, ever fades. The tears flowed again as Leslie told us all about Brooke and her 5 year struggle, her accomplishments, a look into her sense of humor and a Rett parent’s greatest fear- not waking up. It’s a fear I’ve become accustomed to- it’s a part of me, a part of my routine- that first moment I awake and I lie there, take a deep breath and dare myself to…check.
The zoom panel was a wonderful addition to this program, in real time, “face to face” with the FDA, parents responded to comments, added insight, sought to hammer home to those in attendance- the FDA, pharmaceutical companies, doctors, students, researchers, therapists, interested persons, hopefully extended family (who often find it hard to comprehend the challenges faced by their loved ones) and a host of others.
Being able to call in, to write in comments that were read on air was an added plus, enabling the larger, global community to participate. Each and every one was important to be acknowledged and I applaud the parents/caregivers that bravely put themselves out there.
Monica Coenraads, of RSRT, opened the session with a grateful acknowledgement of all those in attendance and discussed the great unmet need of Rett syndrome and the need for caregivers to participate in the program. The moderators were amazing. James Valentine, JD, MHS and Dr. Dominique Pichard, of IRSF, kept it all on track. James asked parents follow-up questions to get to the true point-what the outcome of a single change for the better would mean within the context of their comment. Dr. Pichard seamlessly moved on to comments that were relevant at that point of the program. Behind the scenes Mark and Jana fielded calls and lined up the queue. I cannot imagine the effort, coordination and preparation it took to pull off such an impressive, organized five-hour long program, which felt like half an hour.
HOWEVER, our part is not over yet. ANY caregiver, ANYWHERE in the world still has the chance to contribute to this effort. All the stories, comments, calls will be made into a report which the FDA will use when evaluating drugs, treatments, new therapies and equipment for years to come. This is YOUR chance to make a difference in a truly fundamental, long lasting way. All those children who aren’t eligible for trials- parents you can make their struggles matter to the FDA.
Please, go to https://rettpfdd.org/ and share, not only your story, but your hopes, your opinion about risk vs benefit; talk about how making even just one thing better would impact your child’s life, your life. If your child can speak and tell you where something hurts how many ER visits could be avoided? If your child no longer had severe behavior-you could socialize, people would babysit. If your child could be potty-trained she/he could be in daycares, parents could work and contribute financially to the household and to the economy. A child who can walk may one day not need physical therapy; a child who can speak will not need speech therapy. A child who can be part of the regular academic program may be able to work in fields of study parents hardly dare dream of at all. Think of the domino affect of just a single change and let the FDA know.
Parents outside of the US, you are being given an unthinkable gift- the invitation to affect change in the United States and thereby the world. No one doubts that FDA approval all but guarantees approval in other parts of the world. Take this chance. Trials rarely get performed in smaller countries. Make a difference. The FDA wants to hear from you!
I would like to thank everyone who participated in making this happen- IRSF; RSRT; Hyman, Phelps, McNamara P.C.; the FDA, the sponsors and all those who worked behind the scenes. A special thanks to the parents and caregivers who shared their heartaches, frustrations and hopes.
Let’s all pull together and show the FDA- the Rett community is a whole new level of force.
To learn more about Externally-led Patient Focus Drug Development Meetings, go to the FDA’s page
NEU Price at posting:
$4.48 Sentiment: Hold Disclosure: Held
(20min delay)
