Just to be clear, my posts, my thoughts, hopeful predictions and emotions expressed here today are my own. And, no, I don’t have the benefit of a degree or knowledge of the inside workings of the FDA, nor where it could even be possible that Neuren couldn’t make its case for Breakthrough Designation, but I am a writer and, in that, strive to be honest in how I feel, and today I feel the FDA failed.
How can a treatment show substantial improvement over an existing therapy when there isn’t one??
Why do the way improvements were measured have to be conventional standards? Why can’t improvements be measured in a way that matters for Rett syndrome? Why can’t the way improvements were measured be the NEW standard?
We were there- Us parents who sacrificed our time, incurred financial burden, took a chance that it might not work and we KNOW the true measurements that matter, we saw them, we heard them, we felt them and EVEN YOU, the FDA, agree there’s something there. You just don’t like the way it was measured. Well, I want to just pound on your chests, in your pinstriped suits, with your engraved pens tucked in pockets, the ones that checked that “no” box. I want to show you all the faces that were shared today, hopeful smiling faces, that you let down.
I want to grab you all by the lapels and drag you back in time and SHOW you. How could you?! Really? When you know that it could be rescinded if it wasn’t working? When you know all the promising trials going on right now. Aren’t you aware that you, FDA, just set our countdown clock back up and that timeframe of “too late” became a possibility for more girls and boys?
You FAILED. You failed by not being brave enough to think outside the box of standards that may be out of date or just plain wrong in this situation. You FAILED to look beyond a graph and see the implications. You FAILED to listen to reason and hear our voices crying out for a chance, one we believe in and have seen work with our own eyes. And, you FAILED to be the change in the world of Rett syndrome and to set an example of forward thinking.
Oh, for sure, I don’t know the “whole story” as to why you said no, and right now, today, I don’t care. I am a parent. I know my daugher’s whole story, I know the story of others; those are the whole stories that matter, and yes they’ll all get read, and I’m pretty sure that the last lines of some will go…..and she got Trofinetide and could finally say “I love you, mom.” The End. But, you made a picture book into a novel and not everyone has that kind of time.