https://trailtoatexastrial.wordpress.com/A New Acronym for...

https://trailtoatexastrial.wordpress.com/

A New Acronym for GLOBAL Participation!!Posted on January 29, 2022 by melelllan

This week, I and 116 other participants engaged in a virtual presentation of a pre-FDA EL-PFDD. What a mouthful! So, what the heck does THAT all mean. Well, it means that on March 11, 10-3pm Eastern time I and ANYONE who wishes to attend can participate in a virtual public meeting with the FDA!

EL-PFDD stands for Externally Led-Patient Focus Drug Development Meeting and you can read all about it at Rettpfdd.org. The FDA wants to know more about Rett syndrome. Basically, it is YOUR chance to directly interact with the FDA about the impact on Rett syndrome on your life, your child’s life, your family’s life, so that when they consider future drugs and treatments the FDA has information about what parents and caregivers think/feel about risk/side effects vs benefit.

What is it NOT? It is not the time to bring up pricing, insurance coverage, or why the process takes so long. The FDA does not have any influence on pricing nor insurance coverage. Their process is not open to discussion in this forum. Rett syndrome trials and other rare diseases typically have sped up processes with fewer participants needed. In terms of time, our trials are years quicker than most. Rare diseases and particularly rare PEDIATRIC diseases almost always get expedited review- which is how long the FDA has to consider whether to approve or disapprove a drug, which is four (4) months shorter than than the usual review time of ten (10) months.

Here’s the best part! IT IS OPEN TO ANYONE IN THE WORLD. This is an opportunity for extended family, doctors, teachers, siblings and, just as importantly, those outside of the United States to participate in a unique way. I can honestly say I have never heard of such an important aspect of American life in general being able to be influenced by the voice of those outside of America. I think this is due to the reality that what the FDA does impacts the greater global community and therefore it acknowledges the voice of that global community deserves to be heard.

It is important to note, this is not about any singular drug/treatment. This information will be/can be used when considering drugs, gene therapy, new mechanical treatments, new respiratory treatments etc. This report, when finished, will be an invaluable resource for the FDA in multiple aspects.

Let your voice be heard! Put it on your calendars! For those in other countries, notify your respective Rett Associations so that other parents can find out about it, spread the word! Let’s make a good showing and thereby let the FDA know WE are a committed GLOBAL community!

I would like to thank IRSF and RSRT in co-sponsoring this opportunity.