FDA - New, Innovative?

Posted by: Christine McSherry on April 12, 2013


I had an informative and thought provoking day yesterday at Cold Springs Harbor Labs.  Here, many of us heard the long awaited, and early released data from GSK.  I have comments about the data, but will blog on that in a little while...


Upon conclusion of the presentation, I was approached by a woman and asked if I was one of the "moms" who have been meeting with the FDA on behalf of Eteplirsen, or exon skipping.  Yes, I was one of the three (Mindy and Jenn respectively).  This woman asked me why there was so much community support around Sarepta for Eteplirsen and not GSK and their compound - I simply replied that we are not on any one team, but rather the "boys team". Since GSK/Prosensa had not announced the results of their trial, up until today, as Sarepta had - well, then it makes it difficult to represent them. We are hopeful to see accelerated approval used for any and all promising therapies in Duchenne - Duchenne has unmet medical need and urgency!


Then I was asked about the "stone - faced, unfriendly" FDA officials that we had been meeting with.  I was pleased to respond by letting her know that the officials were hardly stone faced, but to the contrary.  Those that we met with were compassionate about Duchenne.  They understood the disease well.  They agreed that the disorder was not "chronic and manageable", they agreed it was "acute and deadly".  They offered suggestions and ideas related to how the community can help the FDA, help industry, add clarity and support scientific evidence often provided by sponsors.  They were glad to see us, asked about our boys, took time to really listen to our stories and invited us back.  They empowered us, educated us and engaged our opinions and expertise as "experts" in Duchenne...and as parents, all of us as parents - we are the experts.  We talked about patient reported outcomes for the non ambulatory boys, and how that can affect upcoming trials in this demographic.


They discussed the areas where they were flexible, we talked about past successes and where pharmacovigilence played a role in risk/benefit.


We also talked about biological activity of compounds and how it can translate into function.


Last time we met, this past Monday (04/09/13), we talked for two hours...


Is there a shift in the FDA, is this what we are seeing with these interactions?  Based on my interactions with the FDA there is little doubt in my mind. Commissioner Hamburg and Dr. Woodcock often alluded to a “new FDA”. I must say that the FDA has been extremely receptive to listening to parents in the community and open to innovation in the industry. This FDA has been streamlined, responsive, and efficient in their attempt to evaluate life saving therapies. And for that I am extremely grateful.


It is quite exciting to be sitting on the sidelines of this transformation, all the while - looking at a potential therapy to save my son - and many others.


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