Silver Springs, Maryland

12/12/13  8:30 AM 

There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.

Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the Maryland Room at the DoubleTree Hotel in Silver Springs, MD. We opened the morning with Session 1: six individuals who had prepared statements to describe life with Duchenne in the context of the need for therapies and to preserve and protect function at every stage of the disease. These parents and two young men with Duchenne discussed the need to consider the use of a novel clinical trial designs to exclude a placebo group because each day muscle fibers are lost and the fear of placebo for the duration of a trial means the individual will lose muscle fibers and likely function. They talked about urgency. They discussed living with Duchenne and what might make a meaningful difference in their lives. When the discussion opened up to attendees to make a two-minute comment about their own experience, the message was the same. We don't have time. We delivered a cohesive, united statement.

Session 2 discussed natural history and outcomes that would enable trials to be inclusive of patients of all ages. Session 3 discussed requirements for surrogates and biomarkers, the need to have a measure that would predict clinical benefit which could result in shorter trials and more rapid approvals. 

Each session included comments from the community, reiterating the need to avoid placebo, utilize natural history as controls, expedited review, and the use of accelerated approval, getting drugs approved and access for all.


Session 4 was about next steps, the science of writing a draft guidance. The community agreed and has signed on to participate. 

PPMD's Vice President of Research, Sharon Hesterlee, PhD, tweeted notes throughout the day. Those notes will be forthcoming and will give you a more in-depth recap of the day's events. Unfortunately we were not allowed to record the sessions - only stream them online.

And now the work of actually writing the draft guidance begins. Dr. Bastings (FDA) ended the meeting by saying that Duchenne has the highest priority within the FDA.


Goal: Guidance, May, 2014   

The patients are waiting.

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Comment by Ofelia Marin on December 13, 2013 at 1:12pm

Thank you for doing this. Our message was clear and the discussions were very informative!

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