Dr. Temple thinking about innovative trial design in Duchenne

Posted by: Christine McSherry on July 19, 2013


Almost religiously, I have been re-reading Dr. Temple’s transcript from his presentation at the PPMD conference in June. He ended his presentation with the following comment.

“There’s also nothing that says that a company that’s doing its major trial with one group of people
can’t have another trial in a different group that is ineligible for the first. That other trial could be a controlled trial, it could be just observations to get more experience, there’s considerable reason to do all those things, no bar too.”

This statement leads me to believe that he is seriously considering the design of the next trial(s) and how it might not be necessary for a placebo – but instead, to use a cohort of individuals who are not eligible for that particular exon – and use as them as the control.

This sounds like Dr. Temple was listening to the Sarepta presentation from the World Orphan Drug Conference,
where the CEO of Sarepta discussed leveraging a personalized medicine platform to treat all patient
genetic subgroups in rare disease. By allowing and supporting this innovative-type trial design, sponsors could build the foundation of evidence that could be used in considering platform approval for exon skipping technologies – faster. And faster is good.
I like the way Dr. Temple is thinking.

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