"Eteplirsen doubters contend that only with a large clinical trial involving hundreds of DMD children will we be able to collect the data needed to satisfy the regulatory system. But let me tell you, during the face-to-face discussions I and other other DMD advocates had with the FDA (including Commissioner Margaret Hamburg) were were told repeatedly that the agency wanted to see smaller, innovative trial designs that targeted the underlying cause of the disease. FDA is aware of how quickly DMD patients progress and it doesn't want large-scale clinical trials for this disease. It was my sense that the FDA was willing to be flexible and eteplirsen might be the opportunity FDA was looking for to demonstrate their proactive demeanor in accelerated the approval of drugs for diseases like DMD.
At the end of the day, the data speak for itself. The natural history data of DMD patients is undeniable. Left untreated, those diagnosed with this disease follow a predictable decline in muscle function and walking ability. There are few outliers. The eteplirsen dystrophin data are real, the clinical benefit is evident.
Bolstering the case for the drug are anecdotal stories circulating in the DMD community. Eteplirsen patients in the Sarepta trial are beginning do to things physically they never could do before. That rarely, if ever, happens in DMD patients, particularly in this age group. These are important and measurable patient-reported outcomes where parents are seeing gains and stabilization in their DMD kids.
These gains have an enormous impact across the board -- psychologically, sociologically, economically. They are measurable, important and something the DMD "experts" have not taken into consideration. Stopping or slowing the progression of DMD spares families from having to move to a one-story house, buying a handicap van, the fitting for a power-chair or installing ramps to their home. Instead, they are celebrating things like making a basket in basketball ...
Very powerful article. When it gets widely read, it should result in bringing in lots of new investors. It is not only a mother's plea, but it contains very important information about the DMD group's meetings with the FDA. The nay sayers are wrong and Christine drives the point home extremely well.