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PPMD Benefit/Risk Study Results Published in Clinical Therapeutics

PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration’s (FDA’s) benefit–risk assessment of emerging Duchenne therapies.

The study was completed in collaboration with John F P Bridges, PhD, an expert in the application of stated-preference methods and engaging patients and caregivers in benefit-risk analysis.

The study supports what you shared with us in our Share Your Story project (see some examples below). We have been, and will continue to be, aggressively engaged with the FDA to keep this community’s voice in the approval process.

“When it comes to terminal illnesses [the FDA’s] job should be to make sure a product is safe and that the risks and benefits presented by the producer are accurate. Our job should be to determine, given all that information, whether to give it to our children. It is an intensely personal decision that involves the parents and the child with Duchenne.” 

“I understand the need for caution and care, but I also know that our children are dying. Parents should be able to decide the risk/benefit of a drug that has gone through and passed preliminary testing. I would rather my son die trying and fighting than waiting and wondering and wishing….I am one parent willing to take an educated risk!”

“When you have a window of a certain number of years for something to happen that could save your sons' bodies before it is too late, you look at the need for treatment with a longing and a hunger like no other.  What we would risk for the option of having exposure to exon skipping looks very different than what families without this condition might consider.”

PPMD is in the planning stages of our second phase, where we hope to survey teens and adults with Duchenne and clinician caregivers, as well as expand to other countries.

Read the News Release

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