Thank you for all your efforts in The Race to Yes! We wanted to give you an update and ask for your continued help to win this Race.
Earlier this month, natural history data was released that underscores how absurd it is to suggest that the stability of the boys with Duchenne receiving eteplirsen is not due to drug effect. The study states, “The sharper progression with each year … suggest that the relatively stable results on these measures over two or three years, as reported in some of these studies, may be related to the beneficial efficacy of the drug as this is not common in untreated boys” (emphasis added). We believe this natural history data could provide an effective control to measure the efficacy of potential treatments like eteplirsen.
Why is this important to each and every one of us? Because if the FDA avails itself of this study and its data, it could potentially eliminate the argument that more placebo-controlled trials are necessary.
This study, as released, does not include the individual patient data, which is what the FDA would need to see to effectively evaluate the study’s conclusions. The study indicates that this data is available upon request. To date, the FDA has not requested or evaluated the data, and as far as we know, has no plans to do so.
2 Ways You Can Help Now
Tell the FDA directly to get the data, review the data, and use the data! On Twitter @US_FDA, on Facebook, or by phone (888) 463-6332. “Get the data, review the data, and use the data so our kids can get on the drug, not a placebo!”
Ask your Member of Congress to tell the FDA to get the data, review the data, and use the data! Rep. John Culberson (TX) is leading a letter to the FDA, urging them to request and evaluate the data from this new study. A draft of the letter is attached. Ask your Member of Congress to sign this letter. The deadline is October 22.
Thank you for continuing to take action to help get safe, effective treatments to our children.
The Race to Yes Team