The Jett Foundation Fighting Duchenne Muscular Dystrophy

Jett Foundation on April 25th Advisory Committee Meeting

April 27, 2016

I want to thank the Duchenne Community for your effort, your time, and your strength. I want to thank all patients with Duchenne undergoing clinical trials and awaiting a potential treatment for your courage. I want to thank our physicians and researchers for standing up for their patients and for standing by the scientific evidence.

On Monday, we made Duchenne history by changing forever the way patient input is considered by the FDA and the drug development world. Rare disease patients and advocates around the nation will benefit from your groundbreaking actions.

I also want to thank CDER Director Dr. Janet Woodcock and CDER Deputy Director Dr. Robert Temple for emphasizing the importance of the patient perspective, and for underscoring the definition of accelerated approval, which is “reasonably likely to predict a clinical benefit.”

Here is what we know: The FDA was tasked by Congress in FDASIA Section 1137 to take patients’ perspectives into consideration when reviewing a potential treatment for an unmet medical need. This community provided that perspective – vividly and expertly – throughout Monday’s AdComm meeting.

It is important to remember that under FDASIA, the FDA is required to consider our views when it makes a decision on eteplirsen. The independent adcomm panel, however, is not.

Here is what we also know: our patient and expert community brought their A-game. The Duchenne Community came to the table totally prepared, with valid and concrete evidence based in science and fact.  These were not pleas of desperation nor calls for sympathy. The patient perspective on eteplirsen’s meaningfulness was heard by the FDA during the Open Public Hearing and in the first-of-its-kind Patient Reported Outcomes presentation.

The panel’s recommendation was just that-a recommendation based on very narrowly and specifically crafted questions. Those voting questions purposely excluded the patient perspective, and focused only on the FDA’s interpretation of the company’s data.

The panel answered the questions with information from FDA’s largely subjective presentation and analysis, that included no evidence based rational and was filled with the words “may be” “could” appears to be” and “might.”

Monday’s discussion was not scientifically led-it was a clear abuse of power and authority by the Chairman and the Division, facilitated by the silencing of a sponsor that could have answered every single one of the FDA’s misrepresentations, if given the opportunity to do so.

And yet, the vote was close, very close, specifically on question #2 – the accelerated approval question. If dystrophin quantification is validated through the accelerated approval of eteplirsen, the development of treatments for other exons could move much more quickly.

I know we are disappointed. I know we feel silenced or let down, but the patients weren’t silenced.

We were heard. We were loud, we were clear, and most importantly, we were prepared and educated in the science and in the regulatory process even when the panel and the Division were not. The panelists and the agency ended the day with far more information about Duchenne, natural history and eteplirsen than they started with.

Now, in the face of a divided advisory committee panel, I hope – and I want to believe – that yesterday’s unprecedented and intelligent patient participation will encourage the FDA to consider all of the information that it has, including the patient perspective, and make the most ethical and scientific decision on eteplirsen by the May 26th PDUFA date.

Our message to the FDA has always been the same: as long as the world renowned Duchenne researchers and clinicians support eteplirsen’s data, the Duchenne Community will push for approval.










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