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Sarepta Therapeutics, Inc. Message Board

  • speedbll speedbll Dec 17, 2013 12:57 PM Flag

    FDA Policy Forum Speech

    Excerpt (Google the rest)
    FDA Policy Forum Speech

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    Several months ago I was asked to be a presenter at a Duchenne Policy Forum hosted by Parent Project Muscular Dystrophy (PPMD). The intent of this meeting titled, “Optimizing Clinical Trials in Duchenne muscular dystrophy” was to give input to FDA officials as we draft guidance regarding what’s important to the patient community and what risks we are willing to assume in the development of a treatment for Duchenne. The policy forum took place last Thursday just outside of Washington. It was attended by 18 members of the FDA as well as scientists, drug development industry representatives and many parents of children with Duchenne. Because of a recent development in the Duchenne research world – one in which the FDA change its stance on granting accelerated approval to a therapy that has shown efficacy and NO adverse side effects — I took a bit of a different approach with my speech, focusing not simply on the risks associated with administration of a potential treatment to the children, but on the risks in the process of drug approval and the risks in delaying the delivery of a potential treatment to the children who so desperately need a lifeline. Although the potential therapy that I refer to above would not help Joseph as it’s specific to the mutation a child has, it would help about 13% of children with Duchenne. The way in which we get this approved will set precedent for other treatments to come – one of which I believe with all my heart will help Joseph. In order to get the message across, I needed to really paint the picture of what Duchenne does to a child and a family and a community, and I needed to wear my heart on my sleeve. I’m incredibly grateful for the outpouring of support and affirmation after I delivered my speech to the FDA. For those of you who requested a copy, here it is. For those of you who weren’t there, I hope this brings you up to speed and helps you understand our mission.

    Marissa Penrod — FDA Policy Forum speech – December 12, 2013

    When Joseph was just 5 years old, I took him to his pediatrician thinking he might have a mild physical delay and may need physical therapy. Within 24 hours, a blood test and a visit with a neurologist provided us with the most devastating diagnosis imaginable. Duchenne muscular dystrophy. The neurologist explained to us that Joseph’s muscles would rapidly deteriorate, he would lose the ability to walk, to use his arms, to bathe himself, to go to the bathroom on his own. Eventually Duchenne would attack his heart and lungs and the disease would take his life. We have nothing to stop it he told us. It’s 100 % fatal.

    Sentiment: Strong Buy

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    • Rats - thanks for posting the entire speech. Wow.

      reminds me of a quote on my office wall:
      "It is often necessary to make a decision based on information which is sufficient for action, but insufficient to satisfy the intellect" - Emmanual Kant -

      Sentiment: Buy

    • More of the speach:
      Joseph is now 11, and we live each day with the knowledge that because he’s missing exons 8 and 9 of his dystrophin gene, his symptoms continue to progress and he continues on a steady and rapid physical decline.
      Joseph’s stage of progression is one marked by an accelerated decline… It is marked by . . .
      Shock – the shock of his legs buckling without warning, sending him tumbling to the ground. It is marked by the shock of suddenly being unable to life a gallon of milk to put it back in the refrigerator.
      Fatigue – he is plagued by the fatigue of exerting great effort just to do “normal” things – like keeping his balance and getting up from the floor.
      Strategizing – thinking about how we pack his backpack so it’s not too heavy and its weight doesn’t tip him over. We decide what he’ll take to school with him on the bus, and what I will drop off in the office so his load is lighter.
      Disappointment – the searing realization that he can’t play organized sports, my lack of words or adequate comfort when he walks into the house fighting back tears because his friends just rode off on their bikes and he can’t be with them.
      Routine – twice daily stretching sessions, and regularly physical therapy appointments and doctor appointments.
      Hanging on – to what may be the last time he’ll crawl up the steps of the school bus so he can ride with his buddies. Hanging on to the sound of him singing each morning and memorizing the sweet sound of him because I know there will come a day when our house will be deadly silent.
      Adapting — I often watch him play a special version of baseball in the yard with his buddies. It’s a version in which they let him walk to first while they wait patiently. It’s a version in which they help him up and brush him off and run to get him bandaids after his weakening legs have suddenly given out on him and he’s fallen to the ground without warning. Ironically, it’s a version that I’ve learned to be grateful for, because I know that soon this crew of baseball players will transition to pushing him from base to base in a wheelchair.

      Sentiment: Strong Buy

      • 1 Reply to ratshill
      • and More:
        This is the time when kids are moving faster and pushing limits and growing and looking forward to the future. And I’m terrified that Joseph may not have one. Annual events like birthdays and the end of a school year are marked by conflicting emotions – they’re marked by relief that we were given the gift of another year. And by grieving one less year we have together.
        While these symptoms are heartbreaking, I am also filled with the sobering reality that this is MILD compared to what we will face in the very near future. There is a train racing toward my little boy and I’m running as fast as I can to scoop him up and save him, but I’m acutely aware that I may not make it in time. We need you to help stop the train.

        Children at any age – whether they’re 3 or 7 or 11, like my Joseph, should be afraid of the dark. Of the bully on the playground. Of the monsters under their bed. But they should NOT be afraid of needles and biopsies and surgeries and falling and breaking bones. They should NOT be afraid of no longer walking or of being unable to feed themselves or of losing so much strength in their arms that they can no longer hug their moms. Most importantly, they should NOT be afraid of dying.
        We were asked to speak today about what risks would be unacceptable in a clinical trial. The greatest risk in a clinical trial would be a delay in moving into a next phase or into the clinic. I would consider it to be a great risk if there was a potential treatment in clinical trial that showed efficacy and safety and yet more studies were demanded before allowing access to patients. I consider it to be a great risk to require children to be on a placebo arm when they will lose muscle function and strength and for some, the ability to walk, during the course of a trial. I consider it to be a great risk if the design of a clinical trial is approved and the expectations from the FDA change or are unclear after the trial is underway. The VERY greatest risk in a clinical trial, in my opinion, is for the FDA to not use its right and its responsibility to operate under the provisions of FDASAI to use the accelerated approval pathway for a treatment in a disease with unmet need.

        Sentiment: Strong Buy

    • I would love to read the rest of this. Wow. My heart goes out to you. I hope the FDA listened and realized there is NO Risk in approval of this drug but the worst case scenario if they don't. God Bless

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