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Tuesday, April 13, 2021 11:28:26 AM
He continues to perform his number one job and that is to mitigate risk in our trials. He has got us this far and for me nothing has changed. He has funded our trials well and continues to attract 3rd party grants. He continues to execute on trial planning and safety with a DSMB review of our current Ph 2b/3 trials including all OLE’s. Third, he continues to attract bright Dr’s to fill upcoming much needed positions ahead of approval.
When VERY large company’s, with vast resources, continue to fail at what we are targeting, I think that has a major value that people are discounting as well. If you put yourself in Dr. M’s position where he has met with patients, Rare disease organisations, and caregivers and then has witnessed the positive data compounding from each trial, I cant think of any reason why he would unnecessarily delay access to those in dire need of our drug. I actually see just the opposite. He is advocating, accelerating, accommodating, synergising, coordinating all the steps needed to push this drug through as fast as possible against an immovable BP money machine.
Patients in each OLE are receiving the drug..... permanently. This is very positive and imo is what I would consider a “soft start” and is preliminary groundwork for access to the world. It must be very powerful to see the patients and their caregivers faces as a trial organiser. Nothing else matters in this world but disease relief for loved ones.
If you really want to blame someone, try calling your elected leaders and direct your frustrations to those who have been dragging their feet for decades funding trials to know where! Blindly funding broken systems with proven failed targets because everyone is too cowardly to stand up and say “wait, why are we still doing this this way?” True definition of insanity, doing the exact same thing and expecting a different outcome.
I for one continue to applaud Dr M and management and keep their well being in my prayers.
Jmho
Tred
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