TROUBLEposting again, sorry for any formattingissue...
@dolcevita, You certainly raise somevalid points. Unfortunately, I can't see the video perhaps dueto geography?
Again, not to get too bogged down in politics,but to take an opposing view (and I can argue both sides) I wouldsay, yes, there are "advisors and bureaucrats who translate (thePresident's) thought bubbles into policy..." BUT,there are other advisers and bureaucrats who plant thoughts into the bubble,especially on medical matters. The President often speaks of "acure" for various conditions... childhood leukemia, HIV, etc...based on briefings he's heard from his team of experts (who are notalways on his side politically, Dr. Fauci is perhaps a goodexample). So, when the President comes up with something likeHCQ and runs with it (an Americanism from American football), it'snot an idea he pulled out of thin air on his own...Someone told him it held promise... perhaps a bit prematurely?? Finally, on the bleach issue, I would be careful interpreting the President's words based on what the media says... he's often portrayed in the most negative light possible. If youlisten to what he actually said it's much more benign and the wordLIKE as in "like a disinfectant" has to be heard.
Butyes, he sometimes misspeaks.
Leavingthat for a moment, I want to make a few points on a phrase we oftenhear on message boards, namely Fast Track.
Notso much on this board, but sometimes I have the impression postersmay not understand what the term means.
Amedicine may be eligible for Fast Track if"
‘...‘it isintended ... for the treatment of a serious or life-threateningdisease or condition, AND it demonstrates the potential toaddress unmet medical needs for such a disease or condition.’’
So,a company APPLIES to the FDA for this designation and theFDA decision is based not on the medicine but on needs respecting theillness to be treated. That might be GvHD, where remestemcel-Lwas GRANTED Fast Track approval, or it might be C-19 ARDS, where wehave not yet heard anything. A company may apply for thedesignation at the time of or subsequent to the submission of anIND. A new IND means a new opportunity to apply for FastTrack. It means we might have a press release at some pointrelated specifically to C-19 ARDS. Or not.
Theadvantages of Fast Track are opportunities for closerconsultation with the FDA during the clinical trial process,opportunity to submit the BLA using a rolling review process, andincreased consideration for Priority Review once the BLA hasbeen submitted. We saw all of those elements with the aGvHDBLA. Whether Mesoblast will submit a request forFast Track with C-19 ARDS remains to be seen. It seemsessentially all phase 3 C-19 trials are already receiving some FDApriority. Since the Company already has BLA pending, the rollingreview aspect has less benefit. C-19 is more like a labelextension. So, let's see what happens.
I'llpoint out that Fast Track falls under the category of "ExpeditedApproval" mechanisms available through the FDA. There areothers such as "Orphan Drug" or "Accelerated Approval"or "Breakthrough Therapy". The first two are not somuch of interest here, but the Breakthrough Therapy category could beinteresting and might have some advantages over Fast Track. Amedication may be eligible for Breakthrough Therapy if it:
‘‘...isintended... to treat a serious or life-threatening disease orcondition, and preliminary clinical evidence indicates that the drugmay demonstrate substantial improvement over existing therapies on 1or more clinically significant endpoints.’’
So, a littledifferent than Fast Track, with the addition of the "clinicallysignificant endpoints" language, ie (Overall Mortality, forexample compared to existing remdesivir). TheBreakthrough Therapy designation has some advantages including theenlistment of more senior FDA officials in the review processat an earlier stage. Let's see what happens, but a PR on any ofthese designations could come at some point. I believe the FDAhas 60 days to make a decision after the application is made.
I'madding such an announcement to my list of possible press releasesprior to the interim checkpoint review.
A key point is NOT toconfuse Fast Track or Breakthrough Therapy with some kind ofpre-approval of a medication's efficacy. Many postersseem to do that. In fact, the FDA can withdraw the designation ifpriorities change, and a medicine still has to pass throughthe clinical trial process and a full FDA review at the end. Itis no guarantee of eventual success. It's simply the FDAacknowledging the nation has a need for therapeutics in a certainarea, and the medication passes a very preliminary efficacy and moreimportantly safety check up.
Certainly Covid-19 falls inthe category of "unmet need" and I don't thinknational origin enters into such FDA decisions. In fact, theopposite may be true - see my post on "The Swamp" above forfull details.
Bythe way, the UMd doctor was not talking about the phase 3 trial whenhe mentioned the 4 patients, imo. Some universities arestudying MSC's and conducting clinical research. U Minn andUCSF (Dr Matthay) are other examples. They are notcompetitors. The official press release from UMd says it all interms of the university's participation in the phase 3trial.
Meanwhile, Gilead submitted an applicationtoday to the EMA for remdesivir review and approval in Europeunder a "reduced timeline". glta
(20min delay)