Except he's pulling all the examples of why historical and open arm studies are risky of failure and interpretive until randomized trials occur.
Director and deputy director now talking about the FDA requirements. This is like a judicial reading...they are serious about how they feel about the trial designs - historically and bias. This is the best adcom I've ever watched!
Woodcock taking a hard stance.... This is a procedure and data mess up by Sarepta. Why are companies like this still around? Pay some consultants $100,000/year for 10 years to get this right the first time geez...
The opposite actually, a lot of consultants are watching this to understand exactly how rare disease drug trials should be run in the future and a proper nda should be submitted. This is going to set precedence for all companies on historically controlled trials in a very small population of patients. The FDA's risk benefit ratio could be potentially skewed towards AA even if data is limited so long as there is the possibility of a benefit.
Give it a rest Rebecca. That's pretty impressive that the kid is still walking, why are you focusing on the length of the walk?
We're all fathers and mothers with children and we want the best for them agreed! However, the FDA is a regulatory agency that adheres to acts, policies and guidance so that all companies submit meaningful data. I'm mad at Sarepta because of their incompetence. Why rush the NDA with such poor data? It doesn't do the parents and the children any justice if this drug is rejected. I'm investing not on the basis that I want children to suffer, but on the basis that Sarepta rushed their submission. The FDA is put into a corner and advocacy/social media is all the rage today. If everyone did their jobs competently this freakshow of an adcom wouldn't be trending. In the meantime, I've bought puts and will go long large if they vote positive. I want the positive vote because there's a lot of $$$ to be made.
You mean those 36 site investigators? Yes I get their data was published in several leading journals but that doesn't mean it meets the scrutiny of the FDA for a drug approval. This is going to be a policy and guidance rejection due to poor data methodology, poor data collection, not to mention poor data in general.
Personal safety? This is why the whole patient input model is quickly becoming a farce. It's turned into a mob that allows for lobbying which can quickly overturn scientific data. Leave the science to the scientists.
He's the Frances Kelsey of today, not on the question of safety but of efficacy. Both are equally important which is why the media is all over this. He's no doubt had a hard time with this and my respect goes to him. My mind thinks no it won't be approved but my heart hopes for what those Duchenne mom wants. I see this now as a 75 percent potential loss in the 6s but a potential 300 percent potential gain. I bought puts and will buy heavily if certain questions are yes. Those shorts will have to unwind...it won't be quick. Glta and try to have a good weekend.
I did the opposite. I have puts and freed up most of my cash to buy on Tuesday. My position on this is bearish but if the panel votes positive then I'll go long. I know better than to go against the FDA.
The burden is on Sarepta to prove their drug is effective. So far it doesn't look like it's done enough to satisfy the FDA per their trial design. What's interesting is that most Duchenne specialists are vouching for the drug which I think means there is anecdotal evidence that the drug may work. However, the FDA doesn't go off of anecdotal evidence. It goes off of good data from well controlled studies (placebo or historically controlled). This is a good drug executed incorrectly which leaves the FDA with no choice but to reject approval and wait for additional data.
Tricky but this section is what kills the drug approval: As described below, and in Dr. Ling’s
review, Study 201/202 was not designed in a way that allows reliable use of statistical
hypothesis testing (i.e., “p-values”), and is only capable of providing interpretable evidence of
efficacy if the beneficial effect of eteplirsen is so large that it is essentially self-evident, without
the use of statistics. It's the same argument that as used against Biomarin's drug.
Not hoping but don't let the statistical section stump you. No one is denying that a Duchenne drug is despartely needed. This is true for all rare diseases. What people need to understand is that data always matters and trumps emotion. If Sarepta gains approval on lack of substantial evidence, it sets a new precedence for other rare disease drug companies. Then you will see the rest of the big pharmas pile in with their questionable data.
The part where they mention "well-controlled and highly persuasive." To prove this you need to have sound statistical basis. Post hoc evaluations and extended studies are as the FDA says, very difficult and near impossible - or interpretive. So the panel members must decide why I'm voting yes and on what basis. If not statistical then ...you get my drift?
You can refer to this when the adcom is all said and done.
2) No, image enhancement and lack of independent confirmation
4) No, lack of statistical evidence and amended incorrect data introduced bias
6) No effect
7) No, historically controlled study lacked statistical evidence and effects are interpretive
Except panel members can't argue against "adequate and well-controlled studies." FDA just tied panel members hands. End of story.
The FDA rigged the questions to be negative. I agree, this doesn't look good. Sorry longs, this is the last time to get out. And yes, I'm buying puts now and taking a small short now.
"evidence from adequate and well-controlled studies or evidence from a single
highly persuasive adequate and well-controlled study that is accompanied by independent findings that