The response rate did not drop a little, the expected was 15% of subjects with each subject having a 50% or more tumor mass reduction. They submitted an alleged 22.5%, It drop till it was worthless (at least below 15%) and therefore refused to accept application for review. This is complete failure.
Anecdotal evidence do not get a drug approved, many failures and stock debacles are based on anecdotal evidence, the "pressure the FDA" business, all the "needed patients" stories are just soap opera and fairy tales, and will not made one buck of investment (I mean for average long term investors, the ones that trade frequent could learn the ups and downs).
Indeed, it is highly impressive the number of "big shots" willing to talk the drug up about their particular experiences, it does not prove the drug, neither gets it approve.
Anyway, the same Dr Saltz, unquestionably a world expert on the subject, Sloan-Kettering and MD Anderson centers are highly respectable and top of the world trial, those same ones did the trial that got the RTF. The only conclusions is that "IF THE DRUG WORKS" those big names did not performed well, and/or the company was utterly inefficient, non is a good indicator.
Circular reasoning: the drug is good, the anecdotes are good, from good prestigious clinicians, the same that did the trial, the same trial that get RTF, and the trial is accepted as faulty...Conclusion: good drug, bad FDA !!!
It is amazing that the same great anecdote prone ones ARE the same faulty trial designers/runners. If they are so good, my conclusion is the drug is worthless (my oppinion, of course.
"Now you're are claiming out and out fraud"
Not in the execution of the trial, I believe that the drug did not work, and the trial changes, adjustments, and alterationes were done honestly trying to prove it does, that is a very well known phenomenon "scientific and clinical bias", it is non conscious and NO MALICE, I was very clear.
The bias is for drugs to do good, the chemist expects it, the pharmacist expects it, the clinicians expects it, the public, the investors, the company...everyone is for the drug for the good it could produce, like fans of the skaters the juries will vote for thei own, very simple, and even very complex phenomena like clinical trials are full of bias and that is why the "ramdomized, double blind, placebo control, multicenter studies" are the highest level of evidence to Science. History is full of failed therapies of all kind that are so great for decades and when they are subjected to a well designed phase III study they do not.
Just find the recent "Pediatrics" journal, look for the "Committe position on Steroids and Bpd", they are asking for pediatricians to abstained to give any steroids to babies with Bpd unless death its imminent, better reserve the treatment for subjects in phase III trials. All this cause the supposed decreased in mortality is not proven after decades of use AND research, and that the now well known brain deficits from the treatment are not worht the known benefits: less oxygen, faster withdrawal from ventilator breathing machines, impressive improvement in x-rays, and babies comfort improvement. Now, after 30 years of use off label, dexamethasone is back to square one. In the meantime thousands of babies had additional brain deficits. It was bias at the highest level of honesty and compassion, bias nonetheless !!! |
