Quote, "I understand your rationalization here but it is incorrect. Test data does not have a shelf life like that. Results are results just as in Einstein papers. Now if the results submitted need more data for qualifying and it is unavailable then that is different. But in most cases the data is complete FDA is looking for clarification if anything. This is common in cutting edge science as many things are not readily known by all. "
Well, HERE'S THE LIL PROBLEM WITH ALL THAT...IF we wanna talk "Einstein" and the real "scientific method" etc.
The MARVEL TRIAL WAS NEVER COMPLETED. And THAT is the key to the puzzle.
A clinical trial, is a statistical based "model" based on pure mathematics and the "scientific method". Thus, the trial "DESIGN" is determined AHEAD OF TIME, THEN, THEN the "trial" is executed to completion, it MUST REACH THE "PRE DESIGNED END POINTS" for the original "statistical model" to hold any validity, THEN the data collected is "unblinded" and "opened up" for full analysis to test/prove or disprove what was originally postulated, THEN and only THEN can valid "conclusions" which "hold water" under the scientific method, rooted in the "hard science" of probability and statistical modeling be used to draw any valid conclusions.
THUS, the grand problem facing USRM "trying" to present MARVEL to the FDA, is the trial NEVER REACHED FULL enrollment, thus it's 100% IMPOSSIBLE that it ever even tested its original design "postulates" and thus NEVER COULD HAVE HIT ITS END POINTS, thus the data can never be back-tested through the original designed statistical model to see if what they postulated, aka were TRYING TO TEST/PROVE IN THE FIRST PLACE is valid, good, bad, statistically relevant, etc.
An incomplete clinical trial (especially when "double blinded), a halted "trial" is really pretty much NO TRIAL AT ALL, cause every trial hinges on MEETING THE ORIGINAL DESIGN PARAMETERS, it's pre-designed, else, it ain't a valid mathematical, probability based, valid "clinical trial". It becomes anecdotal at best, which is the weakest kind of science based "data" that exists.
The original MARVEL trial "design" needed to hit in the 130 to 150 patient range (really 330 I think based on the original design as submitted to the FDA). The trial STOPPED AT 20 PATIENTS. NEVER CAME CLOSE TO HITTING ITS ENDPOINTS. HALTED FOR LACK OF FUNDING.
More importantly, early in the trial, they had SERIOUS ADVERSE EVENTS, aka at only the NINTH patient, they had to "unblind" the trial and send it to the safety review committee as they had patients w/ serious V-tach occurring, irregular heartbeats of a type and severity that can kill the patient. Thus, they didn't even really get rolling, before they had serious adverse events, then halted at only 20 patients for supposed LACK OF FUNDING, and in SEVEN DAMN YEARS, were never able to find that "funding" to ever restart that trial again, let alone ever drive it to completion, aka end points, then fully unblind the data, properly analyze it in total, etc
THAT IS THE "STORY" OF MARVEL. I have another post, I'll copy and paste it in a little while when I get time. The ENTIRE MARVEL "20 patient" story as written in the "Heart journal" where it was published as an INCOMPLETE, NEVER COMPLETED TRIAL. And the author's who ran that partially completed trial and tried to "analyze/salvage" any data they could from it, they are CRYSTAL CLEAR, that due to the fact the trial was HALTED/STOPPED FOR LACK OF FUNDING, they really can't draw much hard conclusions, other than "maybe" some encouraging data, BUT MORE TRIALS NEEDED TO PROVE OR DISPROVE what we "think" might have occurred in the PARTIALLY COMPLETED/HALTED MARVEL TRIAL, with a micro sample of only 20 patients ever "treated", an early "unblinding" at patient #9 for safety review issues, then a halt FAR SHORT OF THE ORIGINAL TRIAL DESIGN, where they thus state, "WE NEVER HIT THE END POINTS THEN, THUS WE CAN NOT MAKE CONCLUSIONS" per the trial design protocol (paraphrasing).
END OF STORY.
