Hi there. This is an interesting point you raise about pivotal trials being PII. But, I have a question for you. If they are indeed PII activity, why would they be talking about historical controls? Don't you need statistical controls only for efficacy studies? Do safety trials need statistical control group? Thanks...
A pivotal trial is the trial from which the data becomes the basis for the BLA. Most pivotal trials are phase lll's. However, sometimes a company feels the data is so compelling that they expand the phase ll and use that data for the BLA. Imclone did that with C-225. It has it's risks and the very act of a pivotal phase ll is telling the FDA "We don't think we need the normal amount of data required by a phase lll" OR "We think efficacy is satisfactorily established in our phase ll trial". It puts the FDA in a bit of a corner and the data has to be very compelling. "Pivotal" seems to mean the data in which the future of the drug depends on.
P.S. I think IMMU is cheap given it's fundamentals and prospects : )
Because in oncology you can not run a true placebo group, it is unethical not to give the patient any opportunity for cure.
Typically one would use an approved drug or standard of care therapy as the "placebo" group. If nothing else is truly available to compare against then one if left to a historical averages for observed outcomes. The problem with a historical strategy is that it is a lot like pornography, the agency will not and can not clearly define what they are looking for or at, but know it when they see it. Thus, NERX will not know until they submit a protocol or data if the historical argument is accepted.
Finally, one does not truly due a safety in oncology because the agency (and rightly so) will not allow healthy normals be dosed with a candidate like STR. Thus the equivalent of a safety trial in oncology is a dose toleration study that measures observed, adverse, and serious adverse side effects. This is the initial low dose work originally done by NERX.
mygame, i think str produced very good responce rates and give that all mm's are about to die, in the end, str,even with some negative side-effects will be approved. we are not talking millions of ill people but a small group with no alternatives but death.regards-lou