Far too much has been made of Myotrophin being 1 for 2 in Phase III, 'failing to show efficacy' in Europe. That unfortunately
reflects an unrealistically polarized categorical measure...as if significance is all or none. It is not....statistical signifance is a continuum. In the US study, the slowing of functional deterioration
had a greater than 99% chance of being due to the drug, while the 'failed' European study showed slowing that was 93% likely to be due to the drug. The FDA slavishly adheres to what I call the
'tyranny of .05', the notion that 95% likelihood of a true positive effect is necessary for significance, as if that is 'significantly' different from..say 94%..or 93%. That is absurd. Perhaps
for 'me-too' drugs looking to join an armamentarium, like the third or fourth SSRI for depression,one might justify a high hurdle for addition to already existing tx options. But for a disease where there is little or nothing available, and Rilutek qualifies as little tx for ALS, I am not the first to suggest that a 90% likelihood that a drug offers true benefit should be sufficient.If that standard were applied to Myotrophin, it would have two successful trials, and all of this would have been unnecessary.
Cephalon has mishandled the Myotrophin approval process egregiously, it is a textbook case of how not to deal with the FDA. It is perhaps overly kind to describe them as trying to work 'outside
of the box'of the rigid FDA frame; the box exists, but Cephalon's behavior came across more as arrogant than traiblazing. Be that as it may, there is no regulatory or scientific glory to the FDA's rigidity in this matter, it is misguided zeal at best, personality conflicts overriding patient care concerns at worst. But there is
no current court of appeal in this context, and the FDA has the will, and the way, to punish companies who try to expand the FDA's myopic vision of the drug approval process.
NeuroInvestment (www.neuroinv.com)
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