It's great that it appears that DCVax is very safe. Safety is really important obviously, but if the experimental treatment does not work, what's the point? I think in the minds of most clinicians, and the FDA, efficacy and being better than standard if care is most important.
However, there are some cases where the toxicity may not outweigh any perceived marginal benefit. For example, there are some treatments that may have better efficacy and have more toxicity that are used. The chemotherapy regimens BEACOPP vs ABVD in Hodgkin's lymphoma are both very effective. BEACOPP might be a bit better, but it is more toxic. The US feels that it is not worth the toxicity, but in Europe they do and use that regimen.
There's a chance that it could be shown to be safe and as effective as standard of care, but then I doubt it would be put into use. In addition, there's a chance that it could trend towards being better, but not be significantly better. In this case, potentially an argument could be made, if there are potentially good reasons why it did not reach significance. We need to keep in mind that our determination of alpha < 0.05 for statistical significance is arbitrary. This means that there is a 5% risk of concluding that a difference exists when there is no actual difference. In other words 95% of the time what you perceive is not happening by chance alone. An alpha of 0.1 would mean that 90% of the time it's not by chance alone. That sounds pretty good still right? I think so, but many doctors get hung up on a P value <0.05.
If we were to have a close but no cigar statistical significance, but the percent difference appeared to be really trending to be better, the FDA could want to see another Phase III. But there's no way to really know
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