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clock Created with Sketch. 26/04/16
14:14
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Originally posted by Southoz
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Yep cgt2009 this is the paradox of the thing. The trials take too long. So in response you increase the number of sites that you recruit from as NEU did. But then this increases patient heterogeneity which is now recognised as the killer in TBI trials. It also stuffs up the power of the study (which was calculated here nearly an eon ago). The reliability of outcome measures here (including on the primary) are sensitive to site variation in discharge practices as well as site specific rater differences on the cognitive measures. So straight away the power was reducing on you. This combined with a lower base for mortality (reported from the unblinded data) again reduces power. The reality was that there were many factors conspiring against a positive result here. One left field aspect here is that by partnering with DoD Neu had very little wriggle room in terms of sugar coating the results. Which typically occurs by ASX biotechs reporting out of P2. Where to now? Well it will be back to the drawing board for mild TBI. Most likely that study will be abandoned. It may or may not be reinvented. I think the market will be more receptive to the biomarker analyses than people might be expecting. To purists it will look like a post-hoc data mining dredging activity. But I think there is significant difference here in that the FDA is recognising that this kind of approach has benefit to guide the development of drugs where the traditional approach has shown not to be working and wasting enormous amounts of money (eg 30 straight failed P3 TBI trials). There is a legitimate argument that you should control for that patient heterogeneity through biomarkers even though that was not planned at the start of trial. Heresee to some … but because of the time to complete these trials is many years in that period knowledge improves. That knowledge should be used but quite how to go about this in a convincing manner I don’t think anyone knows. On the other hand sentiment will flow over to Retts and Fragile X. At some stage Neu has to move through the exploratory phase and into the confirmatory whether or not the complexities in outcome measurement that NEU seem fascinated by are fully solved. The benefit of the doubt here that I think people have given Neu here will take a bit of a wack. So as usual with Neu … it really is a matter of being as glass half full or empty as you like.
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Ahh a disappointing result but at least it comforts me that they did not spin it and put it as is on the plate. I am actually glad that there is a good support at 7 centish. More efforts will be placed on RETT and FXS which will hopefully improve the study and the outcomes.
(20min delay)