NEU neuren pharmaceuticals limited

Ann: Neuren completes enrolment in Rett syndrome Phase 2 trial, page-16

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    Yep ... this will be the issue for P3 when you will need much larger Ns for a confirmatory trial, where efficacy will be the primary. So basically the P3 trial will go longer, more US sites / other countries.

    I take your point that a win is a win. But not any win will do.

    First without power you have less chance of seeing that it is a win to begin with.

    Second there is a threshold that has been set by the FDA which the P3 trial will have to meet.

    Third not all change is better. Natural variability and measurement error will cause poorer scores for some subjects in absence of true effects. So its not as easy as falling off a log. You have to be going forward just to stay still. Thats the catch.

    But anyway when (assuming it does) get to P3 and the details become available someone can try and back track from the sample size assuming different levels of power to try and guess at what that threshold is.
    Last edited by Southoz: 16/11/16
 
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