On 15 Jan 2003 06:04:49 -0800, [EMAIL PROTECTED] (Lizzy) wrote:

> With a very large sample size (n=15000), what is the validity of
> secondary outcomes if sample size calculations were based on the
> primary outcome only(death)and the primary outcome itself does not
> show differences between treatment arms?

I can think of "secondary outcomes"  as denoting (say) symptoms 
of the disease.  In that case, the failure of the "primary" question
probably undermines every secondary conclusion.  
Outcomes would have to be "exploratory" and "speculative"
even if the p-levels were impressively small.

I have also thought of  "secondary outcomes"  as more 
independent than that.  The hypotheses were secondary,
perhaps, in terms of justifying the funding for the study,
but where rather extrinsic to the other ideas.  
 - That is relevant, I think, in justifying how one splits the
overall error.

We do want to protect a 5% error level on an 
experiment, for instance.   On the other hand, we do 
want to save a passel of money, by tossing in an 
extra data-collection form or two, and testing an
extra hypothesis or two -- instead of re-running the 
experiment, at a cost of millions, in order to preserve
this theoretical virtue.

You have to figure this out.


> 
> One to four out of 13 secondary outcomes/ outcome subgroups showed
> significant difference between treatment arms. Can one state that
> these data are valid, "hard" data, taking into account the very large
> sample size? No adjustments for cut off point for p-values where done.

If these were 13  parallel, equal hypotheses, then you do *need*
to control for multiple tests, or else, label it as  "exploratory",
that whole portion of the followup.

Huge sample size has a drawback, in that it can elevate tiny
biases into being 'significant'  effects.   That is especially
important if this huge N   was observational, and not a 
randomized trial; for convenience samples, 'significant'  
might indicate  that, yes, the outcome was *interesting*;  now
it is time to  explain away all the other possible causes.
(That is very difficult when the literal effect is small.)


> This regards the clinical interpretation of data from one large drug
> trial.

 - Large trials usually have large planning, and that
should have included statements about the philosophy 
of dealing with multiple outcomes.  Epistemology of the
epidemiology...
-- 
Rich Ulrich, [EMAIL PROTECTED]
http://www.pitt.edu/~wpilib/index.html
.
.
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