Biomedical Engineering Reference
In-Depth Information
your study obviously pointless from the start? Even if
you knew, what could you do about it without harming
the validity of the whole effort?
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The problem is to conduct clinical trials so that both the
rigor of the experimental design (RCT) will be maintained
throughout, and program revision can occur, based on the
timeliest data. And, methodologically speaking, that is
precisely the problem cited by Lowe, with reference to the
evaluation of training programs.
As Scott Gottlieb, FDA Deputy Commissioner for Medical
and Scientifi c Affairs, has expressed it, FDA is interested in
“adaptive sampling designs, including response-adaptive
designs for statistical experiments, where the accruing data
from experiments - the observations - are used to adjust the
experiment as it is being run.”
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He goes on to say:
. . . the advantages of these approaches, rigorously
designed, are becoming more evident, including among
the ranks of our experts at FDA. It is essential that we
at the FDA do all we can to facilitate their appropriate
use in modern drug development.
Gottlieb discusses several adaptive approaches to the design
of experiments, including the following:
■
In an adaptive clinical trial, patient outcomes can be used
as they become available to adjust the allocation of future
patients or some other aspect of the study design.
■
A second type of adaptive trial design involves on-going
assessment of the sample size, to avoid under- or over-
allotment of patients.
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■
[Another includes] seamless designs that allow learning to
be more iterative and less method-limited. That allow
