Article

In clinical trials, the choice of the primary outcome affects the study design,
the sample size calculations, the data analysis, and the interpretation of the
study results. Most importantly, it may determine the future of the intervention
being studied. In several clinical and rehabilitation settings, a single primary
outcome measure is often not sufficient to reflect the effect of an intervention
because attention is focused on multiple aspects of patients' recovery. In stroke
and traumatic brain injuries trials, for example, functional recovery is as
important as cognitive recovery. Thus, a trial with a functional scale alone as
the primary outcome would not be informative about the effectiveness of the
intervention on cognitive functions. From the methodologic point of view, the
choice of multiple primary outcomes presents several challenges, including
selecting a measure, among several, to be used for sample size calculations;
dealing with multiple comparisons; and interpreting the results. In this article,
we discuss a global test procedure that allows investigators to use several
binary measures as primary outcomes in a clinical trial. This procedure offers an
efficient solution under very reasonable assumptions, avoids loss of power caused
by multiple comparisons, has greater statistical power than any single outcome
measure, and is easily interpreted and of direct clinical interest.

Langue : ANGLAIS