A few years ago, Prof. Gary G. Koch (University of North Carolina at
Chapel
Hill) gave a talk here in Tokyo on his researches into the statistical
handling of "multiple endpoints" (the term he used for what you
describe) in
clinical trials. One of his articles might be a good entry point
into the
literature on the matter. He gave his talk under the auspices of
the Drug
Information Association, an organization that is largely focused on
industrial
product-development activities (viz., pharmaceuticals and
biologics), but the
approaches he described can be applied more generally, e.g.,
evaluating
benefits of medical or surgical treatments, or life-style changes.
Tony
Lachenbruch already mentioned the positions that the U.S. Food and
Drug
Administration takes for so-called co-primary outcomes and multiple
primary outcomes in the context of granting or denying sales
authorizations
for medical products. Professor Koch's talk covered those, but also
broached
the topic of using the endpoints in a compound, or joint, manner (as
if
combining them into a single index of treatment response), and
addressed some
of the various approaches of doing that and their associated
problems. The
U.S. Food and Drug Administration allows these latter approaches in
selected
cases, for example, I believe that there is such a compounded index
for
rheumatoid arthritis that is widely and frequently used.
Joseph Coveney
Christopher W. Ryan wrote:
I would like to try to do a journal club session for my family
practice
residents on the uses, and especailly the pitfalls, of compound
outcome
variables, like "stroke or myocardial infarction or coronary bypass
surgery." They appear frequently in clinical trial literature. Can
anyone recommend any good articles discussing the issues? Thanks.
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