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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