This is something that I have thought about because I am interested in
asthma for which there is no definition, let alone a diagnostic test. In a
way, for a disease like this where the aim is to relieve symptoms (unlike a
condition with a bad outcome like hypertension or cancer) the real clinical
question is: - how do identify patients who will benefit from the
treatment. Perhaps the gold standard could be based on the outcome of
treatment. For a noisy disease, it could be based on a n of 1 trial
consisting of a sequence of several drug and placebo periods with a tight
definition of what constitutes a response.
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