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Name
Stephen Archer

Wed, 11/26/2014 - 22:27

Thanks Ken. I didn't hear Rob (not enough mitochondria in his talk?) but agree: large RCTs are invaluable. Sadly the opportunity to capitalize on the money spent is often not optimal because only the minimal data set for FDA approval is obtained and the industry sponsors forgoe the potential to acquire a rich data set. Within most RCTs one could prospectively, simultaneously create a patient cohort with a specific phenotypes that would support hypothesis testing. For example the licensing trials for pulmonary hypertension uniformly fail to assess right ventricular function. Were this done, a deeper phenotype, relevant to the natural history of PAH, would be available. A drug that has modest overall benefit on a simple metric, like 6-minute walk duration might have great benefit (or exert harm) on patients with certain RV morphologic or functional characteristic. So I believe Jay and Rob are both right. Large trials should incorporate smaller, mechanistic cohorts with a uniform, clinically relevant deep phenotype that would test the possibility of personalized medical interventions.

Name
Stephen Archer
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