Therapeutic area is Myelodysplastic Syndromes (MDS). My 2 questions are these: For patients in a "watchful waiting" treatment mode (i.e. likely IPSS Low Risk or possibly Int-1 Risk groups), with or without supportive care, 1) what would be clinically meaningful endpoints to build into a clinical trial for a novel drug therapy and, 2) what would be the burden of proof in terms of performance against these endpoints (e.g. x% improvement vs. current treatments) that you would require to significantly adopt the new drug in appropriate settings. I know there is a regulatory angle to consider here, but setting that aside, from a clinical adoption perspective, what would be meaningful and relevant for you?
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Appropriate clinical end points would include such things as overall survival advantage, progression or event free survival, reduced toxicity, reduced transfusion requirements, reduction in infection rates, hospitalizations, or economic parameters. Any end-point which demonstrated improved quality or quantity of life with treatment vs. control.
Thanks. The problem with all of the endpoints you suggest, particularly OS and PFS is that it is impractical to develop a controlled trial to measure these, as the patients (fortunately) live quite long and will subsequently undergo many different treatments as their disease progresses. And as far as reduced transfusion requirements, which is a good one for these kinds of lower risk patients, by the time they are already doing transfusions, they are no longer in the pure "watchful waiting" period and are into "supportive care". But those kinds of metrics may be all that can be constructed. Thanks.
1976, MD Case Western Reserve U. 1979, Certified Internal Medicine, 1983, Certified Medical Oncology