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Abstract:
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Novel therapies are challenging current drug development standards. Agents with specific biologic targets, unknown dose efficacy curves, limited toxicity profiles, and in combination therapy require novel designs. We (and many others) have previously presented designs for identifying optimal doses of single drugs or two drug combinations in Phase I trials utilizing a variety of endpoints. A common theme is the use of model-based methodology to adaptively determine the dose for the next patient or cohort, based on the data observed to date in the trial. The implementation of these new designs in practice is challenging for a variety of reasons, some of which I will discuss in this talk. Specifically, I will describe the approach we have used at Mayo Clinic for implementing these novel designs highlighting the successes, challenges, and lessons learned along the way.
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