Abstract
In a separate paper we have pointed out that in addition to safety concerns which strongly suggest the use of concentration-controlled trials (CCTs) for drugs with narrow therapeutic windows, sample size considerations favor the choice of CCTs in many situations. In this paper, we consider ways in which CCTs can be utilized to streamline the drug development process. In particular, a randomized concentration-controlled titration design is proposed for Phase II of drug development. Such a design would facilitate an assessment of efficacy early in drug development, while providing information on concentration-response for a rational choice of dose or concentration in Phase III-Alternative schemes are considered for comparison. Data analysis and dose selection based on CCTs are also discussed.
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