Phase II - Typically Therapeutic Exploratory
2. Selection of Doses for Proof of Concept (PoC) Studies
As a rule, PoC is tested at the maximum tolerated doses (MTD), or maximal administered dose if MTD was not defined. This is to minimise false negative results, and to provide best test of hypothesis, as well as to maximise pharmacodynamics (PD) effect(s). The evaluation of several dose levels to assess dose response is preferred, but is not required. The assessment of dose response allows researchers to establish a minimum effective and optimal dose, to mitigate the risk of the MTD not being tolerated. Gathering these further data strengthens the weight of evidence, and guides dose selection for subsequent studies, however it increases the time required to complete phase II studies, and thus the cost.