Table 1 Goals of Phase I–III Clinical Trials
CHARACTERISTIC | PHASE OF CLINICAL TRIAL | ||
|---|---|---|---|
PHASE I (DOSE FINDING) | PHASE II (ACTIVITY/EFFICACY) | PHASE III (PIVOTAL) | |
Primary goals | • Determine MTD • Define DLT • Characterize type and severity of adverse events | • Determine activity/efficacy in defined populations • Inform the decision to move to a Phase III trial | • Compare a new drug or combination to therapy currently regarded as standard of care |
Secondary goals | • PK/PD issues • Scheduling issues • Target modulation effects • Preliminary efficacy data • Investigate surrogate biomarkers of response | • Estimate therapeutic index • Expand adverse event data • Evaluate additional dosing groups • Expand target modulation and biomarker data • Quality of life measures | • Quality of life comparisons • Comparative costs |