Which statement best contrasts typical Phase I and Phase II study designs?

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The main idea is how early-phase studies differ in purpose, population, and design. Phase I trials focus on safety, tolerability, pharmacokinetics, and finding a safe dose range, usually in healthy volunteers, and they commonly use dose-escalation to identify how much of the drug can be given safely. Phase II trials move toward efficacy, testing whether the drug shows a therapeutic effect in people who have the disease, and they typically use randomized controlled designs with patients to compare outcomes and better assess efficacy and safety at a chosen dose.

This makes the statement that Phase I uses dose escalation in healthy volunteers and Phase II uses randomized controlled designs with patients the best fit. Other descriptions—such as Phase I being placebo-controlled or double-blind, Phase II being limited to dose-escalation, Phase I being observational, or Phase II being open-label—do not align with the usual aims and methods of the early phases.