Phase 1 trials primarily evaluate...

• A. Tests efficacy in a large population
• B. Phase 1 is primarily about post-marketing surveillance
• C. Phase 1 tests on animals only
• D. Tests a new drug or treatment for the first time on a small group (20-80) to evaluate safety and determine dosage

Answer: (D)

Phase 1 trials focus on safety and dosing in humans. They are the first time a new drug is given to people, and the main goal is to determine how the drug behaves in the body and what adverse effects occur. This helps establish a safe starting dose and how to adjust doses, often through escalating-dose steps. The typical number of participants is small, about 20 to 80, which allows researchers to gather pharmacokinetic and pharmacodynamic data, identify early safety signals, and decide whether it’s reasonable to proceed to larger studies.

Efficacy in a large population is usually the aim of later phases, while post-marketing surveillance occurs after a drug is approved. Animal testing and other preclinical work happen before any human trials.