What is evaluated during Phase 2 clinical testing?

Phase 2 clinical testing is primarily focused on evaluating the effectiveness of a drug and determining the optimal dosing regimen. During this phase, the drug is administered to a larger group of participants—typically several hundred—who have the condition the drug is intended to treat. This allows researchers to assess how well the drug works on the disease or condition and to further refine dosing strategies, exploring different doses to find the most effective one with manageable side effects.

While safety considerations are still important during Phase 2, the main aim is to establish preliminary data on the drug's efficacy. Researchers gather data on various indicators of the drug's performance, including the therapeutic benefits it provides. Additionally, understanding which doses produce these effects without causing unacceptable adverse events is integral to this phase of testing.

In contrast, earlier phases primarily focus on safety and toxicology, while later phases involve more extensive safety evaluations and monitoring for serious adverse reactions when the drug is tested on thousands of participants. Thus, Phase 2 is critical in bridging the exploratory findings from Phase 1 to more comprehensive evaluations in Phase 3.