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Phase 2 Efficacy Trials

Meaning

Phase 2 Efficacy Trials constitute the second stage of clinical investigation, following the successful establishment of a safe dose range in Phase 1 studies. The central objective of this phase is to gather preliminary data on the drug’s effectiveness, or efficacy, for treating the specific disease or condition it is intended for. These trials involve a larger group of patients, typically 100 to 300 individuals who actually have the target condition, and continue to rigorously monitor safety and side effects. This stage is considered the “proof-of-concept” phase, determining if the intervention shows sufficient therapeutic promise to warrant large-scale definitive testing.