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During Phase 2 clinical trials, the primary focus is on assessing dosage optimization and efficacy. This phase aims to determine the optimal dosage that produces the desired therapeutic effect while minimizing side effects. Researchers examine how well the treatment works in a larger group of participants who have the condition the treatment targets, allowing for a better understanding of its effectiveness. This information is critical for determining whether to proceed to the next phase of testing, where larger populations are evaluated and additional factors such as safety are monitored more closely.

Long-term effects of the treatment are typically studied in later phases, while post-marketing safety focuses on surveillance after the product is available on the market. Randomized controlled effectiveness studies may also occur later in the research process to establish how well the treatment performs in real-world conditions compared to alternatives. Thus, the assessment of dosage optimization and efficacy is the key focus during Phase 2 clinical trials.