Regulatory Affairs Certification (RAC) Practice Exam

What is the main goal of Phase 3 clinical trials?

1. Determine initial safety

2. Confirm effectiveness

3. Investigate pharmacokinetics

4. Monitor long-term effects

The correct answer is: Confirm effectiveness

The primary goal of Phase 3 clinical trials is to confirm the effectiveness of a treatment or intervention in a larger population. At this stage, the therapy has typically shown promise in earlier phases regarding its safety and potential effectiveness, but Phase 3 trials are designed to provide more critical evidence to support the drug's benefit-risk profile. Phase 3 trials usually involve a significant number of participants across various demographics, and they often compare the new treatment to a placebo or an existing standard of care. The results from these trials are essential for regulatory submission, as they help to establish the efficacy of the drug in the intended population and support claims made by the manufacturer about its benefits in treating a specific condition. In contrast, earlier phases focus more on safety and dose determination or pharmacokinetics, which involves how the drug is absorbed, metabolized, and excreted in the body. Monitoring long-term effects is typically done in post-marketing surveillance, following the conclusion of Phase 3 trials, to assess the drug's performance over time and in the real-world setting.