In clinical trials, efficacy and safety are measured by means of certain predetermined endpoints, or outcomes. The main objective of a Phase 3 trial is to demonstrate the efficacy of a drug in the actual target population that is proven in a statistically and clinically significant manner. A Phase 3 trial has a clearly defined primary endpoint(s), pre-specified even before study initiation, because they will determine the power of the study and ensure that the research question is specific. Clinical pharmacology, safety and immunogenicity data from early trials provide the framework for the statistical analysis plan, scientific rationale, and selection of endpoints for the Phase 3 study.
In clinical trials, it is essential to be very specific when choosing and defining endpoints. The key characteristics of a good clinical endpoint are;
The endpoint must be clinically relevant and must closely and comprehensively reflect the overall disease being treated without capturing only one aspect of a disease.
The endpoint should capture enough appropriate information for you to analyze and draw appropriate conclusions.
The endpoint should be reliable. “Reliability is the “consistency” or “repeatability” of the endpoint”. Double- checking the measurements for an endpoint
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A positive result on the primary endpoint may be enough to establish a causal relationship between the intervention and outcome, and regardless of how many other endpoints are used in a trial, a negative result on the primary endpoint means that the intervention has not achieved its purported effect. Phase 3 studies generally have only one pre-specified primary endpoint (usually an efficacy endpoint) with an alpha of 0.05 (widely accepted as the threshold for a positive study). A p-value of less than 0.05 establishes causality or an effect, and greater than 0.05 rejects the possibility of an