In a confirmatory clinical trial, there is at least one primary efficacy endpoint prespecified for treatment effect evaluation. In a drug development to treating chronic diseases, definitive understanding of a treatment effect would rely on assessment of a long-term efficacy endpoint or endpoints, which may take longer than three years to measure. However, feasibility of a long-term confirmatory clinical trial has a direct impact to public health at large, especially when there is no treatment availability. In such cases, there is an unmet medical need to consider studies that might help bring a treatment to market sooner than demonstration of an ultimate clinical efficacy benefit outweighing its risks. In this paper presentation, we will introduce post-treatment biomarker endpoints, provide clinical contexts for biomarker endpoints use, and their roles in clinical trials. Some study design and statistical considerations for biomarker endpoints assessments will be given. Some literature examples with results may be used for illustration.
*This abstract reflects the views of the author and should not be construed to represent the views or policies of the U.S. Food and Drug Administration.