Emerging Endpoints: Minimal Residual Disease

Another emerging endpoint is minimal residual disease. This benchmark has emerged because even those in complete remission are known to experience relapses, which is true even when the patient has been in remission for significant periods of time. Emerging technology can now identify malignancies at levels orders of magnitude lower than what was previously conceivable.

 

Minimum residual disease can be used for a number of biomarkers, including diagnostic, prognostic, predictive, efficacy-response or monitoring. The efficacy-response biomarker can act as a surrogate endpoint for purposes of approval, but it does not provide any measurement of primary clinical benefit.

 

Meta-analysis will be required to validate minimum residual disease as a surrogate endpoint. However, MRD isn’t without issues as a surrogate endpoint. For example, MRD might not apply to every subgroup. In addition, the surrogate endpoint and the clinical benefit endpoint might not square up.

This underscores the importances of data analysis and study design. The best way to determine how close these two figures are is to include a variety of mechanisms of action. Missing and unevaluable assessments can distract from this task.

 

Our next blog post will involve placebos and blinding guidelines in randomized clinical trials.

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