Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) discussed the use of biomarkers to monitor efficacy in gene therapies.
As Dr. Marks explains, one benefit of a biomarker is it can act as a comparator instead of using a placebo group. The biomarker can be measured from the same person being treated and it can be a measurement of treatment effect. This is especially important for rare conditions in which the patient population is very small and patient recruitment for a trial is limited. Biomarkers also provide an opportunity to have a noninvasive outcome measure that can ease the patients’ burden, stress, and/or discomfort during the trial.
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