Advancing Pediatric Drug Development

MRD as a surrogate for Overall Survival trending idea

Briefly describe your research question :

MRD as a general measure of tumor burden has multiple potential regulatory and clinical uses as a biomarker; however, the evidence to support the clinical validity of MRD as a biomarker varies across hematologic cancer types and patient populations. In pediatric patients with relapsed/refractory AML, MRD-negative CR has not yet been established as an intermediate endpoint likely to predict overall survival. To investigate this in a trial would be extremely time consuming and costly and could slow down the approval of life saving therapies for patients who have no options.

A retrospective analysis of MRD data that the FDA has received through the review of recently approved agents in the space could help answer the question of whether MRD-negative CR could be an intermediate endpoint likely to predict overall survival. If the results of this retrospective analysis suggest that MRD-negative CR could be an intermediate endpoint, this could accelerate drug development for pediatric patients with AML.

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Idea No. 155