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Abstract:
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Precision medicine presents unique challenges to the design of clinical trials for all cancer types, including rare diseases such as pediatric cancers. In this talk, I will provide an overview of precision medicine features in pediatric cancer trials conducted through Children's Oncology Group, the pediatric cooperative group member of the NCI's National Clinical Trials Network. These features include biomarker-driven stratification in tumor-agnostic basket trials, and marker- and risk-based stratification in other cancer-specific therapeutic trials. A common feature (and challenge) in pediatric trials is the use of non-concurrent control data when randomization is not feasible or ethical, in which case data from recently-completed trials is often used to inform the design or analysis of current and future trials. I will discuss these challenges, as well as examples, experiences, and recommendations from the Children's Oncology Group, highlighting those with broader applicability to precision medicine trials in other rare diseases.
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