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The guidance describes the agency's recommendations on how to group patients with different molecular alterations and approaches for evaluating the benefits and risks of targeted therapies where some molecular alterations may occur at low frequencies.
On October 15, 2018, FDA published guidance providing its recommendations for grouping patients with different molecular alterations for eligibility in clinical trials. The guidance also discusses approaches to evaluating the benefits and risks of targeted therapies within a clinically defined disease where some molecular alterations may occur at low frequencies.
The guidance provides information on the “type and quantity of evidence that can demonstrate efficacy across molecular subsets within a disease, particularly when one or more molecular subsets occur at a low frequency.” The guidance specifically addresses identification of patients to include in clinical trials, benefit and risk determination, and refining the target population after initial approval.