This guidance includes recommendations on the inclusion of pediatric patients in clinical trials for cancer treatments.
FDA states that “Eligibility of a specific pediatric population for a cancer clinical trial should be considered when there is clinical evidence or a strong scientific rationale to suggest that pediatric patients with a specific cancer diagnosis, histologic subtype, or tumor associated with the same relevant molecular target may benefit and when there is compelling nonclinical and/or adequate clinical information to sufficiently justify patient risk.”
FDA encourages including pediatric patients for conditions without known curative options in early-phase trials that assess dose, safety, and PK in a variety of tumor types when compelling nonclinical data and/or early adult clinical data suggest activity.
The minimum age of eligibility specified in late-phase trials should be tailored to the biology of the disease under study, the scientific objectives of the trial, and the existing data regarding the mechanism of action and safety profile of the drug.