Should adult safety studies precede the initiation of pediatric oncology clinical studies?
The Committee noted that there is no single answer to the question. As a general practice, an adult Phase I study should precede pediatric oncology clinical studies in order to determine preliminary safety and dosing information. There may be an exception for some drugs that may be exclusively developed in pediatrics. The committee acknowledged that current preclinical testing may not be able to identify about 1/3 of clinical toxicity; however, the development of newer preclinical models may have better correlation with both activity and toxicity. It may be possible with predictive preclinical data of sufficient scope and plausibility to begin clinical studies directly with pediatric patients, particularly if the planned indication is a pediatric tumor type, and there is a reasonable expectation of clinical benefit in relation to a potential risk.
