Skip to main content
Premium Trial:

Request an Annual Quote

FDA Issues Draft Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs

NEW YORK – The US Food and Drug Administration issued a draft guidance on Thursday to aid in the early planning of studies for molecularly targeted oncology drugs in pediatric patients.

The guidance is aimed at providing the pharmaceutical industry, clinical investigators, and institutional review boards with information to facilitate pediatric oncology studies. It specifically addresses early planning for evaluation of certain molecularly targeted drugs in pediatric patients for which original new drug applications (NDAs) and biologics license applications (BLAs) will be submitted to the FDA on or after Aug. 18, 2020.

If an original NDA or BLA is submitted after that date for a new active ingredient, and the drug is intended for treatment of an adult cancer and directed at a molecular target the FDA has determined to be also relevant for a pediatric cancer, reports on the pediatric cancer investigation must be submitted with the marketing application, the FDA said. The draft guidance contains lists of relevant molecular targets that are intended to serve as a guide to companies and researchers as they consider development plans for new targeted drugs, and the agency said it will update those lists regularly.

"The list includes molecular targets for which adequate data exist to determine their substantial relevance to the growth or progression of one or more pediatric cancers," the draft guidance notes. Categories include targets related to specific gene abnormalities, targets associated with cell lineage determinants, targets on normal immune cells and cellular components of the tumor microenvironment, and other targets associated with specific pathways or functional mechanisms of normal and/or malignant cells.

The guidance also contains considerations for rare cancers, noting the challenges of standalone trials of investigational drugs in pediatric patients with a rare cancer associated with a specific molecular target. In situations where a conventionally designed pediatric trial may be inefficient or difficult to conduct because of the scarcity of affected patients, the agency said, sponsors may consider including a pediatric cohort in an ongoing adult trial or including adolescent patients in existing adult trials by lowering the age of entry.

"Tissue-agnostic studies may facilitate the development of targeted therapies in multiple pediatric cancers with shared genetic aberrations (e.g., MSI-H/dMMR tumors, NTRK-fusion positive tumors) or may incorporate pediatric cohorts in adult studies which share genetic aberrations with pediatric cancers," the agency said.

The guidance also notes that international cooperation in clinical trials and drug development may aid in the efficient development of new pediatric oncology therapies.

"Due to the rarity of pediatric cancers, which are frequently being subdivided into even rarer subpopulations based on underlying molecular features, international collaboration is increasingly important for facilitating the development of new treatments," the agency said, adding that global coordination is important for prioritizing drugs of interest in general and for specific cancers in pediatric patients.