The U.S. Food and Drug Administration (FDA) issued a draft guidance document, “FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act.” The draft guidance addresses early planning for pediatric evaluation of certain molecularly targeted oncology drugs for which original new drug applications (NDAs) and biologics license applications (BLAs) are expected to be submitted to the FDA on or after August 18, 2020, in accordance with section 505B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), which was amended by the FDA Reauthorization Act of 2017 (FDARA).
“When it comes to medical product innovation, children must always remain front-of-mind, particularly those with dire needs. It is important to deliver promising new therapies as quickly as possible, while still ensuring the utmost safety,” said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. “Thanks to amendments to the Federal Food, Drug, and Cosmetic Act made by the FDA Reauthorization Act of 2017, we have a new mechanism to require the evaluation of certain novel cancer medicines for potential pediatric treatment. Our new draft guidance addresses implementation of these amendments, which we anticipate will facilitate early pediatric assessment of certain targeted cancer drugs and accelerate the development of new, safe and effective therapies for pediatric patients. ”
Traditionally, drug development for pediatric cancers lagged, in part, because the requirements to study new cancer drugs in children have been based on whether the cancer occurs in children — and many adult cancers rarely occur in children. New, targeted oncology drugs being developed for adult cancers may prove effective in the treatment of some cancers occurring primarily in pediatric patients with similar molecular targets.
The draft guidance provides the pharmaceutical industry, clinical investigators and institutional review boards with information to facilitate pediatric studies of molecularly targeted oncology drugs. Specifically, if an original NDA or BLA is for a new active ingredient, and the drug that is the subject of the application is intended for treatment of an adult cancer and directed at a molecular target the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer, reports on the required molecularly targeted pediatric cancer investigation must be submitted with the marketing application, unless this requirement is waived or deferred. The draft guidance describes lists, which the FDA sometimes refers to as “The Relevant Molecular Target List” and “The Non-Relevant Molecular Target Leading to Waiver List,” that the FDA plans to update regularly, and that are intended to serve as a guide to sponsors as they consider development plans for new targeted drugs and the need for early pediatric assessments.