On December 12, 2019, the FDA issued draft guidance to implement amendments to the Federal Food, Drug, and Cosmetic (FD&C) Act that will facilitate early assessment of studies of molecularly targeted oncology drugs that may be effective in the treatment of pediatric cancers.
The guidance document was issued in advance of original new drug applications (NDAs) and biologics license applications (BLAs) that the FDA expects to receive on or after August 18, 2020, the date that the provisions of the amendment will become effective.
Titled “FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act—Guidance for Industry,” the document is designed to provide information to pharmaceutical companies, clinical investigators, and institutional review boards ahead of planning their studies of molecularly targeted oncology drugs, with stipulations for early planning for pediatric evaluation of products that have the potential to treat this patient population.
“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 FDA noted in a press release.
According to Adm. Brett P. Giroir, MD, Acting FDA Commissioner, the agency anticipates that the new amendments “will facilitate early pediatric assessment of certain targeted cancer drugs and accelerate the development of new, safe, and effective therapies for pediatric patients.”
The FDA will issue the final version of the guidance in 2020 and invites comments on the draft, preferably by February 11, 2020.
“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,” Dr Giroir added.