When FDA Requires Pediatric Studies for New Cancer Drug Submissions: New Draft Guidance

Regulatory NewsRegulatory News | 12 December 2019 |  By 

Beginning 18 August 2020, biopharma companies will begin submitting pediatric assessments of certain molecularly targeted oncology drugs for which new drug applications (NDAs) and biologics license applications (BLAs) are expected to be submitted to the US Food and Drug Administration (FDA).

New FDA draft guidance published on Thursday explains how sponsors will need provide such submissions and what factors will be considered.

“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 agency said.

The 13-page draft guidance explains how FDA will decide whether a molecular target is “substantially relevant to the growth or progression of pediatric cancer,” including information on waivers and deferrals.

To help with such determinations, FDA’s Oncology Center of Excellence currently offers lists of more than 200 molecular targets that include those 1) for which existing evidence and/or biologic rationale exist to determine their potential relevance to the growth or progression of pediatric cancers and 2) targets for which there is evidence that they are not associated with the growth or progression of pediatric tumors, and for which the requirement for early pediatric evaluations of drugs and biologics which are directed at these targets would be waived.

“Molecular targets that lack sufficient evidence for FDA to determine whether they are ‘substantially relevant’ or ‘not substantially relevant’ will not be included in a target list, however, the lists will be updated regularly to reflect additional determinations regarding the relevance of molecular targets,” the draft notes.

The guidance also includes details on what content should be included in initial pediatric study plans and recommended studies to be included. Additional considerations for rare cancers are also included, in addition to the agency’s work with other international regulators on the issue.

“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,” Acting FDA Commissioner Adm. Brett Giroir said.

FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of the FD&C Act


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