To help with anti-cancer drug development, the FDA has developed two new lists of molecular targets to guide submissions for pediatric study plans.
The two lists, posted Tuesday by the FDA’s Oncology Center of Excellence, are aimed at fostering the development of new oncology drugs or biologics for pediatric populations. They also fulfill a commitment the agency made under the FDA Reauthorization Act of 2017 (FDARA).
One list points to the molecular targets that are likely to contribute to the growth or progression of at least one pediatric cancer, while the other identifies the targets of new drugs currently in development that would be automatically exempted from pediatric cancer study requirements.
“Pediatric cancer drug development has lagged far behind development of cancer drugs for adults,” FDA Commissioner Scott Gottlieb said Tuesday in a Twitter thread announcing the lists.
The creation of these lists is further intended to harness the potential of tumor genetic profiling in pediatrics as well as leverage the amendments made by section 504 of FDARA to section 505B of the FD&C Act, which set forth new requirements on pediatric drug development.
“Until the passage of FDARA, section 505B of the FD&C Act has not typically been a useful mechanism to require the development of drugs for pediatric cancers since most of the oncology drugs approved for adults are used to treat cancers that are very rarely or never occur in children,” the agency said. “Therefore, historically, drug sponsors have requested and obtained waivers for conducting the required assessments of these drugs in pediatric patients.”
The 2017 legislative changes addressed this issue by nixing the FD&C Act’s orphan drug designation exemptions on pediatric assessments. Under FDARA, conducting these pediatric assessments are required “even when the adult indication has received an orphan designation, or when the adult indication does not occur, in the pediatric population,” the FDA added.
The agency identified a total of 205 candidate molecular targets for the development of the new lists. Most of these molecular targets (77) are classified as “others,” followed by those that target a gene abnormality (62), a cell lineage determinant (40) and the tumor microenvironment of the immune system (21). Only five molecular targets are listed as candidates for automatic waivers.
The lists form part of a broader effort at the FDA to incentivize pediatric drug development. Other recent actions in the space include the FDA’s adopted version of an International Council for Harmonization addendum and 2017 guidance that provides policy clarifications on orphan designation status for pediatric subpopulations of common diseases.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email email@example.com for more information.
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