Pediatric Cancer Study Requirements Added To FDA User Fee Bill
User fee legislation headed for a US House vote this week would require pediatric studies of certain molecularly targeted cancer drugs; the requirement, which could apply to some orphan drugs, would not take effect for three years.
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House Energy & Commerce Committee adds Give Kids a Chance Act to user fee reauthorization package during markup. Bill was modified to exclude provision giving the FDA authority to require preclinical studies.
Advisory committee says planned waivers for molecularly targeted studies should take into account comparative adult efficacy and toxicity data for multiple drugs in the same class, as well as differences in product attributes, such as route of administration and dosing schedule; unmet clinical need should have less influence on decision to grant waivers, panelists said.
Early advice meetings allow sponsors to hear the FDA’s current thinking about relevance of a specific molecular target and expectations for early assessment of an adult cancer drug in children unless a waiver or deferral is justified; agency has waived subsequent pediatric studies for some same-in-class products, including PD-1/L1 inhibitors.