Pediatric Cancer: US FDA Will Take ‘Rational’ Approach To Requiring Combination Studies
Agency urged to prioritize studies based on strength of preclinical and adult data, and good mechanistic understanding of how multiple therapies are expected to work together; new pediatric study requirements for molecularly targeted agents take effect in August 2020.
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Findings being presented at ASCO next month could signify major increase in targets relevant to pediatric oncology, meaning sponsors should prepare for more US FDA-required studies under the RACE for Children legislation.
Beginning in August 2020, applications for some novel cancer drugs and biologics will have to include reports on molecularly targeted pediatric investigations; agency is already working with sponsors on pediatric study development plans and expects to issue two draft guidances soon.
Initial draft list of molecular target candidates has industry, investigators and patient advocates asking how the agency intends to prioritize targets and same-in-class molecules for pediatric studies, and how frequently it will update the list; industry representatives divided as to whether ‘broader is better’ when it comes to the target list.