Clinicians and researchers are voicing concerns that the US Food and Drug Administration (FDA) has overemphasized the role for extrapolating adult data in pediatric populations as part of recent draft guidance on ethical considerations for conducting clinical trials that involve children.
In September 2022, FDA issued draft guidance aimed at assisting industry, sponsors and institutional review boards (IRBs) when conducting or reviewing clinical investigations of drugs, biological products and medical devices in pediatric populations. (RELATED: New FDA guidance offers ethical roadmap for including children in clinical trials, Regulatory Focus 26 September 2022)
The guidance explores the principle of scientific necessity, noting that “for products that are being developed for use in adults and children, if effectiveness in adults can be extrapolated to children, then effectiveness studies in adults should be conducted to minimize the need to collect effectiveness data in children.”
In public comments on the draft guidance, the American Academy of Pediatrics (AAP) wrote that the document could give the “impression that if a product has been shown to be effective in adults, it unnecessary to evaluate it in children.” While extrapolation is an important tool, the “framing” in the draft guidance does not address issues of adverse effects and long-term problems that may result from medications only tested in adults. “The guidance should clarify that very often there are important scientific questions in pediatrics that can be addressed through pediatric studies,” wrote AAP President Moira Szilagyi…
