The US Food and Drug Administration (FDA) has issued new draft guidance for sponsors who are developing anti-infective drug products for the pediatric population.
The draft guidance notes that pediatric drug development in general comes with some challenges, including the fact that differences not only in body size but also organ maturation and body fluid composition and distribution can affect drug pharmacokinetics and pharmacodynamics in children. Also, some infectious diseases have different manifestations in neonates and infants. Finally, logistics and ethics may limit the ability for sponsors to obtain certain samples from children.
Efficacy for anti-infective in a pediatric population can be extrapolated under the guidance if both of two requirements are met: First, the course of the infectious disease proposed to be treated must be similar in pediatric patients as adults. “This implies a similar disease process, including the pathogens recovered from the site of infection. Second, the drug’s effect on pediatric patients should be “sufficiently similar” in adults and children.
“Even when efficacy can be extrapolated, however, pediatric data will be needed to assess the safety and pharmacokinetics of the drug product,” according to the draft guidance , with the goal of demonstrating that the dosing regimen proposed achieves similar exposure for pediatric patients as for adults…