Trial design, data and the evidence base behind paediatric prescribing.
Regulators increasingly accept that adult efficacy data can carry over to children when the disease and the drug’s effect are similar enough. The argument is about how much.
Evidence
Small populations, cautious parents and dozens of trials chasing the same patients make recruitment the most common reason paediatric programmes slip.
Evidence
Health records, registries and claims data contain years of experience with medicines in children. Turning that into regulatory-grade evidence is harder than it looks.
Evidence
Physiologically based pharmacokinetic models simulate how a drug moves through a growing body. Regulators now see them in a large share of paediatric submissions.
Evidence
Children cannot fully consent, so research rules ask more of the people designing paediatric trials. Placebo arms are where those rules bite hardest.
Evidence
The babies in neonatal intensive care receive some of the most intensive drug treatment in medicine. Very little of it was ever tested in them.
