Why children still wait years for medicines adults already have
Two decades of incentives and mandates on both sides of the Atlantic have transformed paediatric drug development. They have not closed the gap.
In brief
How the US BPCA and PREA and the EU Paediatric Regulation shape children’s medicines, and why paediatric data still arrive years after adult approval.
- The US pairs a voluntary reward (BPCA) with a legal requirement to study children (PREA).
- The EU requires an agreed paediatric investigation plan before most marketing applications.
- Deferrals are routine, so paediatric data often arrive years after adult approval.
Most medicines are still developed for adults first. Children get access later, often through off-label prescribing, while the evidence that would support a licensed paediatric dose is gathered, deferred or never collected at all.
That gap is not for want of policy. The United States and the European Union have both spent more than twenty years building systems designed to pull paediatric studies earlier into development. They have changed behaviour. They have not changed the basic sequence.
Carrots and sticks in the United States
The US runs two complementary laws. The Best Pharmaceuticals for Children Act (BPCA), first passed in 2002, is voluntary. A sponsor that completes the studies set out in an FDA Written Request can earn six months of additional market exclusivity, attached to all of its existing exclusivities and patents for that active moiety.
The Pediatric Research Equity Act (PREA), from 2003, is a mandate. Applications for a new active ingredient, indication, dosage form, dosing regimen or route of administration must include a paediatric assessment unless the FDA grants a waiver or a deferral.
Europe’s single instrument
The EU took a different route with its Paediatric Regulation, in force since 2007. Companies must agree a paediatric investigation plan (PIP) with the European Medicines Agency’s Paediatric Committee, usually by the end of adult pharmacokinetic studies. A marketing application is not valid without either compliance with an agreed PIP, a waiver or a deferral.
Completing a PIP can unlock a six-month extension of the supplementary protection certificate, or two extra years of market exclusivity for orphan medicines.
Six months of extra exclusivity is a powerful incentive for a blockbuster. For a drug with modest sales, it buys very little.
Where the lag comes from
Both systems allow paediatric studies to be deferred until after adult approval, and deferral is the norm rather than the exception. Trials in children are slower to recruit, need age-appropriate formulations and face ethical constraints that adult trials do not. Each adds time.
The incentive structure also skews towards commercially large products. The medicines children most need, for conditions that mainly or only affect them, often have no adult market to anchor them.
What reform targets
Proposals in Brussels and Washington keep returning to the same pressure points: tying rewards more closely to unmet need, tightening deferral timelines, requiring studies based on a drug’s mechanism of action rather than its adult indication, and making it harder for paediatric commitments to slip quietly past their due dates.
