Early Learning

Clinical trials among neonate and paediatric patients characteristically present a set of unique challenges and opportunities to consider. The various ethical, scientific and practical challenges in performing research among these groups require a specific approach to ensure efficiency of time, money, promotion of goodwill and repeat studies – with full parental participation.

Despite a general consensus that the recruitment of newborns and children into clinical trials is a challenge to overcome, clinical research in paediatric populations is continuing to grow each year. This growth is largely due to recent regulatory and legislative changes in the US, Europe and Japan, which give drug companies financial incentives to conduct drug studies in children balanced against potential penalties if they don’t. These incentives have in turn resulted in a dramatic increase in paediatric drug studies. Prior to these regulatory changes, adult medications were often used in children without being specifically labelled for that use.

One paediatrician has called this practice “an uncontrolled experiment with every paediatric prescription.” The process of regulatory and legislative development began in the US in 1997, culminating in 2003, when Congress authorised the ‘Pediatric Research Equity Act’ (PREA).

A similar regulation has now recently come into effect in the EU, the Paediatric Medicines Regulation. Both regulations mandate that a paediatric plan be submitted with all new investigational new drugs (INDs), although implementation of the plan may be deferred until after adult studies have been completed or waived altogether for drugs that will have no paediatric uses. The PREA also provided for studies on drugs frequently used in children that were off-patent, and thus had no willing research sponsor (no such provision exists under the EU regulation).