Developing medicines

The majority of medicines are not licensed for paediatric use

Although many medicines are given to children, the majority are not licensed for paediatric use. This means that the medicine has not been thoroughly tested in clinical trials in children and has not been approved for use in children by regulatory authorities.

Children are not just small adults

Children are not just small adults – they are still developing and this can affect how their bodies process medicines. The paediatric population itself also varies a lot in terms of age, size and maturation, from small babies to toddlers, to children and adolescents, with each stage having its own needs where medications are concerned. For these reasons, it is important that medicines are specifically licensed for use in children and also that age-appropriate formulations are developed.

Children find it hard to take medicine

Swallowing conventional tablets may be difficult for young children and those with certain conditions. If an age-appropriate formulation is not available, as is often the case, pharmacists or caregivers may have to crush tablets to make liquids. Such practices are not ideal as they can result in the child having a varying amount of the medicine each time, which can affect safety and how well the medicine works.

Children may also refuse to take some medicines if they have an unpleasant taste. In some cases, mixing medicines with food or juice may alter its properties. To make the medicine more palatable for children, special formulations may need to be produced where bitter tastes are masked with other flavours.

Should a medicine be made for a child?

When making formulations for children, it is also important to consider that some inactive substances (‘excipients’) that are added to produce tablets or medicinal liquids may not be suitable for children.

An additional challenge that specifically relates to the development of children’s medicines is the need for accurate dose administration for varying ages and weights, for example, when very small doses are needed for babies and infants.

Age-appropriate formulations overcome these problems by offering easy-to-administer palatable medicines where the dose can be accurately adjusted. Child-friendly medicines are often in the form of liquids and suspensions, but issues with excipients, palatability and a short shelf-life make appropriate liquid formulations difficult to produce. Child-appropriate solid dosage forms, such as orodispersible minitablets (ODMTs), are being increasingly studied. These tiny uncoated tablets disintegrate rapidly in the mouth and offer accurate dosing and convenience, without requiring the ability to swallow.

As every parent knows, giving medicines to children is not easy, particularly when the child is taking multiple medicines frequently. Licensed age-appropriate formulations, with proven safety and effectiveness, can improve compliance and also make life easier for children and their carers.

Reviewed November 2021