Facts about children medicines

There is a clear need for adapted medicines in children. The term “Children” does not mean a unique segmentation of the population; it designates a heterogeneous population with different sub-categories.

Each sub-category has its own metabolism and drugs must be tested in them before being approved. As a consequence, drug dosage must be adapted depending on specific criterion so as to provide the children with the correct treatment.

Certain organs in children mature more rapidly than others. One consequence of this is that at certain age and for specific medicines, children might need stronger doses than adults because they are capable of metabolizing the drug substance faster the adults do. On the contrary, dividing the dose based on the size or weight might lead to overdoses.

Another current issue comes from the pharmaceutical industry which tends to develop medicines for adults. These adult medicines are then adapted in hospitals based on an accurate calculation of the individual needs. However, these of-labeled hospital preparations cannot be done under current regulatory standard requirements which can sometimes lead to mis-manipulations and cause serious adverse events. For example, when using an adult parenteral formulation, hospital pharmacies dilute the preparation several times before achieving the adequate concentration.

The use of unlicensed and off label medicine in children is widespread and it has been an increasing concern over the last years. In the EU, fifty per cent or more of medicines used in children have never been actually studied in this population, but only in adults, and not necessarily in the same indication (EMEA, 2007).

On an ethical standpoint, it is difficult to say whether or not a neonate is willing to participate to a clinical trial and experiment a new drug, or to interpret his/her expressions. It is also difficult to sample blood in this very small population, which is necessary to measure safety, efficacy or treatment response. Some think it is rather unethical not to undertake drug trials in children because without appropriate clinical data, the benefit for the patient of using a drug is diminished as safety concerns arise.