Medicines and children: children medicines qualification project.
Just one third of the medicines on the market has been tested for children (safety and efficaciousness). For the remaining, the medicine therapy comes out to be improper, due to the following causes:
- the medicine was not tested/registered for children (“unlicensed”);
- the medicine is used for different purposes from the ones it was tested/registered (“off-label”);
- it is a product which is not proper to be given/accepted;
- it is about a molecule which is not marketed as a pharmaceutical product (a too rare disease, off market), so they need administer it as a simple chemical preparation.
For these reasons, the paediatrist is faced with difficult decisions to take on therapy, in particular for situations which are not important from the clinical point of view.
The clinical use for children of molecules potentially innovative is to be deduced from the data coming from clinical studies on adults, with many limits as to safety/efficaciousness, as far as pharmacokinetics (the features of absorption, distribution, metabolism, excretion of the medicine examined) and pharmacodynamics (the outcome of the interaction between medicine and body) are concerned. These results might be completely different during childhood.
The Ethical Committees of the single institutions are frequently called to help to take a decision, in relation to using medicines for children without authorisation or for different purposes from the ones indicated.
The modalities to grant protection to children as research subjects is a very delicate matter, as it comes out from directions and guidelines for children clinical studies and from the Declaration of Helsinki.
The awareness of medicines use for children has been growing and growing during last years, taking to new laws and rules coming into force starting from the ‘90s. In Europe, in 1997 the Committee for Proprietary Medicinal Products (CPMP), being part of the European Medicine Evaluation Agency (EMEA) which edited a paper, called Note for Guidance on Clinical Investigation of Medicinal Products in Children.
