Changes in Research and Development of Medicinal Products since the Paediatric Regulation

The lack or incompleteness of evidence of the efficacy and safety of drugs used in children has been a growing concern in the recent past. The great majority of drugs prescribed to children are often given either on an unlicensed or an “off label” basis simply by extrapolating data for adults and without conducting any paediatric clinical, pharmacokinetics, dose finding, or formulation studies in the paediatric population. The paediatric pharmaceutical repertoire therefore comprised pills too large to swallow and extemporaneous formulations containing excipients unsafe or unpalatable to children. Diseases in children, however, are often different from their adult equivalents, and the processes underlying growth and development might lead to a different effect or an adverse drug reaction unseen in adults. The health and, therefore, quality of life of the children in Europe suffer from a lack of testing and authorisation of medicines for their use. It means that children are “orphans” of appropriate medicinal products and children continue to be exposed to risks, and at the same time miss out on therapeutic advances. This is particularly ironic considering that our modern system of medicines regulation, that ensures the high standards of safety, quality and efficacy of medicinal products for use in adults, was developed primarily in response to therapeutic disasters, or “drug catastrophes”, that occurred in children in the past ( such as the numerous cases of icterus induced by sulphanilamide which occurred in 1937 and the well-known phocomelia caused by thalidomide in the 1960s).