Une large proportion de la pharmacopée utilisée en pédiatrie, incluant les psychotropes, est prescrite hors du cadre de leur autorisation de mise sur le marché (AMM), et les enfants et adolescents se voient souvent refuser l’accès à des produits nouveaux ou innovants. Parce que cette situation n’est plus éthiquement acceptable, la nécessité d’obtenir des informations pour les médicaments utilisés en pédiatrie semble aujourd’hui évoluer vers un consensus global. Sur cette base, il était clair dans l’UE, à l’instar de ce qui s’était produit aux États-Unis, qu’il convenait d’instaurer une obligation légale pour l’industrie pharmaceutique à réaliser des programmes pédiatriques. Cette régulation pédiatrique européenne, entrée en vigueur en 2007, ouvrant une nouvelle ère de l’histoire de réglementaire européenne, a pour ambition d’améliorer la santé des enfants et adolescents. Mais le développement pédiatrique reste difficile et nombreux sont les obstacles auxquels se heurte la recherche clinique en population pédiatrique. Cet article présente le nouveau règlement pédiatrique européen, illustre sa raison d’être grâce à l’exemple des antidépresseurs chez les enfants et adolescents, et aborde les nouveaux enjeux de la recherche en psychopharmacologie pédiatrique.

A large proportion of medicines used in children are actually prescribed off-label, which can place children at a direct risk of under- or overdosing and a delayed risk of long-term adverse effects, and children have often been denied access to new or innovative medications. Many generations of paediatricians and other physicians have learned to live with the situation. But because such situation is nowadays considered as unethical, the need to obtain paediatric information for medicines used in children seems a matter of consensus on a global basis. If the therapeutic effects of amphetamines in hyperactive children were first described in 1937, thus, preceding the major discoveries of adult psychopharmacology, since this little innovation has occurred in paediatric psychopharmacology. However, it is widely recognized that mental disorders in children and adolescents lead to a major burden for them and for their families. Over the past decade, under the impulsion of the US paediatric legislation, the number of high quality paediatric psychopharmacological studies has dramatically increased. Like what happened in the US, it was clear in European Union, that there was a need for a legal obligation for Pharmaceutical Companies to perform studies in paediatric populations, and a new Paediatric European Regulation came into force in 2007, opening a new era in the history of European regulation with the ambition of improving the health of children and adolescents. Therefore drug development is changing; the concern of protecting children against clinical research fading away, a new paradigm is now emerging, i.e. protecting children through clinical research. In Europe, paediatric development is no longer an option but needs to be truly integrated in clinical development plans with paediatric evaluations being a regular part of every drug development process. It seems reasonable to anticipate that more research may occur as well in paediatric psychopharmacology and more studies will be enrolling paediatric patients all over the world in the forthcoming years. But paediatric clinical development is difficult and the hurdles of conducting clinical trials in paediatric population are numerous. This article presents briefly the new European Paediatric Regulation, illustrates its purpose through the example of antidepressants in children and adolescents, and discusses new research challenges in paediatric psychopharmacology. Ultimately, it is through well-conducted research that children will gain access to new medications and receive safe and optimal drug therapy.