Background: Mental disorders in children and adolescents lead to a significant burden for them and their families, and pharmacological treatments have been slowly introduced essentially based on adult experiences. Since the 90’s, first with the introduction of the selective serotonin uptake inhibitors and then with the second-generation antipsychotics, constant increased use of psychotropic agents in the paediatric population has been witnessed, not always supported by robust scientific data, leading to a large number of prescriptions being off-label.
Objective: To examine the challenges and opportunities of paediatric psychopharmacological development and to understand the key ethical, clinical and practical considerations in designing clinical trials and programs in paediatric psychopharmacology.
Methods: Review of approved psychotropic agents in Europe for paediatric versus adult indications. Review of key ethical, clinical and practical considerations in paediatric psychopharmacology.
Results: Except for ADHD, compared to what is available for adults, very few psychotropic agents are approved in EU for paediatric use, illustrating the current limitations of paediatric research and development and leading to common worrisome off-label prescriptions. Since the end of the XXth century, both US and EU paediatric regulations have played a significant role transforming drug development, helping the concern of protecting children against clinical research fading away and promoting new paradigms, i.e. protecting children through clinical research and embracing new models of drug development.
Conclusion: Numerous challenges and opportunities for better treatment and prevention of child and adolescent mental conditions remain. The challenges and hurdles of conducting research in paediatric population are numerous and complex including ‘moral’ and ethical issues, scientific, practical (including age appropriate drug formulations) and last but not least financial issues. These challenges may of course differ across paediatric therapeutic areas, however paediatric psychopharmacology can serve as a perfect paradigm for all paediatric pharmacological developments.