Impact of the European paediatric legislation in paediatric rheumatology: past, present and future

Ann Rheum Dis. 2013 Dec;72(12):1893-6. doi: 10.1136/annrheumdis-2013-204168. Epub 2013 Aug 20.


Conducting clinical trials in paediatric rheumatology has been difficult mainly because of the lack of funding for academic studies and the lack of interest by pharmaceutical companies in the small and non-rewarding paediatric market. The situation changed dramatically a few years ago with the introduction of the Best Pharmaceuticals for Children Act in the USA and of specific legislation for the development of paediatric medicines (Paediatric Regulation) in the European Union (EU). The EU Paediatric Regulation had a positive impact in paediatric rheumatology-in particular, on the development of new treatments for children with juvenile idiopathic arthritis (JIA). Some problems remain, however, such as greater harmonisation of the regulatory aspects of medicines, how to handle me-too agents, how to conduct adequate pharmacokinetic studies and develop age-appropriate formulations, ethical problems in study review and implementation, and a change in the current JIA classification. The introduction of specific legislation, coupled with the existence of large international networks such as the Pediatric Rheumatology Collaborative Study Group (PRCSG at, covering North America, and the Paediatric Rheumatology International Trials Organisation (PRINTO at, covering more than 50 countries, has led to great advances in paediatric rheumatology. Future changes might increase the possibility of conducting trials with similar approaches in other paediatric rheumatological conditions and provide evidence-based treatments for children affected by rheumatic diseases.

Keywords: Anti-TNF; Juvenile Idiopathic Arthritis; Methotrexate.

MeSH terms

  • Antirheumatic Agents / therapeutic use
  • Arthritis, Juvenile / drug therapy
  • Child
  • Clinical Trials as Topic / legislation & jurisprudence
  • Clinical Trials as Topic / trends
  • Drug Discovery / legislation & jurisprudence
  • European Union
  • Humans
  • International Cooperation
  • Legislation, Drug
  • Pediatrics / legislation & jurisprudence*
  • Pediatrics / trends
  • Rheumatology / legislation & jurisprudence*
  • Rheumatology / trends


  • Antirheumatic Agents