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

Nicolino Ruperto, Richard Vesely, Agnes Saint-Raymond, Alberto Martini, Tore K. Kvien

Research output: Contribution to journalArticle

Abstract

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 nonrewarding 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 ageappropriate 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 http://www.prcsg.org), covering North America, and the Paediatric Rheumatology International Trials Organisation (PRINTO at http://www.printo.it), 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.

Original languageEnglish
Pages (from-to)1893-1896
Number of pages4
JournalAnnals of the Rheumatic Diseases
Volume72
Issue number12
DOIs
Publication statusPublished - Dec 2013

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Pediatrics
Rheumatology
Legislation
Juvenile Arthritis
European Union
Medicine
Pharmacokinetics
North America
Rheumatic Diseases
Pharmaceutical Preparations
Research Design
Clinical Trials
Organizations

ASJC Scopus subject areas

  • Rheumatology
  • Immunology
  • Biochemistry, Genetics and Molecular Biology(all)
  • Immunology and Allergy

Cite this

Impact of the European paediatric legislation in paediatric rheumatology : Past, present and future. / Ruperto, Nicolino; Vesely, Richard; Saint-Raymond, Agnes; Martini, Alberto; Kvien, Tore K.

In: Annals of the Rheumatic Diseases, Vol. 72, No. 12, 12.2013, p. 1893-1896.

Research output: Contribution to journalArticle

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