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Challenges in clinical trials for children and young people
  1. Florian B Lagler1,2,
  2. Steven Hirschfeld3,
  3. Jenny M Kindblom2,4
  1. 1 Institute for Inherited Metabolic Diseases and Department of Pediatrics, Paracelsus Medical University, Clinical Research Center Salzburg GmBH, Strubergasse 21, Salzburg, Austria
  2. 2 European Society of Developmental, Perinatal and Pediatric Pharmacology (ESDPPP) council, Leuven, Belgium
  3. 3 Uniformed Services University of the Health Sciences, 4201 Jones Bridge Road, Bethesda, Maryland, 20814 USA
  4. 4 Pediatric Clinical Research Center, Queen Silvia Children’s Hospital, Sahlgrenska University Hospital, Goteborg, Sweden
  1. Correspondence to Dr Jenny M Kindblom, Pediatric Clinical Research Center, Queen Silvia Children’s Hospital, Vitaminvägen 21, Sahlgrenska University Hospital, Goteborg 413 45, Sweden; jenny.kindblom{at}


There is a well-known knowledge gap regarding the efficacy and safety of medicines in children of all ages and children are often treated with medicines off-label. Children are thus deprived of treatment based on the same quality of information that guides treatment in adults. The knowledge gap regarding efficacy and safety of medicines in children has been acknowledged by authorities and is reflected in legislation both in North America and in the European Union. Recent reports on the effects of legislation indicates that paediatric clinical trials remain a challenge.

Paediatric clinical trials are needed in the entire developmental age spectrum and are especially needed in certain therapy areas. Paediatric clinical trials have special features compared with trials in adults, and these need to be taken into account. These special features include scientific issues related to small samples and heterogeneity, the consent/assent procedure, the need for age-appropriate study information, specific outcomes and safety issues related to development and maturation. Competence in paediatric clinical trials is required in both designing, planning, co-ordinating and organising paediatric clinical trials, as well as research infrastructure and networks to increase power and disseminate information and expert advice. Strengthening of paediatric clinical research is essential to facilitate generating the data that will let children enjoy new medical advances in a similar manner as adults.

  • evidence-based medicine
  • pharmacology
  • general paediatrics

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  • Contributors All authors performed literature searches and read relevant literature. All authors drafted the manuscript and reviewed it critically. All authors approved the re-submission.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Provenance and peer review Commissioned; externally peer reviewed.

  • Data availability statement There are no data that can be made available with this review article.