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Prerequisites to support high-quality clinical trials in children and young people
  1. Steven Hirschfeld1,
  2. Florian B Lagler2,3,
  3. Jenny M Kindblom3,4
  1. 1 Uniformed Services University of the Health Sciences, 4201 Jones Bridge Road, Bethesda, Maryland, 20814 USA
  2. 2 Institute for Inherited Metabolic Diseases and Department of Pediatrics, Paracelsus Medical University, Clinical Research Center Salzburg GmbH, Strubergasse 21, 5020 Salzburg, Austria
  3. 3 European Society of Developmental, Perinatal and Pediatric Pharmacology (ESDPPP) Council, Leuven, Belgium
  4. 4 Pediatric Clinical Research Center, Queen Silvia Children’s Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden
  1. Correspondence to Dr Jenny M Kindblom, Sahlgrenska University Hospital, S-413 45 Goteborg, Sweden; jenny.kindblom{at}vgregion.se

Abstract

Children have the right to treatment based on the same quality of information that guides treatment in adults. Without the proper evaluation of medicinal products and devices in paediatric clinical trials that are designed to meet the rigorous standards of the competent authorities, children are discriminated from advances in medicine. There are regulatory, scientific and ethical incentives to address the knowledge gap regarding efficacy and safety of medicines in the paediatric population. High-quality clinical trials involving children of all ages can generate data that will ultimately close the knowledge gaps and support decision making.

For clinical trials that enrol children, the needs are specialised and often resource intensive. Prerequisites for successful paediatric clinical trials are personnel with training in both paediatrics and neonatology and expertise in clinical trials in these populations. Moreover, national and international networks for efficient collaboration, dissemination of information, and sharing of resources and expertise are also needed, together with competent, efficient and high-quality local infrastructure with effective processes. Monitoring and oversight bodies with the relevant competence, including expertise in paediatrics, is also an important prerequisite for paediatric clinical trials. Compromise in any of these components will compromise the downstream results.

This paper discusses the structures and competences needed in order to perform effective, high-quality paediatric clinical trials with the ultimate goal of better medicines and treatments for children. We propose a model of examining the process as a series of components that each has to be optimised, then all the components are actively optimised to function together as an ecosystem, and the resulting ecosystem functions well with the general research system and the healthcare delivery system.

  • evidence based medicine
  • general paediatrics
  • outcomes research

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Footnotes

  • 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.

  • Provenance and peer review Commissioned; externally peer reviewed.

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