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Paediatric Pharmacotherapy and Drug Regulation -- Moving Past the Therapeutic Orphan
  • Charlotte Moore-Hepburn,
  • Michael Rieder
Charlotte Moore-Hepburn
University of Toronto
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Michael Rieder
University of Western Ontario
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Abstract

The development of specific drug therapy for children was a paradigm changing event that transformed paediatric medical practice. However a series of tragedies involving drug treatment for children resulted in a gap developing between drug regulation and practice, with the majority of drugs used in child health care being used “off label” rendering children therapeutic orphans. Over the past two decades changes in drug regulation led by the US FDA and followed by the European Union’s EMA have led to substantial changes in how new drugs with potential use in children are studied and labelled. While these changes have substantially improved labeling for new drugs, there has been much less progress with older drugs. As well while the unique challenges of conducting clinical research in children have been addressed by novel clinical trial designs, many of these innovations have not been translated into approaches accepted for the drug approval process. The regulations applying to the need for paediatric studies currently are only applicable in the United States and the European Union, and there is less impetus for paediatric labeling in other jurisdictions. This impacts on a number of issues beyond labeling, including the availability of child-friendly formulations. Finally the impact of Brexit on paediatric drug studies in the UK remains unclear and subject to on-going negotiations between the UK government and the European Union.

Peer review status:IN REVISION

31 Aug 2020Submitted to British Journal of Clinical Pharmacology
02 Sep 2020Assigned to Editor
02 Sep 2020Submission Checks Completed
04 Sep 2020Reviewer(s) Assigned
20 Sep 2020Review(s) Completed, Editorial Evaluation Pending
22 Sep 2020Editorial Decision: Revise Minor