Children are currently underserviced by drug approval regulations in Canada. They need medications that they can easily swallow, are adequately studied in their age group, are safe and effective, allow dosing flexibility and contain safe nonmedicinal ingredients. At present, these requirements are not fully met. We discuss how Health Canada's regulatory reform should include pediatric-focused prescribing considerations.
An estimated 50% to 80% of all medications prescribed to children in Canada are given off label, as their use deviates from the dose, age, route of administration, formulation or indication described in the product monograph. (1-3) For young children, the commercial preparation (often a tablet for adults) has to be modified for administration, a practice called compounding. Compounded formulations prepared at the pharmacy or the patient's home are not in keeping with pharmaceutical manufacturing standards, and have the potential to cause patient harm. (4,5) A recent study showed that only 47% and 28% of product monographs of newly approved medications in Canada have pediatric-specific information and indications, respectively. (6) Another Canadian study at a tertiary pediatric hospital showed that 48% of frequently compounded medications were commercially available as child-friendly formulations in the United States or European Union. (7)
Both the US and EU have implemented pediatric-targeted policies over the past 2 decades to promote clinical drug research involving children to ensure safe, evidence-based pediatric drug labelling and to encourage the development of pediatric formulations. When pediatric use of a product is anticipated, the US Food and Drug Administration (FDA) requires a pediatric study plan as a prerequisite to a new drug application. Similarly, the European Medicines Agency (EMA) mandates that sponsors complete a pediatric investigation plan before their application for market authorization. Both the FDA and EMA also have incentives to promote the development of pediatric medications and formulations. (8,9) Between 2007 and 2015, both the EU and US had twice as many new pediatric medications approved, and 32% and 62% more new pediatric indications, respectively, than Canada. (10) Incentives and legal obligations have strongly affected the submission of pediatric data for appropriate labelling. (8,10) For instance, between 1998 and December 2021, the labels of 967 medications in the US were updated to include pediatric information, mainly to comply with legal requirements. (11)
In contrast to the US and EU, Canada does not have a purposeful regulatory framework for pediatric drug approval. For existing medications (both off- and on-patent), the current framework does not authorize Health Canada to request manufacturers to update product monographs when pediatric efficacy or safety data are available, and does not provide incentives to encourage manufacturers to market pediatric formulations that exist in other trusted foreign jurisdictions. For new drug submissions, manufacturers are not obligated to seek pediatric indications, nor to develop pediatric formulations when pediatric usage is anticipated. Incentives are few and limited to new medications, with a potential additional 6-month extension of data protection for manufacturers conducting pediatric clinical trials. (12) As a result, many medications submitted for approval in Canada are for adult use only,...